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

Official Title

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).

Trial Description

Primary Outcome:

  • Annualized rate of new osteochondromas
Secondary Outcome:
  • Total volume of osteochondromas
  • Annualized rate of new or worsening deformities
  • Annualized rate of MO-related surgeries
  • Palovarotene area under the concentration-time curve (AUC)
  • Palatability
Multiple osteochondromas is a rare condition where children develop multiple benign cartilage-capped bony tumours called osteochondromas on bones throughout the body, resulting in pain, deformity, limb length discrepancy, disability, and eventually arthritis and possible malignancy. The primary objective is to compare the efficacy of two dosage regimens of palovarotene with placebo to prevent the formation of new osteochondromas in pediatric MO subjects with exostosin 1 or exostosin 2 gene mutations. Osteochondroma formation will be assessed by whole body magnetic resonance imaging (MRI). Secondary efficacy objectives are to compare the effect of palovarotene on the volume of osteochondromas as assessed by MRI; and on the annualized rate of new or worsening deformities and MO-related surgeries. The overall safety of palovarotene and the effects of palovarotene on linear growth, bone growth plates, bone mineral density, quality of life, and pain due to osteochondromas will also be studied.

View this trial on

Interested in this trial?

Print this page and take it to your doctor to discuss your eligibilty and treatment options. Only your doctor can refer you to a clinical trial.


Canadian Cancer Society

These resources are provided in partnership with the Canadian Cancer Society