Trial · NCT04371666
Phase 3 Trial of Pamrevlumab or Placebo With Systemic Corticosteroids in Participants With Non-ambulatory Duchenne Muscular Dystrophy (DMD)
NCT04371666, Phase 3, Terminated, Kyntra Bio; results first posted 2024-03-12
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Phase 3 Trial of Pamrevlumab or Placebo With Systemic Corticosteroids in Participants With Non-ambulatory Duchenne Muscular Dystrophy (DMD). Trials, record NCT04371666. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04371666
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase 3 Trial of Pamrevlumab or Placebo With Systemic Corticosteroids in Participants With Non-ambulatory Duchenne Muscular Dystrophy (DMD)
- NCT number
- NCT04371666
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 98 participants, actual
- Lead sponsor
- Kyntra Bio
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Pamrevlumab, Placebo, Corticosteroids
- First posted
- 2020-05-01
- Last updated
- 2024-03-12
- Other registries' IDs
- eudract:2020-000698-26
- Results first posted
- 2024-03-12
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in the Total Score of Performance of Upper Limb (PUL) 2.0 Version at Week 52 | Baseline, Week 52 | The PUL module is an observer-administered performance battery of upper extremity mobility tasks for the shoulder (upper, 6 items, 12 points), elbow (middle, 9 items, 17 points) and wrist/hand (distal, 7 items, 13 points). Higher scores indicate higher level of function. Total score ranges from 0-42 points and is the sum of the scores for the 3 subscales. Analysis was done using a random coefficient model (RCM), which included fixed effects of time (as a continuous variable), treatment, and treatment-by-time interaction, with baseline ordinal PUL entry score as covariate. |
| Secondary | Change From Baseline in Percent Predicted Forced Vital Capacity (ppFVC) at Week 52, Assessed by Spirometry | Baseline, Week 52 | FVC is a standard pulmonary function test used to quantify respiratory muscle weakness. FVC was the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in liters. Predicted FVC is based on a formula using sex, age and height of a person, and is an estimate of healthy lung capacity. Percent of predicted FVC = (observed value)/(predicted value) \* 100%. Analysis was done using an RCM, which included fixed effects of time (as a continuous variable), treatment, and treatment-by-time interaction, with baseline value as covariate. |
| Secondary | Change From Baseline in the Grip Strength of the Hands at Week 52, Assessed by Hand Held Myometry (HHM) | Baseline, Week 52 | The HHM was used to measure distal upper arm strength (grip strength). Data has been presented by dominant and non-dominant hand. Grip Strength was analyzed using a MMRM with fixed effects for treatment, visit (as a factor), treatment-by-visit interaction, and covariates (baseline values). |
| Secondary | Change From Baseline in Left Ventricular Ejection Fraction Percentage (LVEF %) at Week 52, Assessed by Magnetic Resonance Imaging (MRI) | Baseline, Week 52 | LVEF% is an important measure of cardiac function. LVEF is a fraction of blood (in percent) pumped out of the left ventricle of the heart (the main pumping chamber). The LVEF% was analyzed using an analysis of covariance (ANCOVA) model with treatment and baseline value. |
| Secondary | Change From Baseline in Percent Predicted Peak Expiratory Flow (ppPEF) at Week 52, Assessed by Spirometry | Baseline, Week 52 | The ppPEF is a measure of the maximal or peak flow produced during an exhalation with maximal effort and, as such, is the most effort-dependent measure of lung function. The ppFEV1 was analyzed using an RCM including fixed effects of time, treatment, and treatment-by-time interaction, with baseline as covariate. |
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 | Change From Baseline in the Total Score of Performance of Upper Limb (PUL) 2.0 Version at Week 52 | Baseline, Week 52 | The modified intent-to-treat (mITT) population included all randomized participants with a PUL Entry score ≥2 (excluding PUL entry scores of 1) at baseline. Here, 'Overall number of participants analyzed' = participants evaluable for specified outcome measure. | units on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Percent Predicted Forced Vital Capacity (ppFVC) at Week 52, Assessed by Spirometry | Baseline, Week 52 | The mITT population included all randomized participants with a PUL Entry score ≥2 (excluding PUL entry scores of 1) at baseline. Here, 'Overall number of participants analyzed' = participants evaluable for specified outcome measure. | percentage of predicted FVC | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in the Grip Strength of the Hands at Week 52, Assessed by Hand Held Myometry (HHM) | Baseline, Week 52 | The mITT population included all randomized participants with a PUL Entry score ≥2 (excluding PUL entry scores of 1) at baseline. Here, 'Overall number of participants analyzed' = participants evaluable for specified outcome measure. 'Number analyzed' = participants evaluable for specified category. | newton | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Left Ventricular Ejection Fraction Percentage (LVEF %) at Week 52, Assessed by Magnetic Resonance Imaging (MRI) | Baseline, Week 52 | The mITT population included all randomized participants with a PUL Entry score ≥2 (excluding PUL entry scores of 1) at baseline. Here, 'Overall number of participants analyzed' = participants evaluable for specified outcome measure. | percentage of LVEF | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Percent Predicted Peak Expiratory Flow (ppPEF) at Week 52, Assessed by Spirometry | Baseline, Week 52 | The ITT set included all randomized participants. Here, 'Overall number of participants analyzed' = participants evaluable for specified outcome measure. | percentage of predicted PEF | LEAST_SQUARES_MEAN |
Source ClinicalTrials.gov
Results by group12
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in the Total Score of Performance of Upper Limb (PUL) 2.0 Version at Week 52 | Pamrevlumab | -2.036 | Standard Error 0.4471 | units on a scale | |
| Change From Baseline in the Total Score of Performance of Upper Limb (PUL) 2.0 Version at Week 52 | Placebo | -2.119 | Standard Error 0.3367 | units on a scale | |
| Change From Baseline in Percent Predicted Forced Vital Capacity (ppFVC) at Week 52, Assessed by Spirometry | Pamrevlumab | -8.349 | Standard Error 1.5760 | percentage of predicted FVC | |
| Change From Baseline in Percent Predicted Forced Vital Capacity (ppFVC) at Week 52, Assessed by Spirometry | Placebo | -5.989 | Standard Error 1.2233 | percentage of predicted FVC | |
| Change From Baseline in the Grip Strength of the Hands at Week 52, Assessed by Hand Held Myometry (HHM) | Pamrevlumab | Grip Strength by Dominant Hand | -7.570 | Standard Error 2.0989 | newton |
| Change From Baseline in the Grip Strength of the Hands at Week 52, Assessed by Hand Held Myometry (HHM) | Placebo | Grip Strength by Dominant Hand | -0.072 | Standard Error 2.2065 | newton |
| Change From Baseline in the Grip Strength of the Hands at Week 52, Assessed by Hand Held Myometry (HHM) | Pamrevlumab | Grip Strength by Nondominant Hand | -7.627 | Standard Error 1.8893 | newton |
| Change From Baseline in the Grip Strength of the Hands at Week 52, Assessed by Hand Held Myometry (HHM) | Placebo | Grip Strength by Nondominant Hand | -0.012 | Standard Error 1.8546 | newton |
| Change From Baseline in Left Ventricular Ejection Fraction Percentage (LVEF %) at Week 52, Assessed by Magnetic Resonance Imaging (MRI) | Pamrevlumab | -0.499 | Standard Error 0.9750 | percentage of LVEF | |
| Change From Baseline in Left Ventricular Ejection Fraction Percentage (LVEF %) at Week 52, Assessed by Magnetic Resonance Imaging (MRI) | Placebo | -1.114 | Standard Error 0.9204 | percentage of LVEF |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 results by groupParticipant flow16
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| DB Treatment Period (52 Weeks) | STARTED | Pamrevlumab | 49 | |
| DB Treatment Period (52 Weeks) | STARTED | Placebo | 49 | |
| DB Treatment Period (52 Weeks) | Received at Least 1 Dose of Study Drug | Pamrevlumab | 48 | |
| DB Treatment Period (52 Weeks) | Received at Least 1 Dose of Study Drug | Placebo | 49 | |
| DB Treatment Period (52 Weeks) | COMPLETED | Pamrevlumab | 44 | |
| DB Treatment Period (52 Weeks) | COMPLETED | Placebo | 46 | |
| DB Treatment Period (52 Weeks) | NOT COMPLETED | Pamrevlumab | 5 | |
| DB Treatment Period (52 Weeks) | NOT COMPLETED | Placebo | 3 | |
| OLE (Maximum Exposure: 93.4 Weeks) | STARTED | Pamrevlumab | 43 | |
| OLE (Maximum Exposure: 93.4 Weeks) | STARTED | Placebo | 43 |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 participant flowAdverse events147
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 | Cardiomyopathy | Cardiac disorders | DB Period: Pamrevlumab | 1 | 48 | |
| other | Cardiomyopathy | Cardiac disorders | DB Period: Placebo | 5 | 49 | |
| other | Cardiomyopathy | Cardiac disorders | OLE Period: Pamrevlumab | 0 | 86 | |
| other | Cataract | Eye disorders | DB Period: Pamrevlumab | 3 | 48 | |
| other | Cataract | Eye disorders | DB Period: Placebo | 0 | 49 | |
| other | Cataract | Eye disorders | OLE Period: Pamrevlumab | 0 | 86 | |
| other | Vomiting | Gastrointestinal disorders | DB Period: Pamrevlumab | 10 | 48 | |
| other | Vomiting | Gastrointestinal disorders | DB Period: Placebo | 5 | 49 | |
| other | Vomiting | Gastrointestinal disorders | OLE Period: Pamrevlumab | 5 | 86 | |
| other | Nausea | Gastrointestinal disorders | DB Period: Pamrevlumab | 8 | 48 |
1 to 10 of 147Source ClinicalTrials.gov
See all 147 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | DB Period: Pamrevlumab | 1 | 48 |
| Total, all-cause mortality | DB Period: Placebo | 0 | 49 |
| Total, all-cause mortality | OLE Period: Pamrevlumab | 0 | 86 |
| Total, other adverse events | DB Period: Pamrevlumab | 42 | 48 |
| Total, other adverse events | DB Period: Placebo | 44 | 49 |
| Total, other adverse events | OLE Period: Pamrevlumab | 53 | 86 |
| Total, serious adverse events | DB Period: Pamrevlumab | 8 | 48 |
| Total, serious adverse events | DB Period: Placebo | 6 | 49 |
| Total, serious adverse events | OLE Period: Pamrevlumab | 5 | 86 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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/NCT04371666
GET https://conductscience.org/api/trials/v1/NCT04371666/events?page=2