Becker Muscular DystrophyPhase 1/2NCT01856868

Use of (-)-Epicatechin in the Treatment of Becker Muscular Dystrophy (Pilot Study)

A Phase 1/2 study testing (-)-epicatechin in people with Becker Muscular Dystrophy, run by Craig McDonald, MD at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

(-)-Epicatechin will be evaluated for the treatment of progressive muscle loss and impaired skeletal muscle function in Becker Muscular Dystrophy (BMD) patients. Read more about this study

Who is it for?: Ages 18 to 60 years, men only
Conditions the study is for: Becker Muscular Dystrophy
What is being tested?: (-)-epicatechin
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 7 participants
Where: 1 siteUnited States
Study dates: May 2013to Sep 2018
Phase: Phase 1/2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 to 60 years
Men only
No healthy volunteers
Adult

Who may be able to join

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

Show all 13Show fewer

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

Who cannot join

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%

Trial locations and contacts

1 site in 1 country.

United States1 site

University of California, Davis
Sacramento, California, 95817

Other trials in United States

About this study

Official title: An Open-label Pilot Study of Purified Tea-derived Epicatechin to Improve Mitochondrial Function, Strength and Skeletal Muscle Exercise Response in Becker Muscular Dystrophy.

(-)-Epicatechin will be evaluated for the treatment of progressive muscle loss and impaired skeletal muscle function in Becker Muscular Dystrophy (BMD) patients.

Read the detailed description

This is a proof-of-concept phase 1/2a pilot and endpoint development study that is designed to provide initial evidence of biological activity of (-)-epicatechin. Primary endpoints include initial assessment of tissue-specific evidence of efficacy from muscle biopsy samples. Secondary endpoints include measures of strength and physical function, and safety and adverse event data. Pilot endpoints include assessment of mRNA and miRNA peripheral blood profiles and validation of non-invasive near-infrared spectroscopy (NIRS) muscle perfusion studies during exercise and a recumbent cycle exercise test that may be employed as endpoints in future clinical trials.

This single center open-label pilot study will enroll 10 adults with genetically-confirmed Becker muscular dystrophy, who will receive the purified nutritional extract (-)-epicatechin 100mg/day orally for 8 weeks. After screening visits, participants will be enrolled in the study if they meet all inclusion criteria. They will be evaluated at baseline and at screening, day 1, and weeks 1, 2, 4 and 8.

Conditions: Becker Muscular Dystrophy
Being tested: (-)-epicatechin
Keywords: BMD, Becker muscular dystrophy, epicatechin, clinical trial, neuromuscular disease
Medical subject headings: Muscular Dystrophy, Duchenne, Neuromuscular Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

(-)-epicatechinDrug
Also called dietary supplement
purified nutritional extract (-)-epicatechin 100mg/day orally for 8 weeks.

Study groups in NCT01856868
Study group	Role	What they receive
Treatment with Epicatechin	Experimental	Purified nutritional extract (-)-epicatechin 100mg/day orally for 8 weeks. Receives: Drug: (-)-epicatechin

What is being measured

The primary outcome is the main question the study is designed to answer.

Change From Baseline in Muscle Tissue PGC1alpha (AU) at 8 Weeks
Western blot measurement of the transcriptional coactivator gene PGC1alpha involved in mitochondrial biogenesis will be assessed using relative band intensities of the pre-treatment (Baseline) and post-treatment (8 Weeks) specimens with digitally quantified using ImageJ software.
Measured: Baseline and 8 Weeks
Mean Change From Baseline in Muscle Tissue AMPK at 8 Weeks
Western blot measurement of AMPK will be assessed using relative band intensities of the pre-treatment and post-treatment specimens with digitally quantified using ImageJ software).
Measured: 8 weeks
Mean Change From Baseline in Muscle Tissue LKB1 at 8 Weeks
Western blot measurement of LKB1 will be assessed using relative band intensities of the pre-treatment and post-treatment specimens with digitally quantified using ImageJ software) .
Measured: 8 weeks
Mean Change From Baseline in Cristae-associated Mitofillin Levels at 8 Weeks
Western blot measurement of Mitofillin will be assessed using relative band intensities of the pre-treatment and post-treatment specimens with digitally quantified using ImageJ software.
Measured: 8 weeks
Mean Change From Baseline in Muscle Tissue Follistatin at 8 Weeks
Regulators of muscle growth and regeneration including follistatin will be assessed by Western using relative band intensities of the pre-treatment and post-treatment specimens with digitally quantified using ImageJ software).
Measured: 8 weeks
Mean Change From Baseline in Muscle Tissue Myostatin at 8 Weeks
Regulators of muscle growth and regeneration including myostatin will be assessed by Western using relative band intensities of the pre-treatment and post-treatment specimens with digitally quantified using ImageJ software).
Measured: 8 weeks
Mean Change From Baseline in Muscle Tissue Myogenin at 8 Weeks
Modulators of skeletal muscle regeneration by Western will include myogenin will be assessed using relative band intensities of the pre-treatment and post-treatment specimens with digitally quantified using ImageJ software).
Measured: 8 weeks
Mean Change From Baseline in Muscle Tissue Myf5 at 8 Weeks
Modulators of skeletal muscle regeneration My5 will be assessed by Western using relative band intensities of the pre-treatment and post-treatment specimens with digitally quantified using ImageJ software).
Measured: 8 weeks
Mean Change From Baseline in Muscle Tissue MyoD at 8 Weeks
Modulators of skeletal muscle regeneration MyoD will be assessed by Western using relative band intensities of the pre-treatment and post-treatment specimens with digitally quantified using ImageJ software).
Measured: 8 weeks
Mean Change From Baseline in Muscle Tissue MEF2a at 8 Weeks
Modulators of skeletal muscle regeneration MEF2a will be assessed by Western using relative band intensities of the pre-treatment and post-treatment specimens with digitally quantified using ImageJ software).
Measured: 8 weeks
Mean Change From Baseline in Muscle Tissue Dysferlin at 8 Weeks
Structure associated indicators including dysferlin will be assessed by Western using relative band intensities of the pre-treatment and post-treatment specimens with digitally quantified using ImageJ software).
Measured: 8 weeks
Mean Change From Baseline in Muscle Tissue Utrophin at 8 Weeks
Structure associated indicators including utrophin will be assessed by Western using relative band intensities of the pre-treatment and post-treatment specimens with digitally quantified using ImageJ software).
Measured: 8 weeks

Secondary outcomes (6)

-(-)Epicatechin Pharmacokinetics
Pharmacokinetics sequentially after dosing will be measured.
Measured: 8 Weeks
Participants With Abnormal Treatment-Related Laboratory Assessments
Standard safety monitoring of plasma hematologic, hepatologic, renal and metabolic parameters will be assessed. Abnormal will be defined as values outside of typical range for patients with Becker Muscular Dystrophy.
Measured: 8 weeks
Change From Baseline in Knee Extension at 8 Weeks
Knee extension will be assessed using an isokinetic dynamometer.
Measured: Baseline and 8 Weeks
Change From Baseline in 6-Minute Walk Distance at 8 Weeks
Muscle function will be assessed by measuring the 6-minute walk distance
Measured: Baseline and 8 Weeks
Change From Baseline in Stand From Supine at 8 Weeks
Muscle burst function will be assessed by time function tests.
Measured: Baseline and 8 Weeks
Change From Baseline in Elbow Flexion at 8 Weeks
Elbow flexion will be assessed using an isokinetic dynamometer.
Measured: Baseline and 8 Weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

May 2013Sep 2018

First posted17 May 2013 (estimated)
Study startMay 2013
Primary completionSep 2018
Study completionSep 2018

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Becker Muscular DystrophyStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 1/2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 7 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01856868
Sponsor's protocol ID: 454352
Status last verified: Nov 2021
First submitted: 9 May 2013
Last updated: 22 Dec 2021
Results posted: Yes, on ClinicalTrials.gov

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingEarly Phase 1
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
Duchenne Muscular Dystrophy (DMD)
RecruitingPhase 2
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Not yet recruitingPhase 2
A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD)
Not yet recruitingObservational study
Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function
Becker Muscular Dystrophy
Enrolling by invitationObservational study
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
Duchenne Muscular Dystrophy

All Becker Muscular Dystrophy trials

Study at a glance

From the registry record, last updated by the sponsor on 22 Dec 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Open-label Pilot Study of Purified Tea-derived Epicatechin to Improve Mitochondrial Function, Strength and Skeletal Muscle Exercise Response in Becker Muscular Dystrophy.
ClinicalTrials.gov ID: NCT01856868
Acronym / sponsor ID: 454352
Conditions: Becker Muscular Dystrophy
Keywords: BMD, Becker muscular dystrophy, epicatechin, clinical trial, neuromuscular disease
Phase: Phase 1/2
Study type: Interventional
Recruitment status: Completed
Enrolment: 7 participants (actual)
Lead sponsor: Craig McDonald, MDUniversity, hospital or other
Collaborators: Cardero Therapeutics, Inc.
Study start: May 2013
Primary completion: Sep 2018 (actual)
Study completion: Sep 2018 (actual)
First posted: 17 May 2013 (estimated)
Results: Posted 22 Dec 2021

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

(-)-Epicatechin will be evaluated for the treatment of progressive muscle loss and impaired skeletal muscle function in Becker Muscular Dystrophy (BMD) patients.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Treatment with Epicatechin	Experimental	Drug: (-)-epicatechinPurified nutritional extract (-)-epicatechin 100mg/day orally for 8 weeks.

Who may qualify

Ages: 18 to 60 years
Sex: Male only
Healthy volunteers: Not accepted
Age groups: Adult

Key inclusion criteria (13 listed)

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:

Key exclusion criteria (5 listed)

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%

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
(-)-epicatechinAlso: dietary supplementpurified nutritional extract (-)-epicatechin 100mg/day orally for 8 weeks.	Drug	Treatment with Epicatechin

Primary outcome

Change From Baseline in Muscle Tissue PGC1alpha (AU) at 8 Weeks
Time frame: Baseline and 8 Weeks
Western blot measurement of the transcriptional coactivator gene PGC1alpha involved in mitochondrial biogenesis will be assessed using relative band intensities of the pre-treatment (Baseline) and post-treatment (8 Weeks) specimens with digitally quantified using ImageJ software.
Mean Change From Baseline in Muscle Tissue AMPK at 8 Weeks
Time frame: 8 weeks
Western blot measurement of AMPK will be assessed using relative band intensities of the pre-treatment and post-treatment specimens with digitally quantified using ImageJ software).
Mean Change From Baseline in Muscle Tissue LKB1 at 8 Weeks
Time frame: 8 weeks
Western blot measurement of LKB1 will be assessed using relative band intensities of the pre-treatment and post-treatment specimens with digitally quantified using ImageJ software) .
Mean Change From Baseline in Cristae-associated Mitofillin Levels at 8 Weeks
Time frame: 8 weeks
Western blot measurement of Mitofillin will be assessed using relative band intensities of the pre-treatment and post-treatment specimens with digitally quantified using ImageJ software.
Mean Change From Baseline in Muscle Tissue Follistatin at 8 Weeks
Time frame: 8 weeks
Regulators of muscle growth and regeneration including follistatin will be assessed by Western using relative band intensities of the pre-treatment and post-treatment specimens with digitally quantified using ImageJ software).
Mean Change From Baseline in Muscle Tissue Myostatin at 8 Weeks
Time frame: 8 weeks
Regulators of muscle growth and regeneration including myostatin will be assessed by Western using relative band intensities of the pre-treatment and post-treatment specimens with digitally quantified using ImageJ software).
Mean Change From Baseline in Muscle Tissue Myogenin at 8 Weeks
Time frame: 8 weeks
Modulators of skeletal muscle regeneration by Western will include myogenin will be assessed using relative band intensities of the pre-treatment and post-treatment specimens with digitally quantified using ImageJ software).
Mean Change From Baseline in Muscle Tissue Myf5 at 8 Weeks
Time frame: 8 weeks
Modulators of skeletal muscle regeneration My5 will be assessed by Western using relative band intensities of the pre-treatment and post-treatment specimens with digitally quantified using ImageJ software).
Mean Change From Baseline in Muscle Tissue MyoD at 8 Weeks
Time frame: 8 weeks
Modulators of skeletal muscle regeneration MyoD will be assessed by Western using relative band intensities of the pre-treatment and post-treatment specimens with digitally quantified using ImageJ software).
Mean Change From Baseline in Muscle Tissue MEF2a at 8 Weeks
Time frame: 8 weeks
Modulators of skeletal muscle regeneration MEF2a will be assessed by Western using relative band intensities of the pre-treatment and post-treatment specimens with digitally quantified using ImageJ software).
Mean Change From Baseline in Muscle Tissue Dysferlin at 8 Weeks
Time frame: 8 weeks
Structure associated indicators including dysferlin will be assessed by Western using relative band intensities of the pre-treatment and post-treatment specimens with digitally quantified using ImageJ software).
Mean Change From Baseline in Muscle Tissue Utrophin at 8 Weeks
Time frame: 8 weeks
Structure associated indicators including utrophin will be assessed by Western using relative band intensities of the pre-treatment and post-treatment specimens with digitally quantified using ImageJ software).

6 secondary outcomes

-(-)Epicatechin Pharmacokinetics
Time frame: 8 Weeks
Participants With Abnormal Treatment-Related Laboratory Assessments
Time frame: 8 weeks
Change From Baseline in Knee Extension at 8 Weeks
Time frame: Baseline and 8 Weeks
Change From Baseline in 6-Minute Walk Distance at 8 Weeks
Time frame: Baseline and 8 Weeks
Change From Baseline in Stand From Supine at 8 Weeks
Time frame: Baseline and 8 Weeks
Change From Baseline in Elbow Flexion at 8 Weeks
Time frame: Baseline and 8 Weeks

Results and safety

Results posted to the registry on 22 Dec 2021, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Treatment With Epicatechin	7	7

Primary outcome: Change From Baseline in Muscle Tissue PGC1alpha (AU) at 8 Weeks

Time frame: Baseline and 8 Weeks. Mean (Standard Deviation). Unit: AU

Measure	Treatment With Epicatechin n = 6
Baseline	0.55 (0.3)
8 Weeks	0.86 (0.17)

Treatment With Epicatechin: p = 0.0434, t-test, 2 sided

Population analysed: One participant sustained a fall at home that resulted in a minor injury just prior to the 8 week evaluation and declined undergoing the 8 week biopsy. Thus, only 6 participants were analyzed.

Primary outcome: Mean Change From Baseline in Muscle Tissue AMPK at 8 Weeks

Time frame: 8 weeks. Mean (Standard Deviation). Unit: AU

Measure	Treatment With Epicatechin n = 6
Baseline	0.76 (0.48)
Week 8	1.06 (0.52)

Treatment With Epicatechin: p = 0.0185, t-test, 2 sided

Primary outcome: Mean Change From Baseline in Muscle Tissue LKB1 at 8 Weeks

Time frame: 8 weeks. Mean (Standard Deviation). Unit: AU

Measure	Treatment With Epicatechin n = 6
Baseline	0.67 (0.17)
Week 8	0.88 (0.24)

Treatment With Epicatechin: p = 0.0434, t-test, 2 sided

Primary outcome: Mean Change From Baseline in Cristae-associated Mitofillin Levels at 8 Weeks

Time frame: 8 weeks. Mean (Standard Deviation). Unit: AU

Measure	Treatment With Epicatechin n = 6
Baseline	1.05 (0.36)
Week 8	1.33 (0.3)

Treatment With Epicatechin: p = 0.0078, t-test, 2 sided

6 secondary outcomes are reported in the full results.

Adverse events

Time frame: 8 weeks. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Treatment With Epicatechin	0 of 6 (0%)	6 of 6 (100%)	0 of 6

Limitations and caveats (from the sponsor): Limitations of this study include the small number of subjects, the open-label design, and the use of within-subjects controls with BMD rather than a placebo-treated group in a randomized, double-blind, placebo-controlled trial design.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 22 Dec 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
UC Davis Neuromuscular Research CenterLink provided by the sponsor.

Related trials for Becker Muscular Dystrophy

RecruitingEarly Phase 1
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
Duchenne Muscular Dystrophy (DMD)
RecruitingPhase 2
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Not yet recruitingPhase 2
A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD)
Not yet recruitingObservational study
Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function
Becker Muscular Dystrophy
Enrolling by invitationObservational study
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
Duchenne Muscular Dystrophy

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Craig M McDonald, MD principal investigator

University of California, Davis

Erik K Henricson, MPH study director

University of California, Davis

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing (-)-epicatechin in people with Becker Muscular Dystrophy, run by Craig McDonald, MD at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 13 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 22 Dec 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 22 Dec 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change From Baseline in Muscle Tissue PGC1alpha (AU) at 8 Weeks (time frame: Baseline and 8 Weeks). The study lists 12 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01856868.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Dec 2021.