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Clinical trials
catalyst calendar across sponsors →Studies where RGNX is the lead sponsor, as filed with ClinicalTrials.gov. Completion dates are the sponsor's own projected windows and are revised as a study runs. Active studies first, then completed and stopped — newest readout first within each.
7 interventional · 10 observational · 1 expanded access · 2 with posted results
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| Study | Phase | Status | Interventions | Conditions | Enrollment | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|---|
| Long Term Follow-up for RGX-202NCT06491927observationalEvaluation of long-term safety of RGX-202 | — | Enrolling by invitation | Genetic: No Intervention | Duchenne Muscular Dystrophy | 66 | 2029-12-01 | in 1,200 d | 2026-03-24 |
| Long-term Follow-Up for RGX-121NCT04597385observationalLong-term Safety of RGX-121 | — | Active, not recruiting | Other: Long-term Follow-Up | Mucopolysaccharidosis II | 27 | Oct 2028 | ≤ 804 d | 2026-08-19 |
| RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)NCT07236606Efficacy of RGX-121 on Neurodevelopmental Function (as measured by the Bayley Scales of Infant and Toddler Development, 3rd Edition)Open-label · Treatment | Phase 3 | Active, not recruiting | Genetic: RGX-121-3102 | MPS II, Hunter Syndrome (MPS II) | 2 | 2028-04-30 | in 620 d | 2026-06-04 |
| Long Term Follow-Up for RGX-111NCT06103487observationalEvaluation of the long-term safety of RGX-111 | — | Enrolling by invitation | Genetic: No Intervention | Mucopolysaccharidosis I | 21 | Sep 2027 | ≤ 407 d | 2026-04-29 |
| AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)NCT05693142Part 1 Safety measured by incidence of Adverse Events and Serious Adverse EventsNon-randomized · Open-label · Treatment | Phase 2/3 | Active, not recruiting | Genetic: RGX-202 | Duchenne Muscular Dystrophy | 65 | Sep 2026 | ≤ 42 d | 2026-07-21 |
| AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMDNCT05683379observationalPrevalence of anti-AAV8 antibodies in patients with DMD | — | Active, not recruiting | Diagnostic test: AAV8 DetectCDx | Duchenne Muscular Dystrophy | 200 | 2026-06-30actual | — | 2026-07-07 |
| CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)NCT03566043Part 1 SafetyNon-randomized · Open-label · Treatment | Phase 2/3 | Active, not recruiting | Genetic: RGX-121 | Mucopolysaccharidosis Type II (MPS II) | 48 | 2023-11-27actual | — | 2025-01-28 |
| RGX-111 Gene Therapy in Patients With MPS INCT03580083Safety and TolerabilityNon-randomized · Open-label · Treatment | Phase 1/2 | Suspendedsponsor's stated reason: The FDA placed a clinical hold on RGX-111 investigational gene therapy following an initial single case report of neoplasm (intraventricular CNS tumor) in a participant treated in its phase I/II study four years prior. | Genetic: RGX-111 | Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome, Hurler-Scheie Syndrome | 21 | 2027-07-07 | in 322 d | 2026-03-12 |
| Mucopolysaccharidosis Type II ObservationalNCT04591834observationalChanges in neurodevelopmental parameters of cognitive function over time | — | Withdrawnsponsor's stated reason: Sponsor decision | Other: Observational | Mucopolysaccharidosis II | 0 | Jul 2025 | — | 2022-10-10 |
| A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501NCT04080050observationalNumber of incidents of new and unexpected adverse events and serious adverse events. | — | Unknown status | Drug: Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy | Homozygous Familial Hypercholesterolemia (HoFH) | 8 | 2024-09-29 | — | 2023-03-09 |
| An Observational Study in Children With CLN2 Batten DiseaseNCT04462692observationalChange in retinal structure in children with CLN2 Batten disease | — | Withdrawnsponsor's stated reason: study stopped due to company decision | — | Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) | 0 | Oct 2023 | — | 2021-11-01 |
| RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)NCT04571970Number of participants with treatment-related adverse events and serious adverse eventsOpen-label · Treatment | Phase 1/2 | Completed | Genetic: RGX-121 | Mucopolysaccharidosis Type II (MPS II) | 6 | 2023-05-09actual | — | 2025-01-28 |
| A Retrospective, Natural History Study in Children With CLN2NCT04480476observationalCharacterize retinal structural changes in children with CLN2 | — | Withdrawnsponsor's stated reason: study stopped due to company decision | — | Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) | 0 | Apr 2022 | — | 2021-11-01 |
| Mucopolysaccharidosis Type II Natural HistoryNCT03529786observationalCognitive function over time, as indicated by results of neurocognitive measures documented in medical chart. | — | Completed | — | Mucopolysaccharidosis II | 36 | 2022-03-22actual | — | 2022-04-26 |
| A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)NCT02651675results2023-07-13Number of Participants With IP (Investigational Product) Related Adverse EventsNon-randomized · Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 1/2 | Terminatedsponsor's stated reason: Terminated by Sponsor for Business Reasons | Genetic: AAV directed hLDLR gene therapy | Homozygous Familial Hypercholesterolemia (HoFH) | 9 | 2020-11-27actual | — | 2023-07-13 |
| Identifying and Genotyping Homozygous Familial Hypercholesterolemia (HoFH) PatientsNCT04148001observationalThe number of participants who have HoFH due to mutations in the LDLR gene measured by genetic testing | — | Completed | — | Homozygous Familial Hypercholesterolemia (HoFH) | 4 | 2020-04-08actual | — | 2021-02-02 |
| Safety and Tolerability of RGX-314 (Investigational Product) Gene Therapy for Neovascular AMD TrialNCT03066258results2023-05-16Safety (Participants With Ocular and Non-ocular AEs (Adverse Events) and SAEs (Serious Adverse Events))Non-randomized · Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 1/2 | Completed | Genetic: RGX-314 | Neovascular Age-related Macular Degeneration, Wet Age-related Macular Degeneration | 42 | 2019-11-24actual | — | 2023-05-16 |
| Expanded Access to RGX-202NCT07652606expanded access | — | Temporarily not available | Genetic: RGX-202 | — | — | — | — | 2026-06-17 |
Primary completion is the date the sponsor filed with the registry — their own projection, revised whenever they revise it, unless the row is marked actual.
Source: ClinicalTrials.gov, retrieved 2026-08-19