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

• Status: Terminated
• Trial end date: 2020-10-30
• Target enrollment: 0
• Participant gender: All

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).

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Clementia Pharmaceuticals Inc.

Criteria

Key Inclusion Criteria:

• 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.

Key Exclusion Criteria:

• Weight under 10 kg.

• Other syndromic conditions such as Langer-Giedion or Potocki-Shaffer.

• Any subject with neurologic signs suggestive of spinal cord impingement.

• Concomitant medications that are strong inhibitors or inducers of cytochrome P450 3A4 activity.

• Amylase or lipase >2 times the above the upper limit of normal (>2×ULN) or with a history of chronic pancreatitis.

• Elevated aspartate aminotransferase or alanine aminotransferase above 2.5×ULN.

• Any surgical implant that is contraindicated for MRI.