Trial · NCT03312634
An Efficacy and Safety Study of Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva.
NCT03312634, Phase 3, Completed, Clementia Pharmaceuticals Inc.; results first posted 2023-03-14
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. An Efficacy and Safety Study of Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva. Trials, record NCT03312634. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03312634
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- An Efficacy and Safety Study of Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva.
- NCT number
- NCT03312634
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 107 participants, actual
- Lead sponsor
- Clementia Pharmaceuticals Inc.
- Conditions
- Fibrodysplasia Ossificans Progressiva
- Interventions
- Palovarotene
- First posted
- 2017-10-18
- Last updated
- 2023-11-29
- Other registries' IDs
- eudract:2017-002541-29
- Results first posted
- 2023-03-14
- Limitations and caveats
- A valid comparison of AEs from observational study (PVO-1A-001) was not made since AEs were only captured as related to study procedures.
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Annualized New Heterotopic Ossification (HO) | Baseline (within one month of screening/Day 1) and up to 24 months | The annualized new HO was assessed by low-dose, whole body computed tomography (WBCT), excluding head. The weighted linear mixed effect method without square-root transformation and negatives included was used for annualized new HO analysis. |
| Secondary | Percentage of Participants With Any New HO | From Baseline (Day 1) up to end of 4-year follow-up period (approximately 57 months) | The new HO was assessed by WBCT scan. The percentage of participants with any new HO (volume \> 0 mm\^3) were analyzed using the Bayesian distribution. Results are presented for overall ITT period. |
| Secondary | Number of Body Regions With New HO | From Baseline (Day 1) up to end of 4-year follow-up period (approximately 57 months) | All participants were analyzed for number of body regions with any new HO (new HO \> 0 mm\^3). The presence of HO across various body regions was analyzed using WBCT scan. Results are presented for overall ITT period |
| Secondary | Percentage of Participants With Flare-Ups | Month 12 | Flare-up as an event with one or more flare-up symptoms, and regardless of flare-up symptom onset. Flare-up was evaluated remotely, or by telephone or video-conferencing, unless the Investigator deemed that a site visit was necessary. |
| Secondary | Ratio of Flare-Up Per Participant-Month of Exposure | From Baseline (Day 1) up to end of 4-year follow-up period (approximately 57 months) | Flare-up as an event with one or more flare-up symptoms, and regardless of flare-up symptom onset. Flare-up was evaluated remotely, or by telephone or video-conferencing, unless the Investigator deemed that a site visit was necessary. The flare-up rate per participant-month exposure was analyzed using a negative binomial regression. Results are presented for overall ITT period. |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Annualized New Heterotopic Ossification (HO) | Baseline (within one month of screening/Day 1) and up to 24 months | The Principal FAS includes all enrolled participants in the Principal EP who had a baseline HO volume measurement and at least 1 post-baseline HO volume measurement in study PVO-1A-301. For study PVO-1A-001, the Principal FAS included participants enrolled in study PVO-1A-001 with available baseline and at least 1 post-baseline HO volume measurement. Study PVO-1A-001 was used as an external control. | cubic millimeters (mm^3) | MEAN |
| SECONDARY | Percentage of Participants With Any New HO | From Baseline (Day 1) up to end of 4-year follow-up period (approximately 57 months) | The Principal FAS included all enrolled participants in the Principal EP who had a baseline HO volume measurement and at least 1 post-baseline HO volume measurement in study PVO-1A-301. | percentage of participants | NUMBER |
| SECONDARY | Number of Body Regions With New HO | From Baseline (Day 1) up to end of 4-year follow-up period (approximately 57 months) | The Principal FAS included all enrolled participants in the Principal EP who had a baseline HO volume measurement and at least 1 post-baseline HO volume measurement in the study PVO-1A-301. Only data from the participants analyzed were reported. | body regions | MEAN |
| SECONDARY | Percentage of Participants With Flare-Ups | Month 12 | The Principal SS included all enrolled participants in the Principal EP set (ie, participants with the R206H ACVR1 mutation) receiving at least 1 dose of palovarotene in study PVO-1A-301. Only data from the participants analyzed at Month 12 reported. | percentage of participants | NUMBER |
| SECONDARY | Ratio of Flare-Up Per Participant-Month of Exposure | From Baseline (Day 1) up to end of 4-year follow-up period (approximately 57 months) | The Safety analysis set included all enrolled participants who received at least 1 dose of palovarotene in study PVO-1A-301. | ratio of flare-up | MEAN |
Source ClinicalTrials.gov
Results by group6
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Annualized New Heterotopic Ossification (HO) | Palovarotene | 9427.1 | Standard Error 3084.0 | cubic millimeters (mm^3) | |
| Annualized New Heterotopic Ossification (HO) | Untreated (PVO-1A-001) | 23720.2 | Standard Error 4850.0 | cubic millimeters (mm^3) | |
| Percentage of Participants With Any New HO | Palovarotene | 83.5 | percentage of participants | ||
| Number of Body Regions With New HO | Palovarotene | 3.0 | Standard Deviation 1.68 | body regions | |
| Percentage of Participants With Flare-Ups | Palovarotene | 64.6 | percentage of participants | ||
| Ratio of Flare-Up Per Participant-Month of Exposure | Palovarotene | 0.2 | Standard Deviation 0.40 | ratio of flare-up |
Source ClinicalTrials.gov
Participant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Palovarotene | 107 | |
| Overall Study | STARTED | Untreated (PVO-1A-001) | 114 | |
| Overall Study | COMPLETED | Palovarotene | 49 | |
| Overall Study | COMPLETED | Untreated (PVO-1A-001) | 33 | |
| Overall Study | NOT COMPLETED | Palovarotene | 58 | |
| Overall Study | NOT COMPLETED | Untreated (PVO-1A-001) | 81 |
Source ClinicalTrials.gov
Adverse events222
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 5 mg | 60 | 107 | 95 |
| other | Dry skin | Skin and subcutaneous tissue disorders | Palovarotene 20/10 mg | 37 | 81 | 77 |
| other | Dry skin | Skin and subcutaneous tissue disorders | Untreated (PVO-1A-001) | 0 | 114 | 0 |
| other | Alopecia | Skin and subcutaneous tissue disorders | Palovarotene 5 mg | 19 | 107 | 23 |
| other | Alopecia | Skin and subcutaneous tissue disorders | Palovarotene 20/10 mg | 21 | 81 | 28 |
| other | Alopecia | Skin and subcutaneous tissue disorders | Untreated (PVO-1A-001) | 0 | 114 | 0 |
| other | Drug eruption | Skin and subcutaneous tissue disorders | Palovarotene 5 mg | 15 | 107 | 24 |
| other | Drug eruption | Skin and subcutaneous tissue disorders | Palovarotene 20/10 mg | 26 | 81 | 40 |
| other | Drug eruption | Skin and subcutaneous tissue disorders | Untreated (PVO-1A-001) | 0 | 114 | |
| other | Pruritus | Skin and subcutaneous tissue disorders | Palovarotene 5 mg | 19 | 107 | 29 |
1 to 10 of 222Source ClinicalTrials.gov
See all 222 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Palovarotene 5 mg | 0 | 107 |
| Total, all-cause mortality | Palovarotene 20/10 mg | 0 | 81 |
| Total, all-cause mortality | Untreated (PVO-1A-001) | 1 | 114 |
| Total, other adverse events | Palovarotene 5 mg | 105 | 107 |
| Total, other adverse events | Palovarotene 20/10 mg | 77 | 81 |
| Total, other adverse events | Untreated (PVO-1A-001) | 0 | 114 |
| Total, serious adverse events | Palovarotene 5 mg | 34 | 107 |
| Total, serious adverse events | Palovarotene 20/10 mg | 19 | 81 |
| Total, serious adverse events | Untreated (PVO-1A-001) | 0 | 114 |
Source ClinicalTrials.gov
Papers3
The papers the trial lists on ClinicalTrials.gov.
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/NCT03312634
GET https://conductscience.org/api/trials/v1/NCT03312634/events?page=2