A Phase 2, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Palovarotene in Subjects With Multiple Osteochondromas
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).
• Written, signed, and dated informed subject/parent consent and age-appropriate assent (performed according to local regulations).
• A clinical diagnosis of MO with disease-causing exostosin 1 or 2 gene mutations.
• Male or female from 2 to 14 years of age.
• Female subjects must be premenarchal at screening.
• A bone age at screening of 14 years or less.
• Symptomatic MO, defined as five or more clinically evident osteochondromas and a new or enlarged osteochondroma that occurred in the preceding 12 months, five or more clinically evident osteochondromas and the presence of a painful osteochondroma, a skeletal deformity, a joint limitation, or prior surgery for a MO-related complication.
• The ability to undergo whole body MRI with or without sedation/general anesthesia.
• Use of two effective methods of birth control during treatment, and for 1 month after treatment discontinuation, unless committed to true abstinence from heterosexual sex. Sexually active females of child-bearing potential must also agree to start effective methods of birth control at screening.