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

Official title

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

Overview

A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1

Detailed description

The study consists of a Screening Period of up to either 4-weeks or 8-weeks depending on the prior parent trial, and up to a 4-year Treatment Period. The anticipated duration is 50 months and 2 weeks (4 years and 2.5 months).

Participants will receive an intravenous infusion of del-desiran at the clinical study site every 8 weeks for a total of 7 doses per year. The final dose will occur at Year 4, Visit 7, followed by a final assessment 8 weeks after the last dose.

An additional subgroup of de novo participants will also be included in a Fixed-Dose PK cohort.

An Independent Data Monitoring Committee (IDMC) comprised of members independent and external to the Sponsor will review safety, tolerability, and efficacy (as needed) data of this study at regular intervals.

Interventions

• Drug Del-desiran (AOC 1001)
  Del-desiran will be administered by intravenous (IV) infusion.

Primary outcome measures

• TEAEs [Time frame: From enrollment to the end of treatment at 4 years]

Eligibility criteria

FROM A PARENT STUDY

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

• 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.

FIXED-DOSE PK COHORT

Inclusion criteria

• Clinical and genetic diagnosis of DM1 and clinically significant hand myotonia.

Exclusion criteria

• Breastfeeding, pregnancy, or intent to become pregnant during the study
• Unwilling to comply with contraceptive requirements
• Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study
• Diabetes that is not adequately controlled
• History of decompensated heart failure within 3 months of screening. Participants with preexisting pacemaker/ICD are not excluded
• Body Mass Index > 35 kg/m2 at Screening
• Recently treated with an investigational drug or biological agent

Note: Additional protocol defined Inclusion and Exclusion criteria apply

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

N/A

Model

Single group

Masking

Open label

Primary purpose

Treatment

Study locations

United States · 19 centers

• University of California, Los Angeles (UCLA) — Los Angeles
• Stanford University — Stanford
• University of Colorado — Denver
• University of Florida — Gainesville
• University Research Center of South Florida — Tampa
• Indiana University (IU) — Indianapolis
• Kansas University Medical Center — Kansas City
• Kennedy Krieger Institute — Baltimore
• … and 11 more centers

Japan · 4 centers

• Aomori Hospital — Aomori
• National Hospital Organization Osaka Toneyama Medical Center — Osaka
• Osaka University Hospital — Osaka
• National Center of Neurology and Psychiatry — Tokyo

Canada · 2 centers

• The Ottawa Hospital — Ottawa
• Montreal Neurological Institute — Montreal

Denmark · 2 centers

• Aarhus University Hospital — Aarhus
• Rigshospitalet — Copenhagen

Netherlands · 2 centers

• Maastricht University Medical Center — Maastricht
• Stichting Radboud Universitair Medisch Centrum — Nijmegen

United Kingdom · 2 centers

• University College London Hospital — London
• St. Georges University Hospitals NHS Foundation Trust — London

France · 1 center

• AP-HP Hopital Pitie-Salpetriere — Paris

Germany · 1 center

• Klinikum der Ludwig-Maximilians-Universitaet Muenchen — München

Italy · 1 center

• Fondazione Serena Onlus - Centro Clinico NeMO Milano — Milan

Spain · 1 center

• Hospital Universitario Donostia — San Sebastián

Identifiers

NCT: NCT07008469 · AOC 1001-CS4

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗