drugset / Trial / NCT03442985
An Efficacy and Safety Study of Palovarotene for the Treatment of MO
RandomizedParallel-groupQuadruple-blindTreatment
Summary
This is a randomized, double-blind, placebo-controlled study comparing the safety and efficacy of 2 dosage regimens of palovarotene versus placebo in preventing disease progression in pediatric subjects with multiple osteochondromas (MO).
Timeline
- Start
- 2018-03-22
- Primary completion
- 2020-03-24
- Completion
- 2020-10-30
Outcome
Missed primary endpoint
Stopped: “Trial was terminated early to analyze the accumulated data and evaluate the efficacy, safety and future of palovarotene in MO.”
paper No significant differences in the annualized rate of new OCs PMID 41188357 ↗
registry Placebo vs Palovarotene 5.0 mg; p = 0.6570; Risk Ratio (RR) 1.441 (95% CI 0.287 to 7.234) NCT03442985 ↗
Publications
- Background Inubushi T, Lemire I, Irie F, Yamaguchi Y. Palovarotene Inhibits Osteochondroma Formation in a Mouse Model of Multiple Hereditary Exostoses. J Bone Miner Res. 2018 Apr;33(4):658-666. doi: 10.1002/jbmr.3341. Epub 2017 Nov 30.
- Sangiorgi L, Conrad EU, Shih F, Strahs A, Feldman DS. Palovarotene for patients with multiple hereditary exostosis: results of MO-Ped, a terminated, randomized, placebo-controlled, double-blind phase 2 trial. Sci Rep. 2025 Nov 4;15(1):38563. doi: 10.1038/s41598-025-22554-6.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Palovarotene | Small molecule | 2.5 mg | — |
| Subject | Palovarotene | Small molecule | 5 mg | — |