Long-term Follow-Up for RGX-121
NCT04597385 · Status: UNKNOWN · Type: OBSERVATIONAL · Enrollment: 12
Last updated 2021-08-10
Summary
RGX-121-5101 is the long-term follow-up study to the RGX-121-101 first in human study where participants received RGX-121, a gene therapy intended to deliver a functional copy of the iduronate-2-sulfatase gene (IDS) to the central nervous system. This study will evaluate the long-term safety and efficacy of RGX-121.
Conditions
- Mucopolysaccharidosis II
Interventions
- OTHER
-
Long-term Follow-Up
No intervention
Sponsors & Collaborators
- lead INDUSTRY
Eligibility
- Min Age
- 28 Months
- Sex
- MALE
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2021-03-14
- Primary Completion
- 2025-09-30
- Completion
- 2025-09-30
Countries
- United States
Study Locations
More Related Trials
-
A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ
NCT05422482 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1
-
A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
NCT05967351 ·Status: ENROLLING_BY_INVITATION ·Phase: PHASE3
-
Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer
NCT04273243 ·Status: ACTIVE_NOT_RECRUITING
-
Phase 1/2 Study of FRF-001, an AAV-9 Gene Therapy, in Patients With FOXG1 Syndrome (FS)
NCT07293546 ·Status: NOT_YET_RECRUITING ·Phase: PHASE1/PHASE2
-
Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS I
NCT03071341 ·Status: COMPLETED ·Phase: PHASE1/PHASE2
-
Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Syndrome
NCT02262338 ·Status: COMPLETED ·Phase: PHASE1
-
A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)
NCT02432144 ·Status: COMPLETED ·Phase: PHASE3
-
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II
NCT03041324 ·Status: TERMINATED ·Phase: PHASE1/PHASE2
-
Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181
NCT02597114 ·Status: COMPLETED ·Phase: PHASE1
-
Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)
NCT03612869 ·Status: UNKNOWN ·Phase: PHASE2/PHASE3
-
Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome)
NCT00004454 ·Status: COMPLETED ·Phase: PHASE1/PHASE2
-
RG2133 (2',3',5'-Tri-O-Acetyluridine) in Mitochondrial Disease
NCT00060515 ·Status: TERMINATED ·Phase: PHASE1
-
A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria
NCT07241234 ·Status: RECRUITING ·Phase: PHASE1
-
Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients
NCT03614234 ·Status: COMPLETED ·Phase: PHASE3
-
An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)
NCT01856218 ·Status: COMPLETED ·Phase: PHASE1/PHASE2
-
A Long-term, 3-Year, Follow-up Study for Patients Completing the KS-GIG-001-01 Study
NCT05613569 ·Status: ACTIVE_NOT_RECRUITING
-
An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient
NCT02097251 ·Status: NO_LONGER_AVAILABLE
-
Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type II
NCT05665166 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2
-
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74)
NCT06241950 ·Status: TERMINATED ·Phase: PHASE1
-
Study of rhASB in Patients With Mucopolysaccharidosis VI
NCT00104234 ·Status: COMPLETED ·Phase: PHASE3
-
Extension Study of PRX-102 for up to 60 Months
NCT01981720 ·Status: COMPLETED ·Phase: PHASE1/PHASE2
-
Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks
NCT03180840 ·Status: COMPLETED ·Phase: PHASE3
-
A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)
NCT02230566 ·Status: COMPLETED ·Phase: PHASE3
-
Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I
NCT02371226 ·Status: COMPLETED ·Phase: PHASE1
-
Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age
NCT02418455 ·Status: COMPLETED ·Phase: PHASE2