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In-Home Evaluation of Episodic Administration of Palovarotene in Fibrodysplasia Ossificans Progressiva (FOP) Subjects

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ClinicalTrials.gov Identifier: NCT02521792
Recruitment Status : Terminated (Subjects were enrolled into a different Phase 2 study (PVO-1A-202, NCT02279095).)
First Posted : August 13, 2015
Results First Posted : October 19, 2020
Last Update Posted : October 19, 2020
Sponsor:
Information provided by (Responsible Party):
Ipsen ( Clementia Pharmaceuticals Inc. )

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Fibrodysplasia Ossificans Progressiva
Intervention Drug: Palovarotene
Enrollment 6
Recruitment Details Fibrodysplasia Ossificans Progressiva (FOP) subjects treated with palovarotene for 2 flare-ups in Study PVO-1A-202/Part A were enrolled. During enrolment, the dosage regimen for Study PVO-1A-202/Part B was optimized. Study PVO-1A-203 was terminated and participating subjects enrolled into Study PVO-1A-202/Part B to receive the modified regimen.
Pre-assignment Details The screening visit for this study (PVO-1A-203) was the same day as the end of study visit for Study PVO-1A-202/Part A. Subjects experiencing an eligible flare-up received open-label palovarotene (treatment period of at least 6 weeks). Subjects were to be followed for 36 months with telephone/video-conferencing assessments performed every 6 months.
Arm/Group Title Palovarotene
Hide Arm/Group Description Subjects experiencing an eligible flare-up received a weight-based equivalent dose of palovarotene 10 milligram (mg) orally once daily for 14 days, followed by 5 mg once daily for 28 days. If treatment was extended beyond 6 weeks (if deemed necessary by the Investigator), the palovarotene dose-equivalent of 5 mg was to be administered (in 2-week increments) until the flare-up resolved.
Period Title: Overall Study
Started 6
Received Treatment 4
Completed 0
Not Completed 6
Reason Not Completed
Study Terminated             6
Arm/Group Title Palovarotene
Hide Arm/Group Description Subjects experiencing an eligible flare-up received a weight-based equivalent dose of palovarotene 10 mg orally once daily for 14 days, followed by 5 mg once daily for 28 days. If treatment was extended beyond 6 weeks (if deemed necessary by the Investigator), the palovarotene dose-equivalent of 5 mg was to be administered (in 2-week increments) until the flare-up resolved.
Overall Number of Baseline Participants 6
Hide Baseline Analysis Population Description
All enrolled subjects were included in the baseline analysis.
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants
<=18 years
0
   0.0%
Between 18 and 65 years
6
 100.0%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants
Female
1
  16.7%
Male
5
  83.3%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 6 participants
6
1.Primary Outcome
Title Number of Subjects With Treatment-emergent Adverse Events (TEAEs)
Hide Description The primary endpoint was the safety of palovarotene as assessed by the incidence of TEAEs (including those known to be associated with retinoids) and serious adverse event (SAEs) monitored throughout the treatment period. TEAEs were adverse events reported during treatment with palovarotene or within 6 weeks after the end of treatment. Day 1 was the first day that study drug was administered for a flare-up. The number of subjects experiencing at least one TEAE or treatment-emergent SAE are presented.
Time Frame Day 1 until 6 weeks after the end of treatment (an expected average treatment of 6 weeks). Assessed until data cut-off for study termination (maximum of 35 days).
Hide Outcome Measure Data
Hide Analysis Population Description
The safety population included all subjects who received at least 1 dose of study drug.
Arm/Group Title Palovarotene
Hide Arm/Group Description:
Subjects experiencing an eligible flare-up received a weight-based equivalent dose of palovarotene 10 mg orally once daily for 14 days, followed by 5 mg once daily for 28 days. If treatment was extended beyond 6 weeks (if deemed necessary by the Investigator), the palovarotene dose-equivalent of 5 mg was to be administered (in 2-week increments) until the flare-up resolved.
Overall Number of Participants Analyzed 4
Measure Type: Count of Participants
Unit of Measure: Participants
TEAE
4
 100.0%
Treatment-emergent SAE
1
  25.0%
2.Secondary Outcome
Title Subject Global Assessment of Movement as Determined by a Subject Completed Questionnaire, or Proxy Completed Questionnaire in Subjects Under 8 Years of Age
Hide Description [Not Specified]
Time Frame Every 6 weeks while on study drug, at the end of treatment (should treatment be extended beyond 6 weeks), and 6 weeks after the end of treatment.
Hide Outcome Measure Data
Hide Analysis Population Description
The study was stopped prematurely and only 4 subjects received treatment. No summary statistics are available due to the limited data from the small number of evaluable subjects and individual subject data are not presented to protect the privacy of the individuals.
Arm/Group Title Palovarotene
Hide Arm/Group Description:
Subjects experiencing an eligible flare-up received a weight-based equivalent dose of palovarotene 10 mg orally once daily for 14 days, followed by 5 mg once daily for 28 days. If treatment was extended beyond 6 weeks (if deemed necessary by the Investigator), the palovarotene dose-equivalent of 5 mg was to be administered (in 2-week increments) until the flare-up resolved.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
3.Secondary Outcome
Title Change From Baseline in Cumulative Analogue Joint Involvement Scale for FOP as Assessed by the Investigator Using Remote Video-conferencing
Hide Description [Not Specified]
Time Frame Baseline (flare-up screening), every 6 weeks while on study drug, at the end of treatment (should treatment be extended beyond 6 weeks), and 6 weeks after the end of treatment.
Hide Outcome Measure Data
Hide Analysis Population Description
The study was stopped prematurely and only 4 subjects received treatment. No summary statistics are available due to the limited data from the small number of evaluable subjects and individual subject data are not presented to protect the privacy of the individuals.
Arm/Group Title Palovarotene
Hide Arm/Group Description:
Subjects experiencing an eligible flare-up received a weight-based equivalent dose of palovarotene 10 mg orally once daily for 14 days, followed by 5 mg once daily for 28 days. If treatment was extended beyond 6 weeks (if deemed necessary by the Investigator), the palovarotene dose-equivalent of 5 mg was to be administered (in 2-week increments) until the flare-up resolved.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
4.Secondary Outcome
Title Change From Baseline in Extent of Heterotopic Ossification (HO) by Whole Body Low-dose Computerized Tomography (CT) Scan, Excluding the Head
Hide Description [Not Specified]
Time Frame Baseline (final visit for Study PVO-1A-202/Part A) and at end of study (36 months).
Hide Outcome Measure Data
Hide Analysis Population Description
The study was stopped prematurely and only 4 subjects received treatment. No summary statistics are available due to the limited data from the small number of evaluable subjects and individual subject data are not presented to protect the privacy of the individuals.
Arm/Group Title Palovarotene
Hide Arm/Group Description:
Subjects experiencing an eligible flare-up received a weight-based equivalent dose of palovarotene 10 mg orally once daily for 14 days, followed by 5 mg once daily for 28 days. If treatment was extended beyond 6 weeks (if deemed necessary by the Investigator), the palovarotene dose-equivalent of 5 mg was to be administered (in 2-week increments) until the flare-up resolved.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
5.Secondary Outcome
Title Change From Baseline in Extent of HO by Whole Body Dual Energy X-ray Absorptiometry (DEXA) Scan
Hide Description [Not Specified]
Time Frame Baseline (screening/enrollment visit) and at end of study (36 months).
Hide Outcome Measure Data
Hide Analysis Population Description
The study was stopped prematurely and only 4 subjects received treatment. No summary statistics are available due to the limited data from the small number of evaluable subjects and individual subject data are not presented to protect the privacy of the individuals.
Arm/Group Title Palovarotene
Hide Arm/Group Description:
Subjects experiencing an eligible flare-up received a weight-based equivalent dose of palovarotene 10 mg orally once daily for 14 days, followed by 5 mg once daily for 28 days. If treatment was extended beyond 6 weeks (if deemed necessary by the Investigator), the palovarotene dose-equivalent of 5 mg was to be administered (in 2-week increments) until the flare-up resolved.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
6.Secondary Outcome
Title Change From Baseline in Pain and Swelling at the Flare-up Site Using Numeric Rating Scales, or Faces Pain Scale-Revised in Subjects Under 8 Years of Age
Hide Description [Not Specified]
Time Frame Baseline (flare-up screening), every 2 weeks while on study drug, at the end of treatment (should treatment be extended beyond 6 weeks), and 6 weeks after the end of treatment.
Hide Outcome Measure Data
Hide Analysis Population Description
The study was stopped prematurely and only 4 subjects received treatment. No summary statistics are available due to the limited data from the small number of evaluable subjects and individual subject data are not presented to protect the privacy of the individuals.
Arm/Group Title Palovarotene
Hide Arm/Group Description:
Subjects experiencing an eligible flare-up received a weight-based equivalent dose of palovarotene 10 mg orally once daily for 14 days, followed by 5 mg once daily for 28 days. If treatment was extended beyond 6 weeks (if deemed necessary by the Investigator), the palovarotene dose-equivalent of 5 mg was to be administered (in 2-week increments) until the flare-up resolved.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
7.Secondary Outcome
Title Change From Baseline in Physical Function Using Age-appropriate Forms of the FOP-Physical Function Questionnaire
Hide Description [Not Specified]
Time Frame Baseline (flare-up screening), every 6 weeks while on study drug, at the end of treatment (should treatment be extended beyond 6 weeks), and 6 weeks after the end of treatment, and 6-month intervals for duration of study.
Hide Outcome Measure Data
Hide Analysis Population Description
The study was stopped prematurely and only 4 subjects received treatment. No summary statistics are available due to the limited data from the small number of evaluable subjects and individual subject data are not presented to protect the privacy of the individuals.
Arm/Group Title Palovarotene
Hide Arm/Group Description:
Subjects experiencing an eligible flare-up received a weight-based equivalent dose of palovarotene 10 mg orally once daily for 14 days, followed by 5 mg once daily for 28 days. If treatment was extended beyond 6 weeks (if deemed necessary by the Investigator), the palovarotene dose-equivalent of 5 mg was to be administered (in 2-week increments) until the flare-up resolved.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
8.Secondary Outcome
Title Change From Baseline in Physical and Mental Health Using Age-appropriate Forms of the Patient-Reported Outcomes Measurement Information System (PROMIS) Global Health Scale
Hide Description [Not Specified]
Time Frame Baseline (flare-up screening), every 6 weeks while on study drug, at the end of treatment (should treatment be extended beyond 6 weeks), 6 weeks after the end of treatment, and 6-month intervals for duration of study.
Hide Outcome Measure Data
Hide Analysis Population Description
The study was stopped prematurely and only 4 subjects received treatment. No summary statistics are available due to the limited data from the small number of evaluable subjects and individual subject data are not presented to protect the privacy of the individuals.
Arm/Group Title Palovarotene
Hide Arm/Group Description:
Subjects experiencing an eligible flare-up received a weight-based equivalent dose of palovarotene 10 mg orally once daily for 14 days, followed by 5 mg once daily for 28 days. If treatment was extended beyond 6 weeks (if deemed necessary by the Investigator), the palovarotene dose-equivalent of 5 mg was to be administered (in 2-week increments) until the flare-up resolved.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
9.Secondary Outcome
Title Duration of Active, Symptomatic Flare-up as Assessed by the Subject and the Investigator
Hide Description [Not Specified]
Time Frame Baseline (flare-up screening), after 6 weeks on study drug, and every 2 weeks after Week 6 until flare-up resolution.
Hide Outcome Measure Data
Hide Analysis Population Description
The study was stopped prematurely and only 4 subjects received treatment. No summary statistics are available due to the limited data from the small number of evaluable subjects and individual subject data are not presented to protect the privacy of the individuals.
Arm/Group Title Palovarotene
Hide Arm/Group Description:
Subjects experiencing an eligible flare-up received a weight-based equivalent dose of palovarotene 10 mg orally once daily for 14 days, followed by 5 mg once daily for 28 days. If treatment was extended beyond 6 weeks (if deemed necessary by the Investigator), the palovarotene dose-equivalent of 5 mg was to be administered (in 2-week increments) until the flare-up resolved.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
10.Secondary Outcome
Title Change From Baseline in the Use of Assistive Devices and Adaptations for Daily Living by FOP Subjects
Hide Description [Not Specified]
Time Frame Baseline (flare-up screening), 6 weeks after the end of treatment, and 6-month intervals for duration of study.
Hide Outcome Measure Data
Hide Analysis Population Description
The study was stopped prematurely and only 4 subjects received treatment. No summary statistics are available due to the limited data from the small number of evaluable subjects and individual subject data are not presented to protect the privacy of the individuals.
Arm/Group Title Palovarotene
Hide Arm/Group Description:
Subjects experiencing an eligible flare-up received a weight-based equivalent dose of palovarotene 10 mg orally once daily for 14 days, followed by 5 mg once daily for 28 days. If treatment was extended beyond 6 weeks (if deemed necessary by the Investigator), the palovarotene dose-equivalent of 5 mg was to be administered (in 2-week increments) until the flare-up resolved.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame TEAEs were to be collected from Day 1 until 6 weeks after the end of treatment (an expected average treatment of 6 weeks). Assessed until data cut-off for study termination (maximum of 35 days).
Adverse Event Reporting Description The safety population included all subjects who received at least one dose of study drug.
 
Arm/Group Title Palovarotene
Hide Arm/Group Description Eligible subjects received a weight-based equivalent dose of palovarotene 10 mg orally once daily for 14 days, followed by 5 mg once daily for 28 days during flare-ups. If treatment was extended beyond 6 weeks (if deemed necessary by the Investigator), a weight-based equivalent dose of 5 mg was to be administered in 2-week increments.
All-Cause Mortality
Palovarotene
Affected / at Risk (%)
Total   0/4 (0.00%)    
Hide Serious Adverse Events
Palovarotene
Affected / at Risk (%) # Events
Total   1/4 (25.00%)    
Musculoskeletal and connective tissue disorders   
Myalgia †  1/4 (25.00%)  1
Nervous system disorders   
Myoclonus †  1/4 (25.00%)  3
†
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Palovarotene
Affected / at Risk (%) # Events
Total   4/4 (100.00%)    
Ear and labyrinth disorders   
Tinnitus †  1/4 (25.00%)  1
Eye disorders   
Dry eye †  1/4 (25.00%)  1
Gastrointestinal disorders   
Lip dry †  2/4 (50.00%)  2
Infections and infestations   
Pneumonia †  1/4 (25.00%)  1
Skin and subcutaneous tissue disorders   
Dry skin †  2/4 (50.00%)  3
Eczema †  1/4 (25.00%)  1
†
Indicates events were collected by systematic assessment
The study was stopped prematurely and due to the small number of evaluable subjects, there was insufficient data to conduct any efficacy analyses. Consequently, no summary statistics are available for any of the secondary outcome measures.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Medical Director
Organization: Ipsen
Phone: see email
EMail: clinical.trials@ipsen.com
Publications:
Layout table for additonal information
Responsible Party: Ipsen ( Clementia Pharmaceuticals Inc. )
ClinicalTrials.gov Identifier: NCT02521792    
Other Study ID Numbers: PVO-1A-203
First Submitted: August 6, 2015
First Posted: August 13, 2015
Results First Submitted: July 17, 2020
Results First Posted: October 19, 2020
Last Update Posted: October 19, 2020