NCT03442985

An Efficacy and Safety Study of Palovarotene for the Treatment of MO

A Phase 2, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Palovarotene in Subjects With Multiple Osteochondromas

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 193 (actual)
• Sponsor: Clementia Pharmaceuticals Inc. · Industry
• Sex: All
• Age: 2 Years – 14 Years
• Healthy volunteers: Not accepted

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).

Detailed description

Multiple osteochondromas is a rare condition where children develop multiple benign cartilage-capped bony tumors called osteochondromas on bones throughout the body, resulting in pain, deformity, limb length discrepancy, disability, and eventually arthritis and possible malignancy. The primary objective is to compare the efficacy of two dosage regimens of palovarotene with placebo to prevent the formation of new osteochondromas in pediatric MO subjects with exostosin 1 or exostosin 2 gene mutations. Osteochondroma formation was assessed by whole body magnetic resonance imaging (MRI). Secondary efficacy objectives were to compare the effects of palovarotene with placebo on the volume of osteochondromas as assessed by MRI; the proportion of subjects with no new osteochondromas as assessed by whole-body MRI; the annualized rate of new or worsening deformities; the annualized rate of MO-related surgeries; and palatability. The overall safety and pharmacokinetics of palovarotene and the effects of palovarotene on linear growth, bone growth plates, bone mineral density, quality of life, and pain due to osteochondromas was also studied.

Conditions

• Exostoses, Multiple Hereditary

Interventions

Type	Name	Description
DRUG	Palovarotene 2.5 mg	Subjects received a weight-adjusted dose equivalent of 2.5 mg palovarotene, once daily, for up to 24 months.
DRUG	Palovarotene 5.0 mg	Subjects received a weight-adjusted dose equivalent of 5.0 mg palovarotene, once daily, for up to 24 months.
OTHER	Placebo	Subjects received placebo, once daily, for up to 24 months.

Timeline

• Start date: 2018-03-22
• Primary completion: 2020-03-24
• Completion: 2020-10-30
• First posted: 2018-02-22
• Last updated: 2022-08-01
• Results posted: 2022-08-01

Locations

31 sites across 12 countries: United States, Australia, Belgium, Canada, France, Italy, Japan, Netherlands, Portugal, Spain, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study