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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03442985
• Date of registration: 11/10/2017
• Prospective Registration: Yes
• Primary sponsor: Clementia Pharmaceuticals Inc.
• Public title: An Efficacy and Safety Study of Palovarotene for the Treatment of MO MO-Ped
• Scientific title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Palovarotene in Subjects With Multiple Osteochondromas
• Date of first enrolment: April 20, 2018
• Target sample size: 194
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03442985
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Australia, Belgium, Canada, France, Italy, Japan, Netherlands, Portugal, Spain, Turkey, United Kingdom, United States

Contacts

• Name: Ipsen Medical Director
• Affiliation: Ipsen

Key inclusion & exclusion 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.



Age minimum: 2 Years
Age maximum: 14 Years
Gender: All

Health Condition(s) or Problem(s) studied

Exostoses, Multiple Hereditary

Intervention(s)

Drug: Palovarotene 5.0 mg

Drug: Palovarotene 2.5 mg

Other: Placebo

Primary Outcome(s)

Annualized rate of new osteochondromas [Time Frame: Month 24]

Secondary Outcome(s)

Annualized rate of MO-related surgeries [Time Frame: Months 12 and 24/EOT]

Annualized rate of new or worsening deformities [Time Frame: Months 12 and 24/EOT]

Palatability [Time Frame: Day 1 and Month 1]

Palovarotene area under the concentration-time curve (AUC) [Time Frame: Month 1]

Total volume of osteochondromas [Time Frame: Months 12 and 24/end-of-treatment (EOT)]

Secondary ID(s)

2017-002751-28

PVO-2A-201

Source(s) of Monetary Support

Please refer to primary and secondary sponsors