Safety and Efficacy Trial of MYD-0124 for Myotonic Dystrophy Type 1

JRCT ID: jRCT2051190069

Registered date:29/10/2019

Basic Information

• Recruitment status: Complete
• Health condition(s) or Problem(s) studied: Myotonic Dystrophy Type 1
• Date of first enrollment: 29/11/2019
• Target sample size: 30
• Study type: Interventional
• Intervention(s): Placebo, MYD-0124 500 mg/day, or 800 mg/day is administrated orally over the course of 24 weeks.

Outcome(s)

• Primary Outcome: Safety (the number of participants with adverse events)

Secondary Outcome

1. Mean Change from Baseline in Splicing Abnormality .
2. Mean Change From Baseline in EMG score.
3. Mean Change From Baseline in Muscle Strength.
4. Mean Change From Ambulation Using the 6 Minute Walk Distance.
5. Mean Change From Baseline in Patient-Reported Quality of Life.
6. Mean Change From Baseline in Clinical Global Impression.
7. Mean Change From Baseline in CK Value.

Key inclusion & exclusion criteria

• Age minimum: >= 20age old
• Age maximum: <= 55age old
• Gender: Both

Include criteria

1. Males or females aged 20 to 55 years old at the time of informed consent
2. Genetic confirmation of DM1
3. Ambulatory (orthoses allowed, canes and walkers not allowed) and able to walk at least 25 meters at screening

Exclude criteria

1. Treatment with macrolide antibiotics medication within 60 days prior to screening.
2. Clinically significant abnormal ECG or significant symptoms of cardiac dysfunction at Screening
3. Intellectual disability, or significant behavioral neuropsychiatric manifestations
4. Clinically significant abnormalities in screening laboratory values that would render the subject unsuitable for inclusion
5. Treatment with anti-myotonia medication within 30 days prior to screening. May include, but not be limited to: Phenytoin, Carbamazepine, Procainamide, Mexiletine
6. Recent history of or current drug or alcohol abuse
7. Have any condition, which, in the opinion of the investigator would make the subject unsuitable for inclusion, or could interfere with the subject participating in or completing the study

Related Information

• Primary Sponsor: Nakamori Masayuki
• Source(s) of Monetary Support: Japan Agency for Medical Research and Development