Duchenne Muscular DystrophyPhase 1NCT02246478
A Study of TAS-205 for Duchenne Muscular Dystrophy
A Phase 1 study testing TAS-205 in people with Duchenne Muscular Dystrophy, run by Taiho Pharmaceutical Co., Ltd. at 1 site in Japan.
Trial summary
The objective of this study is to evaluate the safety and pharmacokinetic of TAS-205 in patients with Duchenne Muscular Dystrophy. Read more about this study
- Who is it for?
- Ages 5 to 15 years, 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
- 23 participants
- Where
- 1 siteJapan
- Study dates
- Sep 2014to Sep 2015
- Phase
- Phase 1
- 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 to 15 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Able to give an informed consent. If applicable, able to give an informed assent.
- Male and >= 5 years and < 16 years of age.
- Bodyweight of >= 15.0 kg and < 75.0 kg.
- Phenotypic evidence of DMD.
- Able to take tablets.
- If taking oral glucocorticosteroids no significant change in total daily dosage or dosing regimen after enrollment.
- Confirmed the urinary PD marker over its criteria.
- Able to follow the study protocol.
Who cannot join
- Current diagnosis or history of any drug allergy.
- A forced vital capacity (FVC) < 50% of predicted value.
- A left ventricular ejection fraction (EF) < 50% or fractional shortening (FS) < 25% based on echocardiogram (ECHO).
- Ongoing immunosuppressive therapy (other than corticosteroids).
- With severe disease such as hepatic disease, kidney disease and others.
- With any systemic allergic disease or any chronic inflammatory disease.
- Treated with any other investigational agents within 90 days.
- Positive reaction in hepatitis B surface antigen (HbsAg), hepatitis C antibody test (HCV), or human immunodeficiency virus (HIV) test.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase I Study of Single and Multiple Doses of TAS-205 in Patients With Duchenne Muscular Dystrophy
The objective of this study is to evaluate the safety and pharmacokinetic of TAS-205 in patients with Duchenne Muscular Dystrophy.
Read the detailed description
Duchenne Muscular Dystrophy (DMD) is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 3500 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 loss of upper body function. The objective of this study is to evaluate the safety and pharmacokinetic of TAS-205 after single and multiple doses in DMD patients. It is also evaluated if TAS-205 affects the urinary excretion of pharmacodynamic (PD) marker 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
- Single-dose phase: 3 steps (low dose, middle dose or high dose group), 5 patients/step, single oral administration after meals
- Multiple-dose phase: 3 steps (low dose, middle dose or high dose group), 5 patients/step (the same patients between single- and multiple-dose phases), repeated oral administration for 7 days, BID after meals
PlaceboDrug
- Single-dose phase: 3steps (low dose, middle dose or high dose group), 2 patients/step, single oral administration after meals
- Multiple-dose phase: 3 steps (low dose, middle dose or high dose group), 2 patients/step (the same patients between single- and multiple-dose phases), repeated oral administration for 7 days, BID after meals
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
| TAS-205 low dose | Active comparator | Not described Receives: Drug: TAS-205 |
| TAS-205 middle dose | Active comparator | Not described Receives: Drug: TAS-205 |
| TAS-205 high dose | Active comparator | Not described Receives: Drug: TAS-205 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Adverse Events
Source Vocabulary Name for Table Default: CTCAE (4.03)
Measured: From the first administration day to the end of the observation period (ie. single-dose phase: 8 days, multiple-doses phase: 14 days)
Secondary outcomes (3)
Peak Plasma Concentration (Cmax) of TAS-205
Due to inspection missing, some data were not analyzed.
Measured: Single-dose phase: immediately before dosing, 0, 0.5, 1, 2, 4, 8, 24, 48 hours post-dose, Multiple-dose phase: Days 1 and 7, immediately before morning dose, 0.5, 1, 2, 4, and 8 hours post-dose and Day 4, immediately before morning dose.
Area Under the Plasma Concentration Versus Time Curve (AUC) of TAS-205
Due to inspection missing, some data were not analyzed.
Measured: Administration period (ie. single-dose phase: from single administration day to 48 hours after the administration, multiple-dose phase: from the first administration day to 8 hours after the last administration)
The Urinary Excretion of PD Marker
Ratio of prostaglandin E2 metabolite / creatinine Due to inspection missing, some data were not analyzed.
Measured: Single-dose: Day -1 before administration, 0-24 hr post-dose, and 24-48 hr post-dose, Multiple-doses: Day -1 before administration, 0 hr after administration on Day 1 and 4 to the following day (Day 2 and 5), and 0-24 hr after administration on Day 7.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Sep 2014 (estimated)
- Study startSep 2014
- Primary completionJun 2015
- Study completionSep 2015
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 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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 23 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
- NCT02246478
- Sponsor's protocol ID
- Taiho10053030
- Status last verified
- May 2021
- First submitted
- 9 Sep 2014
- Last updated
- 4 Jun 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 4 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Study of Single and Multiple Doses of TAS-205 in Patients With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT02246478
- Acronym / sponsor ID
- Taiho10053030
- Conditions
- Duchenne Muscular Dystrophy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 23 participants (actual)
- Lead sponsor
- Taiho Pharmaceutical Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- Sep 2014
- Primary completion
- Jun 2015 (actual)
- Study completion
- Sep 2015 (actual)
- First posted
- 22 Sep 2014 (estimated)
- Results
- Posted 4 Jun 2021
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 safety and pharmacokinetic of TAS-205 in patients with Duchenne Muscular Dystrophy.
Detailed description
Duchenne Muscular Dystrophy (DMD) is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 3500 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 loss of upper body function. The objective of this study is to evaluate the safety and pharmacokinetic of TAS-205 after single and multiple doses in DMD patients. It is also evaluated if TAS-205 affects the urinary excretion of pharmacodynamic (PD) marker 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
- 4
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: Placebo |
| TAS-205 low dose | Active comparator | Drug: TAS-205 |
| TAS-205 middle dose | Active comparator | Drug: TAS-205 |
| TAS-205 high dose | Active comparator | Drug: TAS-205 |
Who may qualify
- Ages
- 5 to 15 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- Able to give an informed consent. If applicable, able to give an informed assent.
- Male and >= 5 years and < 16 years of age.
- Bodyweight of >= 15.0 kg and < 75.0 kg.
- Phenotypic evidence of DMD.
- Able to take tablets.
Key exclusion criteria (8 listed)
- Current diagnosis or history of any drug allergy.
- A forced vital capacity (FVC) < 50% of predicted value.
- A left ventricular ejection fraction (EF) < 50% or fractional shortening (FS) < 25% based on echocardiogram (ECHO).
- Ongoing immunosuppressive therapy (other than corticosteroids).
- With severe disease such as hepatic disease, kidney disease and others.
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-205* Single-dose phase: 3 steps (low dose, middle dose or high dose group), 5 patients/step, single oral administration after meals * Multiple-dose phase: 3 steps (low dose, middle dose or high dose group), 5 patients/step (the same patients between single- and multiple-dose phases), repeated oral administration for 7 days, BID after meals | Drug | TAS-205 high dose, TAS-205 low dose, TAS-205 middle dose |
| Placebo* Single-dose phase: 3steps (low dose, middle dose or high dose group), 2 patients/step, single oral administration after meals * Multiple-dose phase: 3 steps (low dose, middle dose or high dose group), 2 patients/step (the same patients between single- and multiple-dose phases), repeated oral administration for 7 days, BID after meals | Drug | Placebo |
Primary outcome
Incidence of Adverse Events
Time frame: From the first administration day to the end of the observation period (ie. single-dose phase: 8 days, multiple-doses phase: 14 days)
Source Vocabulary Name for Table Default: CTCAE (4.03)
3 secondary outcomes
Peak Plasma Concentration (Cmax) of TAS-205
Time frame: Single-dose phase: immediately before dosing, 0, 0.5, 1, 2, 4, 8, 24, 48 hours post-dose, Multiple-dose phase: Days 1 and 7, immediately before morning dose, 0.5, 1, 2, 4, and 8 hours post-dose and Day 4, immediately before morning dose.
Area Under the Plasma Concentration Versus Time Curve (AUC) of TAS-205
Time frame: Administration period (ie. single-dose phase: from single administration day to 48 hours after the administration, multiple-dose phase: from the first administration day to 8 hours after the last administration)
The Urinary Excretion of PD Marker
Time frame: Single-dose: Day -1 before administration, 0-24 hr post-dose, and 24-48 hr post-dose, Multiple-doses: Day -1 before administration, 0 hr after administration on Day 1 and 4 to the following day (Day 2 and 5), and 0-24 hr after administration on Day 7.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Jun 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.
Taiho Pharmaceutical Co.,Ltd. study director
Taiho Pharmaceutical Co., Ltd.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing TAS-205 in people with Duchenne Muscular Dystrophy, run by Taiho Pharmaceutical Co., Ltd. at 1 site 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 8 inclusion and 8 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 4 Jun 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 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 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?
Yes. Results are posted in the registry (first posted 4 Jun 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?
Incidence of Adverse Events (time frame: From the first administration day to the end of the observation period (ie. single-dose phase: 8 days, multiple-doses phase: 14 days)). 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 4 Jun 2021.