Ultragenyx Pharmaceutical (RARE) is the lead sponsor of 17 active clinical trials listed on ClinicalTrials.gov[4], including 9 Phase 3[1], 4 Phase 2[2], 1 Phase 1[3].
Trial NCT05768854[5] evaluates Bisphosphonate in Osteogenesis Imperfecta with a target enrollment of 69 participants. Trial NCT05933200[6] evaluates Triheptanoin in Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) with a target enrollment of 69 participants. Trial NCT05345171[7] evaluates DTX301 in OTC Deficiency with a target enrollment of 32 participants.
RARE has 1 Form 4 insider filing recorded at the SEC in the past 30 days[8].
Sources
- ClinicalTrials.gov (2026-04-11) ↗
- ClinicalTrials.gov (2026-04-11) ↗
- ClinicalTrials.gov (2026-04-11) ↗
- ClinicalTrials.gov (2026-04-11) ↗
- ClinicalTrials.gov · NCT05768854 (2026-02-25) ↗
- ClinicalTrials.gov · NCT05933200 (2026-04-07) ↗
- ClinicalTrials.gov · NCT05345171 (2026-02-17) ↗
- SEC EDGAR · 0001515673 (2026-04-11) ↗