Trial reportMuscle & nerve2025
Relationship Between Quantitative Magnetic Resonance Imaging Measures and Functional Changes in Patients With Duchenne Muscular Dystrophy.
Trial report in Muscle & nerve, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02310763 (A PHASE 2 RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTIPLE ASCENDING DOSE STUDY TO EVALUATE THE SAFETY, EFFICACY, PHARMACOKINETICS AND PHARMACODYNAMICS OF PF-06252616 IN AMBULATORY BOYS WITH DUCHENNE MUSCULAR DYSTROPHY), which is not on this map. Cited by 3 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A phase 2 randomized, double-blind, placebo-controlled, multiple ascending dose study to evaluate the safety, efficacy, pharmacokinetics and pharmacodynamics of pf-06252616 in ambulatory boys with duchenne muscular dystrophy
Who cites it
3 citing papers in PubMed.
- Observational
- Elbow Flexors Muscle Fat Fraction Is a Sensitive and Relevant Outcome Measure in Nonambulant Patients With DMD.NMR in biomedicine · 2026Article
- 3D finite element models reveal regional fatty infiltration modulates tibialis anterior force generating capacity in FSHD.PloS one · 2025Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
8 authors.
Funding
Abstract
INTRODUCTION/
aimsImproved methodologies to monitor the progression of Duchenne muscular dystrophy (DMD) are needed, especially in the context of clinical trials. We report changes in muscle magnetic resonance imaging (MRI) parameters in participants with DMD, including changes in lean muscle volume (LMV), muscle fat fraction (MFF), and muscle fat infiltration (MFI) and their relationship to changes in functional parameters.
methodsMRI data were obtained as part of a clinical study (NCT02310763) of domagrozumab, an antibody-targeting myostatin that negatively regulates skeletal muscle mass. This post hoc analysis evaluated participants with Dixon MRI data and corresponding functional data at baseline and weeks 49 and 97. Images were analyzed to evaluate changes in adductors, hamstrings, and quadriceps.
resultsThere was a positive correlation between increases in LMV and function. LMV changes in adductors (R = 0.51) and quadriceps (R = 0.54) showed a stronger correlation with function than LMV changes in hamstrings (R = 0.30). There was a negative correlation between MFF and MFI, respectively, and function in adductors (R = -0.57, R = -0.42), quadriceps (R = -0.59, R = -0.50), and hamstrings (R = -0.53, R = -048). Participants with preserved North Star Ambulatory Assessment scores had high total LMV (LMV DISCUSSION: These findings support the use of MRI biomarkers as potential surrogate endpoints in clinical trials of patients with DMD.
trial registrationClinicalTrials.gov identifiers: NCT02310763.
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