UCSF Benioff Children's Hospital
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 24 Trials 
39 Trials

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Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Walters, Mark
GRASP, NCT05353647: A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (, BMT CTN 2001)

Active, not recruiting
2
25
US
Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a
David Williams, National Heart, Lung, and Blood Institute (NHLBI), California Institute for Regenerative Medicine (CIRM), bluebird bio, Blood and Marrow Transplant Clinical Trials Network
Sickle Cell Disease
05/26
05/26
Kohn, Donald B
RP-L201-0318, NCT03812263 / 2020-000517-33: A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I

Completed
1/2
9
Europe, US
RP-L201
Rocket Pharmaceuticals Inc., California Institute for Regenerative Medicine (CIRM)
Leukocyte Adhesion Defect - Type I
09/23
09/23
NCT06282432: Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I)

Enrolling by invitation
N/A
9
Europe, US
Rocket Pharmaceuticals Inc.
Leukocyte Adhesion Deficiency
03/37
03/37
Harmatz, Paul
NCT05494593: A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II)

Recruiting
4
5
US
ELAPRASE, Idursulfase, Rituximab, Methotrexate, Intravenous Immunoglobulin (IVIG)
Takeda, Takeda Development Center Americas, Inc.
Mucopolysaccharidosis (MPS), Hunter Syndrome
07/28
07/28
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
NCT05594992: An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in Mucopolysaccharidosis Type II (Hunter Syndrome) Subjects

Enrolling by invitation
3
80
Europe, US, RoW
JR-141
JCR Pharmaceuticals Co., Ltd.
Mucopolysaccharidosis II
02/28
02/28
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
PROPEL 2, NCT04265651 / 2019-002954-21: Study of Infigratinib in Children with Achondroplasia

Active, not recruiting
2
84
Europe, Canada, US, RoW
Infigratinib 0.016 mg/kg, Infigratinib 0.032 mg/kg, Infigratinib 0.064 mg/kg, Infigratinib 0.128 mg/kg, Infigratinib 0.25 mg/kg
QED Therapeutics, Inc.
Achondroplasia
10/24
12/24
NCT04571970: RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

Completed
1/2
6
Canada, US
RGX-121
REGENXBIO Inc.
Mucopolysaccharidosis Type II (MPS II)
05/23
05/24
NCT06181136: Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)

Recruiting
1/2
20
US
DNL126
Denali Therapeutics Inc.
Mucopolysaccharidosis Type IIIA
08/28
08/28
NCT04783181: A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)

Active, not recruiting
1/2
8
US
AAV BBP-631
Adrenas Therapeutics Inc
Congenital Adrenal Hyperplasia
02/29
02/29
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
NCT05682144: ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I

Recruiting
1
2
US
Autologous Plasmablasts (B cells)
Immusoft of CA, Inc.
Mucopolysaccharidosis IH/S, Mucopolysaccharidosis IS
06/25
06/39
NCT05238324: Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II

Withdrawn
1
9
Canada, US
Genetic HMI-203
Homology Medicines, Inc
Mucopolysaccharidosis II
10/24
01/29
NCT06567769: Phase 1 Study of GC1130A in Pediatric Patients with Sanfilippo Syndrome Type a (MPS IIIA)

Recruiting
1
9
US, RoW
GC1130A
GC Biopharma Corp, Novel Pharma Inc.
Sanfilippo Syndrome Type a
06/27
06/27
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
 
 
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
PRONTO, NCT05109793: GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study

Active, not recruiting
N/A
31
Europe, US, RoW
Azafaros A.G.
GM1 Gangliosidosis, Sandhoff Disease, Tay-Sachs Disease
05/26
05/26
PROPEL, NCT04035811: Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)

Recruiting
N/A
250
Europe, Canada, US, RoW
QED Therapeutics, Inc.
Achondroplasia
06/26
06/26
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
Vichinsky, Elliot P
THRIVE-131, NCT04935879: A Study to Assess the Safety and Efficacy of Inclacumab in Participants With Sickle Cell Disease Experiencing Vaso-occlusive Crises

Completed
3
243
Europe, US, RoW
Inclacumab, Placebo
Pfizer
Sickle Cell Disease, Vaso-occlusive Pain Episode in Sickle Cell Disease, Vaso-occlusive Crisis
06/24
06/24
Moriarty, Marci
003SCD101, NCT03653247: A Study to Assess the Safety, Tolerability, and Efficacy of BIVV003 for Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Sickle Cell Disease

Checkmark Initial data from PRECIZN-1 trial for autologous hematopoietic stem cell transplantation in patients with severe sickle cell disease at ASH 2021
Nov 2021 - Nov 2021: Initial data from PRECIZN-1 trial for autologous hematopoietic stem cell transplantation in patients with severe sickle cell disease at ASH 2021
Checkmark Initial data from PRECIZN-1 trial for sickle cell disease
Mar 2021 - Mar 2021: Initial data from PRECIZN-1 trial for sickle cell disease
Active, not recruiting
1/2
8
US
Plerixafor, Busulfan, BIVV003, Autologous CD34 + hematopoietic stem cells
Sangamo Therapeutics
Sickle Cell Disease
07/25
07/25
Decaris, Julie
THRIVE-132, NCT04927247: A Study of a Single Dose of Inclacumab to Reduce Re-admission in Participants With Sickle Cell Disease and Recurrent Vaso-occlusive Crises

Terminated
3
72
Europe, US, RoW
Inclacumab, Placebo
Pfizer
Sickle Cell Disease, Vaso-occlusive Crisis, Vaso-occlusive Pain Episode in Sickle Cell Disease
11/23
11/23
THRIVE-131, NCT04935879: A Study to Assess the Safety and Efficacy of Inclacumab in Participants With Sickle Cell Disease Experiencing Vaso-occlusive Crises

Completed
3
243
Europe, US, RoW
Inclacumab, Placebo
Pfizer
Sickle Cell Disease, Vaso-occlusive Pain Episode in Sickle Cell Disease, Vaso-occlusive Crisis
06/24
06/24
HIBISCUS, NCT04624659 / 2020-003884-25: A Study of Etavopivat in Adults and Adolescents With Sickle Cell Disease

Recruiting
2/3
344
Europe, Canada, US, RoW
Etavopivat Tablets Low dose, FT-4202, Etavopivat Tablets High dose, Placebo Tablets, placebo, Etavopivat Tablets
Forma Therapeutics, Inc., Novo Nordisk A/S
Sickle Cell Disease
03/26
04/27
Phan, Huong
NCT03692897: An Observational Study of Patients With Chronic Hepatitis B (CHB) Infection

Recruiting
N/A
5000
Canada, US
All approved therapies for the treatment of Chronic Hepatitis B (CHB)
Target PharmaSolutions, Inc.
Hepatitis B, Hepatitis, Liver Diseases
01/26
01/26
Bascon, Cyrus
Imagine-1, NCT04713475 / 2020-001109-22: Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis

Active, not recruiting
1/2
26
Europe, Canada, US, RoW
PBGM01
Passage Bio, Inc.
GM1 Gangliosidosis, GM1 Gangliosidosis, Type I, GM1 Gangliosidosis, Type 2, Beta-Galactosidase-1 (GLB1) Deficiency
02/26
02/29
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
Mahuvakar, Shivani
STEALTH, NCT05432986: Assessment of CSF Shunt Flow With Thermal Measurements B

Completed
N/A
141
US
Thermal Anisotropy Measurement Device, FlowSense
Rhaeos, Inc.
Hydrocephalus
11/23
11/23
Lynch, Leslie
PROPEL 2, NCT04265651 / 2019-002954-21: Study of Infigratinib in Children with Achondroplasia

Active, not recruiting
2
84
Europe, Canada, US, RoW
Infigratinib 0.016 mg/kg, Infigratinib 0.032 mg/kg, Infigratinib 0.064 mg/kg, Infigratinib 0.128 mg/kg, Infigratinib 0.25 mg/kg
QED Therapeutics, Inc.
Achondroplasia
10/24
12/24
PROPEL, NCT04035811: Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)

Recruiting
N/A
250
Europe, Canada, US, RoW
QED Therapeutics, Inc.
Achondroplasia
06/26
06/26
Roth, Danielle
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
Sako, Annie
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
CANaspire, NCT04998396: A Study of AAV9 Gene Therapy in Participants With Canavan Disease ( Clinical Trial)

Recruiting
1/2
26
US
AAV9 BBP-812
Aspa Therapeutics
Canavan Disease
10/26
10/30
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
Fields, Jenna
NCT05238324: Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II

Withdrawn
1
9
Canada, US
Genetic HMI-203
Homology Medicines, Inc
Mucopolysaccharidosis II
10/24
01/29
NCT06567769: Phase 1 Study of GC1130A in Pediatric Patients with Sanfilippo Syndrome Type a (MPS IIIA)

Recruiting
1
9
US, RoW
GC1130A
GC Biopharma Corp, Novel Pharma Inc.
Sanfilippo Syndrome Type a
06/27
06/27
Elzie-Tuttle, Nicole
No trials found

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