An Efficacy and Safety Study of Palovarotene for the Treatment of MO (MO-Ped)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03442985|
Recruitment Status : Active, not recruiting
First Posted : February 22, 2018
Last Update Posted : December 11, 2019
|Condition or disease||Intervention/treatment||Phase|
|Exostoses, Multiple Hereditary||Drug: Palovarotene 2.5 mg Drug: Palovarotene 5.0 mg Other: Placebo||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||240 participants|
|Intervention Model:||Parallel Assignment|
|Intervention Model Description:||Multicenter, randomized, double-blind, placebo-controlled|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||A Phase 2, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Palovarotene in Subjects With Multiple Osteochondromas|
|Actual Study Start Date :||April 20, 2018|
|Estimated Primary Completion Date :||January 2021|
|Estimated Study Completion Date :||February 2021|
|Experimental: Palovarotene 2.5 mg daily regimen||
Drug: Palovarotene 2.5 mg
Subjects will receive a weight-adjusted dose equivalent of 2.5 mg palovarotene, once daily, for up to 24 months.
|Experimental: Palovarotene 5.0 mg daily regimen||
Drug: Palovarotene 5.0 mg
Subjects will receive a weight-adjusted dose equivalent of 5.0 mg palovarotene, once daily, for up to 24 months.
|Placebo Comparator: Placebo regimen||
Subjects will receive placebo, once daily, for up to 24 months.
- Annualized rate of new osteochondromas [ Time Frame: Month 24 ]The annualized rate of new osteochondromas as assessed by whole body magnetic resonance imaging (MRI).
- Total volume of osteochondromas [ Time Frame: Months 12 and 24/end-of-treatment (EOT) ]The change from baseline in the total volume of osteochondromas as assessed by whole body MRI.
- Annualized rate of new or worsening deformities [ Time Frame: Months 12 and 24/EOT ]The annualized rate of new or worsening deformities as assessed by radiographic imaging of both upper and lower limbs.
- Annualized rate of MO-related surgeries [ Time Frame: Months 12 and 24/EOT ]The annualized rate of MO-related surgeries. Surgeries include surgical excisions of a symptomatic osteochondroma and surgical procedures to correct a joint deformities or functional limitations.
- Palovarotene area under the concentration-time curve (AUC) [ Time Frame: Month 1 ]Determination of AUC at steady-state assessed during treatment.
- Palatability [ Time Frame: Day 1 and Month 1 ]The palatability of the drug product versus placebo when sprinkled onto specific foods as assessed by a five-point hedonic face scale.
- Safety of Palovarotene: Monitoring of adverse events [ Time Frame: Study Day 1; and from Months 1 to 30/ET at every subject contact ]Monitoring of adverse events.
- Volume of osteochondroma cartilage cap [ Time Frame: Months 12 and 24 ]Change from baseline in the total volume of cartilage cap of osteochondromas as assessed by whole body MRI
- Annualized rate of new or worsening functional limitations [ Time Frame: Months 12 and 24/EOT ]The annualized rate of new or worsening functional limitations. Functional limitations are defined as restrictions in joint range of motion.
- PedsQL [ Time Frame: Months 6, 12, 18, and 24/EOT ]The Pediatric Quality of Life Inventory (PedsQL, version 4.0) questionnaire to assess quality of life.
- PROMIS Global Health Scale [ Time Frame: Months 6, 12, 18, and 24/EOT ]The Patient-Reported Outcomes Measurement Information System (PROMIS) pain interference pediatric item bank to assess the effect of pain on daily activities.
- FPS-R [ Time Frame: Months 6, 12, 18, and 24/EOT ]The Faces Pain Scale - Revised (FPS-R) to assess pain intensity.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03442985