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

A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1 - HARBOR-OLE

Status

Recruiting

Phases

Phase 3

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT2021250036

Enrollment

Registered

2025-12-10

Start date

2025-12-11

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Myotonic Dystrophy Type 1 (DM1)

Interventions

AOC 1001 will be administered via IV infusion at 4 mg/kg siRNA weight.

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion 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.

Exclusion criteria

Exclusion 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.

Design outcomes

Primary

Measure	Time frame
TEAEs (Incidence of treatment emergent adverse events)	—

Countries

Canada, Denmark, France, Germany, Italy, Japan, Netherlands, Spain, United Kingdom, United States

Contacts

Public ContactYuya Yamada

Medpace Japan K.K.

RSJapan1@medpace.com+81-3-4563-7000

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026