Global Study of Del-desiran for the Treatment of DM1

JRCT ID: jRCT2051240140

Registered date:03/10/2024

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Myotonic Dystrophy Type 1 (DM1)
• Date of first enrollment: 24/09/2024
• Target sample size: 16
• Countries of recruitment: United States,Japan,United Kingdom,Japan,Spain,Japan,Netherlands,Japan,Italy,Japan,Ireland,Japan,Germany,Japan,France,Japan,Denmark,Japan,Canada,Japan
• Study type: Interventional
• Intervention(s): AOC 1001 will be administered by intravenous (IV) infusion every 8 weeks. The first dose will be 2 mg/kg of AOC 1001, all subsequent doses will be 4 mg/kg of AOC 1001.

Outcome(s)

• Primary Outcome: Hand function
• Secondary Outcome: Hand grip strength, Quantitative Muscle Testing composite score, Myotonic Dystrophy Type 1 activity and participation scale c

Key inclusion & exclusion criteria

• Age minimum: >= 16age old
• Age maximum: <= 65age old
• Gender: Both

Include criteria

1. Clinical and genetic diagnosis (CTG repeat >= 100) of DM1
2. Ability to walk independently (orthoses and ankle braces allowed) for at least 10 meters at screening

Exclude criteria

1. Breastfeeding, pregnancy, or intent to become pregnant during the study
2. Unwilling or unable to comply with contraceptive requirements
3. Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study
4. Diabetes that is not adequately controlled
5. History of decompensated heart failure within 3 months of screening. Participants with preexisting pacemaker/ICD are not excluded.
6. Body Mass Index > 35 kg/m2 at Screening
7. Recently treated with an investigational drug or biological agent
8. Treatment with anti-myotonic medication within 5 half-lives or 14 days of baseline, whichever is longer, prior to baseline.

Related Information

• Primary Sponsor: Fowler Marcie
• Secondary ID(s): NCT06411288