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ClinicalTrials.gov public records Last synced Jul 23, 2026, 8:54 AM EDT

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Showing 1–21 of 21 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Conditions
Diabetic Macular Edema
Interventions
RGX-314 Dose 1, RGX-314 Dose 2, Aflibercept (2.0 mg)
Genetic · Biological
Lead sponsor
Sierra Eye Associates
Other
Eligibility
25 Years to 89 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Reno, Nevada
Conditions
Mucopolysaccharidosis Type II (MPS II)
Interventions
RGX-121
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
5 Years to 17 Years · Male only
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
1
States / cities
Oakland, California
Conditions
Duchenne Muscular Dystrophy
Interventions
AAV8 DetectCDx
Diagnostic Test
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
0 Years to 25 Years · Male only
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Atlanta, Georgia
Conditions
AMD, nAMD, Wet Age-related Macular Degeneration, wAMD, Wet AMD, CNV, Neovascular AMD, Neovascular Age-related Macular Degeneration, Choroidal Neovascularization
Interventions
ABBV-RGX-314, Ranibizumab (LUCENTIS®)
Genetic · Biological
Lead sponsor
AbbVie
Industry
Eligibility
50 Years to 89 Years
Enrollment
671 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
89
States / cities
Phoenix, Arizona • Sun City, Arizona • Little Rock, Arkansas + 77 more
Conditions
Mucopolysaccharidosis Type II (MPS II)
Interventions
RGX-121
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
4 Months to 5 Years · Male only
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
4
States / cities
Oakland, California • New Brunswick, New Jersey • Philadelphia, Pennsylvania + 1 more
Conditions
Homozygous Familial Hypercholesterolemia (HoFH)
Interventions
AAV directed hLDLR gene therapy
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
5
States / cities
Boca Raton, Florida • Kansas City, Kansas • Portland, Oregon + 2 more
Conditions
Mucopolysaccharidosis II
Interventions
Observational
Other
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
1 Month to 8 Years · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
2
States / cities
Oakland, California • Philadelphia, Pennsylvania
Conditions
Homozygous Familial Hypercholesterolemia (HoFH)
Interventions
Not listed
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
1
States / cities
Boca Raton, Florida
Conditions
Mucopolysaccharidosis II
Interventions
Not listed
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
Male only
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Conditions
MPS II, Hunter Syndrome (MPS II)
Interventions
RGX-121-3102
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
4 Months to 5 Years · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2031
U.S. locations
1
States / cities
New Brunswick, New Jersey
Conditions
Diabetic Retinopathy (DR), Center-Involved Diabetic Macular Edema (CI-DME)
Interventions
ABBV-RGX-314 Dose 1, ABBV-RGX-314 Dose 2, ABBV-RGX-314 Dose 3, Topical Steroid, ABBV-RGX-314 Dose 4, Aflibercept
Genetic · Drug · Biological
Lead sponsor
AbbVie
Industry
Eligibility
25 Years to 89 Years
Enrollment
139 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
25
States / cities
Phoenix, Arizona • Bakersfield, California • Beverly Hills, California + 21 more
Temporarily Not Available No phase listed Expanded access

Expanded Access to RGX-202

NCT07652606
Conditions
Not listed
Interventions
RGX-202
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
Not listed
U.S. locations
1
States / cities
Rockville, Maryland
Conditions
Duchenne Muscular Dystrophy
Interventions
RGX-202
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
1 Year and older · Male only
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
19
States / cities
Little Rock, Arkansas • Orange, California • Palo Alto, California + 16 more
Conditions
AMD, nAMD, Wet Age-related Macular Degeneration, wAMD, WetAMD, CNV
Interventions
ABBV-RGX-314 Dose 1, ABBV-RGX-314 Dose 2, Aflibercept (EYLEA®)
Genetic · Biological
Lead sponsor
AbbVie
Industry
Eligibility
50 Years to 89 Years
Enrollment
714 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
107
States / cities
Phoenix, Arizona • Scottsdale, Arizona • Bakersfield, California + 94 more
Conditions
Homozygous Familial Hypercholesterolemia (HoFH)
Interventions
Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
Drug
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
4
States / cities
Columbus, Ohio • Portland, Oregon • Philadelphia, Pennsylvania + 1 more
Conditions
Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome, Hurler-Scheie Syndrome
Interventions
RGX-111
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
4 Months and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2029
U.S. locations
1
States / cities
Orange, California
Conditions
Neovascular Age-Related Macular Degeneration (nAMD)
Interventions
Ranibizumab, ABBV-RGX-314 Dose 1, ABBV-RGX-314 Dose 2, ABBV-RGX-314 Dose 3, Local Steroid, Topical Steroid, ABBV-RGX-314 Dose 4
Biological · Genetic · Drug
Lead sponsor
AbbVie
Industry
Eligibility
50 Years to 89 Years
Enrollment
146 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
18
States / cities
Phoenix, Arizona • Bakersfield, California • Beverly Hills, California + 15 more
Not listed No phase listed Observational

Long-term Follow-Up for RGX-121

NCT04597385
Conditions
Mucopolysaccharidosis II
Interventions
Long-term Follow-Up
Other
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
28 Months and older · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Conditions
Neovascular Age-related Macular Degeneration, Wet Age-related Macular Degeneration
Interventions
RGX-314
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
50 Years to 89 Years
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
8
States / cities
Santa Barbara, California • Baltimore, Maryland • Boston, Massachusetts + 4 more
Enrolling by invitation No phase listed Observational

Long Term Follow-up for RGX-202

NCT06491927
Conditions
Duchenne Muscular Dystrophy
Interventions
No Intervention
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
Male only
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
5
States / cities
Little Rock, Arkansas • Palo Alto, California • Chicago, Illinois + 2 more
Enrolling by invitation No phase listed Observational

Long Term Follow-Up for RGX-111

NCT06103487
Conditions
Mucopolysaccharidosis I
Interventions
No Intervention
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
Not listed
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
Orange, California