| Detected | Trial | Change |
|---|---|---|
| 2026-06-12 | NCT02716246 Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH |
date change Primary completion pushed: 2027-07 -> 2029-03 |
| 2026-06-12 | NCT02716246 Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH |
date change Completion pushed: 2027-07 -> 2029-03 |
| 2026-06-12 | NCT03604835 Mucopolysaccharidosis VII Disease Monitoring Program |
date change Primary completion moved earlier: 2033-05 -> 2032-04 |
| 2026-06-12 | NCT03604835 Mucopolysaccharidosis VII Disease Monitoring Program |
date change Completion moved earlier: 2033-05 -> 2032-04 |
| 2026-06-03 | NCT05345171 Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC)… |
Enrollment increased: 32 -> 37 participants |
| NCT ID | Title | Condition | Status |
|---|---|---|---|
| Phase 3 | |||
| NCT05768854 | Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta | Osteogenesis Imperfecta | Active, Not Recruiting |
| NCT06636071 | Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta | Osteogenesis Imperfecta | Active, Not Recruiting |
| NCT05933200 | A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in… | Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | Active, Not Recruiting |
| NCT04360265 | Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for… | Mucopolysaccharidosis IIIA | Enrolling by Invitation |
| NCT06415344 | Long-term Extension of GTX-102 in Angelman Syndrome | Angelman Syndrome | Enrolling by Invitation |
| NCT06617429 | Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome… | Angelman Syndrome | Active, Not Recruiting |
| NCT05345171 | Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC)… | OTC Deficiency | Active, Not Recruiting |
| Phase 2/3 | |||
| NCT02716246 | Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH | MPS IIIA | Recruiting |
| NCT05125809 | Setrusumab vs Placebo for Osteogenesis Imperfecta | Osteogenesis Imperfecta | Active, Not Recruiting |
| Phase 2 | |||
| NCT07157254 | A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type… | Angelman Syndrome | Recruiting |
| Phase 1/2 | |||
| NCT04884815 | A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease | Wilson Disease | Active, Not Recruiting |
| NCT07511556 | First-in-human Study of UX016 in GNEM | GNE Myopathy | Not Yet Recruiting |
| Not specified | |||
| NCT06636383 | Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program | Glycogen Storage Disease Type Ia | Recruiting |
| NCT03604835 | Mucopolysaccharidosis VII Disease Monitoring Program | Mucopolysaccharidosis VII | Recruiting |
| NCT03651505 | X-linked Hypophosphatemia Disease Monitoring Program | X-linked Hypophosphatemia | Active, Not Recruiting |
| NCT03636438 | Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency | Ornithine Transcarbamylase (OTC) Deficiency | Active, Not Recruiting |
| NCT04632953 | Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program | Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | Active, Not Recruiting |
| NCT04783428 | Tumor-induced Osteomalacia Disease Monitoring Program | Tumor-induced Osteomalacia (TIO) | Active, Not Recruiting |