Duchenne Muscular Dystrophy (DMD)Phase 1NCT04184882
A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Participants With Duchenne Muscular Dystrophy (DMD)
A Phase 1 study testing Bocidelpar in people with Duchenne Muscular Dystrophy (DMD), run by Astellas Pharma Inc at 5 sites in United States.
Trial summary
The primary purpose of this study is to evaluate the safety and tolerability of ASP0367. This study will also evaluate the pharmacokinetics, pharmacodynamics and efficacy on muscle function of ASP0367. Read more about this study
- Who is it for?
- Ages 8 to 16 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy (DMD)
- What is being tested?
- Bocidelpar
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 8 participants
- Where
- 5 sitesUnited States
- Study dates
- 24 Feb 2021to 4 Sep 2022
- Phase
- Phase 1
- 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 8 to 16 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Subject has a diagnosis of Duchenne muscular dystrophy (DMD) (confirmed by Central Genetic Counselor) defined as a clinical picture consistent with typical DMD and 1 of the following:
- Dystrophin immunofluorescence and/or Western blot showing severe dystrophin deficiency consistent with the diagnosis of DMD.
- Identifiable mutation within the DMD gene (deletion/duplication of 1 or more exons), where reading frame can be predicted as "out-of-frame"
- Complete dystrophin gene sequencing showing an alteration (point mutation, duplication or other) that is expected to preclude production of the functional dystrophin protein (i.e., nonsense mutation or deletion/duplication leading to a downstream stop codon).
- A male subject of reproductive potential (Tanner Stage 2 and above) must agree to do either of the following from screening throughout the study until 30 days after the last dose of the investigational product (IP):
- Abstain from sexual intercourse, OR
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- If having heterosexual intercourse, must use a condom and their female partners who are of childbearing potential must use a highly effective contraception method.
- Subject has been on a stable regimen of corticosteroids for 6 months prior to the time of enrollment (at baseline).
- Subject has been on stable cardiac therapy for 3 months prior to the time of enrollment (at baseline), if used, which may include prophylactic angiotensin-converting enzyme inhibitors (ACE), angiotensin II receptor blocker (ARB), aldosterone receptor antagonists (e.g., spironolactone, eplerenone), and/or beta-blocker therapy or a combination therapy thereof.
- Subject is unable to complete the 10 meter run/walk in <6 seconds at screening.
- Subject has a PUL 2.0 entry item A score of 4, 5 or 6 at screening.
- Subject and subject's parent(s) or legal guardian agrees not to participate in another interventional study while participating in the present study.
- For those subjects receiving exon-skipping therapy, the subject has been on a stable dose regimen with a single commercially-available product for at least 6 months prior to randomization at baseline.
- For those subjects using metformin, the subject has been on a stable dose of metformin for 3 months prior to the time of enrollment (at baseline) and the investigator expects the subject to maintain the current metformin dose.
Who cannot join
- Subject has had an acute illness (i.e., upper respiratory or viral infection) within 4 weeks prior to study enrollment (at baseline), which precludes participation.
- Subject has a cardiac ejection fraction < 53% on echocardiogram at screening.
- Subject has a mean QT interval from triplicate electrocardiogram (ECG) using Fridericia's correction (QTcF) of > 450 msec at screening. If the mean QTcF exceeds the limits stated above, 1 additional triplicate ECG can be taken and utilized at screening.
- Subject has cardiac troponin I (cTnI) above the upper limit of normal (ULN) at screening and is assessed clinically significant.
- Subject has used coenzyme Q10 (CoQ10), idebenone, carnitine, or other mitochondrial focused supplements or drugs within 4 weeks prior to randomization at baseline. In addition, subject has used any peroxisome proliferator-activated receptors (PPAR) ligands such as fibrates and thiazolidinediones 4 weeks prior to randomization at baseline.
- Subject has a known or suspected hypersensitivity to ASP0367, or any components of the formulation used.
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- Subject has inadequate renal function, as defined by serum Cystatin C > 2 x ULN at screening.
- Subject who has any of the following liver function tests elevated: gamma-glutamyl transferase [GGT] and/or total bilirubin [TBL]) > 1.5 x ULN at screening.
- Subject who has a positive test result for hepatitis A virus (HAV) antibodies (immunoglobulin M [IgM]), hepatitis B surface (HBs) antigen, hepatitis C virus (HCV) antibody or human immunodeficiency virus (HIV) antigen/antibody at screening.
- Subject has mental conditions such as schizophrenia, bipolar disorder or major depressive disorder.
- Subject has a history of suicide attempt, suicidal behavior or has any suicidal ideation within 1 year prior to screening that meets criteria at a level of 4 or 5 by using the Columbia Suicide Severity Rating Scale (C-SSRS) or who is at significant risk to commit suicide, as assessed at screening or at baseline.
- Subject has severe behavioral or cognitive problems that preclude participation in the study.
- Subject has any condition, which makes the subject unsuitable for study participation.
- Subject is taking any other investigational therapy currently or has taken any other investigational therapy within 3 months prior to the time of enrollment (at baseline).
- Subject and parent/guardian are unwilling and unable to comply with scheduled visits, IP administration plan and study procedures.
- Subject tested positive for coronavirus (SARS-CoV-2) infection without any clinical signs or symptoms within 2 weeks prior to randomization at baseline and/or has had clinical signs and symptoms consistent with coronavirus (SARS-CoV-2) infection and fully recovered within 4 weeks prior to randomization at baseline.
- Subject whose parent(s) and/or caregiver(s) have increased risk of coronavirus (SARS-CoV-2) exposure from work history (e.g., nursing home, meat processing facility and correctional facility) or recent travel history unless the subject's parent(s) and/or caregiver(s) have been appropriately vaccinated with one of the COVID-19 vaccines.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Kennedy Krieger Institute
Baltimore, Maryland, 21201
University of Kansas Medical Center
Kansas City, Kansas, 66160
Children's Hospital of Richmond at VCU
Richmond, Virginia, 23219
University of California Davis Health
Sacramento, California, 95817
UMass Memorial Medical Center
Worcester, Massachusetts, 01655
About this study
Official title: A Randomized, Double Blind, Placebo-Controlled Phase 1b Study With Open-Label Extension to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Patients With Duchenne Muscular Dystrophy (DMD)
The primary purpose of this study is to evaluate the safety and tolerability of ASP0367.
This study will also evaluate the pharmacokinetics, pharmacodynamics and efficacy on muscle function of ASP0367.
Read the detailed description
This study is comprised of a 4-week pre-treatment screening period, 24-week treatment period and 4-week post-treatment follow-up period. The 24-week treatment period consists of a 12-week double-blind (DB) part and 12-week open-label extension (OLE) part and each part includes a 2 week Low dose Period and a 10-week High-dose Period.
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Being tested
- Bocidelpar, Placebo
- Keywords
- tolerability, ASP0367, MA-0211, safety
- 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.
BocidelparDrug
Also called MA-0211, ASP0367
Oral
PlaceboDrug
Oral
| Study group | Role | What they receive |
|---|---|---|
| ASP0367 group | Experimental | Participants will be dosed investigational product (IP) at the low dose for 2 weeks and then at the high dose for 10 weeks with the total duration of 12 weeks in the DB part and OLE part, respectively. Receives: Drug: Bocidelpar |
| Placebo to ASP0367 group | Placebo comparator | Participants will be dosed matching placebo in the DB part. In OLE part, participants will dosed IP at the low dose for 2 weeks and then at the high dose for 10 weeks with the total duration of 12 weeks. Receives: Drug: Bocidelpar; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with Treatment Emergent Adverse Events (TEAEs)
An AE is any untoward medical occurrence in a subject administered an investigational product (IP), and which does not necessarily have to have a causal relationship with this treatment.
A TEAE is defined as an AE observed after starting administration of the investigational product (IP) to 28 days after the last dose of IP for the double blind part or moving to the open-label extension part, whichever comes first.
An IP-related TEAE is defined as any TEAE with a causal relationship of "yes" by the investigator.
Measured: Up to Week 28
Number of participants with vital sign abnormalities and/or AEs
Number of participants with potentially clinically significant vital sign values.
Measured: Up to Week 28
Number of participants with body weight change abnormalities and/or AEs
Number of participants with potentially clinically significant body weight.
Measured: Up to Week 28
Number of participants with electrocardiogram (ECG) abnormalities
Number of participants with potentially clinically significant 12-ECG values.
Measured: Up to Week 28
Number of participants with echocardiography abnormalities and/or AEs
Number of participants with potentially clinically significant echocardiography values.
Measured: Up to Week 28
Number of participants with laboratory value abnormalities and/or AEs
Number of participants with potentially clinically significant laboratory values.
Measured: Up to Week 28
Number of participants with suicidal ideation and/or behavior as assessed by Columbia-Suicide Severity Rating Scale (C-SSRS) Change from baseline in Columbia-Suicide Severity Rating Scale (C-SSRS)
The Columbia-Suicide Severity Rating Scale (C-SSRS) is a clinician administered assessment tool that evaluates suicidal ideation and behavior. Number of participants that have an affirmative response provided to the 5 items for suicidal ideation (1. Wish to be dead, 2. Non-specific active suicidal thoughts, 3. Active suicidal ideation with any methods (not plan) without intent to act, 4. Active suicidal ideation with some intent to act, without specific plan, 5. Active suicidal ideation with specific plan and intent) and/or to the 6 items for suicidal behavior (1. Actual attempt, 2. Interrupted attempt, 3. Aborted attempt, 4. Preparatory acts or behavior, 5. Suicidal Behavior 6. Completed suicide,) will be reported.
Measured: Baseline and up to Week 28
Change from baseline in digit span test
The Digit span test is a subtest of Wechsler Intelligence Scale for children (WISC). This test comprises 3 parts on the fifth edition (WISC-V). Digit Span Forward requires the subject to repeat numbers in the same order as presented by the interviewer. Digit Span Backward requires the subject to repeat the numbers in the reverse order of that presented by the interviewer. Digit Span Sequencing requires the subject to sequentially order the numbers presented by the interviewer. Scores of this test are based on each raw score and total raw score.
Measured: Baseline and up to Week 24
Secondary outcomes (10)
Pharmacokinetics (PK) of ASP0367 in plasma: AUC from the time of dosing to the start of next dosing interval (AUCtau)
AUCtau will be recorded from the PK plasma samples collected.
Measured: Up to Week 2
PK of ASP0367 in plasma: maximum concentration (Cmax)
Cmax will be recorded from the PK plasma samples collected.
Measured: Up to Week 2
Pharmacodynamics (PD) of ASP0367: Percent change from baseline in peroxisome proliferator-activated receptor (PPAR) delta target genes expression levels in blood
Whole blood cell samples will be collected to measure percent change in target gene expressions.
Measured: Baseline and up to Week 4
PD of ASP0367: Percent change from baseline in serum myostatin/follistatin ratio
Serum samples will be collected to record myostatin.
Measured: Baseline and up to Week 12
Change from baseline in Performance of Upper Limb Module (PUL) (v2.0) assessment score
The PUL Assessment v2.0 includes a total of 23 upper limb test items, with the first entry item A used to define the starting functional level. The remaining 22 items are subdivided into 3 major dimension levels as following; shoulder level (6 items, maximum score of 12), elbow level (9 items, maximum score of 17) and distal level dimension (7 items, maximum score of 13). Positive change in scores indicates an improvement.
Measured: Baseline and up to Week 12
Change from baseline on Pediatric Quality of Life (PedsQL) Multidimensional Fatigue Scale
PedsQL Multidimensional Fatigue Scale comprises the General Fatigue Scale (6 items), Sleep/Rest Fatigue Scale (6 items) and Cognitive Fatigue Scale (6 items). A 5-point response scale in each item is utilized (0 never a problem; 1 almost never a problem; 2 sometimes a problem; 3 often a problem; 4 almost always a problem). Negative change in scores indicates an improvement. This scale is based on the subject's age and will be assessed by both subject and parent or legal guardian.
Measured: Baseline and up to Week 12
Change from baseline in distance walked in 2 minutes assessed in meters
The 2-minute walk test (2MWT) is a measurement of endurance that assesses walking distance over 2 minutes. Only ambulatory subjects conduct the 2MWT in this study.
Measured: Baseline and up to Week 12
Percent change from baseline in the assisted 6 minute cycling test (a6MCT) maximal attained revolutions
The a6MCT has been developed as a submaximal endurance test for both legs (leg-cycling) and arms (arm-cranking) for children who are expected to lose their walking ability in the near future or are wheel chair dependent. Only arm-cranking will be applied to this study.
Measured: Baseline and up to Week 12
Change from baseline in the a6MCT maximal attained revolutions
The a6MCT has been developed as a submaximal endurance test for both legs (leg-cycling) and arms (arm-cranking) for children who are expected to lose their walking ability in the near future or are wheel chair dependent. Only arm-cranking will be applied to this study.
Measured: Baseline and up to Week 12
Change from baseline in fat fraction by magnetic resonance spectroscopy (MRS)
The fat fraction by MRS will be assessed for the vastus lateralis (VL) and soleus (SOL) muscles.
Measured: Baseline, Week 12 and Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Dec 2019
- Study start24 Feb 2021
- Primary completion4 Sep 2022
- Study completion4 Sep 2022
Why it stopped: Operational Reasons
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular Dystrophy (DMD)Studies 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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Astellas Pharma Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Associate Director, Study director, Astellas Pharma Inc
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04184882
- Sponsor's protocol ID
- 0367-CL-0102
- Status last verified
- Oct 2024
- First submitted
- 2 Dec 2019
- Last updated
- 22 Oct 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: Access to anonymized individual participant level data will not be provided for this trial. Further details on Astellas' data sharing policy can be found at https://www.clinicaltrials.astellas.com/transparency/.
Study at a glance
From the registry record, last updated by the sponsor on 22 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double Blind, Placebo-Controlled Phase 1b Study With Open-Label Extension to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Patients With Duchenne Muscular Dystrophy (DMD)
- ClinicalTrials.gov ID
- NCT04184882
- Acronym / sponsor ID
- 0367-CL-0102
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Keywords
- tolerability, ASP0367, MA-0211, safety
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Operational Reasons
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Astellas Pharma IncIndustry (pharma, biotech, devices)
- Study start
- 24 Feb 2021 (actual)
- Primary completion
- 4 Sep 2022 (actual)
- Study completion
- 4 Sep 2022 (actual)
- First posted
- 4 Dec 2019 (actual)
- 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.
The primary purpose of this study is to evaluate the safety and tolerability of ASP0367.
This study will also evaluate the pharmacokinetics, pharmacodynamics and efficacy on muscle function of ASP0367.
Detailed description
This study is comprised of a 4-week pre-treatment screening period, 24-week treatment period and 4-week post-treatment follow-up period. The 24-week treatment period consists of a 12-week double-blind (DB) part and 12-week open-label extension (OLE) part and each part includes a 2 week Low dose Period and a 10-week High-dose Period.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ASP0367 group | Experimental | Drug: BocidelparParticipants will be dosed investigational product (IP) at the low dose for 2 weeks and then at the high dose for 10 weeks with the total duration of 12 weeks in the DB part and OLE part, respectively. |
| Placebo to ASP0367 group | Placebo comparator | Drug: Bocidelpar, Drug: PlaceboParticipants will be dosed matching placebo in the DB part. In OLE part, participants will dosed IP at the low dose for 2 weeks and then at the high dose for 10 weeks with the total duration of 12 weeks. |
Who may qualify
- Ages
- 8 to 16 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (14 listed)
- Subject has a diagnosis of Duchenne muscular dystrophy (DMD) (confirmed by Central Genetic Counselor) defined as a clinical picture consistent with typical DMD and 1 of the following:
- A male subject of reproductive potential (Tanner Stage 2 and above) must agree to do either of the following from screening throughout the study until 30 days after the last dose of the investigational product (IP):
- Subject has been on a stable regimen of corticosteroids for 6 months prior to the time of enrollment (at baseline).
- Subject has been on stable cardiac therapy for 3 months prior to the time of enrollment (at baseline), if used, which may include prophylactic angiotensin-converting enzyme inhibitors (ACE), angiotensin II receptor blocker (ARB), aldosterone receptor antagonists (e.g., spironolactone, eplerenone), and/or beta-blocker therapy or a combination therapy thereof.
- Subject is unable to complete the 10 meter run/walk in <6 seconds at screening.
Key exclusion criteria (17 listed)
- Subject has had an acute illness (i.e., upper respiratory or viral infection) within 4 weeks prior to study enrollment (at baseline), which precludes participation.
- Subject has a cardiac ejection fraction < 53% on echocardiogram at screening.
- Subject has a mean QT interval from triplicate electrocardiogram (ECG) using Fridericia's correction (QTcF) of > 450 msec at screening. If the mean QTcF exceeds the limits stated above, 1 additional triplicate ECG can be taken and utilized at screening.
- Subject has cardiac troponin I (cTnI) above the upper limit of normal (ULN) at screening and is assessed clinically significant.
- Subject has used coenzyme Q10 (CoQ10), idebenone, carnitine, or other mitochondrial focused supplements or drugs within 4 weeks prior to randomization at baseline. In addition, subject has used any peroxisome proliferator-activated receptors (PPAR) ligands such as fibrates and thiazolidinediones 4 weeks prior to randomization at baseline.
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 |
|---|---|---|
| BocidelparAlso: MA-0211, ASP0367Oral | Drug | ASP0367 group, Placebo to ASP0367 group |
| PlaceboOral | Drug | Placebo to ASP0367 group |
Primary outcome
Number of participants with Treatment Emergent Adverse Events (TEAEs)
Time frame: Up to Week 28
An AE is any untoward medical occurrence in a subject administered an investigational product (IP), and which does not necessarily have to have a causal relationship with this treatment. A TEAE is defined as an AE observed after starting administration of the investigational product (IP) to 28 days after the last dose of IP for the double blind part or moving to the open-label extension part, whichever comes first. An IP-related TEAE is defined as any TEAE with a causal relationship of "yes" by the investigator.
Number of participants with vital sign abnormalities and/or AEs
Time frame: Up to Week 28
Number of participants with potentially clinically significant vital sign values.
Number of participants with body weight change abnormalities and/or AEs
Time frame: Up to Week 28
Number of participants with potentially clinically significant body weight.
Number of participants with electrocardiogram (ECG) abnormalities
Time frame: Up to Week 28
Number of participants with potentially clinically significant 12-ECG values.
Number of participants with echocardiography abnormalities and/or AEs
Time frame: Up to Week 28
Number of participants with potentially clinically significant echocardiography values.
Number of participants with laboratory value abnormalities and/or AEs
Time frame: Up to Week 28
Number of participants with potentially clinically significant laboratory values.
Number of participants with suicidal ideation and/or behavior as assessed by Columbia-Suicide Severity Rating Scale (C-SSRS) Change from baseline in Columbia-Suicide Severity Rating Scale (C-SSRS)
Time frame: Baseline and up to Week 28
The Columbia-Suicide Severity Rating Scale (C-SSRS) is a clinician administered assessment tool that evaluates suicidal ideation and behavior. Number of participants that have an affirmative response provided to the 5 items for suicidal ideation (1. Wish to be dead, 2. Non-specific active suicidal thoughts, 3. Active suicidal ideation with any methods (not plan) without intent to act, 4. Active suicidal ideation with some intent to act, without specific plan, 5. Active suicidal ideation with specific plan and intent) and/or to the 6 items for suicidal behavior (1. Actual attempt, 2. Interrupted attempt, 3. Aborted attempt, 4. Preparatory acts or behavior, 5. Suicidal Behavior 6. Completed suicide,) will be reported.
Change from baseline in digit span test
Time frame: Baseline and up to Week 24
The Digit span test is a subtest of Wechsler Intelligence Scale for children (WISC). This test comprises 3 parts on the fifth edition (WISC-V). Digit Span Forward requires the subject to repeat numbers in the same order as presented by the interviewer. Digit Span Backward requires the subject to repeat the numbers in the reverse order of that presented by the interviewer. Digit Span Sequencing requires the subject to sequentially order the numbers presented by the interviewer. Scores of this test are based on each raw score and total raw score.
10 secondary outcomes
Pharmacokinetics (PK) of ASP0367 in plasma: AUC from the time of dosing to the start of next dosing interval (AUCtau)
Time frame: Up to Week 2
PK of ASP0367 in plasma: maximum concentration (Cmax)
Time frame: Up to Week 2
Pharmacodynamics (PD) of ASP0367: Percent change from baseline in peroxisome proliferator-activated receptor (PPAR) delta target genes expression levels in blood
Time frame: Baseline and up to Week 4
PD of ASP0367: Percent change from baseline in serum myostatin/follistatin ratio
Time frame: Baseline and up to Week 12
Change from baseline in Performance of Upper Limb Module (PUL) (v2.0) assessment score
Time frame: Baseline and up to Week 12
Change from baseline on Pediatric Quality of Life (PedsQL) Multidimensional Fatigue Scale
Time frame: Baseline and up to Week 12
Change from baseline in distance walked in 2 minutes assessed in meters
Time frame: Baseline and up to Week 12
Percent change from baseline in the assisted 6 minute cycling test (a6MCT) maximal attained revolutions
Time frame: Baseline and up to Week 12
Change from baseline in the a6MCT maximal attained revolutions
Time frame: Baseline and up to Week 12
Change from baseline in fat fraction by magnetic resonance spectroscopy (MRS)
Time frame: Baseline, Week 12 and Week 24
Results and safety
The study was stopped early (reason given: Operational Reasons). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Oct 2024.
- Link to plain language summary of the study on the Trial Results Summaries website.Link provided by the sponsor.
- Link to results and other applicable study documents on the Astellas Clinical Trials website.Link provided by the sponsor.
- Individual participant dataSharing planned: No. Access to anonymized individual participant level data will not be provided for this trial. Further details on Astellas' data sharing policy can be found at https://www.clinicaltrials.astellas.com/transparency/.
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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.
Associate Director study director
Astellas Pharma Inc
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Bocidelpar in people with Duchenne Muscular Dystrophy (DMD), run by Astellas Pharma Inc at 5 sites 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 17 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 22 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites 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. Some participants in this study may receive a placebo. 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 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.
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: Operational Reasons). 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 participants with Treatment Emergent Adverse Events (TEAEs) (time frame: Up to Week 28). The study lists 8 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 22 Oct 2024.