9 active not recruiting, 4 completed, 4 enrolling by invitation, 3 withdrawn. 12 Phase 2, 5 Phase 3, 4 Phase 1.
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Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome) RGX-121-3102 - MPS II, Hunter Syndrome (MPS II) | Phase 3 | Active not recruiting | REGENXBIO Inc. | 2 | 2025-11-25 | 2026-06-04 |
| Phase 2 Trial of RGX-314 in Adults With Center Involved - Diabetic Macular Edema (CI - DME) RGX-314 Dose 1, RGX-314 Dose 2, Aflibercept (2.0 mg) - Diabetic Macular Edema | Phase 2 | Recruiting | Sierra Eye Associates | 18 | 2025-03-18 | 2025-04-24 |
| Long Term Follow-up for RGX-202 No Intervention - Duchenne Muscular Dystrophy | Enrolling by invitation | REGENXBIO Inc. | 66 | 2024-05-08 | 2026-03-24 | |
| Long Term Follow-Up for RGX-111 No Intervention - Mucopolysaccharidosis I | Enrolling by invitation | REGENXBIO Inc. | 21 | 2023-07-24 | 2026-04-29 | |
| AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD) RGX-202 - Duchenne Muscular Dystrophy | Phase 2 / Phase 3 | Active not recruiting | REGENXBIO Inc. | 65 | 2023-01-04 | 2026-07-21 |
| AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMD AAV8 DetectCDx - Duchenne Muscular Dystrophy | Active not recruiting | REGENXBIO Inc. | 200 | 2022-12-20 | 2026-07-07 | |
| Long-Term Follow-Up Study of RGX-314 Administered in the Suprachoroidal Space for Participants With and Without Diabetic Retinopathy Standard of Care, Aflibercept - Diabetic Retinopathy, DR | Phase 2 | Enrolling by invitation | AbbVie | 100 | 2022-04-04 | 2026-08-05 |
| Mucopolysaccharidosis Type II Observational Observational - Mucopolysaccharidosis II | Withdrawn | REGENXBIO Inc. | 2022-03-01 | 2022-10-10 | ||
| Pivotal 2 Study of RGX-314 Gene Therapy in Participants With nAMD ABBV-RGX-314 Dose 1, ABBV-RGX-314 Dose 2, Aflibercept (EYLEA®) - AMD, nAMD | Phase 3 | Active not recruiting | AbbVie | 735 | 2022-01-13 | 2026-08-03 |
| Long-Term Follow-Up Study of RGX-314 Administered in the Suprachoroidal Space for Participants With nAMD Standard of Care, Ranibizumab - Neovascular Age-Related Macular Degeneration (nAMD) | Phase 2 | Enrolling by invitation | AbbVie | 116 | 2021-12-20 | 2026-08-05 |
| An Observational Study in Children With CLN2 Batten Disease Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) | Withdrawn | REGENXBIO Inc. | 2021-03-31 | 2021-11-01 | ||
| A Retrospective, Natural History Study in Children With CLN2 Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) | Withdrawn | REGENXBIO Inc. | 2021-03-31 | 2021-11-01 | ||
| Long-term Follow-Up for RGX-121 Long-term Follow-Up - Mucopolysaccharidosis II | Active not recruiting | REGENXBIO Inc. | 27 | 2021-03-14 | 2026-08-19 | |
| RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome) RGX-121 - Mucopolysaccharidosis Type II (MPS II) | Phase 1 / Phase 2 | Completed | REGENXBIO Inc. | 6 | 2021-03-11 | 2025-01-28 |
| Pivotal 1 Study of ABBV-RGX-314 (Also Known as RGX-314) Gene Therapy Administered Via Subretinal Delivery One Time in Participants With nAMD ABBV-RGX-314, Ranibizumab (LUCENTIS®) - AMD, nAMD | Phase 2 / Phase 3 | Active not recruiting | AbbVie | 671 | 2020-12-29 | 2026-04-28 |
| RGX-314 Gene Therapy Administered in the Suprachoroidal Space for Participants With Diabetic Retinopathy (DR) With and Without Center Involved-Diabetic Macular Edema (CI-DME) ABBV-RGX-314 Dose 1, ABBV-RGX-314 Dose 2, ABBV-RGX-314 Dose 3 - Diabetic Retinopathy (DR), Center-Involved Diabetic Macular Edema (CI-DME) | Phase 2 | Active not recruiting | AbbVie | 139 | 2020-11-20 | 2026-08-19 |
| RGX-314 Gene Therapy Administered in the Suprachoroidal Space for Participants With Neovascular Age-Related Macular Degeneration (nAMD) Ranibizumab, ABBV-RGX-314 Dose 1, ABBV-RGX-314 Dose 2 - Neovascular Age-Related Macular Degeneration (nAMD) | Phase 2 | Active not recruiting | AbbVie | 146 | 2020-08-25 | 2026-02-27 |
| Identifying and Genotyping Homozygous Familial Hypercholesterolemia (HoFH) Patients Homozygous Familial Hypercholesterolemia (HoFH) | Completed | REGENXBIO Inc. | 4 | 2019-12-04 | 2021-02-02 | |
| A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501 Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy - Homozygous Familial Hypercholesterolemia (HoFH) | Unknown | REGENXBIO Inc. | 8 | 2019-09-30 | 2023-03-09 | |
| RGX-111 Gene Therapy in Patients With MPS I RGX-111 - Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome | Phase 1 / Phase 2 | Suspended | REGENXBIO Inc. | 21 | 2019-04-03 | 2026-03-12 |
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Mucopolysaccharidosis II (6) · Sudden Infant Death (6) · Atherosclerosis (3) · Cardiovascular Diseases (3) · Genetic Diseases, Inborn (3) · Homozygous Familial Hypercholesterolemia (3) · Hyperlipoproteinemia Type II (3) · Metabolic Diseases (3) · Muscular Dystrophy, Duchenne (3) · Rare Diseases (3) · Choroidal Neovascularization (2) · Diabetic Retinopathy (2) · Eye Diseases (2) · Macular Degeneration (2) · Mucopolysaccharidosis I (2)
aflibercept (4) · Ranibizumab (3) · Standard of Care (2) · Steroids (2) · Genetic Therapy (1) · Piperacillin, Tazobactam Drug Combination (1) · Watchful Waiting (1)
United States (23) · Canada (6) · Brazil (5) · Italy (2) · Netherlands (2) · United Kingdom (2) · France (1) · Germany (1) · Hungary (1) · Japan (1) · Puerto Rico (1) · Spain (1)
Alexander Schramm (1) · Jeffrey Heier, MD (1)
P01HL059407 (1)