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58 Trials

   

 
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
setrusumab (UX143) / Mereo Biopharma, Ultragenyx
NCT06636071: Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta

Active, not recruiting
3
6
Japan
setrusumab, BPS804, UX143
Ultragenyx Pharmaceutical Inc
Osteogenesis Imperfecta
01/27
01/28
ENGULF, NCT05768854: Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta

Active, not recruiting
3
69
Europe, Canada, US, RoW
Bisphosphonate, Setrusumab, BPS804, UX143
Ultragenyx Pharmaceutical Inc
Osteogenesis Imperfecta
12/25
06/26
NCT05125809 / 2021-006597-23: Setrusumab vs Placebo for Osteogenesis Imperfecta

Active, not recruiting
2/3
182
Europe, Canada, US, RoW
Setrusumab, BPS804, UX143, Placebo
Ultragenyx Pharmaceutical Inc, Mereo BioPharma
Osteogenesis Imperfecta
03/26
04/27
Dojolvi (triheptanoin) / Ultragenyx
2022-001539-10: A Randomized Study to Determine the Effect of Triheptanoin in Pediatric Patients with Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Estudio aleatorizado para determinar el efecto de la triheptanoína en pacientes pediátricos con trastornos de la oxidación de ácidos grasos de cadena larga (LC-FAOD)

Ongoing
3
60
Europe, RoW
Dojolvi®, Miglyol® 812 N, UX007, NA, Oral liquid, Dojolvi®, Miglyol® 812 N
Ultragenyx Pharmaceutical Inc., Ultragenyx Pharmaceutical Inc.
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Trastornos de la oxidación de ácidos grasos de cadena larga (LC-FAOD), Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Trastornos de la oxidación de ácidos grasos de cadena larga (LC-FAOD), Diseases [C] - Nutritional and Metabolic Diseases [C18]
 
 
NCT05933200 / 2022-001539-10: A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD

Recruiting
3
60
Europe, Japan, RoW
Triheptanoin, UX007, Dojolvi, MCT Oil, Medium-chain Triglyceride
Ultragenyx Pharmaceutical Inc, Ultragenyx Pharmaceutical Inc.
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
08/27
08/27
A-TC7, NCT04513002: Ataxia-telangiectasia: Treating Mitochondrial Dysfunction With a Novel Form of Anaplerosis

Completed
2a/2b
30
RoW
Triheptanoin
The University of Queensland, National Health and Medical Research Council, Australia
Ataxia Telangiectasia
03/23
07/23
NCT03301532: Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D

Completed
2
10
US
Triheptanoin, C7 oil
University of Texas Southwestern Medical Center, National Institute of Neurological Disorders and Stroke (NINDS)
GLUT1DS1
06/21
07/22
NCT03181399: Diet Treatment Glucose Transporter Type 1 Deficiency (G1D)

Completed
2
45
US
Triheptanoin, C7
University of Texas Southwestern Medical Center, National Institute of Neurological Disorders and Stroke (NINDS)
GLUT1DS1, Epilepsy, Glut1 Deficiency Syndrome 1, Autosomal Recessive, Glucose Metabolism Disorders, Glucose Transport Defect, Glucose Transporter Type 1 Deficiency Syndrome, Glucose Transporter Protein Type 1 Deficiency Syndrome
09/23
09/23
NCT06067802: Study of Triheptanoin for the Prevention of Hypoglycemia in Patients with Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

Recruiting
2
8
US
Triheptanoin, Dojolvi
Jerry Vockley, MD, PhD, Ultragenyx Pharmaceutical Inc
Medium-chain Acyl-CoA Dehydrogenase Deficiency
03/27
07/27
ACTRN12614000082606: Pilot study of the treatment of patients with sporadic Inclusion Body Myositis with the Anaplerotic medication Triheptanoin

Not yet recruiting
1
3
 
Ultragenyx
Sporadic Inclusion body Myositis
 
 
NCT06340685: Triheptanoin for Children with Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency

Recruiting
1
6
US
Triheptanoin, Dojolvi
Jirair Krikor Bedoyan, Ultragenyx Pharmaceutical Inc
Pyruvate Dehydrogenase Complex Deficiency
12/26
06/27
NCT02500082: Triheptanoin (UX007) to Treat Citrate Transporter Deficiency

No Longer Available
N/A
US
triheptanoin, UX007
Irina A Anselm
Citrate Transporter Deficiency, SLC13A5 Gene Mutation
 
 
NCT02968953: Treatment With UX007 for a Single Patient With GLUT1 Deficiency Syndrome

No Longer Available
N/A
US
Triheptanoin, UX007
Jerry Vockley, MD, PhD, Ultragenyx Pharmaceutical Inc
Glucose Transporter 1 Deficiency Syndrome
 
 
NCT02018302: Post Study Continuation of C7 for G1D

No Longer Available
N/A
US
Triheptanoin, C7 oil, Heptanoate, heptanoic acid
Juan Pascual
Glut1 Deficiency Syndrome, Glucose Transporter Type 1 Deficiency Syndrome
 
 
NCT01461304: Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism

No Longer Available
N/A
US
triheptanoin, C7
Jerry Vockley, MD, PhD, Ultragenyx Pharmaceutical Inc
Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2), Mitochondrial Trifunctional Protein Deficiency, Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency, Glycogen Storage Disorders, Pyruvate Carboxylase Deficiency Disease, ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of, Barth Syndrome
 
 
NCT03773770: Expanded Access to Triheptanoin

Available
N/A
NA
Triheptanoin, UX007
Ultragenyx Pharmaceutical Inc
Long Chain Fatty Acid Oxidation Disorders
 
 
Evkeeza (evinacumab-dgnb) / Regeneron
NCT04233918 / 2019-001931-30: Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia

Completed
3
20
Europe, US, RoW
Evinacumab, REGN1500, Evkeeza™
Regeneron Pharmaceuticals
Homozygous Familial Hypercholesterolemia
01/22
05/23
NCT03409744 / 2017-003170-13: Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia

Completed
3
116
Europe, Canada, Japan, US, RoW
evinacumab, REGN1500, EVKEEZA®
Regeneron Pharmaceuticals
Homozygous Familial Hypercholesterolemia
04/23
04/23
NCT05611528: Safety and Effectiveness of Evinacumab for the Treatment of Homozygous Familial Hypercholesterolemia

Completed
3
10
Canada
Evinacumab
Daniel Gaudet, Ultragenyx Pharmaceutical Inc
Homozygous Familial Hypercholesterolemia
12/24
03/25
NCT04863014 / 2021-000437-13: Efficacy and Safety of Evinacumab in Adult Patients With Severe Hypertriglyceridemia for the Prevention of Recurrent Acute Pancreatitis

Terminated
2
21
Europe, Canada, US
evinacumab, REGN1500, Evkeeza™, Placebo
Regeneron Pharmaceuticals, Regeneron Pharmaceuticals, Inc.
Hypertriglyceridemia
02/23
02/23
2021-000437-13: Efficacy and Safety of Evinacumab in Adult Patients with Severe Hypertriglyceridemia for the Prevention of Recurrent Acute Pancreatitis

Not yet recruiting
2
120
Europe
Evinacumab, REGN1500, Concentrate for solution for infusion, Evkeeza
Regeneron Pharmaceuticals, Inc., Regeneron Pharmaceuticals, Inc.
Severe Hypertriglyceridemia for the Prevention of Recurrent Acute Pancreatitis, Hypertriglyceridemia is the presence of high levels of fats called triglycerides in the blood which can cause inflammation of the pancreas, Body processes [G] - Metabolic Phenomena [G03]
 
 
NCT06500598: Compassionate Use of Evinacumab

Available
N/A
NA
Evinacumab, REGN1500, EVKEEZA®
Regeneron Pharmaceuticals
Pediatric Homozygous Familial Hypercholesterolemia (HoFH), Severe Hypertriglyceridemia (HTG), Dysbetalipoproteinemia (DBL)
 
 
Crysvita (burosumab-twza) / Ultragenyx, Kyowa Kirin
NCT04419363: Burosumab in Children and Adolescents With X-linked Hypophosphatemia

Recruiting
4
57
Europe
Burosumab Injection
Bicetre Hospital
Rare Diseases, X-linked Hypophosphatemia
03/19
09/22
NCT04842019: Study to Assess the Safety, Pharmacokinetics and Efficacy of KRN23 in Adult Chinese Patients With XLH

Completed
4
18
RoW
KRN23, Burosumab, Crysvita
Kyowa Kirin Co., Ltd.
X-linked Hypophosphatemia (XLH)
08/23
08/23
NCT04842032: Study to Assess the Safety, Pharmacokinetics and Efficacy of KRN23 in Pediatric Chinese Patients With XLH

Completed
4
28
RoW
KRN23, Burosumab, Crysvita
Kyowa Kirin Co., Ltd.
X-linked Hypophosphatemia (XLH)
12/23
12/23
NCT05357573: Study to Assess the Safety, Pharmacokinetics and Efficacy of KRN23 in Adult Chinese Patients With TIO

Completed
4
9
RoW
KRN23, Burosumab, Crysvita
Kyowa Kirin Co., Ltd.
Tumor-Induced Osteomalacia (TIO)
11/23
12/23
BurGER, NCT04695860: Anti-FGF23 (Burosumab) in Adult Patients With XLH

Completed
3b
36
Europe
Burosumab
Wuerzburg University Hospital, Kyowa Kirin, Inc.
X-linked Hypophosphatemia
03/23
03/23
2019-003190-26: A study to test an antibody (Antibody Burosumab (KRN23)) to treat Hypophosphatemiazu (decreased phosphat level in the blood) in adults Eine Studie, um den Effekt eines Antikörpers (Burosumab (KRN23)) bei der Anwendung in erwachsenen Patienten mit Hypophosphatämie (zu niedriger Phosphatspiegel im Blut) zu testen

Not yet recruiting
3
34
Europe
Crysvita, Solution for injection, Crysvita
Julius-Maximilian University of Würzburg, Kyowa Kirin GmbH
X-linked hypophosphatemia (XLH) is a disorder of renal phosphate wasting, and the most common heritable form of rickets. In XLH patients, high circulating levels of fibroblast growth factor 23 (FGF23) impair normal phosphate reabsorption in the kidney. Low serum phosphorus levels result in hypomineralization of bone and associated abnormalities including rickets, bowing of the legs, and short stature. X-chromosomale Hypophosphatämie (XLH) ist eine Störung der Nierenphosphatverschwendung und die häufigste vererbbare Form von Rachitis. Bei XLH-Patienten beeinträchtigen hohe zirkulierende Spiegel des Fibroblasten-Wachstumsfaktors 23 (FGF23) die normale Phosphat-Reabsorption in der Niere. Niedrige Serumphosphorspiegel führen zu einer Hypomineralisierung des Knochens und damit verbundenen Anomalien wie Rachitis, Beugung der Beine und Kleinwuchs., The disease leads to a low phosphate level in the blood and thus to a demineralization of the bones with associated malformations in bones. Die Krankheit zeigt einen niedrigen Phosphatspiegel im Blut und führt zu einer Demineralisierung der Knochen mit damit verbundenen Fehlbildungen bei Knochen., Diseases [C] - Nutritional and Metabolic Diseases [C18]
 
 
NCT05509595: Burosumab for Fibroblast Growth Factor-23 Mediated Hypophosphatemia in Fibrous Dysplasia

Completed
2
12
US
Burosumab
National Institute of Dental and Craniofacial Research (NIDCR)
Fibrous Dysplasia Of Bone
11/24
11/24
NCT04188964 / 2019-000469-19: Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Patients Less Than 1 Year of Age

Completed
1/2
16
Europe
Burosumab, KRN23, Crysvita
Kyowa Kirin Pharmaceutical Development Ltd
X-linked Hypophosphatemia (XLH)
10/23
03/24
NCT03993821: Burosumab for CSHS

Active, not recruiting
1
1
US
Burosumab, Crysvita
Laura Tosi, Children's National Research Institute, Ultragenyx Pharmaceutical Inc
Cutaneous Skeletal Hypophosphatemia Syndrome (CSHS), Epidermal Nevus Syndrome
10/22
03/23
NCT06202027: Post Marketing Surveillance Study to Observe Safety and Effectiveness of CRYSVITA® in S. Korean Patients

Recruiting
N/A
100
RoW
FGF23-related hypophosphataemic rickets and osteomalacia
Kyowa Kirin Korea Co., Ltd.
FGF23-related Hypophosphataemic Rickets and Osteomalacia
06/31
07/31
NCT03775187: Expanded Access to Burosumab

Available
N/A
NA
Burosumab, UX023, Crysvita®, KRN23
Kyowa Kirin Co., Ltd., Kyowa Kirin Co., Ltd.
X-linked Hypophosphatemia, Tumor-Induced Osteomalacia
 
 
NCT05181839: A Study to Describe the Lived Experience of XLH for Adolescents at End of Skeletal Growth

Completed
N/A
25
Europe
Burosumab
Kyowa Kirin Pharmaceutical Development Ltd
X-Linked Hypophosphatemia
05/24
05/24
FLAM-XLH, NCT06248632: Effect of Burosumab on the Inflammatory Profile of Patients With X-linked Hypophosphatemic Rickets

Not yet recruiting
N/A
20
NA
Expression of inflammatory markers (Il6, Il8, Il1β, CXCL1, CCL2, CXCR3, Il1R, Il6R)
Hospices Civils de Lyon
X-Linked Hypophosphatemic Rickets
04/25
04/25
HYPO-BLASTE, NCT04159675: Burosumab and 1-25 (OH) Vitamin D on Human Osteoblasts

Recruiting
N/A
20
Europe
osteoblast biology study
Hospices Civils de Lyon
Craniosynostoses
04/26
04/26
DTX101 / Ultragenyx
2016-003430-25: A clinical study to learn about the effects of a virus that transfers the gene for human coagulation factor IX in adults with moderate/severe to severe inherited coagulations defects in the long term

Not yet recruiting
1/2
6
Europe
DTX101, Concentrate for solution for infusion
Ultragenyx Pharmaceutical, Inc., Ultragenyx Pharmaceutical, Inc.
Moderate/severe to severe hemophilia B, Moderate/severe to severe inherited blood coagulation disorder, Diseases [C] - Blood and lymphatic diseases [C15]
 
 
rebisufligene etisparvovec (UX111) / Ultragenyx
NCT04360265 / 2019-002979-34: Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA

Enrolling by invitation
3
41
Europe, US, RoW
No Investigational Product, Adjuvant Immunomodulatory (IM) Therapy
Ultragenyx Pharmaceutical Inc, Abeona Therapeutics, Inc
Mucopolysaccharidosis IIIA, MPS IIIA, Sanfilippo Syndrome, Sanfilippo A
08/27
08/27
NCT02716246 / 2015-003904-21: Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

Recruiting
2/3
36
Europe, US, RoW
UX111, scAAV9.U1a.hSGSH, ABO-102, rebisufligene etisparvovec, Prophylactic Immunomodulatory (IM) Therapy, Optimized Prophylactic IM Therapy, Adjuvant IM Therapy
Ultragenyx Pharmaceutical Inc, Abeona Therapeutics, Inc
MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
07/27
07/27
2018-000504-42: Gene Therapy Clinical Trial for Mucopolysaccharidosis IIIA in patient with middle and advanced phases of the disease Ensayo Clínico de terapia génica para la Mucopolisacaridosis tipo IIIA en pacientes con enfermedad MPS IIIA media y avanzada

Not yet recruiting
1/2
12
Europe
scAAV9.U1A.SGSH, ABO-102, Suspension for injection
Abeona Therapeutics Inc, Abeona Therapeutics Inc
MPS IIIA is a devastating lysosomal storage disease, caused by a Nsulfoglucosamine sulfohydrolase gene defect. Infants with MPS IIIA appear normal at birth, but the disease is relentlessly progressive, withdeterioration of social and adaptive abilities, neurocognitive decline, and premature death. Death typically occurs by end of the second or beginning of the third decade. Quite importantly, there is no treatmentcurrently available for the disease. MPS IIIA es una enfermedad de depósito lisosomal, causada por un defecto genético de la enzima N-sulfoglucosamina sulfohidrolasa. Los niños parecen normales al nacer, pero la enfermedad es progresiva, conel deterioro de las habilidades sociales y de adaptación, la disminución neurocognitiva y la muerte prematura. La muerte se produce normalmente a finales de la segunda o principios de la tercera década. Es de destacar que no existe un tratamiento disponible actualmente para la enfermedad., Mucopolysaccharidosis type IIIA is a genetic disease in children, caused by the toxicity of an accumulation of substances in the body that generate a progressive deterioration. La mucopolisacaridosis tipo IIIA es una enfermedad genética en niños, causada por la toxicidad de un acúmulo de sustancias en el organismo que generan un deterioro progresivo., Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
 
 
NCT06577532: Study of KRAS Neoantigen mRNA Vaccine (ABO2102) in Patients With KRAS -Mutated Solid Tumors

Recruiting
1
56
RoW
ABO2102, Toripalimab
Ruijin Hospital, Suzhou Abogen Biosciences Co., Ltd.
Pancreatic Neoplasms, Other Solid Tumors
12/26
08/27
peboctocogene camaparvovec (DTX201) / Ultragenyx
2017-000806-39: Study to test the safety and how well patients with severe hemophilia A respond to treatment with BAY 2599023 (DTX 201), a drug therapy that delivers a healthy version of the defective Factor VIII gene into the nucleus of liver cells using an altered, non-infectious virus (AAV) as a “shuttle”.

Not yet recruiting
1/2
30
Europe, RoW
recombinant AAV vector with hu37 serotype capsid (AAVhu37) encoding human FVIII, BAY 2599023 (DTX201), Concentrate for solution for infusion
Bayer AG, Bayer AG
Hemophilia A, Hemophilia A is an X-linked congenital bleeding disorder causing frequent bleedings and recurrent spontaneous bleeds into the soft tissue and joints, leading to joint damage and severe disability., Diseases [C] - Blood and lymphatic diseases [C15]
 
 
NCT03588299 / 2017-000806-39: Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle"

Active, not recruiting
1/2
11
Europe, US
BAY2599023 (DTX201)
Bayer, Ultragenix pharmaceutical
Hemophilia A
11/26
11/26
pariglasgene brecaparvovec (DTX401) / Ultragenyx
NCT05139316 / 2020-004184-12: A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)

Active, not recruiting
3
49
Europe, Canada, Japan, US, RoW
DTX401, pariglasgene brecaparvovec, Placebo, Oral corticosteroids, prednisolone, Placebo for oral corticosteroids
Ultragenyx Pharmaceutical Inc
Glycogen Storage Disease Type IA
02/24
02/26
avalotcagene ontaparvovec (DTX301) / Ultragenyx
2020-003384-25: A clinical study of a virus transferring the gene for human Ornithine Transcarbamylase (OTC) in patients older than 12 years old with late-onset OTC deficiency

Not yet recruiting
3
50
Europe
DTX301, (1-13C) Sodium Acetate, Concentrate for solution for infusion, Powder for oral solution
Ultragenyx Pharmaceutical Inc., Ultragenyx Pharmaceutical, Inc.
Late-onset Ornithine transcarbamylase (OTC) deficiency, Inherited disorder causing accumulation of ammonia, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
 
 
NCT05345171 / 2020-003384-25: Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency

Active, not recruiting
3
32
Europe, Canada, Japan, US, RoW
DTX301, avalotcagene ontaparvovec, Placebo, Oral Corticosteroids, Prednisolone, Placebo for oral corticosteroids, Sodium Acetate
Ultragenyx Pharmaceutical Inc, Ultragenyx Pharmaceutical, Inc.
OTC Deficiency
02/25
03/31
CAPtivate, NCT03636438 / 2018-000156-18: Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency

Active, not recruiting
N/A
11
Europe, Canada, US
No Intervention
Ultragenyx Pharmaceutical Inc
Ornithine Transcarbamylase (OTC) Deficiency
12/29
12/29
apazunersen (GTX-102) / Foundation for Angelman Syndrome Therap, Ultragenyx
GTX-102-CL 302, NCT06415344: Long-term Extension of GTX-102 in Angelman Syndrome

Enrolling by invitation
3
75
Europe, Canada, US, RoW
GTX-102
Ultragenyx Pharmaceutical Inc
Angelman Syndrome
02/29
02/29
Aspire, NCT06617429: Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With AS

Recruiting
3
120
Europe, Canada, Japan, US, RoW
GTX-102, Sham-LP
Ultragenyx Pharmaceutical Inc
Angelman Syndrome
07/26
11/27
2021-001793-36: A study of the safety of GTX-102 in pediatric patients with Angelman Syndrome (AS)

Not yet recruiting
1/2
83
Europe
GTX-102, GTX-102, Concentrate and solvent for solution for injection, ELLIOTTS B® SOLUTION (buffered intrathecal electrolyte/dextrose injection)
Ultragenyx Pharmaceutical Inc., Ultragenyx Pharmceutical Inc.
Angelman Syndrome, A genetic condition that affects the nervous system and causes severe physical and learning disabilities, Diseases [C] - Nervous System Diseases [C10]
 
 
KIK-AS, NCT04259281 / 2021-001793-36: A Study of the Safety and Tolerability of GTX-102 in Children with Angelman Syndrome

Completed
1/2
74
Europe, Canada, US, RoW
GTX-102
Ultragenyx Pharmaceutical Inc, Ultragenyx Pharmceutical Inc.
Angelman Syndrome
01/25
01/25
rivunatpagene miziparvovec (UX701) / Ultragenyx
2020-005266-34: A study to understand the safety and the effects of a virus that transfers a modified protein responsible for copper metabolism (copper-transporting P-type adenosine triphosphatase, ATP7B) in adults with Wilson disease. Un estudio para entender la seguridad y los efectos del virus que transfiere a proteína modificada responsable del metabolismo del cobre (el cobre transporta la Adenosina Triphosphatase P-type, ATP7B) en adultos con la enfermedad de Wilson.

Not yet recruiting
2/3
94
Europe
UX701 (5.0 × 10^12 genome copies/kg), UX701 (1.0 × 10^13 genome copies/kg), UX701 (2.0 × 10^13 genome copies/kg), Concentrate for solution for infusion
Ultragenyx Pharmaceutical Inc., Ultragenyx Pharmaceutical Inc.
Wilson disease Enfermedad de Wilson, Inherited disorder of copper metabolism Trastorno hereditario del metabolismo del cobre, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
 
 
NCT04884815 / 2020-005266-34: A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease

Recruiting
1/2
82
Europe, Canada, US
UX701, rivunatpagene miziparvovec, Standard of Care (SOC)
Ultragenyx Pharmaceutical Inc, Ultragenyx Pharmaceutical Inc.
Wilson Disease
03/29
03/34
Mepsevii (vestronidase alfa) / Ultragenyx, Amicus
IUERT, NCT04532047: PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

Recruiting
1
10
US
Aldurazyme (laronidase), Elaprase (idursulfase), Vimizim (elosulfase alfa), Naglazyme (galsulfase), Mepsevii (vestronidase alfa-vjbk), Lumizyme (alglucosidase alfa), Kanuma (sebelipase alfa)
University of California, San Francisco, Duke University
MPS I, MPS II, MPS IVA, MPS VI, Mps VII, Gaucher Disease, Type 2, Gaucher Disease, Type 3, Pompe Disease Infantile-Onset, Wolman Disease
07/31
07/32
NCT02097251: An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient

No Longer Available
N/A
US
UX003, recombinant human beta glucuronidase, rhGUS
Joyce Fox, Ultragenyx Pharmaceutical Inc
Mucopolysaccharidosis Type 7
 
 
NCT03775174: Expanded Access to Mepsevii

Available
N/A
NA
Mepsevii, UX003, recombinant human beta-glucuronidase, rhGUS, vestronidase alfa
Ultragenyx Pharmaceutical Inc
MPS VII, Mucopolysaccharidosis VII, Sly Syndrome
 
 
UX053 / Ultragenyx, Arcturus Therap
2021-000903-19: A first-in-human study of UX053 in Patients with Glycogen Storage Disease type III (GSD III) Première administration à l'Homme d'UX053 chez des patients atteints de glycogénose de type III

Not yet recruiting
1/2
30
Europe
mRNA encoding the human glycogen debranching enzyme formulated in a lipid nanoparticle delivery, UX053, Solution for infusion
Ultragenyx Pharmaceutical Inc., ULTRAGENYX PHARMACEUTICAL INC., Ultragenyx Pharmaceutical Inc.
Glycogen Storage Disease Type III (GSD III) Glycogénose de type III, A disease where the body cannot break down glycogen (stored form of glucose). It affects the liver, heart muscle, and skeletal muscle. It is also known as Cori disease or Forbes disease. Maladie où le corps ne peut pas décomposer le glycogène (forme stockée de glucose). Il affecte le foie, le muscle cardiaque et le muscle squelettique. On l'appelle aussi maladie de Cori ou de Forbes., Diseases [C] - Nutritional and Metabolic Diseases [C18]
 
 
NCT04990388 / 2021-000903-19: Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III)

Terminated
1/2
9
Europe, US
UX053, Placebo, Antipyretic, paracetamol, acetaminophen, ibuprofen, H2 Blocker, famotidine, H1 Blocker, cetirizine
Ultragenyx Pharmaceutical Inc, Ultragenyx Pharmaceutical Inc.
Glycogen Storage Disease Type III
03/23
03/23
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
setrusumab (UX143) / Mereo Biopharma, Ultragenyx
NCT06636071: Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta

Active, not recruiting
3
6
Japan
setrusumab, BPS804, UX143
Ultragenyx Pharmaceutical Inc
Osteogenesis Imperfecta
01/27
01/28
ENGULF, NCT05768854: Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta

Active, not recruiting
3
69
Europe, Canada, US, RoW
Bisphosphonate, Setrusumab, BPS804, UX143
Ultragenyx Pharmaceutical Inc
Osteogenesis Imperfecta
12/25
06/26
NCT05125809 / 2021-006597-23: Setrusumab vs Placebo for Osteogenesis Imperfecta

Active, not recruiting
2/3
182
Europe, Canada, US, RoW
Setrusumab, BPS804, UX143, Placebo
Ultragenyx Pharmaceutical Inc, Mereo BioPharma
Osteogenesis Imperfecta
03/26
04/27
Dojolvi (triheptanoin) / Ultragenyx
2022-001539-10: A Randomized Study to Determine the Effect of Triheptanoin in Pediatric Patients with Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Estudio aleatorizado para determinar el efecto de la triheptanoína en pacientes pediátricos con trastornos de la oxidación de ácidos grasos de cadena larga (LC-FAOD)

Ongoing
3
60
Europe, RoW
Dojolvi®, Miglyol® 812 N, UX007, NA, Oral liquid, Dojolvi®, Miglyol® 812 N
Ultragenyx Pharmaceutical Inc., Ultragenyx Pharmaceutical Inc.
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Trastornos de la oxidación de ácidos grasos de cadena larga (LC-FAOD), Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Trastornos de la oxidación de ácidos grasos de cadena larga (LC-FAOD), Diseases [C] - Nutritional and Metabolic Diseases [C18]
 
 
NCT05933200 / 2022-001539-10: A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD

Recruiting
3
60
Europe, Japan, RoW
Triheptanoin, UX007, Dojolvi, MCT Oil, Medium-chain Triglyceride
Ultragenyx Pharmaceutical Inc, Ultragenyx Pharmaceutical Inc.
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
08/27
08/27
A-TC7, NCT04513002: Ataxia-telangiectasia: Treating Mitochondrial Dysfunction With a Novel Form of Anaplerosis

Completed
2a/2b
30
RoW
Triheptanoin
The University of Queensland, National Health and Medical Research Council, Australia
Ataxia Telangiectasia
03/23
07/23
NCT03301532: Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D

Completed
2
10
US
Triheptanoin, C7 oil
University of Texas Southwestern Medical Center, National Institute of Neurological Disorders and Stroke (NINDS)
GLUT1DS1
06/21
07/22
NCT03181399: Diet Treatment Glucose Transporter Type 1 Deficiency (G1D)

Completed
2
45
US
Triheptanoin, C7
University of Texas Southwestern Medical Center, National Institute of Neurological Disorders and Stroke (NINDS)
GLUT1DS1, Epilepsy, Glut1 Deficiency Syndrome 1, Autosomal Recessive, Glucose Metabolism Disorders, Glucose Transport Defect, Glucose Transporter Type 1 Deficiency Syndrome, Glucose Transporter Protein Type 1 Deficiency Syndrome
09/23
09/23
NCT06067802: Study of Triheptanoin for the Prevention of Hypoglycemia in Patients with Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

Recruiting
2
8
US
Triheptanoin, Dojolvi
Jerry Vockley, MD, PhD, Ultragenyx Pharmaceutical Inc
Medium-chain Acyl-CoA Dehydrogenase Deficiency
03/27
07/27
ACTRN12614000082606: Pilot study of the treatment of patients with sporadic Inclusion Body Myositis with the Anaplerotic medication Triheptanoin

Not yet recruiting
1
3
 
Ultragenyx
Sporadic Inclusion body Myositis
 
 
NCT06340685: Triheptanoin for Children with Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency

Recruiting
1
6
US
Triheptanoin, Dojolvi
Jirair Krikor Bedoyan, Ultragenyx Pharmaceutical Inc
Pyruvate Dehydrogenase Complex Deficiency
12/26
06/27
NCT02500082: Triheptanoin (UX007) to Treat Citrate Transporter Deficiency

No Longer Available
N/A
US
triheptanoin, UX007
Irina A Anselm
Citrate Transporter Deficiency, SLC13A5 Gene Mutation
 
 
NCT02968953: Treatment With UX007 for a Single Patient With GLUT1 Deficiency Syndrome

No Longer Available
N/A
US
Triheptanoin, UX007
Jerry Vockley, MD, PhD, Ultragenyx Pharmaceutical Inc
Glucose Transporter 1 Deficiency Syndrome
 
 
NCT02018302: Post Study Continuation of C7 for G1D

No Longer Available
N/A
US
Triheptanoin, C7 oil, Heptanoate, heptanoic acid
Juan Pascual
Glut1 Deficiency Syndrome, Glucose Transporter Type 1 Deficiency Syndrome
 
 
NCT01461304: Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism

No Longer Available
N/A
US
triheptanoin, C7
Jerry Vockley, MD, PhD, Ultragenyx Pharmaceutical Inc
Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2), Mitochondrial Trifunctional Protein Deficiency, Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency, Glycogen Storage Disorders, Pyruvate Carboxylase Deficiency Disease, ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of, Barth Syndrome
 
 
NCT03773770: Expanded Access to Triheptanoin

Available
N/A
NA
Triheptanoin, UX007
Ultragenyx Pharmaceutical Inc
Long Chain Fatty Acid Oxidation Disorders
 
 
Evkeeza (evinacumab-dgnb) / Regeneron
NCT04233918 / 2019-001931-30: Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia

Completed
3
20
Europe, US, RoW
Evinacumab, REGN1500, Evkeeza™
Regeneron Pharmaceuticals
Homozygous Familial Hypercholesterolemia
01/22
05/23
NCT03409744 / 2017-003170-13: Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia

Completed
3
116
Europe, Canada, Japan, US, RoW
evinacumab, REGN1500, EVKEEZA®
Regeneron Pharmaceuticals
Homozygous Familial Hypercholesterolemia
04/23
04/23
NCT05611528: Safety and Effectiveness of Evinacumab for the Treatment of Homozygous Familial Hypercholesterolemia

Completed
3
10
Canada
Evinacumab
Daniel Gaudet, Ultragenyx Pharmaceutical Inc
Homozygous Familial Hypercholesterolemia
12/24
03/25
NCT04863014 / 2021-000437-13: Efficacy and Safety of Evinacumab in Adult Patients With Severe Hypertriglyceridemia for the Prevention of Recurrent Acute Pancreatitis

Terminated
2
21
Europe, Canada, US
evinacumab, REGN1500, Evkeeza™, Placebo
Regeneron Pharmaceuticals, Regeneron Pharmaceuticals, Inc.
Hypertriglyceridemia
02/23
02/23
2021-000437-13: Efficacy and Safety of Evinacumab in Adult Patients with Severe Hypertriglyceridemia for the Prevention of Recurrent Acute Pancreatitis

Not yet recruiting
2
120
Europe
Evinacumab, REGN1500, Concentrate for solution for infusion, Evkeeza
Regeneron Pharmaceuticals, Inc., Regeneron Pharmaceuticals, Inc.
Severe Hypertriglyceridemia for the Prevention of Recurrent Acute Pancreatitis, Hypertriglyceridemia is the presence of high levels of fats called triglycerides in the blood which can cause inflammation of the pancreas, Body processes [G] - Metabolic Phenomena [G03]
 
 
NCT06500598: Compassionate Use of Evinacumab

Available
N/A
NA
Evinacumab, REGN1500, EVKEEZA®
Regeneron Pharmaceuticals
Pediatric Homozygous Familial Hypercholesterolemia (HoFH), Severe Hypertriglyceridemia (HTG), Dysbetalipoproteinemia (DBL)
 
 
Crysvita (burosumab-twza) / Ultragenyx, Kyowa Kirin
NCT04419363: Burosumab in Children and Adolescents With X-linked Hypophosphatemia

Recruiting
4
57
Europe
Burosumab Injection
Bicetre Hospital
Rare Diseases, X-linked Hypophosphatemia
03/19
09/22
NCT04842019: Study to Assess the Safety, Pharmacokinetics and Efficacy of KRN23 in Adult Chinese Patients With XLH

Completed
4
18
RoW
KRN23, Burosumab, Crysvita
Kyowa Kirin Co., Ltd.
X-linked Hypophosphatemia (XLH)
08/23
08/23
NCT04842032: Study to Assess the Safety, Pharmacokinetics and Efficacy of KRN23 in Pediatric Chinese Patients With XLH

Completed
4
28
RoW
KRN23, Burosumab, Crysvita
Kyowa Kirin Co., Ltd.
X-linked Hypophosphatemia (XLH)
12/23
12/23
NCT05357573: Study to Assess the Safety, Pharmacokinetics and Efficacy of KRN23 in Adult Chinese Patients With TIO

Completed
4
9
RoW
KRN23, Burosumab, Crysvita
Kyowa Kirin Co., Ltd.
Tumor-Induced Osteomalacia (TIO)
11/23
12/23
BurGER, NCT04695860: Anti-FGF23 (Burosumab) in Adult Patients With XLH

Completed
3b
36
Europe
Burosumab
Wuerzburg University Hospital, Kyowa Kirin, Inc.
X-linked Hypophosphatemia
03/23
03/23
2019-003190-26: A study to test an antibody (Antibody Burosumab (KRN23)) to treat Hypophosphatemiazu (decreased phosphat level in the blood) in adults Eine Studie, um den Effekt eines Antikörpers (Burosumab (KRN23)) bei der Anwendung in erwachsenen Patienten mit Hypophosphatämie (zu niedriger Phosphatspiegel im Blut) zu testen

Not yet recruiting
3
34
Europe
Crysvita, Solution for injection, Crysvita
Julius-Maximilian University of Würzburg, Kyowa Kirin GmbH
X-linked hypophosphatemia (XLH) is a disorder of renal phosphate wasting, and the most common heritable form of rickets. In XLH patients, high circulating levels of fibroblast growth factor 23 (FGF23) impair normal phosphate reabsorption in the kidney. Low serum phosphorus levels result in hypomineralization of bone and associated abnormalities including rickets, bowing of the legs, and short stature. X-chromosomale Hypophosphatämie (XLH) ist eine Störung der Nierenphosphatverschwendung und die häufigste vererbbare Form von Rachitis. Bei XLH-Patienten beeinträchtigen hohe zirkulierende Spiegel des Fibroblasten-Wachstumsfaktors 23 (FGF23) die normale Phosphat-Reabsorption in der Niere. Niedrige Serumphosphorspiegel führen zu einer Hypomineralisierung des Knochens und damit verbundenen Anomalien wie Rachitis, Beugung der Beine und Kleinwuchs., The disease leads to a low phosphate level in the blood and thus to a demineralization of the bones with associated malformations in bones. Die Krankheit zeigt einen niedrigen Phosphatspiegel im Blut und führt zu einer Demineralisierung der Knochen mit damit verbundenen Fehlbildungen bei Knochen., Diseases [C] - Nutritional and Metabolic Diseases [C18]
 
 
NCT05509595: Burosumab for Fibroblast Growth Factor-23 Mediated Hypophosphatemia in Fibrous Dysplasia

Completed
2
12
US
Burosumab
National Institute of Dental and Craniofacial Research (NIDCR)
Fibrous Dysplasia Of Bone
11/24
11/24
NCT04188964 / 2019-000469-19: Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Patients Less Than 1 Year of Age

Completed
1/2
16
Europe
Burosumab, KRN23, Crysvita
Kyowa Kirin Pharmaceutical Development Ltd
X-linked Hypophosphatemia (XLH)
10/23
03/24
NCT03993821: Burosumab for CSHS

Active, not recruiting
1
1
US
Burosumab, Crysvita
Laura Tosi, Children's National Research Institute, Ultragenyx Pharmaceutical Inc
Cutaneous Skeletal Hypophosphatemia Syndrome (CSHS), Epidermal Nevus Syndrome
10/22
03/23
NCT06202027: Post Marketing Surveillance Study to Observe Safety and Effectiveness of CRYSVITA® in S. Korean Patients

Recruiting
N/A
100
RoW
FGF23-related hypophosphataemic rickets and osteomalacia
Kyowa Kirin Korea Co., Ltd.
FGF23-related Hypophosphataemic Rickets and Osteomalacia
06/31
07/31
NCT03775187: Expanded Access to Burosumab

Available
N/A
NA
Burosumab, UX023, Crysvita®, KRN23
Kyowa Kirin Co., Ltd., Kyowa Kirin Co., Ltd.
X-linked Hypophosphatemia, Tumor-Induced Osteomalacia
 
 
NCT05181839: A Study to Describe the Lived Experience of XLH for Adolescents at End of Skeletal Growth

Completed
N/A
25
Europe
Burosumab
Kyowa Kirin Pharmaceutical Development Ltd
X-Linked Hypophosphatemia
05/24
05/24
FLAM-XLH, NCT06248632: Effect of Burosumab on the Inflammatory Profile of Patients With X-linked Hypophosphatemic Rickets

Not yet recruiting
N/A
20
NA
Expression of inflammatory markers (Il6, Il8, Il1β, CXCL1, CCL2, CXCR3, Il1R, Il6R)
Hospices Civils de Lyon
X-Linked Hypophosphatemic Rickets
04/25
04/25
HYPO-BLASTE, NCT04159675: Burosumab and 1-25 (OH) Vitamin D on Human Osteoblasts

Recruiting
N/A
20
Europe
osteoblast biology study
Hospices Civils de Lyon
Craniosynostoses
04/26
04/26
DTX101 / Ultragenyx
2016-003430-25: A clinical study to learn about the effects of a virus that transfers the gene for human coagulation factor IX in adults with moderate/severe to severe inherited coagulations defects in the long term

Not yet recruiting
1/2
6
Europe
DTX101, Concentrate for solution for infusion
Ultragenyx Pharmaceutical, Inc., Ultragenyx Pharmaceutical, Inc.
Moderate/severe to severe hemophilia B, Moderate/severe to severe inherited blood coagulation disorder, Diseases [C] - Blood and lymphatic diseases [C15]
 
 
rebisufligene etisparvovec (UX111) / Ultragenyx
NCT04360265 / 2019-002979-34: Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA

Enrolling by invitation
3
41
Europe, US, RoW
No Investigational Product, Adjuvant Immunomodulatory (IM) Therapy
Ultragenyx Pharmaceutical Inc, Abeona Therapeutics, Inc
Mucopolysaccharidosis IIIA, MPS IIIA, Sanfilippo Syndrome, Sanfilippo A
08/27
08/27
NCT02716246 / 2015-003904-21: Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

Recruiting
2/3
36
Europe, US, RoW
UX111, scAAV9.U1a.hSGSH, ABO-102, rebisufligene etisparvovec, Prophylactic Immunomodulatory (IM) Therapy, Optimized Prophylactic IM Therapy, Adjuvant IM Therapy
Ultragenyx Pharmaceutical Inc, Abeona Therapeutics, Inc
MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
07/27
07/27
2018-000504-42: Gene Therapy Clinical Trial for Mucopolysaccharidosis IIIA in patient with middle and advanced phases of the disease Ensayo Clínico de terapia génica para la Mucopolisacaridosis tipo IIIA en pacientes con enfermedad MPS IIIA media y avanzada

Not yet recruiting
1/2
12
Europe
scAAV9.U1A.SGSH, ABO-102, Suspension for injection
Abeona Therapeutics Inc, Abeona Therapeutics Inc
MPS IIIA is a devastating lysosomal storage disease, caused by a Nsulfoglucosamine sulfohydrolase gene defect. Infants with MPS IIIA appear normal at birth, but the disease is relentlessly progressive, withdeterioration of social and adaptive abilities, neurocognitive decline, and premature death. Death typically occurs by end of the second or beginning of the third decade. Quite importantly, there is no treatmentcurrently available for the disease. MPS IIIA es una enfermedad de depósito lisosomal, causada por un defecto genético de la enzima N-sulfoglucosamina sulfohidrolasa. Los niños parecen normales al nacer, pero la enfermedad es progresiva, conel deterioro de las habilidades sociales y de adaptación, la disminución neurocognitiva y la muerte prematura. La muerte se produce normalmente a finales de la segunda o principios de la tercera década. Es de destacar que no existe un tratamiento disponible actualmente para la enfermedad., Mucopolysaccharidosis type IIIA is a genetic disease in children, caused by the toxicity of an accumulation of substances in the body that generate a progressive deterioration. La mucopolisacaridosis tipo IIIA es una enfermedad genética en niños, causada por la toxicidad de un acúmulo de sustancias en el organismo que generan un deterioro progresivo., Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
 
 
NCT06577532: Study of KRAS Neoantigen mRNA Vaccine (ABO2102) in Patients With KRAS -Mutated Solid Tumors

Recruiting
1
56
RoW
ABO2102, Toripalimab
Ruijin Hospital, Suzhou Abogen Biosciences Co., Ltd.
Pancreatic Neoplasms, Other Solid Tumors
12/26
08/27
peboctocogene camaparvovec (DTX201) / Ultragenyx
2017-000806-39: Study to test the safety and how well patients with severe hemophilia A respond to treatment with BAY 2599023 (DTX 201), a drug therapy that delivers a healthy version of the defective Factor VIII gene into the nucleus of liver cells using an altered, non-infectious virus (AAV) as a “shuttle”.

Not yet recruiting
1/2
30
Europe, RoW
recombinant AAV vector with hu37 serotype capsid (AAVhu37) encoding human FVIII, BAY 2599023 (DTX201), Concentrate for solution for infusion
Bayer AG, Bayer AG
Hemophilia A, Hemophilia A is an X-linked congenital bleeding disorder causing frequent bleedings and recurrent spontaneous bleeds into the soft tissue and joints, leading to joint damage and severe disability., Diseases [C] - Blood and lymphatic diseases [C15]
 
 
NCT03588299 / 2017-000806-39: Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle"

Active, not recruiting
1/2
11
Europe, US
BAY2599023 (DTX201)
Bayer, Ultragenix pharmaceutical
Hemophilia A
11/26
11/26
pariglasgene brecaparvovec (DTX401) / Ultragenyx
NCT05139316 / 2020-004184-12: A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)

Active, not recruiting
3
49
Europe, Canada, Japan, US, RoW
DTX401, pariglasgene brecaparvovec, Placebo, Oral corticosteroids, prednisolone, Placebo for oral corticosteroids
Ultragenyx Pharmaceutical Inc
Glycogen Storage Disease Type IA
02/24
02/26
avalotcagene ontaparvovec (DTX301) / Ultragenyx
2020-003384-25: A clinical study of a virus transferring the gene for human Ornithine Transcarbamylase (OTC) in patients older than 12 years old with late-onset OTC deficiency

Not yet recruiting
3
50
Europe
DTX301, (1-13C) Sodium Acetate, Concentrate for solution for infusion, Powder for oral solution
Ultragenyx Pharmaceutical Inc., Ultragenyx Pharmaceutical, Inc.
Late-onset Ornithine transcarbamylase (OTC) deficiency, Inherited disorder causing accumulation of ammonia, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
 
 
NCT05345171 / 2020-003384-25: Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency

Active, not recruiting
3
32
Europe, Canada, Japan, US, RoW
DTX301, avalotcagene ontaparvovec, Placebo, Oral Corticosteroids, Prednisolone, Placebo for oral corticosteroids, Sodium Acetate
Ultragenyx Pharmaceutical Inc, Ultragenyx Pharmaceutical, Inc.
OTC Deficiency
02/25
03/31
CAPtivate, NCT03636438 / 2018-000156-18: Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency

Active, not recruiting
N/A
11
Europe, Canada, US
No Intervention
Ultragenyx Pharmaceutical Inc
Ornithine Transcarbamylase (OTC) Deficiency
12/29
12/29
apazunersen (GTX-102) / Foundation for Angelman Syndrome Therap, Ultragenyx
GTX-102-CL 302, NCT06415344: Long-term Extension of GTX-102 in Angelman Syndrome

Enrolling by invitation
3
75
Europe, Canada, US, RoW
GTX-102
Ultragenyx Pharmaceutical Inc
Angelman Syndrome
02/29
02/29
Aspire, NCT06617429: Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With AS

Recruiting
3
120
Europe, Canada, Japan, US, RoW
GTX-102, Sham-LP
Ultragenyx Pharmaceutical Inc
Angelman Syndrome
07/26
11/27
2021-001793-36: A study of the safety of GTX-102 in pediatric patients with Angelman Syndrome (AS)

Not yet recruiting
1/2
83
Europe
GTX-102, GTX-102, Concentrate and solvent for solution for injection, ELLIOTTS B® SOLUTION (buffered intrathecal electrolyte/dextrose injection)
Ultragenyx Pharmaceutical Inc., Ultragenyx Pharmceutical Inc.
Angelman Syndrome, A genetic condition that affects the nervous system and causes severe physical and learning disabilities, Diseases [C] - Nervous System Diseases [C10]
 
 
KIK-AS, NCT04259281 / 2021-001793-36: A Study of the Safety and Tolerability of GTX-102 in Children with Angelman Syndrome

Completed
1/2
74
Europe, Canada, US, RoW
GTX-102
Ultragenyx Pharmaceutical Inc, Ultragenyx Pharmceutical Inc.
Angelman Syndrome
01/25
01/25
rivunatpagene miziparvovec (UX701) / Ultragenyx
2020-005266-34: A study to understand the safety and the effects of a virus that transfers a modified protein responsible for copper metabolism (copper-transporting P-type adenosine triphosphatase, ATP7B) in adults with Wilson disease. Un estudio para entender la seguridad y los efectos del virus que transfiere a proteína modificada responsable del metabolismo del cobre (el cobre transporta la Adenosina Triphosphatase P-type, ATP7B) en adultos con la enfermedad de Wilson.

Not yet recruiting
2/3
94
Europe
UX701 (5.0 × 10^12 genome copies/kg), UX701 (1.0 × 10^13 genome copies/kg), UX701 (2.0 × 10^13 genome copies/kg), Concentrate for solution for infusion
Ultragenyx Pharmaceutical Inc., Ultragenyx Pharmaceutical Inc.
Wilson disease Enfermedad de Wilson, Inherited disorder of copper metabolism Trastorno hereditario del metabolismo del cobre, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
 
 
NCT04884815 / 2020-005266-34: A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease

Recruiting
1/2
82
Europe, Canada, US
UX701, rivunatpagene miziparvovec, Standard of Care (SOC)
Ultragenyx Pharmaceutical Inc, Ultragenyx Pharmaceutical Inc.
Wilson Disease
03/29
03/34
Mepsevii (vestronidase alfa) / Ultragenyx, Amicus
IUERT, NCT04532047: PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

Recruiting
1
10
US
Aldurazyme (laronidase), Elaprase (idursulfase), Vimizim (elosulfase alfa), Naglazyme (galsulfase), Mepsevii (vestronidase alfa-vjbk), Lumizyme (alglucosidase alfa), Kanuma (sebelipase alfa)
University of California, San Francisco, Duke University
MPS I, MPS II, MPS IVA, MPS VI, Mps VII, Gaucher Disease, Type 2, Gaucher Disease, Type 3, Pompe Disease Infantile-Onset, Wolman Disease
07/31
07/32
NCT02097251: An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient

No Longer Available
N/A
US
UX003, recombinant human beta glucuronidase, rhGUS
Joyce Fox, Ultragenyx Pharmaceutical Inc
Mucopolysaccharidosis Type 7
 
 
NCT03775174: Expanded Access to Mepsevii

Available
N/A
NA
Mepsevii, UX003, recombinant human beta-glucuronidase, rhGUS, vestronidase alfa
Ultragenyx Pharmaceutical Inc
MPS VII, Mucopolysaccharidosis VII, Sly Syndrome
 
 
UX053 / Ultragenyx, Arcturus Therap
2021-000903-19: A first-in-human study of UX053 in Patients with Glycogen Storage Disease type III (GSD III) Première administration à l'Homme d'UX053 chez des patients atteints de glycogénose de type III

Not yet recruiting
1/2
30
Europe
mRNA encoding the human glycogen debranching enzyme formulated in a lipid nanoparticle delivery, UX053, Solution for infusion
Ultragenyx Pharmaceutical Inc., ULTRAGENYX PHARMACEUTICAL INC., Ultragenyx Pharmaceutical Inc.
Glycogen Storage Disease Type III (GSD III) Glycogénose de type III, A disease where the body cannot break down glycogen (stored form of glucose). It affects the liver, heart muscle, and skeletal muscle. It is also known as Cori disease or Forbes disease. Maladie où le corps ne peut pas décomposer le glycogène (forme stockée de glucose). Il affecte le foie, le muscle cardiaque et le muscle squelettique. On l'appelle aussi maladie de Cori ou de Forbes., Diseases [C] - Nutritional and Metabolic Diseases [C18]
 
 
NCT04990388 / 2021-000903-19: Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III)

Terminated
1/2
9
Europe, US
UX053, Placebo, Antipyretic, paracetamol, acetaminophen, ibuprofen, H2 Blocker, famotidine, H1 Blocker, cetirizine
Ultragenyx Pharmaceutical Inc, Ultragenyx Pharmaceutical Inc.
Glycogen Storage Disease Type III
03/23
03/23

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