University of Alberta - Faculty of Medicine & Dentistry
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 9 Trials 
35 Trials

   

 
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Ficicioglu, Can
NCT05099640 / 2021-000474-29: A Study of PTC923 in Participants With Phenylketonuria

Completed
3
157
Europe, Canada, US, RoW
PTC923, Placebo
PTC Therapeutics
Phenylketonuria
04/23
05/23
LEAP2MONO, NCT05222906 / 2021-005402-10: Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3

Active, not recruiting
3
43
Europe, Canada, Japan, US, RoW
Venglustat, imiglucerase, Cerezyme®
Sanofi, Sanofi-aventis recherche & développement
Gaucher's Disease Type III
09/25
10/26
SYNPHENY-3, NCT05764239: Efficacy and Safety of SYNB1934 in Patients With PKU

Terminated
3
35
Canada, US, RoW
SYNB1934v1, Placebo
Synlogic
Phenylketonuria
03/24
03/24
NCT05166161 / 2021-000497-28: A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria

Recruiting
3
200
Europe, Canada, Japan, US, RoW
PTC923, Sepiapterin
PTC Therapeutics
Phenylketonuria
04/26
04/26
STARLIGHT, NCT04573023 / 2020-003200-14: A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II

Recruiting
3
80
Europe, US, RoW
JR-141, Idursulfase, JR-141 or Idursulfase
JCR Pharmaceuticals Co., Ltd., JCR Pharmaceuticals Co., Ltd.
Mucopolysaccharidosis II
01/26
01/26
NCT03566043: CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)

Active, not recruiting
2/3
48
US, RoW
RGX-121
REGENXBIO Inc.
Mucopolysaccharidosis Type II (MPS II)
11/23
08/25
COMPASS, NCT05371613 / 2021-005200-35: A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) Vs Idursulfase in Pediatric and Young Adult Participants with Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

Recruiting
2/3
54
Europe, Canada, US, RoW
tividenofusp alfa, idursulfase
Denali Therapeutics Inc.
Mucopolysaccharidosis II
12/25
12/25
NCT05795361: Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome

Available
N/A
Europe, US, RoW
Idursulfase-IT, HGT-2310, TAK-609
Takeda
Hunter Syndrome
 
 
OR-ARI-EAP-NPC, NCT04316637: Early Access Program With Arimoclomol in US Patients With NPC

Available
N/A
US
Arimoclomol
ZevraDenmark
Niemann-Pick Disease, Type C
 
 
NCT04612764: Liver Disease in Urea Cycle Disorders

Recruiting
N/A
62
US
Baylor College of Medicine, Children's National Research Institute, Seattle Children's Hospital, Children's Hospital Colorado, Children's Hospital of Philadelphia
Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency, Citrullinemia 1, ARGI Deficiency, ASL Deficiency, Argininosuccinic Aciduria, ASS Deficiency, Hyperargininemia
12/24
12/24
NCT00237315: Longitudinal Study of Urea Cycle Disorders

Recruiting
N/A
1500
Europe, Canada, US
Andrea Gropman, National Center for Research Resources (NCRR), Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), Rare Diseases Clinical Research Network
Brain Diseases, Metabolic, Inborn, Amino Acid Metabolism, Inborn Errors, Urea Cycle Disorders
07/25
07/26
NCT04041102: Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients

Recruiting
N/A
40
Europe, Canada, US, RoW
University of Pennsylvania, Passage Bio, Inc.
GM1 Gangliosidosis
06/26
12/26
Rajan, Deepa Soundara
COMPASS, NCT05371613 / 2021-005200-35: A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) Vs Idursulfase in Pediatric and Young Adult Participants with Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

Recruiting
2/3
54
Europe, Canada, US, RoW
tividenofusp alfa, idursulfase
Denali Therapeutics Inc.
Mucopolysaccharidosis II
12/25
12/25
NCT04251026 / 2019-004909-27: A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome

Active, not recruiting
1/2
47
Europe, Canada, US
tividenofusp alfa
Denali Therapeutics Inc.
Mucopolysaccharidosis II
07/27
07/27
NCT04007536: A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome

Completed
N/A
18
Europe, US
No Intervention
Denali Therapeutics Inc.
Mucopolysaccharidosis II
03/24
03/24
NCT06776341: Natural History Study of GEMIN-5 Related Neurodevelopmental Disorder

Not yet recruiting
N/A
500
US
GEMIN5-Related Neurodevelopmental Disorder
University of Pittsburgh
SMN Complex Proteins, GEMIN5 Protein, Human, Neurodevelopmental Disorders
12/50
12/50
NCT02995538: Neurogenetics Patient Registry

Recruiting
N/A
1000
US
University of Pittsburgh
Neurogenetic Disorders
01/27
01/28
NCT00787865: Diffusion Tensor Imaging (DTI) in Infants With Krabbe Disease

Active, not recruiting
N/A
100
US
University of Pittsburgh, National Institute of Neurological Disorders and Stroke (NINDS)
Krabbe Disease
04/27
04/27
NCT03333200: Longitudinal Study of Neurodegenerative Disorders

Recruiting
N/A
1500
US
Palliative Care, Hematopoetic Stem Cell Transplantation
University of Pittsburgh
MLD, Krabbe Disease, ALD, MPS I, MPS II, MPS III, Vanishing White Matter Disease, GM3 Gangliosidosis, PKAN, Tay-Sachs Disease, NP Deficiency, Osteopetrosis, Alpha-Mannosidosis, Sandhoff Disease, Niemann-Pick Diseases, MPS IV, Gaucher Disease, GAN, GM1 Gangliosidoses, Morquio Disease, S-Adenosylhomocysteine Hydrolase Deficiency, Batten Disease, Pelizaeus-Merzbacher Disease, Leukodystrophy, Lysosomal Storage Diseases, Purine Nucleoside Phosphorylase Deficiency, Multiple Sulfatase Deficiency Disease
01/30
01/35
NCT03047369: The Myelin Disorders Biorepository Project

Recruiting
N/A
12000
US
Children's Hospital of Philadelphia, National Institutes of Health (NIH), National Institute of Neurological Disorders and Stroke (NINDS), National Center for Advancing Translational Sciences (NCATS), Biogen, Eli Lilly and Company, Myrtelle Inc., Orchard Therapeutics Ltd., Passage Bio, Inc., Synaptix Biotherapeutics Ltd., Takeda, Boehringer Ingelheim, Ionis Pharmaceuticals, Inc., Sanofi Winthrop Industrie, Sana Biotechnology, Yaya Foundation for 4H Leukodystrophy, University of Pennsylvania, United MSD Foundation, Foundation to Fight H-ABC, Calliope Joy Foundation, Don't Forget Me Foundation
Leukodystrophy, White Matter Disease, Leukoencephalopathies, 4H Syndrome, Adrenoleukodystrophy, AMN, ALD, ALD Gene Mutation, ALD (Adrenoleukodystrophy), X-linked Adrenoleukodystrophy, X-ALD, Adrenomyeloneuropathy, Aicardi Goutieres Syndrome, AGS, Alexander Disease, Alexanders Leukodystrophy, AxD, ADLD, Canavan Disease, CTX, Cerebrotendinous Xanthomatoses, Krabbe Disease, GALC Deficiency, Globoid Leukodystrophy, TUBB4A-Related Leukodystrophy, H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum, HBSL, HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity, LBSL, Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder), Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation, ALSP, CSF1R Gene Mutation, HCC - Hypomyelination and Congenital Cataract, MLC1, Megalencephalic Leukoencephalopathy With Subcortical Cysts, MLD, Metachromatic Leukodystrophy, PMD, Pelizaeus-Merzbacher Disease, PLP1 Null Syndrome, PLP1 Gene Duplication | Blood or Tissue | Mutations, Pelizaeus Merzbacher Like Disease, Peroxisomal Biogenesis Disorder, Zellweger Syndrome, Refsum Disease, Salla Disease, Sialic Storage Disease, Sjögren, Sjogren-Larsson Syndrome, Van Der Knapp Disease, Vanishing White Matter Disease, Charcot-Marie-Tooth, CMT, Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency, Allan-Herndon-Dudley Syndrome, Cadasil, Cockayne Syndrome, Multiple Sulfatase Deficiency, Gangliosidoses, GM2 Gangliosidosis, BPAN, Labrune Syndrome, LCC, Mucopolysaccharidoses, TBCK-Related Intellectual Disability Syndrome
12/30
12/30
Leslie, Nancy
COMPASS, NCT05371613 / 2021-005200-35: A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) Vs Idursulfase in Pediatric and Young Adult Participants with Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

Recruiting
2/3
54
Europe, Canada, US, RoW
tividenofusp alfa, idursulfase
Denali Therapeutics Inc.
Mucopolysaccharidosis II
12/25
12/25
NCT06075537: An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007

Enrolling by invitation
2/3
99
Europe, Canada, US, RoW
tividenofusp alfa
Denali Therapeutics Inc.
Mucopolysaccharidosis II
06/27
06/27
OR-ARI-EAP-NPC, NCT04316637: Early Access Program With Arimoclomol in US Patients With NPC

Available
N/A
US
Arimoclomol
ZevraDenmark
Niemann-Pick Disease, Type C
 
 
Hillman, Paul
COMPASS, NCT05371613 / 2021-005200-35: A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) Vs Idursulfase in Pediatric and Young Adult Participants with Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

Recruiting
2/3
54
Europe, Canada, US, RoW
tividenofusp alfa, idursulfase
Denali Therapeutics Inc.
Mucopolysaccharidosis II
12/25
12/25
OR-ARI-EAP-NPC, NCT04316637: Early Access Program With Arimoclomol in US Patients With NPC

Available
N/A
US
Arimoclomol
ZevraDenmark
Niemann-Pick Disease, Type C
 
 
Andrews, Saadet
COMPASS, NCT05371613 / 2021-005200-35: A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) Vs Idursulfase in Pediatric and Young Adult Participants with Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

Recruiting
2/3
54
Europe, Canada, US, RoW
tividenofusp alfa, idursulfase
Denali Therapeutics Inc.
Mucopolysaccharidosis II
12/25
12/25
Kolar, Dawn
COMPASS, NCT05371613 / 2021-005200-35: A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) Vs Idursulfase in Pediatric and Young Adult Participants with Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

Recruiting
2/3
54
Europe, Canada, US, RoW
tividenofusp alfa, idursulfase
Denali Therapeutics Inc.
Mucopolysaccharidosis II
12/25
12/25
NCT04251026 / 2019-004909-27: A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome

Active, not recruiting
1/2
47
Europe, Canada, US
tividenofusp alfa
Denali Therapeutics Inc.
Mucopolysaccharidosis II
07/27
07/27
PROVIDE, NCT04411654: Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease

Active, not recruiting
1/2
15
Europe, US
LY3884961, Methylprednisolone, Sirolimus, Prednisone
Prevail Therapeutics, Eli Lilly and Company
Gaucher Disease, Type 2
05/28
05/28
NCT04007536: A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome

Completed
N/A
18
Europe, US
No Intervention
Denali Therapeutics Inc.
Mucopolysaccharidosis II
03/24
03/24
Daumeyer, Henry
COMPASS, NCT05371613 / 2021-005200-35: A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) Vs Idursulfase in Pediatric and Young Adult Participants with Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

Recruiting
2/3
54
Europe, Canada, US, RoW
tividenofusp alfa, idursulfase
Denali Therapeutics Inc.
Mucopolysaccharidosis II
12/25
12/25
Garcia, Marilyn
COMPASS, NCT05371613 / 2021-005200-35: A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) Vs Idursulfase in Pediatric and Young Adult Participants with Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

Recruiting
2/3
54
Europe, Canada, US, RoW
tividenofusp alfa, idursulfase
Denali Therapeutics Inc.
Mucopolysaccharidosis II
12/25
12/25
HERO, NCT04732429 / 2023-000785-33: Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia

Terminated
2
26
US, RoW
HST5040, Placebo
HemoShear Therapeutics, HemoShear Therapeutics
Methylmalonic Acidemia, Propionic Acidemia
10/23
10/23
OR-ARI-EAP-NPC, NCT04316637: Early Access Program With Arimoclomol in US Patients With NPC

Available
N/A
US
Arimoclomol
ZevraDenmark
Niemann-Pick Disease, Type C
 
 
Hassan, Farah
COMPASS, NCT05371613 / 2021-005200-35: A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) Vs Idursulfase in Pediatric and Young Adult Participants with Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

Recruiting
2/3
54
Europe, Canada, US, RoW
tividenofusp alfa, idursulfase
Denali Therapeutics Inc.
Mucopolysaccharidosis II
12/25
12/25

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