Trial · NCT02310763
A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy
NCT02310763, Phase 2, Terminated, Pfizer; results first posted 2019-07-23
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy. Trials, record NCT02310763. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02310763
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy
- NCT number
- NCT02310763
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 121 participants, actual
- Lead sponsor
- Pfizer
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- PF-06252616, Placebo
- First posted
- 2014-12-08
- Last updated
- 2020-12-07
- Other registries' IDs
- eudract:2014-002072-92
- Results first posted
- 2019-07-23
- Limitations and caveats
This study was terminated because the primary efficacy objective (the efficacy of treatment with domagrozumab based on a mean change from baseline on 4 Stair Climb as compared to placebo following 48 weeks of treatment) was not met.
Source ClinicalTrials.gov
Registered outcomes75
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment-emergent Adverse Events (TEAEs) by Week 49 | Study Day 1 to Week 49 visit | An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a product; the event did not need to have a causal relationship with the treatment. A serious adverse event (SAE) was any untoward medical occurrence at any dose that resulted in death; was life threatening; required inpatient hospitalization or prolongation of existing hospitalization; resulted in persistent or significant disability/incapacity; resulted in congenital anomaly/birth defect. AEs included both SAEs and AEs. TEAEs were AEs occurred following the start of treatment or AEs increasing in severity during treatment. Severe TEAEs were TEAEs that interfered significantly with participants' usual function. Treatment-related TEAEs were determined by the investigator. |
| Primary | Number of Participants Who Discontinued From the Study Due to TEAEs by Week 49 | Study Day 1 to Week 49 visit | An AE was any untoward medical occurrence in a clinical investigation participant administered a product; the event did not need to have a causal relationship with the treatment. TEAEs were AEs occurred following the start of treatment or AEs increasing in severity during treatment. Treatment-related TEAEs were determined by the investigator. |
| Primary | Number of Participants With Dose Reduced or Temporary Discontinuation Due to TEAEs by Week 49 | Study Day 1 to Week 49 visit | An AE was any untoward medical occurrence in a clinical investigation participant administered a product; the event did not need to have a causal relationship with the treatment. TEAEs were AEs occurred following the start of treatment or AEs increasing in severity during treatment. Treatment-related TEAEs were determined by the investigator. |
| Primary | Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormality) by Week 49 - Hematology | Baseline to Week 49 visit | Hematology evaluation included: hemoglobin, hematocrit, red blood cell (RBC) count, platelets, RBC morphology, white blood cell (WBC) count, absolute lymphocytes, absolute atypical lymphocytes, absolute total neutrophils, absolute total neutrophils count, absolute band cells, absolute basophils, absolute eosinophils, absolute monocytes and absolute myelocytes. |
| Primary | Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormality) by Week 49 - Coagulation | Baseline to Week 49 visit | Coagulation evaluation included activated partial thromboplastin time (aPTT) and prothrombin time (PT). |
| Primary | Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormality) by Week 49 - Liver Function | Baseline to Week 49 visit | Liver function evaluation included: total/direct/indirect bilirubin, aspartate aminotransferase (AST), alanine aminotransferase (ALT), gamma-glutamyl transferase (GGT), alkaline phosphatase, total protein, albumin and glutamate dehydrogenase. |
| Primary | Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormality) by Week 49 - Renal Function | Baseline to Week 49 visit | Renal function evaluation included: blood urea nitrogen (BUN), creatinine and uric acid. |
| Primary | Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormality) by Week 49 - Electrolytes | Baseline to Week 49 visit | Electrolytes evaluation included: sodium, potassium, chloride, calcium, phosphate, bicarbonate, ferritin, transferrin saturation, iron, iron binding capacity and unsaturated iron binding capacity. Number of participants with iron abnormalities was reported in different age groups. |
| Primary | Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormality) by Week 49 - Hormones | Baseline to Week 49 visit | Hormone evaluations included free thyroxine (T4), thyroid stimulating hormone (TSH), lutenizing hormone (LH), follicle stimulating hormone (FSH), and androstenedione. Numbers of participants with abnormalities of LH, FSH and androstenedione were reported in different age groups. |
| Primary | Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormality) by Week 49 - Clinical Chemistry | Baseline to Week 49 visit | Clinical chemistry evaluation included glucose, creatine kinase (CK), troponin I, and amylase. |
1 to 10 of 75Source ClinicalTrials.gov
See all 75 registered outcomesOutcomes75
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Treatment-emergent Adverse Events (TEAEs) by Week 49 | Study Day 1 to Week 49 visit | The analysis population included all participants who received at least 1 dose of investigational drug. Number of participants analyzed signifies number of participants who were evaluable for this outcome measure. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants Who Discontinued From the Study Due to TEAEs by Week 49 | Study Day 1 to Week 49 visit | The analysis population included all participants who received at least 1 dose of investigational drug. Number of participants analyzed signifies number of participants who were evaluable for this outcome measure. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Dose Reduced or Temporary Discontinuation Due to TEAEs by Week 49 | Study Day 1 to Week 49 visit | The analysis population included all participants who received at least 1 dose of investigational drug. Number of participants analyzed signifies number of participants who were evaluable for this outcome measure. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormality) by Week 49 - Hematology | Baseline to Week 49 visit | This analysis population included all participants who received at least 1 dose of investigational drug. Number of participants analyzed signifies number of participants who were evaluable for this outcome measure. Number analyzed refers to number of participants evaluable for specified rows of categories. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormality) by Week 49 - Coagulation | Baseline to Week 49 visit | This analysis population included all participants who received at least 1 dose of investigational drug. Number of participants analyzed signifies number of participants who were evaluable for this outcome measure. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormality) by Week 49 - Liver Function | Baseline to Week 49 visit | This analysis population included all participants who received at least 1 dose of investigational drug. Number of participants analyzed signifies number of participants who were evaluable for this outcome measure. Number analyzed refers to number of participants evaluable for specified rows of categories. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormality) by Week 49 - Renal Function | Baseline to Week 49 visit | This analysis population included all participants who received at least 1 dose of investigational drug. Number of participants analyzed signifies number of participants who were evaluable for this outcome measure. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormality) by Week 49 - Electrolytes | Baseline to Week 49 visit | This analysis population included all participants who received at least 1 dose of investigational drug. Number of participants analyzed signifies number of participants who were evaluable for this outcome measure. Number analyzed refers to number of participants evaluable for specified rows of categories. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormality) by Week 49 - Hormones | Baseline to Week 49 visit | This analysis population included all participants who received at least 1 dose of investigational drug. Number of participants analyzed signifies number of participants who were evaluable for this outcome measure. Number analyzed refers to number of participants evaluable for specified rows of categories. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormality) by Week 49 - Clinical Chemistry | Baseline to Week 49 visit | This analysis population included all participants who received at least 1 dose of investigational drug. Number of participants analyzed signifies number of participants who were evaluable for this outcome measure. Number analyzed refers to number of participants evaluable for specified rows of categories. | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 75Source ClinicalTrials.gov
See all 75 outcomesResults by group1,246
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) by Week 49 | Placebo | All-causalities TEAE | 38 | Participants | |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) by Week 49 | Domagrozumab 5 mg/kg | All-causalities TEAE | 66 | Participants | |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) by Week 49 | Domagrozumab 20 mg/kg | All-causalities TEAE | 57 | Participants | |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) by Week 49 | Domagrozumab 40 mg/kg | All-causalities TEAE | 59 | Participants | |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) by Week 49 | Placebo | Treatment-related TEAE | 14 | Participants | |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) by Week 49 | Domagrozumab 5 mg/kg | Treatment-related TEAE | 18 | Participants | |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) by Week 49 | Domagrozumab 20 mg/kg | Treatment-related TEAE | 14 | Participants | |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) by Week 49 | Domagrozumab 40 mg/kg | Treatment-related TEAE | 16 | Participants | |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) by Week 49 | Placebo | All-causalities serious TEAE | 0 | Participants | |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) by Week 49 | Domagrozumab 5 mg/kg | All-causalities serious TEAE | 1 | Participants |
1 to 10 of 1,246Source ClinicalTrials.gov
See all 1,246 results by groupParticipant flow18
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Period 1 (Weeks 1 to 48) | STARTED | Sequence 1 | 41 | |
| Period 1 (Weeks 1 to 48) | STARTED | Sequence 2 | 39 | |
| Period 1 (Weeks 1 to 48) | STARTED | Sequence 3 | 40 | |
| Period 1 (Weeks 1 to 48) | COMPLETED | Sequence 1 | 38 | |
| Period 1 (Weeks 1 to 48) | COMPLETED | Sequence 2 | 37 | |
| Period 1 (Weeks 1 to 48) | COMPLETED | Sequence 3 | 38 | |
| Period 1 (Weeks 1 to 48) | NOT COMPLETED | Sequence 1 | 3 | |
| Period 1 (Weeks 1 to 48) | NOT COMPLETED | Sequence 2 | 2 | |
| Period 1 (Weeks 1 to 48) | NOT COMPLETED | Sequence 3 | 2 | |
| Period 2 (Weeks 49 to 96) | STARTED | Sequence 1 | 38 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 participant flowAdverse events368
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 | Abdominal pain | Gastrointestinal disorders | Domagrozumab 5 mg/kg, Period 1 | 3 | 80 | 3 |
| other | Abdominal pain | Gastrointestinal disorders | Domagrozumab 20 mg/kg, Period 1 | 1 | 78 | 8 |
| other | Abdominal pain | Gastrointestinal disorders | Domagrozumab 40 mg/kg, Period 1 | 1 | 76 | 1 |
| other | Abdominal pain | Gastrointestinal disorders | Placebo, Period 1 | 3 | 40 | 3 |
| other | Abdominal pain | Gastrointestinal disorders | Domagrozumab 5 mg/kg, Period 2 | 0 | 38 | 0 |
| other | Abdominal pain | Gastrointestinal disorders | Domagrozumab 20 mg/kg, Period 2 | 1 | 38 | 1 |
| other | Abdominal pain | Gastrointestinal disorders | Domagrozumab 40 mg/kg, Period 2 | 0 | 66 | 0 |
| other | Abdominal pain | Gastrointestinal disorders | Placebo, Period 2 | 0 | 37 | 0 |
| other | Abdominal pain upper | Gastrointestinal disorders | Domagrozumab 5 mg/kg, Period 1 | 3 | 80 | 6 |
| other | Abdominal pain upper | Gastrointestinal disorders | Domagrozumab 20 mg/kg, Period 1 | 3 | 78 | 4 |
1 to 10 of 368Source ClinicalTrials.gov
See all 368 adverse eventsAdverse event totals24
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Domagrozumab 5 mg/kg, Period 1 | 0 | 80 |
| Total, all-cause mortality | Domagrozumab 20 mg/kg, Period 1 | 0 | 78 |
| Total, all-cause mortality | Domagrozumab 40 mg/kg, Period 1 | 0 | 76 |
| Total, all-cause mortality | Placebo, Period 1 | 0 | 40 |
| Total, all-cause mortality | Domagrozumab 5 mg/kg, Period 2 | 0 | 38 |
| Total, all-cause mortality | Domagrozumab 20 mg/kg, Period 2 | 0 | 38 |
| Total, all-cause mortality | Domagrozumab 40 mg/kg, Period 2 | 0 | 66 |
| Total, all-cause mortality | Placebo, Period 2 | 0 | 37 |
| Total, other adverse events | Domagrozumab 5 mg/kg, Period 1 | 58 | 80 |
| Total, other adverse events | Domagrozumab 20 mg/kg, Period 1 | 43 | 78 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 adverse event totalsPapers8
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 comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Domagrozumab vs Placebo: Number of Participants With Vital Signs Findings Reported as SAEs by Week 49 |
Source Trial comparisons
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/NCT02310763
GET https://conductscience.org/api/trials/v1/NCT02310763/registered?page=2