University of California San Francisco, Benioff Children's Hospital
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 13 Trials 
21 Trials

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Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
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
Nicholas, Jill
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
Thura, Matt
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
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

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