NIPH Clinical Trials Search

Global Open-Label Extension Study of Del-desiran for the Treatment of DM1

Basic Information

Recruitment status	Recruiting
Health condition(s) or Problem(s) studied	Myotonic Dystrophy Type 1 (DM1)
Date of first enrollment	11/12/2025
Target sample size	16
Countries of recruitment	United States,Japan,United Kingdom,Japan,Spain,Japan,Netherlands,Japan,Italy,Japan,Germany,Japan,France,Japan,Denmark,Japan,Canada,Japan
Study type	Interventional
Intervention(s)	AOC 1001 will be administered via IV infusion at 4 mg/kg siRNA weight.

Outcome(s)

Primary Outcome	TEAEs (Incidence of treatment emergent adverse events)
Secondary Outcome

Key inclusion & exclusion criteria

Age minimum	>= 16age old
Age maximum	Not applicable
Gender	Both
Include criteria	- Completion of any prior AOC 1001 studies with satisfactory completion of dosing and follow-up assessments and satisfactory compliance with the protocol requirements of the parent study, as determined by the Investigator.
Exclude criteria	- Breastfeeding, pregnancy, or intent to become pregnant during the study. - Unwilling to comply with contraceptive requirements. - Any new conditions or worsening of existing condition that in the opinion of the Investigator would make the participant unsuitable for the study.