Long Term Follow-up for RGX-202
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Genetic: No Intervention
- First Posted Date
- 2024-07-09
- Last Posted Date
- 2025-05-13
- Lead Sponsor
- REGENXBIO Inc.
- Target Recruit Count
- 19
- Registration Number
- NCT06491927
- Locations
- 🇺🇸
Arkansas Children's Hospital, Little Rock, Arkansas, United States
🇺🇸Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States
Long Term Follow-Up for RGX-111
- Conditions
- Mucopolysaccharidosis I
- Interventions
- Genetic: No Intervention
- First Posted Date
- 2023-10-26
- Last Posted Date
- 2024-07-03
- Lead Sponsor
- REGENXBIO Inc.
- Target Recruit Count
- 7
- Registration Number
- NCT06103487
- Locations
- 🇺🇸
Children's Hospital of Orange County, Orange, California, United States
🇧🇷Hospital de Clinicas de Porto Alegre, Porto Alegre, RS, Brazil
A First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Tolerability of Gene Therapy with TTX-381 for the Ocular Manifestations Associated with Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease
- Conditions
- Neuronal Ceroid Lipofuscinosis Type 2
- Interventions
- Genetic: RGX-381
- First Posted Date
- 2023-03-30
- Last Posted Date
- 2025-02-13
- Lead Sponsor
- Tern Therapeutics, LLC
- Target Recruit Count
- 16
- Registration Number
- NCT05791864
- Locations
- 🇬🇧
Greater Ormond Street Hospital, London, United Kingdom
AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Genetic: RGX-202
- First Posted Date
- 2023-01-20
- Last Posted Date
- 2025-05-13
- Lead Sponsor
- REGENXBIO Inc.
- Target Recruit Count
- 65
- Registration Number
- NCT05693142
- Locations
- 🇺🇸
Arkansas Children's Hospital, Little Rock, Arkansas, United States
🇺🇸Stanford School of Medicine /Division of Neuromuscular Medicine, Palo Alto, California, United States
🇺🇸Children's Hospital Colorado, Aurora, Colorado, United States
AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Boys With DMD
- Conditions
- Duchenne Muscular Dystrophy
- First Posted Date
- 2023-01-13
- Last Posted Date
- 2025-04-06
- Lead Sponsor
- REGENXBIO Inc.
- Target Recruit Count
- 200
- Registration Number
- NCT05683379
- Locations
- 🇺🇸
Rare Disease Research, Atlanta, Georgia, United States
Long-term Follow-Up for RGX-121
- Conditions
- Mucopolysaccharidosis II
- Interventions
- Other: Long-term Follow-Up
- First Posted Date
- 2020-10-22
- Last Posted Date
- 2021-08-10
- Lead Sponsor
- REGENXBIO Inc.
- Target Recruit Count
- 12
- Registration Number
- NCT04597385
- Locations
- 🇺🇸
Children's Hospital of Pittsburgh - UPMC: Program for Neurodevelopment in Rare Disorders, Pittsburgh, Pennsylvania, United States
Mucopolysaccharidosis Type II Observational
- Conditions
- Mucopolysaccharidosis II
- Interventions
- Other: Observational
- First Posted Date
- 2020-10-19
- Last Posted Date
- 2022-10-10
- Lead Sponsor
- REGENXBIO Inc.
- Registration Number
- NCT04591834
- Locations
- 🇺🇸
Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
🇺🇸University of California San Francisco, Benioff Children's Hospital, Oakland, California, United States
🇨🇦McGill University Health Center, Montréal, Quebec, Canada
RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)
- Conditions
- Mucopolysaccharidosis Type II (MPS II)
- Interventions
- Genetic: RGX-121
- First Posted Date
- 2020-10-01
- Last Posted Date
- 2025-01-28
- Lead Sponsor
- REGENXBIO Inc.
- Target Recruit Count
- 6
- Registration Number
- NCT04571970
- Locations
- 🇨🇦
McGill University Heath Center, Montréal, Quebec, Canada
🇺🇸University of California San Francisco, Benioff Children's Hospital, Oakland, California, United States
A Retrospective, Natural History Study in Children With CLN2
- Conditions
- Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)
- First Posted Date
- 2020-07-21
- Last Posted Date
- 2021-11-01
- Lead Sponsor
- REGENXBIO Inc.
- Registration Number
- NCT04480476
An Observational Study in Children With CLN2 Batten Disease
- Conditions
- Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)
- First Posted Date
- 2020-07-08
- Last Posted Date
- 2021-11-01
- Lead Sponsor
- REGENXBIO Inc.
- Registration Number
- NCT04462692