Ultragenyx Pharmaceutical (RARE) clinical trials: pipeline and status changes, no readout expected in the next 12 months
$RARE · Ultragenyx Pharmaceutical IncTrials that could report in the next 12 months: 0BiopharmaCTG
Catalyst timeline
No upcoming catalysts tracked for RARE. Estimated readout windows appear automatically as tracked trials approach their registered completion dates.
Pipeline
| Program | Phase | Status | Primary completion | Trial |
|---|---|---|---|---|
| A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Diseas… | Phase 1 | Active Not Recruitingenrolled, still running | NCT04884815 | |
| A Study to Determine the Effect of Triheptanoin Compared With Even-Cha… | Phase 3 | Active Not Recruitingenrolled, still running | NCT05933200 | |
| Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known… | Phase 3 | Enrolling By Invitation | NCT04360265 | |
| Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Tran… | Phase 3 | Active Not Recruitingenrolled, still running | NCT05345171 | |
| Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH | Phase 2 | Recruitingstill enrolling patients | NCT02716246 | |
| Long-term Extension of GTX-102 in Angelman Syndrome | Phase 3 | Enrolling By Invitation | NCT06415344 | |
| Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta | Phase 3 | Active Not Recruitingenrolled, still running | NCT06636071 | |
| A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or N… | Phase 2 | Recruitingstill enrolling patients | NCT07157254 |
+ 1 more tracked trial
| Program | Phase | Status | Primary completion | Trial |
|---|---|---|---|---|
| First-in-human Study of UX016 in GNEM | Phase 1 | Not Yet Recruitingnot open yet | NCT07511556 |
Status-change history
Every ClinicalTrials.gov change recorded for RARE's trials — a running history no other tracker shows.
No status changes detected yet for this ticker.
Frequently asked
When is RARE's next PDUFA date?
No disclosed PDUFA date is currently tracked for this ticker.
What phase is RARE's lead program in?
Phase 3 — active not recruiting.
Where does this data come from?
ClinicalTrials.gov v2 API (trial status + diffs) and company 8-K/press-release disclosures (PDUFA dates). Every number links to its source.