Duchenne Muscular DystrophyPhase 2NCT02752048
A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy
A Phase 2 study testing TAS-205 in people with Duchenne Muscular Dystrophy, run by Taiho Pharmaceutical Co., Ltd. at 11 sites in Japan.
Trial summary
The objective of this study is to evaluate the efficacy after 24-week repeated oral doses of TAS-205 in patients with Duchenne Muscular Dystrophy (DMD) in an exploratory manner. Read more about this study
- Who is it for?
- Ages 5 years and older, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- TAS-205
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 36 participants
- Where
- 11 sitesJapan
- Study dates
- May 2016to 17 Oct 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 5 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Able to give an informed consent. If applicable, able to give an informed assent.
- Phenotypic evidence of DMD.
- Male and ≧5 years of age.
- Bodyweight ≧7.5 kg and <60 kg.
- Able to complete the 6MWD test with a distance of at least 75 m.
- Able to take tablets.
- If taking oral glucocorticoids no significant change in the total daily or dosing 6 months before enrollment.
Who cannot join
- Any serious drug allergy.
- A forced vital capacity (FVC) of <50% of predicted value.
- Wearing a respirator continuously (except for the use during sleep).
- A left ventricular ejection fraction (EF) of <40% or fractional shortening (FS) of <25% on echocardiogram.
- Clinically significant cardiac failure and respiratory failure.
- Ongoing immunosuppressive therapy (other than corticosteroids) .
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- Surgical history or plan for surgery that may affect muscular strength or motor function.
- Any injury that may affect muscular strength or motor function.
- With any systemic allergic disease or any chronic inflammatory disease.
- Previous gene therapy (exon skipping, or stop codon read through therapy), cell-based therapy, or any other investigational agents.
Trial locations and contacts
11 sites in 1 country.
11 sites in 1 country
Japan11 sites
Nagoya City University Hospital
Aichi, 467-8601
National Hospital Organization Nagara Medical Center
Gifu, 502-8558
Kobe University Hospital
Hyōgo, 650-0017
National Hospital Organization Utano Hospital
Kyoto, 616-8255
Shinshu University Hospital
Nagano, 390-8621
National Hospital Organization Niigata National Hospital
Niigata, 945-8585
National Hospital Organization Toneyama National Hospital
Osaka, 560-8552
National Hospital Organization Higashisaitama Hospital
Saitama, 349-0196
Tokyo Women's Medical University Hospital
Tokyo, 162-8666
National Center of Neurology and Psychiatry
Tokyo, 187-8551
Tottori University Hospital
Tottori, 683-8504
About this study
Official title: A Randomized Phase IIa Study of TAS-205 in Patients With Duchenne Muscular Dystrophy
The objective of this study is to evaluate the efficacy after 24-week repeated oral doses of TAS-205 in patients with Duchenne Muscular Dystrophy (DMD) in an exploratory manner.
Read the detailed description
Duchenne Muscular Dystrophy (DMD) is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in 3,500 lives male births. DMD patients suffer from a relentless decline in muscle strength that impairs the ability of walking and breathing, resulting in their lives with wheelchairs and then loss of upper body function. The main objective of this study is to evaluate the efficacy after 24-week repeated oral doses of TAS-205 in patients with DMD in an exploratory manner. The objective of this study is also to evaluate the safety, the dose-response and the urinary excretion of pharmacodynamic (PD) marker after 24-week repeated oral doses of TAS-205 in DMD patients.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- TAS-205, Placebo
- 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.
TAS-205Drug
2 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal
PlaceboDrug
1 group: Placebo group. Oral administration for 24 weeks, BID after meal
| Study group | Role | What they receive |
|---|---|---|
| TAS-205（Low dose group） | Experimental | Low dose group：Oral administration of tablets for 24 weeks, bis in die (BID) after meal The number of tablets of the study drug corresponding to the dosage (6.67-13.33 mg/kg/dose) by body weight within 14 days before enrollment was to be administered within 30 minutes after breakfast and dinner. Receives: Drug: TAS-205 |
| TAS-205（High dose group） | Experimental | High dose group: Oral administration of tablets for 24 weeks, bis in die (BID) after meal The number of tablets of the study drug corresponding to the dosage (13.33-26.67 mg/kg/dose) by body weight within 14 days before enrollment was to be administered within 30 minutes after breakfast and dinner. Receives: Drug: TAS-205 |
| Placebo | Placebo comparator | Placebo group: Oral administration of tablets for 24 weeks, BID after meal Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean Change From Baseline to 24 Weeks in the 6-minute Walk Distance (6MWD)
The distance the subject can walk as fast as possible in 6 minutes will be evaluated.
Measured: baseline, 24 weeks
Secondary outcomes (3)
Mean Change From Baseline in Time to Rise From the Floor
The time required for the subject to rise from a supine position on the floor as quickly as possible will be evaluated.
Measured: baseline, and 24 weeks
Mean Change From Baseline in Time to Walk/Run for 10meters
The time required for the subject to run or walk as quickly as possible a 10 m-wide passage with marks affixed on the floor will be evaluated.
Measured: baseline, and 24 weeks
Mean Change From Baseline in Time to up and go (TUG)
This test will assess the extent of the subject's composite mobility, including standing up, walking, repositioning the body, and balancing.
Measured: baseline, and 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Apr 2016 (estimated)
- Study startMay 2016
- Primary completion15 May 2017
- Study completion17 Oct 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.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- Taiho Pharmaceutical Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Taiho Pharmaceutical Co., Ltd., Study director, Taiho Pharmaceutical Co., Ltd.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02752048
- Sponsor's protocol ID
- Taiho10053040
- Status last verified
- Mar 2020
- First submitted
- 6 Apr 2016
- Last updated
- 20 Apr 2020
- Results posted
- Yes, on ClinicalTrials.gov
Publications
Komaki H, Maegaki Y, Matsumura T, Shiraishi K, Awano H, Nakamura A, Kinoshita S, Ogata K, Ishigaki K, Saitoh S, Funato M, Kuru S, Nakayama T, Iwata Y, Yajima H, Takeda S. Early phase 2 trial of TAS-205 in patients with Duchenne muscular dystrophy. Ann Clin Transl Neurol. 2020 Feb;7(2):181-190. doi: 10.1002/acn3.50978. Epub 2020 Jan 20.
PubMed 31957953
Study at a glance
From the registry record, last updated by the sponsor on 20 Apr 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized Phase IIa Study of TAS-205 in Patients With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT02752048
- Acronym / sponsor ID
- Taiho10053040
- Conditions
- Duchenne Muscular Dystrophy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- Taiho Pharmaceutical Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- May 2016
- Primary completion
- 15 May 2017 (actual)
- Study completion
- 17 Oct 2017 (actual)
- First posted
- 26 Apr 2016 (estimated)
- Results
- Posted 20 Apr 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The objective of this study is to evaluate the efficacy after 24-week repeated oral doses of TAS-205 in patients with Duchenne Muscular Dystrophy (DMD) in an exploratory manner.
Detailed description
Duchenne Muscular Dystrophy (DMD) is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in 3,500 lives male births. DMD patients suffer from a relentless decline in muscle strength that impairs the ability of walking and breathing, resulting in their lives with wheelchairs and then loss of upper body function. The main objective of this study is to evaluate the efficacy after 24-week repeated oral doses of TAS-205 in patients with DMD in an exploratory manner. The objective of this study is also to evaluate the safety, the dose-response and the urinary excretion of pharmacodynamic (PD) marker after 24-week repeated oral doses of TAS-205 in DMD patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| TAS-205（Low dose group） | Experimental | Drug: TAS-205Low dose group：Oral administration of tablets for 24 weeks, bis in die (BID) after meal The number of tablets of the study drug corresponding to the dosage (6.67-13.33 mg/kg/dose) by body weight within 14 days before enrollment was to be administered within 30 minutes after breakfast and dinner. |
| TAS-205（High dose group） | Experimental | Drug: TAS-205High dose group: Oral administration of tablets for 24 weeks, bis in die (BID) after meal The number of tablets of the study drug corresponding to the dosage (13.33-26.67 mg/kg/dose) by body weight within 14 days before enrollment was to be administered within 30 minutes after breakfast and dinner. |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo group: Oral administration of tablets for 24 weeks, BID after meal |
Who may qualify
- Ages
- 5 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Able to give an informed consent. If applicable, able to give an informed assent.
- Phenotypic evidence of DMD.
- Male and ≧5 years of age.
- Bodyweight ≧7.5 kg and <60 kg.
- Able to complete the 6MWD test with a distance of at least 75 m.
Key exclusion criteria (10 listed)
- Any serious drug allergy.
- A forced vital capacity (FVC) of <50% of predicted value.
- Wearing a respirator continuously (except for the use during sleep).
- A left ventricular ejection fraction (EF) of <40% or fractional shortening (FS) of <25% on echocardiogram.
- Clinically significant cardiac failure and respiratory failure.
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 |
|---|---|---|
| TAS-2052 groups: Low dose group, High dose group. Oral administration for 24 weeks, bis in die (BID) after meal | Drug | TAS-205（High dose group）, TAS-205（Low dose group） |
| Placebo1 group: Placebo group. Oral administration for 24 weeks, BID after meal | Drug | Placebo |
Primary outcome
Mean Change From Baseline to 24 Weeks in the 6-minute Walk Distance (6MWD)
Time frame: baseline, 24 weeks
The distance the subject can walk as fast as possible in 6 minutes will be evaluated.
3 secondary outcomes
Mean Change From Baseline in Time to Rise From the Floor
Time frame: baseline, and 24 weeks
Mean Change From Baseline in Time to Walk/Run for 10meters
Time frame: baseline, and 24 weeks
Mean Change From Baseline in Time to up and go (TUG)
Time frame: baseline, and 24 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Apr 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 31957953Komaki H, Maegaki Y, Matsumura T, Shiraishi K, Awano H, Nakamura A, Kinoshita S, Ogata K, Ishigaki K, Saitoh S, Funato M, Kuru S, Nakayama T, Iwata Y, Yajima H, Takeda S. Early phase 2 trial of TAS-205 in patients with Duchenne muscular dystrophy. Ann Clin Transl Neurol. 2020 Feb;7(2):181-190. doi: 10.1002/acn3.50978. Epub 2020 Jan 20.
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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.
Taiho Pharmaceutical Co., Ltd. study director
Taiho Pharmaceutical Co., Ltd.
11 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 TAS-205 in people with Duchenne Muscular Dystrophy, run by Taiho Pharmaceutical Co., Ltd. at 11 sites in Japan.
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 7 inclusion and 10 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 20 Apr 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in Japan. 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 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 20 Apr 2020). 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?
Mean Change From Baseline to 24 Weeks in the 6-minute Walk Distance (6MWD) (time frame: baseline, 24 weeks). 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 NCT02752048, which also lists 1 linked publication.
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 20 Apr 2020.