Duchenne Muscular DystrophyPhase 2NCT01239758
Extension Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy
A Phase 2 study testing ACE-031 (Extension of cohort 1 from core study, A031-03), ACE-031 (Extension of cohort 2 from core study, A031-03) and ACE-031 (Extension of cohort 3 from core study, A031-03) in people with Duchenne Muscular Dystrophy, run by Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA at 4 sites in Canada.
Trial summary
To evaluate the long-term safety and tolerability of ACE-031 administration in subjects with Duchenne muscular dystrophy (DMD) who participated in Study A031-03. [Note: This study was terminated based on preliminary safety data. Read more about this study
- Who is it for?
- Ages 4 years and older, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- ACE-031 (Extension of cohort 1 from core study, A031-03), ACE-031 (Extension of cohort 2 from core study, A031-03) and ACE-031 (Extension of cohort 3 from core study, A031-03)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 4 sitesCanada
- Study dates
- Oct 2010to May 2011
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 4 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Completion of participation in Study A031-03 and Investigator approval
- Continuation of corticosteroid therapy at the same absolute dose and schedule as on Study A031-03
Who cannot join
- Participation in any other therapeutic clinical trial
- Plans to have surgery during the course of the study
Trial locations and contacts
4 sites in 1 country.
Canada4 sites
Acceleron Investigative Site
Calgary, Alberta
Acceleron Investigative Site
Hamilton, Ontario
Acceleron Investigative Site
London, Ontario
Acceleron Investigative Site
Ottawa, Ontario
About this study
Official title: An Open-Label Extension Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of ACE-031 (ActRIIB-IgG1) in Subjects With Duchenne Muscular Dystrophy
To evaluate the long-term safety and tolerability of ACE-031 administration in subjects with Duchenne muscular dystrophy (DMD) who participated in Study A031-03. [Note: This study was terminated based on preliminary safety data. Pending further analysis of safety data and discussion with health authorities, a new ACE-031 trial will be planned.]
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- ACE-031 (Extension of cohort 1 from core study, A031-03), ACE-031 (Extension of cohort 2 from core study, A031-03), ACE-031 (Extension of cohort 3 from core study, A031-03)
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ACE-031 (Extension of cohort 1 from core study, A031-03)Biological
ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 24 weeks.
ACE-031 (Extension of cohort 2 from core study, A031-03)Biological
Up to 1.0 mg/kg subcutaneously once every 2 weeks for 24 weeks.
ACE-031 (Extension of cohort 3 from core study, A031-03)Biological
Up to 2.5 mg/kg subcutaneously once every 4 weeks for 24 weeks.
| Study group | Role | What they receive |
|---|---|---|
| ACE-031 (Extension of cohort 1 from core study, A031-03) | Experimental | Not described Receives: Biological: ACE-031 (Extension of cohort 1 from core study, A031-03) |
| ACE-031 (Extension of cohort 2 from core study, A031-03) | Experimental | Not described Receives: Biological: ACE-031 (Extension of cohort 2 from core study, A031-03) |
| ACE-031 (Extension of cohort 3 from core study, A031-03) | Experimental | Not described Receives: Biological: ACE-031 (Extension of cohort 3 from core study, A031-03) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of patients with adverse events.
Measured: From treatment initiation to End-of-Study Visit, approximately 24 weeks later.
Change in laboratory parameters and vital signs.
Measured: Baseline to End-of-Study Visit, approximately 24 weeks later.
Secondary outcomes (6)
Percent change in total lean body mass by DXA scan.
Measured: Baseline to End-of-Study Visit, approximately 24 weeks later.
Percent change in total body and lumbar spine bone mineral density by DXA scan.
Measured: Baseline to End-of-Study Visit, approximately 24 weeks later.
Percent change in muscle strength score by hand-held myometry.
Measured: Baseline to End-of-Study Visit, approximately 24 weeks later.
Change in distance traveled in 6 minutes (standardized 6-Minute-Walk Test).
Measured: Baseline to End-of-Study Visit, approximately 24 weeks later.
Change in time to travel 10 meters (standardized 10-Meter-Walk/Run test).
Measured: Baseline to End-of-Study Visit, approximately 24 weeks later.
Change in pulmonary function tests.
Measured: Baseline to End-of-Study Visit, approximately 24 weeks later.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Nov 2010 (estimated)
- Study startOct 2010
- Primary completionMay 2011
- Study completionMay 2011
Why it stopped: This study was terminated based on preliminary safety data.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne 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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01239758
- Sponsor's protocol ID
- A031-06
- Status last verified
- Jan 2013
- First submitted
- 1 Nov 2010
- Last updated
- 1 Feb 2013
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 1 Feb 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Extension Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of ACE-031 (ActRIIB-IgG1) in Subjects With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT01239758
- Acronym / sponsor ID
- A031-06
- Conditions
- Duchenne Muscular Dystrophy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: This study was terminated based on preliminary safety data.
- Enrolment
- 11 participants (actual)
- Lead sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USAIndustry (pharma, biotech, devices)
- Study start
- Oct 2010
- Primary completion
- May 2011 (actual)
- Study completion
- May 2011 (actual)
- First posted
- 11 Nov 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To evaluate the long-term safety and tolerability of ACE-031 administration in subjects with Duchenne muscular dystrophy (DMD) who participated in Study A031-03. [Note: This study was terminated based on preliminary safety data. Pending further analysis of safety data and discussion with health authorities, a new ACE-031 trial will be planned.]
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| ACE-031 (Extension of cohort 1 from core study, A031-03) | Experimental | Biological: ACE-031 (Extension of cohort 1 from core study, A031-03) |
| ACE-031 (Extension of cohort 2 from core study, A031-03) | Experimental | Biological: ACE-031 (Extension of cohort 2 from core study, A031-03) |
| ACE-031 (Extension of cohort 3 from core study, A031-03) | Experimental | Biological: ACE-031 (Extension of cohort 3 from core study, A031-03) |
Who may qualify
- Ages
- 4 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Completion of participation in Study A031-03 and Investigator approval
- Continuation of corticosteroid therapy at the same absolute dose and schedule as on Study A031-03
Key exclusion criteria (2 listed)
- Participation in any other therapeutic clinical trial
- Plans to have surgery during the course of the study
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 |
|---|---|---|
| ACE-031 (Extension of cohort 1 from core study, A031-03)ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 24 weeks. | Biological | ACE-031 (Extension of cohort 1 from core study, A031-03) |
| ACE-031 (Extension of cohort 2 from core study, A031-03)Up to 1.0 mg/kg subcutaneously once every 2 weeks for 24 weeks. | Biological | ACE-031 (Extension of cohort 2 from core study, A031-03) |
| ACE-031 (Extension of cohort 3 from core study, A031-03)Up to 2.5 mg/kg subcutaneously once every 4 weeks for 24 weeks. | Biological | ACE-031 (Extension of cohort 3 from core study, A031-03) |
Primary outcome
Number of patients with adverse events.
Time frame: From treatment initiation to End-of-Study Visit, approximately 24 weeks later.
Change in laboratory parameters and vital signs.
Time frame: Baseline to End-of-Study Visit, approximately 24 weeks later.
6 secondary outcomes
Percent change in total lean body mass by DXA scan.
Time frame: Baseline to End-of-Study Visit, approximately 24 weeks later.
Percent change in total body and lumbar spine bone mineral density by DXA scan.
Time frame: Baseline to End-of-Study Visit, approximately 24 weeks later.
Percent change in muscle strength score by hand-held myometry.
Time frame: Baseline to End-of-Study Visit, approximately 24 weeks later.
Change in distance traveled in 6 minutes (standardized 6-Minute-Walk Test).
Time frame: Baseline to End-of-Study Visit, approximately 24 weeks later.
Change in time to travel 10 meters (standardized 10-Meter-Walk/Run test).
Time frame: Baseline to End-of-Study Visit, approximately 24 weeks later.
Change in pulmonary function tests.
Time frame: Baseline to End-of-Study Visit, approximately 24 weeks later.
Results and safety
The study was stopped early (reason given: This study was terminated based on preliminary safety data.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Feb 2013.
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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.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ACE-031 (Extension of cohort 1 from core study, A031-03), ACE-031 (Extension of cohort 2 from core study, A031-03) and ACE-031 (Extension of cohort 3 from core study, A031-03) in people with Duchenne Muscular Dystrophy, run by Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA at 4 sites in Canada.
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 2 inclusion and 2 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: stopped early, as of the sponsor's last update on 1 Feb 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Canada. 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?
The study was stopped early (reason given: This study was terminated based on preliminary safety data.). Results are not available in the source record.
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?
Number of patients with adverse events. (time frame: From treatment initiation to End-of-Study Visit, approximately 24 weeks later.). The study lists 2 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 1 Feb 2013.