A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy
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ClinicalTrials.gov Identifier: NCT02310763 |
Recruitment Status :
Terminated
(Termination Date 30AUG2018: Reason for termination: Lack of Efficacy)
First Posted : December 8, 2014
Results First Posted : July 23, 2019
Last Update Posted : December 7, 2020
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Sponsor:
Pfizer
Information provided by (Responsible Party):
Pfizer
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Study Type | Interventional |
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Study Design | Allocation: Randomized; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment |
Condition |
Duchenne Muscular Dystrophy |
Interventions |
Biological: PF-06252616 Drug: Placebo |
Enrollment | 121 |
Participant Flow
Recruitment Details | |
Pre-assignment Details | A total of 162 participants were screened, 121 participants were enrolled in the study and assigned to 1 of 3 sequences. Only 120 participants received the study treatment and 1 participant withdrew prior to dosing. |
Arm/Group Title | Sequence 1 | Sequence 2 | Sequence 3 |
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Participants in this sequence received domagrozumab in a dose escalating fashion (5, 20 and 40 mg/kg) for 48 weeks (Period 1). At each dose level, dosing was administered over 2 hours by intravenous infusion every 4 weeks for a total of 16 weeks (4 doses). From Week 49 (Period 2), participants continued to receive domagrozumab at the maximum tolerated dose (40 mg/kg) every 4 weeks for additional 48 weeks or until early termination of the study. | Participants in this sequence received domagrozumab in a dose escalating fashion (5, 20 and 40mg/kg) for 48 weeks (Period 1). At each dose level, dosing was administered over 2 hours by intravenous infusion every 4 weeks for a total of 16 weeks (4 doses). From Week 49 (Period 2), participants received placebo for additional 48 weeks or until early termination of the study. | Participants in this sequence received placebo for 48 weeks (Period 1). From Week 49 (Period 2), participants received domagrozumab in a dose escalating fashion (5, 20 and 40 mg/kg) for additional 48 weeks or until early termination of the study. At each dose level, dosing was administered over 2 hours by intravenous infusion every 4 weeks for a total of 16 weeks (4 doses). |
Period Title: Period 1 (Weeks 1 to 48) | |||
Started | 41 | 39 | 40 |
Completed | 38 | 37 | 38 |
Not Completed | 3 | 2 | 2 |
Reason Not Completed | |||
Withdrawal by Subject | 1 | 1 | 1 |
Unable to comply with study procedures | 0 | 1 | 1 |
Lost to Follow-up | 1 | 0 | 0 |
Adverse Event | 1 | 0 | 0 |
Period Title: Period 2 (Weeks 49 to 96) | |||
Started | 38 | 37 | 38 |
Completed | 22 | 21 | 22 |
Not Completed | 16 | 16 | 16 |
Reason Not Completed | |||
Study terminated by sponsor | 16 | 16 | 16 |
Baseline Characteristics
Arm/Group Title | Sequence 1 | Sequence 2 | Sequence 3 | Total | |
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Participants in this sequence received domagrozumab in a dose escalating fashion (5, 20 and 40 mg/kg) for 48 weeks (Period 1). At each dose level, dosing was administered over 2 hours by intravenous infusion every 4 weeks for a total of 16 weeks (4 doses). From Week 49 (Period 2), participants continued to receive domagrozumab at the maximum tolerated dose (40 mg/kg) every 4 weeks for additional 48 weeks or until early termination of the study. | Participants in this sequence received domagrozumab in a dose escalating fashion (5, 20 and 40mg/kg) for 48 weeks (Period 1). At each dose level, dosing was administered over 2 hours by intravenous infusion every 4 weeks for a total of 16 weeks (4 doses). From Week 49 (Period 2), participants received placebo for additional 48 weeks or until early termination of the study. | Participants in this sequence received placebo for 48 weeks (Period 1). From Week 49 (Period 2), participants received domagrozumab in a dose escalating fashion (5, 20 and 40 mg/kg) for additional 48 weeks or until early termination of the study. At each dose level, dosing was administered over 2 hours by intravenous infusion every 4 weeks for a total of 16 weeks (4 doses). | Total of all reporting groups | |
Overall Number of Baseline Participants | 41 | 39 | 40 | 120 | |
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Baseline analysis population included all participants who received at least 1 dose of study treatment.
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Age, Categorical
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 41 participants | 39 participants | 40 participants | 120 participants | |
<=18 years |
41 100.0%
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39 100.0%
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40 100.0%
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120 100.0%
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Between 18 and 65 years |
0 0.0%
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0 0.0%
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0 0.0%
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0 0.0%
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>=65 years |
0 0.0%
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0 0.0%
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0 0.0%
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0 0.0%
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Age, Continuous
Mean (Standard Deviation) Unit of measure: Years |
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Number Analyzed | 41 participants | 39 participants | 40 participants | 120 participants | |
8.3 (1.9) | 8.5 (1.5) | 9.3 (2.3) | 8.7 (2.0) | ||
Sex: Female, Male
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 41 participants | 39 participants | 40 participants | 120 participants | |
Female |
0 0.0%
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0 0.0%
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0 0.0%
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0 0.0%
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Male |
41 100.0%
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39 100.0%
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40 100.0%
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120 100.0%
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Race/Ethnicity, Customized
Measure Type: Number Unit of measure: Participants |
Number Analyzed | 41 participants | 39 participants | 40 participants | 120 participants |
White | 33 | 33 | 35 | 101 | |
Black | 1 | 0 | 1 | 2 | |
Asian | 6 | 5 | 4 | 15 | |
Other | 1 | 1 | 0 | 2 |