Status:
TERMINATED
An Efficacy and Safety Study of Palovarotene for the Treatment of MO
Lead Sponsor:
Clementia Pharmaceuticals Inc.
Conditions:
Exostoses, Multiple Hereditary
Eligibility:
All Genders
2-14 years
Phase:
PHASE2
Brief 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 subjec...
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...
Eligibility Criteria
Inclusion
- Key
- 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
- 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.
Key Trial Info
Start Date :
March 22 2018
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 30 2020
Estimated Enrollment :
193 Patients enrolled
Trial Details
Trial ID
NCT03442985
Start Date
March 22 2018
End Date
October 30 2020
Last Update
August 1 2022
Active Locations (31)
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1
Children's Orthopaedic Center
Los Angeles, California, United States, 90027
2
Shriners Hospital for Children - Sacramento
Sacramento, California, United States, 95817
3
University of California-San Francisco
San Francisco, California, United States, 94158
4
Children's National Medical Center
Washington D.C., District of Columbia, United States, 20010