Becker Muscular DystrophyPhase 2NCT03236662
(-)- Epicatechin Becker Muscular Dystrophy
A Phase 2 study testing (-)-Epicatechin in people with Becker Muscular Dystrophy, run by Craig McDonald, MD at 1 site in United States.
Trial summary
This is a 48-week open-label extension of our initial proof-of-concept study (UCD0113) in patients with Becker muscular dystrophy who participated in the earlier trial. This single center study will enroll up to 10 adults who will receive the purified nutritional extract (-)-epicatechin 100mg/day orally for 8 weeks. Read more about this study
- Who is it for?
- Ages 18 to 70 years, men only
- Conditions the study is for
- Becker Muscular Dystrophy
- What is being tested?
- (-)-Epicatechin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2016to Dec 2017
- Phase
- Phase 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 70 years
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Prior participation in UCD0113 BMD epicatechin pilot study
- Male
- Age 18 years to 70 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:
Show all 14Show fewer
- 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.
- Hematology profile within normal range
- Baseline laboratory safety chemistry profile within normal range
- No plan to change exercise regimen during study participation
- 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).
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
UC Davis Medical Center
Sacramento, California, 95817
About this study
Official title: UCD0115B: An Open-label Extension Study of Purified Epicatechin to Improve Mitochondrial Function, Strength and Skeletal Muscle Exercise Response in Becker Muscular Dystrophy
This is a 48-week open-label extension of our initial proof-of-concept study (UCD0113) in patients with Becker muscular dystrophy who participated in the earlier trial. This single center study will enroll up to 10 adults 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 screening, baseline, and weeks 4, 8, 12, 24, 16 and 48. The main criterion for success of the study will be presence of one or more biologic or strength and performance outcome measures that yield a response magnitude that allows for sufficient power in a Phase II B study with a sample size of 30 individuals.
- 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
| Study group | Role | What they receive |
|---|---|---|
| Treatment | Experimental | (-)-epicatechin 50mg twice per day (100mg per day total dose) Receives: Drug: (-)-Epicatechin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Plasma Follistatin
blood biomarker concentration
Measured: 48 weeks
Plasma Myostatin
blood biomarker concentration
Measured: 48 weeks
Plasma Nitrates/ SNO
blood biomarker concentration
Measured: 48 weeks
Plasma BNP
blood biomarker concentration
Measured: 48 weeks
Plasma Creatine Kinase
blood biomarker concentration
Measured: 48 weeks
Plasma MMP-9
blood biomarker concentration
Measured: 48 weeks
Plasma TNF-Alpha
blood biomarker concentration
Measured: 48 weeks
Plasma TGF-Beta
blood biomarker concentration
Measured: 48 weeks
Plasma Follistatin:Myostain Ratio
Ratio of plasma follistatin to plasma myostatin
Measured: 48 weeks
Secondary outcomes (2)
Graded Exercise Test Using a Recumbent Cycle Ergometer
blood lactate measured
Measured: baseline and at 2-minute intervals
6-minute Walk Test
Measurements recorded will include 25-meter split times and total distance traveled.
Measured: 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Aug 2017
- Study startNov 2016
- Primary completionNov 2017
- Study completionDec 2017
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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Craig McDonald, MD
- Sponsor type
- University, hospital or other
- Collaborators
- Cardero Therapeutics, Inc.
- Responsible party
- Sponsor-investigator, Craig McDonald, MD, University of California, Davis
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03236662
- Sponsor's protocol ID
- 767161
- Status last verified
- Nov 2021
- First submitted
- 7 Nov 2016
- Last updated
- 24 Nov 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Nov 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- UCD0115B: An Open-label Extension Study of Purified Epicatechin to Improve Mitochondrial Function, Strength and Skeletal Muscle Exercise Response in Becker Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT03236662
- Acronym / sponsor ID
- 767161
- Conditions
- Becker Muscular Dystrophy
- Keywords
- BMD, Becker muscular dystrophy, epicatechin, clinical trial, neuromuscular disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Craig McDonald, MDUniversity, hospital or other
- Collaborators
- Cardero Therapeutics, Inc.
- Study start
- Nov 2016 (actual)
- Primary completion
- Nov 2017 (actual)
- Study completion
- Dec 2017 (actual)
- First posted
- 2 Aug 2017 (actual)
- Results
- Posted 26 Jul 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a 48-week open-label extension of our initial proof-of-concept study (UCD0113) in patients with Becker muscular dystrophy who participated in the earlier trial. This single center study will enroll up to 10 adults 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 screening, baseline, and weeks 4, 8, 12, 24, 16 and 48. The main criterion for success of the study will be presence of one or more biologic or strength and performance outcome measures that yield a response magnitude that allows for sufficient power in a Phase II B study with a sample size of 30 individuals.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment | Experimental | Drug: (-)-Epicatechin(-)-epicatechin 50mg twice per day (100mg per day total dose) |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- Prior participation in UCD0113 BMD epicatechin pilot study
- Male
- Age 18 years to 70 years
- Average to low daily physical activity
- Ability to ambulate for 75 meters without assistive devices
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 |
|---|---|---|
| (-)-Epicatechin | Drug | Treatment |
Primary outcome
Plasma Follistatin
Time frame: 48 weeks
blood biomarker concentration
Plasma Myostatin
Time frame: 48 weeks
blood biomarker concentration
Plasma Nitrates/ SNO
Time frame: 48 weeks
blood biomarker concentration
Plasma BNP
Time frame: 48 weeks
blood biomarker concentration
Plasma Creatine Kinase
Time frame: 48 weeks
blood biomarker concentration
Plasma MMP-9
Time frame: 48 weeks
blood biomarker concentration
Plasma TNF-Alpha
Time frame: 48 weeks
blood biomarker concentration
Plasma TGF-Beta
Time frame: 48 weeks
blood biomarker concentration
Plasma Follistatin:Myostain Ratio
Time frame: 48 weeks
Ratio of plasma follistatin to plasma myostatin
2 secondary outcomes
Graded Exercise Test Using a Recumbent Cycle Ergometer
Time frame: baseline and at 2-minute intervals
6-minute Walk Test
Time frame: 48 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Nov 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 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 14 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 24 Nov 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 phase mean?
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 26 Jul 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?
Plasma Follistatin (time frame: 48 weeks). The study lists 9 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 24 Nov 2021.