Trial · NCT02521792
In-Home Evaluation of Episodic Administration of Palovarotene in Fibrodysplasia Ossificans Progressiva (FOP) Subjects
NCT02521792, Phase 2, Terminated, Clementia Pharmaceuticals Inc.; results first posted 2020-10-19
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. In-Home Evaluation of Episodic Administration of Palovarotene in Fibrodysplasia Ossificans Progressiva (FOP) Subjects. Trials, record NCT02521792. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02521792
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- In-Home Evaluation of Episodic Administration of Palovarotene in Fibrodysplasia Ossificans Progressiva (FOP) Subjects
- NCT number
- NCT02521792
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 6 participants, actual
- Lead sponsor
- Clementia Pharmaceuticals Inc.
- Conditions
- Fibrodysplasia Ossificans Progressiva
- Interventions
- Palovarotene
- First posted
- 2015-08-13
- Last updated
- 2020-10-19
- Results first posted
- 2020-10-19
- Limitations and caveats
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.
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Subjects With Treatment-emergent Adverse Events (TEAEs) | 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). | 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. |
| Secondary | Subject Global Assessment of Movement as Determined by a Subject Completed Questionnaire, or Proxy Completed Questionnaire in Subjects Under 8 Years of Age | 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. | |
| Secondary | Change From Baseline in Cumulative Analogue Joint Involvement Scale for FOP as Assessed by the Investigator Using Remote Video-conferencing | 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. | |
| Secondary | Change From Baseline in Extent of Heterotopic Ossification (HO) by Whole Body Low-dose Computerized Tomography (CT) Scan, Excluding the Head | Baseline (final visit for Study PVO-1A-202/Part A) and at end of study (36 months). | |
| Secondary | Change From Baseline in Extent of HO by Whole Body Dual Energy X-ray Absorptiometry (DEXA) Scan | Baseline (screening/enrollment visit) and at end of study (36 months). | |
| Secondary | 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 | 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. | |
| Secondary | Change From Baseline in Physical Function Using Age-appropriate Forms of the FOP-Physical Function Questionnaire | 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. | |
| Secondary | Change From Baseline in Physical and Mental Health Using Age-appropriate Forms of the Patient-Reported Outcomes Measurement Information System (PROMIS) Global Health Scale | 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. | |
| Secondary | Duration of Active, Symptomatic Flare-up as Assessed by the Subject and the Investigator | Baseline (flare-up screening), after 6 weeks on study drug, and every 2 weeks after Week 6 until flare-up resolution. | |
| Secondary | Change From Baseline in the Use of Assistive Devices and Adaptations for Daily Living by FOP Subjects | Baseline (flare-up screening), 6 weeks after the end of treatment, and 6-month intervals for duration of study. |
Source ClinicalTrials.gov
Outcomes10
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Subjects With Treatment-emergent Adverse Events (TEAEs) | 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). | The safety population included all subjects who received at least 1 dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Subject Global Assessment of Movement as Determined by a Subject Completed Questionnaire, or Proxy Completed Questionnaire in Subjects Under 8 Years of Age | 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. | 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. | ||
| SECONDARY | Change From Baseline in Cumulative Analogue Joint Involvement Scale for FOP as Assessed by the Investigator Using Remote Video-conferencing | 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. | 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. | ||
| SECONDARY | Change From Baseline in Extent of Heterotopic Ossification (HO) by Whole Body Low-dose Computerized Tomography (CT) Scan, Excluding the Head | Baseline (final visit for Study PVO-1A-202/Part A) and at end of study (36 months). | 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. | ||
| SECONDARY | Change From Baseline in Extent of HO by Whole Body Dual Energy X-ray Absorptiometry (DEXA) Scan | Baseline (screening/enrollment visit) and at end of study (36 months). | 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. | ||
| SECONDARY | 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 | 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. | 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. | ||
| SECONDARY | Change From Baseline in Physical Function Using Age-appropriate Forms of the FOP-Physical Function Questionnaire | 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. | 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. | ||
| SECONDARY | Change From Baseline in Physical and Mental Health Using Age-appropriate Forms of the Patient-Reported Outcomes Measurement Information System (PROMIS) Global Health Scale | 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. | 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. | ||
| SECONDARY | Duration of Active, Symptomatic Flare-up as Assessed by the Subject and the Investigator | Baseline (flare-up screening), after 6 weeks on study drug, and every 2 weeks after Week 6 until flare-up resolution. | 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. | ||
| SECONDARY | Change From Baseline in the Use of Assistive Devices and Adaptations for Daily Living by FOP Subjects | Baseline (flare-up screening), 6 weeks after the end of treatment, and 6-month intervals for duration of study. | 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. |
Source ClinicalTrials.gov
Results by group2
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Subjects With Treatment-emergent Adverse Events (TEAEs) | Palovarotene | TEAE | 4 | Participants | |
| Number of Subjects With Treatment-emergent Adverse Events (TEAEs) | Palovarotene | Treatment-emergent SAE | 1 | Participants |
Source ClinicalTrials.gov
Participant flow4
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Palovarotene | 6 | |
| Overall Study | Received Treatment | Palovarotene | 4 | |
| Overall Study | COMPLETED | Palovarotene | 0 | |
| Overall Study | NOT COMPLETED | Palovarotene | 6 |
Source ClinicalTrials.gov
Adverse events8
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Dry skin | Skin and subcutaneous tissue disorders | Palovarotene | 2 | 4 | 3 |
| other | Lip dry | Gastrointestinal disorders | Palovarotene | 2 | 4 | 2 |
| other | Dry eye | Eye disorders | Palovarotene | 1 | 4 | 1 |
| other | Eczema | Skin and subcutaneous tissue disorders | Palovarotene | 1 | 4 | 1 |
| other | Tinnitus | Ear and labyrinth disorders | Palovarotene | 1 | 4 | 1 |
| other | Pneumonia | Infections and infestations | Palovarotene | 1 | 4 | 1 |
| serious | Myoclonus | Nervous system disorders | Palovarotene | 1 | 4 | 3 |
| serious | Myalgia | Musculoskeletal and connective tissue disorders | Palovarotene | 1 | 4 | 1 |
Source ClinicalTrials.gov
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Palovarotene | 0 | 4 |
| Total, other adverse events | Palovarotene | 4 | 4 |
| Total, serious adverse events | Palovarotene | 1 | 4 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 21460849 | BACKGROUND |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02521792
GET https://conductscience.org/api/trials/v1/NCT02521792/registered