Trial · NCT03188666
A Study to Examine the Safety, Tolerability and Effects on Abnormal Bone Formation of REGN2477 in Patients With Fibrodysplasia Ossificans Progressiva
NCT03188666, Phase 2, Completed, Regeneron Pharmaceuticals; results first posted 2022-12-02
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study to Examine the Safety, Tolerability and Effects on Abnormal Bone Formation of REGN2477 in Patients With Fibrodysplasia Ossificans Progressiva. Trials, record NCT03188666. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03188666
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Examine the Safety, Tolerability and Effects on Abnormal Bone Formation of REGN2477 in Patients With Fibrodysplasia Ossificans Progressiva
- NCT number
- NCT03188666
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 44 participants, actual
- Lead sponsor
- Regeneron Pharmaceuticals
- Conditions
- Fibrodysplasia Ossificans Progressiva
- Interventions
- REGN2477, Matching placebo
- First posted
- 2017-06-15
- Last updated
- 2022-12-02
- Other registries' IDs
- eudract:2016-005035-33
- Results first posted
- 2022-12-02
Source ClinicalTrials.gov
Registered outcomes43
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Period 1: Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious TEAEs | Up to Week 28 | Treatment-emergent adverse events (TEAEs) are adverse events not present at baseline or represent the exacerbation of a pre-existing condition during the on-treatment period. A serious TEAE was defined as any untoward medical occurrence that resulted in any of following outcomes not present at baseline or represent the exacerbation of a pre-existing condition during the on-treatment period: death, life-threatening, required initial/prolonged in-participant hospitalization, persistent/significant disability/incapacity, congenital anomaly/birth defect/considered as medically important event. Number of participants with TEAEs and Serious TEAEs are reported. |
| Primary | Period 1: Number of Participants With TEAEs by Severity | Up to Week 28 | Severity of TEAEs were graded as follows: Mild: Does not interfere in a significant manner with the participant's normal functioning level. It may be an annoyance. Prescription drugs are not ordinarily needed for relief of symptoms but may be given because of personality of the participants. Moderate: Produces some impairment of functioning but is not hazardous to health. It was uncomfortable or an embarrassment. Treatment for symptom may be needed. Severe: Produces significant impairment of functioning or incapacitation and was a definite hazard to the participant's health. Treatment for symptom may be given and/or participants hospitalized. Number of participants with TEAEs by severity is reported. |
| Primary | Period 1: Time-Weighted Average (Standardized Area Under the Curve [AUC]) of the Percent Change From Baseline in Total Lesion Activity by Fluorine-18-labeled Sodium Fluoride (18^F-NaF) Positron Emission Tomography (PET) at Week 28 (AHO) | Baseline and Week 28 | 18\^F-NaF PET is used to assess lesion and disease activity. Time-weighted average (standardized area under the curve \[AUC\]) of the percent change from baseline in total lesion activity by 18\^F-NaF PET up to Week 28 in AHO analysis set is reported. |
| Primary | Period 1: Percent Change From Baseline in the Total Volume of HO Lesions as Assessed by Computed Tomography (CT) at Week 28 (AHO) | Week 28 | CT is a diagnostic imaging test used to create detailed images of internal organs, bones, soft tissue, and blood vessels. CT scan acquired contemporaneously to the PET scan. Percent change from baseline in the total volume of HO lesions as assessed by CT during Period 1 at Week 28 is reported. |
| Primary | Period 2: Number of New HO Lesions as Assessed by CT at Week 56 Relative to Week 28 Scan (AHO COVID-19 mITT) | Week 28, Week 56 | CT is a diagnostic imaging test used to create detailed images of internal organs, bones, soft tissue, and blood vessels. CT scan acquired contemporaneously to the PET scan. HO detectable by CT that developed after baseline are referred to as "new HO lesions." Number of new HO lesions as assessed by CT at Week 56 relative to Week 28 scan is reported. |
| Primary | Period 1: Time-weighted Average (Standardized AUC) of the Percent Change From Baseline in Total Lesion Activity Assessed by 18^F-NaF PET at Week 28 (AHOC) | Week 28 | 18\^F-NaF PET is used to assess lesion and disease activity. Time-weighted average (Standardized AUC) of the percent change from baseline in total lesion activity as assessed by 18\^F-NaF PET in Active HO Classic ACVR1 Mutation (AHOC) analysis set up to Week 28 is reported. |
| Primary | Period 1: Percent Change From Baseline in the Total Volume of HO Lesions as Assessed by CT at Week 28 (AHOC) | Week 28 | CT is a diagnostic imaging test used to create detailed images of internal organs, bones, soft tissue, and blood vessels. CT scan acquired contemporaneously to the PET scan. Percent change from baseline in the total volume of HO lesions was assessed by CT at Week 28 in AHOC analysis set is reported. |
| Secondary | Period 1: Time-weighted Average (Standardized AUC) of the Change From Baseline in Daily Pain Due to Fibrodysplasia Ossificans Progressiva (FOP) Assessed by Daily Numeric Rating Scale (NRS) at Week 28 (AHO) | Week 28 | The pain NRS is a patient reported outcome (PRO) used by participants to rate their pain associated with FOP. Participants were asked to rate their pain on a scale that ranges from "0" (no pain) to "10" (worst possible pain), where the highest score indicated worst outcome. Time-weighted average (Standardized AUC) of the change from baseline in daily pain due to FOP assessed by daily NRS at Week 28 in AHO analysis set is reported. |
| Secondary | Period 1: Time-weighted Average (Standardized AUC) of the Change From Baseline in Daily Pain Due to FOP, Assessed by Daily NRS at Week 28 (AHOC) | Week 28 | The pain NRS is a PRO used by participants to rate their pain associated with FOP. Participants were asked to rate their pain on a scale that ranges from "0" (no pain) to "10" (worst possible pain), where the highest score indicated worst outcome. Time-Weighted average (standardized AUC) of the change from baseline in daily pain due to FOP assessed by daily NRS at Week 28 in AHOC analysis set is reported. |
| Secondary | Period 1: Percent Change From Baseline in 18^F-NaF SUVmax of Individual Active HO Site(s) Assessed by 18^F-NaF PET at Week 8 (AHOC) | Week 8 | Standardized uptake value max (SUVmax) was a measurement of the maximum radiopharmaceutical uptake within the volume of interest. Relative accuracy of a particular radiotracer in a particular tissue is determined by expressing the absolute accuracy (obtained in the primary outcome measure) in terms of percent difference between SUVmax values obtained from PET/CT. Percent Change in 18\^F-NaF SUVmax of Individual Active HO Site(s) assessed by 18\^F-NaF PET in AHOC analysis set is reported. |
1 to 10 of 43Source ClinicalTrials.gov
See all 43 registered outcomesOutcomes43
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Period 1: Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious TEAEs | Up to Week 28 | The safety analysis set included all randomized participants who received any study drug and was analyzed as treated. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Period 1: Number of Participants With TEAEs by Severity | Up to Week 28 | The safety analysis set included all randomized participants who received any study drug and was analyzed as treated. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Period 1: Time-Weighted Average (Standardized Area Under the Curve [AUC]) of the Percent Change From Baseline in Total Lesion Activity by Fluorine-18-labeled Sodium Fluoride (18^F-NaF) Positron Emission Tomography (PET) at Week 28 (AHO) | Baseline and Week 28 | Baseline-active heterotopic ossification analysis set (AHO) included all randomized participants who had at least one active HO lesion at baseline; and was based on the treatment allocated (as randomized). | Percent Change | LEAST_SQUARES_MEAN |
| PRIMARY | Period 1: Percent Change From Baseline in the Total Volume of HO Lesions as Assessed by Computed Tomography (CT) at Week 28 (AHO) | Week 28 | AHO analysis set included all randomized participants who had at least one active HO lesion at baseline was based on the treatment allocated (as randomized). | Percent Change | LEAST_SQUARES_MEAN |
| PRIMARY | Period 2: Number of New HO Lesions as Assessed by CT at Week 56 Relative to Week 28 Scan (AHO COVID-19 mITT) | Week 28, Week 56 | COVID-19 mITT: All AHO participants who received a treatment in Period 2 for whom at least 1 post-Week 28 scan was collected and the period between any consecutive doses was less than 9 weeks (63 days) before the 1st post-week 28 scan. Overall number of participants analyzed are the participants who were evaluable for this outcome measure. Separate analyses were conducted from week 56 relative to baseline for participants on treatment during both periods (REGN2477/REGN2477) as planned. | New HO Lesions | NUMBER |
| PRIMARY | Period 1: Time-weighted Average (Standardized AUC) of the Percent Change From Baseline in Total Lesion Activity Assessed by 18^F-NaF PET at Week 28 (AHOC) | Week 28 | AHOC analysis set included all randomized participants with the classic ACVR1 \[R206H\] mutation and who had at least one AHO at baseline, as defined by 18\^F-NaF PET positivity; and was based on the treatment allocated (as randomized). Overall number of participants analyzed are the participants who were evaluable for this outcome measure. | Percent Change | LEAST_SQUARES_MEAN |
| PRIMARY | Period 1: Percent Change From Baseline in the Total Volume of HO Lesions as Assessed by CT at Week 28 (AHOC) | Week 28 | AHOC analysis set included all randomized participants with the classic ACVR1 \[R206H\] mutation and who had at least one AHO at baseline, as defined by 18\^F-NaF PET positivity; and was based on the treatment allocated (as randomized). Overall number of participants analyzed are the participants who were evaluable for this outcome measure. | Percent Change | LEAST_SQUARES_MEAN |
| SECONDARY | Period 1: Time-weighted Average (Standardized AUC) of the Change From Baseline in Daily Pain Due to Fibrodysplasia Ossificans Progressiva (FOP) Assessed by Daily Numeric Rating Scale (NRS) at Week 28 (AHO) | Week 28 | AHO analysis set included all randomized participants who had at least one active HO lesion at baseline; based on the treatment allocated (as randomized). Overall number of participants analyzed are the participants who were evaluable for this outcome measure. | Score on a Scale | LEAST_SQUARES_MEAN |
| SECONDARY | Period 1: Time-weighted Average (Standardized AUC) of the Change From Baseline in Daily Pain Due to FOP, Assessed by Daily NRS at Week 28 (AHOC) | Week 28 | AHOC analysis set included all randomized participants with the classic ACVR1 \[R206H\] mutation and who had at least one AHO at baseline, as defined by 18\^F-NaF PET positivity; and was based on the treatment allocated (as randomized). Overall number of participants analyzed are the participants who were evaluable for this outcome measure. | Score on a Scale | LEAST_SQUARES_MEAN |
| SECONDARY | Period 1: Percent Change From Baseline in 18^F-NaF SUVmax of Individual Active HO Site(s) Assessed by 18^F-NaF PET at Week 8 (AHOC) | Week 8 | AHOC analysis set included all randomized participants with the classic ACVR1 \[R206H\] mutation and who had at least one AHO at baseline, as defined by 18\^F-NaF PET positivity; and was based on the treatment allocated (as randomized). Overall number of participants analyzed are the participants who were evaluable for this outcome measure. | Percent Change | MEAN |
1 to 10 of 43Source ClinicalTrials.gov
See all 43 outcomesResults by group84
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Period 1: Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious TEAEs | Placebo | Participants with at least one TEAE | 24 | Participants | |
| Period 1: Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious TEAEs | REGN2477 10 mg/kg Q4W | Participants with at least one TEAE | 20 | Participants | |
| Period 1: Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious TEAEs | Placebo | Participants with at least one serious TEAE | 2 | Participants | |
| Period 1: Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious TEAEs | REGN2477 10 mg/kg Q4W | Participants with at least one serious TEAE | 4 | Participants | |
| Period 1: Number of Participants With TEAEs by Severity | Placebo | Participants with at least one Mild TEAE | 9 | Participants | |
| Period 1: Number of Participants With TEAEs by Severity | REGN2477 10 mg/kg Q4W | Participants with at least one Mild TEAE | 7 | Participants | |
| Period 1: Number of Participants With TEAEs by Severity | Placebo | Participants with at least one Moderate TEAE | 12 | Participants | |
| Period 1: Number of Participants With TEAEs by Severity | REGN2477 10 mg/kg Q4W | Participants with at least one Moderate TEAE | 10 | Participants | |
| Period 1: Number of Participants With TEAEs by Severity | Placebo | Participants with at least one Severe TEAE | 3 | Participants | |
| Period 1: Number of Participants With TEAEs by Severity | REGN2477 10 mg/kg Q4W | Participants with at least one Severe TEAE | 3 | Participants |
1 to 10 of 84Source ClinicalTrials.gov
See all 84 results by groupParticipant flow36
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Period 1 (28 Weeks Double-blind) | STARTED | Placebo | 24 | |
| Period 1 (28 Weeks Double-blind) | STARTED | REGN2477 10 mg/kg Q4W | 20 | |
| Period 1 (28 Weeks Double-blind) | STARTED | Placebo/REGN2477 10 mg/kg Q4W | 0 | |
| Period 1 (28 Weeks Double-blind) | STARTED | REGN2477/REGN2477 10 mg/kg Q4W | 0 | |
| Period 1 (28 Weeks Double-blind) | COMPLETED | Placebo | 24 | |
| Period 1 (28 Weeks Double-blind) | COMPLETED | REGN2477 10 mg/kg Q4W | 19 | |
| Period 1 (28 Weeks Double-blind) | COMPLETED | Placebo/REGN2477 10 mg/kg Q4W | 0 | |
| Period 1 (28 Weeks Double-blind) | COMPLETED | REGN2477/REGN2477 10 mg/kg Q4W | 0 | |
| Period 1 (28 Weeks Double-blind) | NOT COMPLETED | Placebo | 0 | |
| Period 1 (28 Weeks Double-blind) | NOT COMPLETED | REGN2477 10 mg/kg Q4W | 1 |
1 to 10 of 36Source ClinicalTrials.gov
See all 36 participant flowAdverse events992
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 | Arthralgia | Musculoskeletal and connective tissue disorders | Placebo | 9 | 24 | 13 |
| other | Arthralgia | Musculoskeletal and connective tissue disorders | REGN2477 10 mg/kg Q4W | 7 | 20 | 8 |
| other | Arthralgia | Musculoskeletal and connective tissue disorders | Placebo/REGN2477 10 mg/kg Q4W | 8 | 24 | 16 |
| other | Arthralgia | Musculoskeletal and connective tissue disorders | REGN2477/REGN2477 10 mg/kg Q4W | 9 | 19 | 24 |
| other | Pain in extremity | Musculoskeletal and connective tissue disorders | Placebo | 9 | 24 | 19 |
| other | Pain in extremity | Musculoskeletal and connective tissue disorders | REGN2477 10 mg/kg Q4W | 5 | 20 | 6 |
| other | Pain in extremity | Musculoskeletal and connective tissue disorders | Placebo/REGN2477 10 mg/kg Q4W | 6 | 24 | 18 |
| other | Pain in extremity | Musculoskeletal and connective tissue disorders | REGN2477/REGN2477 10 mg/kg Q4W | 8 | 19 | 22 |
| other | Myalgia | Musculoskeletal and connective tissue disorders | Placebo | 3 | 24 | 3 |
| other | Myalgia | Musculoskeletal and connective tissue disorders | REGN2477 10 mg/kg Q4W | 1 | 20 | 1 |
1 to 10 of 992Source ClinicalTrials.gov
See all 992 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Placebo | 0 | 24 |
| Total, all-cause mortality | REGN2477 10 mg/kg Q4W | 0 | 20 |
| Total, all-cause mortality | Placebo/REGN2477 10 mg/kg Q4W | 2 | 24 |
| Total, all-cause mortality | REGN2477/REGN2477 10 mg/kg Q4W | 3 | 19 |
| Total, other adverse events | Placebo | 24 | 24 |
| Total, other adverse events | REGN2477 10 mg/kg Q4W | 20 | 20 |
| Total, other adverse events | Placebo/REGN2477 10 mg/kg Q4W | 24 | 24 |
| Total, other adverse events | REGN2477/REGN2477 10 mg/kg Q4W | 18 | 19 |
| Total, serious adverse events | Placebo | 2 | 24 |
| Total, serious adverse events | REGN2477 10 mg/kg Q4W | 4 | 20 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers6
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/NCT03188666
GET https://conductscience.org/api/trials/v1/NCT03188666/registered?page=2