Trials Sponsors Conditions Drugs Pricing

Conditions

Keywords

Multiple osteochondromasOsteochondromaPalovaroteneHereditary multiple exostosesHMEMORetinoic acid receptor gamma agonistRetinoic acid receptor agonist

Outcome Measures

Primary Outcomes (1)

Annualized Rate of New Osteochondromas (OCs)
The annualized rate of new OCs was assessed by whole-body magnetic resonance imaging (MRI) (that is, the total number of new OCs divided by the time in years between the baseline and latest post-baseline MRI).
Month 12

Secondary Outcomes (9)

Mean Change From Baseline in the Total Volume of New OCs at Month 12
Baseline (Day 1) and Month 12
Percentage of Participants With No New OCs
Month 12
Annualized Rate of New or Worsening Deformities
Month 12
Annualized Rate of MO-Related Surgeries
Month 12
Maximum Observed Plasma Drug Concentrations at Steady State (Cmax,ss) of Palovarotene
Month 1: pre-dose and 3, 6, 10 and 24 hours post-dose
+4 more secondary outcomes

Study Arms (3)

Palovarotene 2.5 mg daily regimen

EXPERIMENTAL

Drug: Palovarotene 2.5 mg

Palovarotene 5.0 mg daily regimen

EXPERIMENTAL

Drug: Palovarotene 5.0 mg

Placebo regimen

PLACEBO COMPARATOR

Other: Placebo

Interventions

Palovarotene 2.5 mgDrug

Subjects received a weight-adjusted dose equivalent of 2.5 mg palovarotene, once daily, for up to 24 months.

Palovarotene 2.5 mg daily regimen

Palovarotene 5.0 mgDrug

Subjects received a weight-adjusted dose equivalent of 5.0 mg palovarotene, once daily, for up to 24 months.

Palovarotene 5.0 mg daily regimen

Eligibility Criteria

Age2 Years - 14 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17)

You may qualify if:

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.

Sponsors & Collaborators

Clementia Pharmaceuticals Inc.
Industrylead

Study Sites (31)

Children's Orthopaedic Center

Los Angeles, California, 90027, United States

Shriners Hospital for Children - Sacramento

Sacramento, California, 95817, United States

University of California-San Francisco

San Francisco, California, 94158, United States

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

The Paley Institute

West Palm Beach, Florida, 330407, United States

Shriners Hospital for Children - Chicago

Chicago, Illinois, 60707, United States

Johns Hopkins University

Related Publications (2)

Inubushi T, Lemire I, Irie F, Yamaguchi Y. Palovarotene Inhibits Osteochondroma Formation in a Mouse Model of Multiple Hereditary Exostoses. J Bone Miner Res. 2018 Apr;33(4):658-666. doi: 10.1002/jbmr.3341. Epub 2017 Nov 30.
PMID: 29120519BACKGROUND
Sangiorgi L, Conrad EU, Shih F, Strahs A, Feldman DS. Palovarotene for patients with multiple hereditary exostosis: results of MO-Ped, a terminated, randomized, placebo-controlled, double-blind phase 2 trial. Sci Rep. 2025 Nov 4;15(1):38563. doi: 10.1038/s41598-025-22554-6.
PMID: 41188357DERIVED

MeSH Terms

Conditions

Exostoses, Multiple HereditaryOsteochondroma

Interventions

Palovarotene

Condition Hierarchy (Ancestors)

OsteochondromatosisNeoplasms, Bone TissueNeoplasms, Connective TissueNeoplasms, Connective and Soft TissueNeoplasms by Histologic TypeNeoplasmsNeoplastic Syndromes, HereditaryOsteochondrodysplasiasBone Diseases, DevelopmentalBone DiseasesMusculoskeletal Diseases

Limitations and Caveats

The Sponsor terminated the study early due to a partial clinical hold instituted by the Food and Drug Administration. Recruitment was stopped before full enrollment was reached, and study drug administration was discontinued.

Results Point of Contact

Title: Medical Director
Organization: Ipsen

Study Officials

Ipsen Medical Director
Ipsen
STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee: No
Restrictive Agreement: Yes

Enrollment

193 participants (actual enrollment reported by the registry).

Study Design

Study Type: interventional
Phase: phase 2
Allocation: RANDOMIZED
Masking: QUADRUPLE
Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose: TREATMENT
Intervention Model: PARALLEL
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

October 11, 2017

First Posted

February 22, 2018

Study Start

March 22, 2018

Actual (reported)

Study Stopped

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

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Results Posted

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (9)

Study Arms (3)

Palovarotene 2.5 mg daily regimen

Palovarotene 5.0 mg daily regimen

Placebo regimen

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (31)

Related Publications (2)

Related Links

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Limitations and Caveats

Results Point of Contact

Study Officials

Publication Agreements

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Documents

Locations