| Phase 3 |
| NCT06636071 |
Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta |
Osteogenesis Imperfecta |
Active, Not Recruiting |
2026-09-28 |
| 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 |
2026-09-17 |
| NCT05345171 |
Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC)… |
OTC Deficiency |
Active, Not Recruiting |
2026-09-09 |
| NCT06415344 |
Long-term Extension of GTX-102 in Angelman Syndrome |
Angelman Syndrome |
Active, Not Recruiting |
2026-09-09 |
| NCT06617429 |
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome… |
Angelman Syndrome |
Active, Not Recruiting |
2026-08-06 |
| NCT04360265 |
Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for… |
Mucopolysaccharidosis IIIA |
Enrolling by Invitation |
2026-07-17 |
| NCT05768854 |
Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta |
Osteogenesis Imperfecta |
Active, Not Recruiting |
2026-02-25 |
| Phase 2/3 |
| NCT02716246 |
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH |
MPS IIIA |
Recruiting |
2026-09-22 |
| NCT05125809 |
Setrusumab vs Placebo for Osteogenesis Imperfecta |
Osteogenesis Imperfecta |
Active, Not Recruiting |
2026-10-06 |
| Phase 2 |
| NCT07157254 |
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type… |
Angelman Syndrome |
Active, Not Recruiting |
2026-09-09 |
| Phase 1/2 |
| NCT07511556 |
First-in-human Study of UX016 in GNEM |
GNE Myopathy |
Recruiting |
2026-09-03 |
| NCT04884815 |
A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease |
Wilson Disease |
Active, Not Recruiting |
2026-09-25 |
| Not specified |
| NCT03604835 |
Mucopolysaccharidosis VII Disease Monitoring Program |
Mucopolysaccharidosis VII |
Recruiting |
2026-08-27 |
| NCT06636383 |
Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program |
Glycogen Storage Disease Type Ia |
Recruiting |
2026-08-26 |
| NCT07855562 |
MPS IIIA Disease Monitoring Program |
MPS IIIA |
Not Yet Recruiting |
2026-10-02 |
| NCT03651505 |
X-linked Hypophosphatemia Disease Monitoring Program |
X-linked Hypophosphatemia |
Active, Not Recruiting |
2026-08-28 |
| NCT04632953 |
Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program |
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) |
Active, Not Recruiting |
2026-06-03 |
| NCT04783428 |
Tumor-induced Osteomalacia Disease Monitoring Program |
Tumor-induced Osteomalacia (TIO) |
Active, Not Recruiting |
2026-06-03 |
| NCT03636438 |
Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency |
Ornithine Transcarbamylase (OTC) Deficiency |
Active, Not Recruiting |
2026-06-01 |