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
- ๐บ๐ธ
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
- ๐บ๐ธ
Columbus Location, Columbus, Ohio, United States
๐บ๐ธPhiladelphia location, Philadelphia, Pennsylvania, United States
๐จ๐ฆMontreal 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
- ๐ง๐ท
Hospital de Clinicas de Porto Alegre, Porto Alegre, RS, Brazil
๐ฎ๐ฑSheba Medical Center, Tel HaShomer, Israel
๐บ๐ธChildren's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
CAMPSIITEโข 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
- 2023-11-18
- Lead Sponsor
- REGENXBIO Inc.
- Target Recruit Count
- 48
- Registration Number
- NCT03566043
- Locations
- ๐บ๐ธ
Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
๐บ๐ธUniversity of California San Francisco, Benioff Children's Hospital, Oakland, California, United States
๐ง๐ทHospital 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
- ๐ง๐ท
Hospital de Clinicas de Porto Alegre, Porto Alegre, RS, Brazil
๐ฌ๐งManchester Centre for Genomic Medicine, Manchester, United Kingdom
๐บ๐ธChildren'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
- ๐บ๐ธ
Reno location, Reno, Nevada, United States
๐บ๐ธBaltimore location, Baltimore, Maryland, United States
๐บ๐ธSanta 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
- ๐บ๐ธ
Kansas City Location, Kansas City, Kansas, United States
๐ฎ๐นPalermo location, Palermo, PA, Italy
๐บ๐ธPortland location, Portland, Oregon, United States