Identifying and Genotyping Homozygous Familial Hypercholesterolemia (HoFH) Patients
- Conditions
- Homozygous Familial Hypercholesterolemia (HoFH)
- First Posted Date
- 2019-11-01
- Last Posted Date
- 2021-02-02
- Lead Sponsor
- REGENXBIO Inc.
- Target Recruit Count
- 4
- Registration Number
- NCT04148001
- Locations
- 🇺🇸
Excel Medical Clinical Trials, LLC, Boca Raton, Florida, United States
A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501
- Conditions
- Homozygous Familial Hypercholesterolemia (HoFH)
- Interventions
- Drug: Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
- First Posted Date
- 2019-09-06
- Last Posted Date
- 2023-03-09
- Lead Sponsor
- REGENXBIO Inc.
- Target Recruit Count
- 8
- Registration Number
- NCT04080050
- Locations
- 🇺🇸
Columbus Location, Columbus, Ohio, United States
🇺🇸Philadelphia location, Philadelphia, Pennsylvania, United States
🇨🇦Montreal location, Montreal, Quebec, Canada
RGX-111 Gene Therapy in Patients With MPS I
- Conditions
- Hurler-Scheie SyndromeMucopolysaccharidosis Type I (MPS I)Hurler Syndrome
- Interventions
- Genetic: RGX-111
- First Posted Date
- 2018-07-09
- Last Posted Date
- 2023-12-06
- Lead Sponsor
- REGENXBIO Inc.
- Target Recruit Count
- 8
- Registration Number
- NCT03580083
- Locations
- 🇧🇷
Hospital de Clinicas de Porto Alegre, Porto Alegre, RS, Brazil
🇮🇱Sheba Medical Center, Tel HaShomer, Israel
🇺🇸Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
CAMPSIITEâ„¢ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)
- Conditions
- Mucopolysaccharidosis Type II (MPS II)
- Interventions
- Genetic: RGX-121
- First Posted Date
- 2018-06-21
- Last Posted Date
- 2025-01-28
- Lead Sponsor
- REGENXBIO Inc.
- Target Recruit Count
- 48
- Registration Number
- NCT03566043
- Locations
- 🇺🇸
Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
🇺🇸University of California San Francisco, Benioff Children's Hospital, Oakland, California, United States
🇧🇷Hospital de Clinicas de Porto Alegre, Porto Alegre, RS, Brazil
Mucopolysaccharidosis Type II Natural History
- Conditions
- Mucopolysaccharidosis II
- First Posted Date
- 2018-05-18
- Last Posted Date
- 2022-04-26
- Lead Sponsor
- REGENXBIO Inc.
- Target Recruit Count
- 36
- Registration Number
- NCT03529786
- Locations
- 🇧🇷
Hospital de Clinicas de Porto Alegre, Porto Alegre, RS, Brazil
🇬🇧Manchester Centre for Genomic Medicine, Manchester, United Kingdom
🇺🇸Children's Hospital of Pittsburgh - UPMC: Program for Neurodevelopment in Rare Disorders, Pittsburgh, Pennsylvania, United States
Safety and Tolerability of RGX-314 (Investigational Product) Gene Therapy for Neovascular AMD Trial
- Conditions
- Neovascular Age-related Macular DegenerationWet Age-related Macular Degeneration
- Interventions
- Genetic: RGX-314
- First Posted Date
- 2017-02-28
- Last Posted Date
- 2023-05-16
- Lead Sponsor
- REGENXBIO Inc.
- Target Recruit Count
- 42
- Registration Number
- NCT03066258
- Locations
- 🇺🇸
Reno location, Reno, Nevada, United States
🇺🇸Baltimore location, Baltimore, Maryland, United States
🇺🇸Santa Barbara location, Santa Barbara, California, United States
A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)
- Conditions
- Homozygous Familial Hypercholesterolemia (HoFH)
- Interventions
- Genetic: AAV directed hLDLR gene therapy
- First Posted Date
- 2016-01-11
- Last Posted Date
- 2023-07-13
- Lead Sponsor
- REGENXBIO Inc.
- Target Recruit Count
- 9
- Registration Number
- NCT02651675
- Locations
- 🇺🇸
Kansas City Location, Kansas City, Kansas, United States
🇮🇹Palermo location, Palermo, PA, Italy
🇺🇸Portland location, Portland, Oregon, United States