Phase 2
N=7
Use of (-)-Epicatechin in the Treatment of Becker Muscular Dystrophy (Pilot Study)
Becker Muscular Dystrophy
Bottom Line
View on ClinicalTrials.gov: NCT01856868 ↗Enrolled (actual)
7
Serious AEs
0.0%
Results posted
Dec 2021
Primary outcome: Primary: Change From Baseline in Muscle Tissue PGC1alpha (AU) at 8 Weeks — 0.55; 0.86 AU — p=0.0434
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- (-)-epicatechin (Drug)
- Age
- Adult · 18+ yrs
- Sex
- Male
- Sponsor
- Craig McDonald, MD
- Primary completion
- Sep 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in Muscle Tissue PGC1alpha (AU) at 8 Weeks |
0.55; 0.86 | 0.0434 sig |
| PRIMARY Mean Change From Baseline in Muscle Tissue AMPK at 8 Weeks |
0.76; 1.06 | 0.0185 sig |
| PRIMARY Mean Change From Baseline in Muscle Tissue LKB1 at 8 Weeks |
0.67; 0.88 | 0.0434 sig |
| PRIMARY Mean Change From Baseline in Cristae-associated Mitofillin Levels at 8 Weeks |
1.05; 1.33 | 0.0078 sig |
| PRIMARY Mean Change From Baseline in Muscle Tissue Follistatin at 8 Weeks |
0.83; 1.11 | 0.0025 sig |
| PRIMARY Mean Change From Baseline in Muscle Tissue Myostatin at 8 Weeks |
0.79; 0.51 | 0.0082 sig |
| PRIMARY Mean Change From Baseline in Muscle Tissue Myogenin at 8 Weeks |
0.89; 1.20 | 0.031 sig |
| PRIMARY Mean Change From Baseline in Muscle Tissue Myf5 at 8 Weeks |
1.07; 1.232 | 0.0238 sig |
| PRIMARY Mean Change From Baseline in Muscle Tissue MyoD at 8 Weeks |
0.87; 1.21 | 0.0321 sig |
| PRIMARY Mean Change From Baseline in Muscle Tissue MEF2a at 8 Weeks |
0.63; 0.78 | 0.0182 sig |
| PRIMARY Mean Change From Baseline in Muscle Tissue Dysferlin at 8 Weeks |
0.65; 0.91 | 0.0371 sig |
| PRIMARY Mean Change From Baseline in Muscle Tissue Utrophin at 8 Weeks |
0.63; 0.8 | 0.0257 sig |
| SECONDARY -(-)Epicatechin Pharmacokinetics |
— | — |
| SECONDARY Participants With Abnormal Treatment-Related Laboratory Assessments |
— | — |
| SECONDARY Change From Baseline in Knee Extension at 8 Weeks |
48.1; -1.82 | 0.47 |
| SECONDARY Change From Baseline in 6-Minute Walk Distance at 8 Weeks |
11.2 | 0.50 |
| SECONDARY Change From Baseline in Stand From Supine at 8 Weeks |
9.621667; 12.145 | — |
| SECONDARY Change From Baseline in Elbow Flexion at 8 Weeks |
27.7; 5.33 | 0.159 |
Summary
(-)-Epicatechin will be evaluated for the treatment of progressive muscle loss and impaired skeletal muscle function in Becker Muscular Dystrophy (BMD) patients.
Eligibility Criteria
Inclusion Criteria
- Male
- Age 18 years to 60 years
- Average to low daily physical activity
- Ability to ambulate for 75 meters without assistive devices
- Diagnosis of BMD confirmed by at least one the following:
- Dystrophin immunofluorescence and/or immunoblot showing partial dystrophin deficiency, and clinical picture consistent with typical BMD, or
- Gene deletions test positive (missing one or more exons) of the dystrophin gene, where reading frame can be predicted as 'in-frame', and clinical picture consistent with typical BMD, or
- Complete dystrophin gene sequencing showing an alteration (point mutation, duplication, or other mutation resulting in a stop codon mutation) that can be definitely associated with BMD, with a typical clinical picture of BMD, or
- Positive family history of BMD confirmed by one of the criteria listed above in a sibling or maternal uncle, and clinical picture typical of BMD.
- Nutritional, herbal and antioxidant supplements taken with the intent of maintaining or improving skeletal muscle strength or functional mobility have been discontinued at least 2 weeks prior to screening (daily multivitamin use is acceptable).
- Hematology profile within normal range
- Baseline laboratory safety chemistry profile within normal range
- No plan to change exercise regimen during study participation
Exclusion Criteria
- Currently enrolled in another treatment clinical trial.
- History of significant concomitant illness or significant impairment of renal or hepatic function.
- Use of regular daily aspirin or other medication with antiplatelet effects within 3 weeks of first dose of study medication.
- Regular participation in vigorous exercise.
- Symptomatic heart failure with cardiac ejection fraction <25%
Data sourced from ClinicalTrials.gov (NCT01856868). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.