Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy

NCT06138743 | Recruiting Phase 1 FDA Drug

• 2 other identifiers: SRP-1003-1012024-513579-42
• Study Type: interventional
• Target: 78
• Locations: 11 countries
• Sites: 35

Brief Summary

This is a phase 1/2a double-blinded, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of single and multiple ascending doses of SRP-1003 compared to placebo in male and female participants with type 1 myotonic dystrophy (DM1). Participants who have provided written informed consent and met all protocol eligibility requirements will be randomized to receive single (Part 1) or multiple (Part 2) doses of SRP-1003 or placebo.

Conditions

Myotonic Dystrophy 1

Keywords

Myotonic Dystrophy 1; SRP-1003; ARO-DM1

Outcome Measures

Primary Outcomes (1)

• Number of Participants with Treatment-emergent Adverse Events Over Time Through End of Study (EOS)

  Single-dose phase (Part 1): Up to Day 90 (EOS); multiple-dose phase (Part 2): Up to Day 180 (EOS)

Secondary Outcomes (11)

• PK of SRP-1003: Maximum Observed Plasma Concentration (Cmax)

  Single-dose phase (Part 1): Up 24 hours post-dose; multiple-dose phase (Part 2): Through 24 hours post first and second dose

• PK of SRP-1003: Area Under the Plasma Concentration Versus Time Curve from Zero to 24 Hours (AUC0-24)

  Single-dose phase (Part 1): Up 24 hours post-dose; multiple-dose phase (Part 2): Through 24 hours post first and second dose

• PK of SRP-1003: Area Under the Plasma Concentration Versus Time Curve from Zero to the Last Quantifiable Plasma Concentration (AUClast)

  Single-dose phase (Part 1): Up 24 hours post-dose; multiple-dose phase (Part 2): Through 24 hours post first and second dose

• PK of SRP-1003: Area Under the Plasma Concentration Versus Time Curve from Zero to Infinity (AUCinf)

  Single-dose phase (Part 1): Up 24 hours post-dose; multiple-dose phase (Part 2): Through 24 hours post first and second dose

• Change from Baseline at Day 120 for Video Hand Opening Time (vHOT)

  (Part 2): Baseline, Day 120

Study Arms (4)

SRP-1003 IV Infusion

EXPERIMENTAL

Single or multiple doses of SRP-1003 by intravenous (IV) infusion

Drug: SRP-1003 IV Infusion

Placebo by IV Infusion

PLACEBO COMPARATOR

Single or multiple doses of placebo by IV infusion

Drug: Placebo IV Infusion

SRP-1003 SC Injection

EXPERIMENTAL

Single or multiple doses of SRP-1003 by subcutaneous (SC) injection

Drug: SRP-1003 SC Injection

Placebo by SC Injection

PLACEBO COMPARATOR

Single or multiple doses of placebo by SC injection

Drug: Placebo SC Injection

Interventions

SRP-1003 IV Infusion DRUG

SRP-1003 by IV infusion

Also known as: ARO-DM1 IV Infusion

SRP-1003 IV Infusion

Placebo SC Injection DRUG

0.9% NaCl calculated volume to match active treatment by SC injection(s)

Placebo by SC Injection

Placebo IV Infusion DRUG

0.9% sodium chloride (NaCl) calculated volume to match active treatment by IV infusion

Placebo by IV Infusion

SRP-1003 SC Injection DRUG

SRP-1003 by SC injection(s)

Also known as: ARO-DM1 SC Injection

SRP-1003 SC Injection

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Genetically confirmed diagnosis of DM1
• Clinician-assessed signs of DM1 including clinically apparent myotonia
• Onset of DM1 symptoms occurred after the age of 12 years
• Walk for at least 10 meters independently at screening
• Participants of childbearing potential must agree to use highly effective contraception in addition to a condom during the study and for at least 90 days following the end of study or last dose of study drug, whichever is later. Participants must not donate sperm or eggs during the study and for at least 90 days following the end of study or last dose of study drug whichever is later.

You may not qualify if:

• Inadequately controlled diabetes
• Confirmed diagnosis of congenital DM1
• Uncontrolled hypertension
• History of tibialis anterior (TA) biopsy within 3 months of Day 1 or planning to undergo TA biopsies during the study period
• Clinically significant cardiac, liver or renal disease
• Human immunodeficiency virus infection (seropositive) at screening
• Seropositive for hepatitis B or hepatitis C at screening
• Untreated or poorly controlled epilepsy
• Treatment with anti-myotonia medication within a period of 5 half-lives of the medication prior to screening.
• Abnormal coagulation parameters at screening including platelet count, international normalized ratio, prothrombin time, and activated partial thromboplastin time

Sponsors & Collaborators

• Sarepta Therapeutics, Inc.: lead

Study Sites (35)

Liverpool Hospital

Liverpool, New South Wales, 2170, Australia

RECRUITING

Sunshine Coast University Hospital

Birtinya, Queensland, 4575, Australia

RECRUITING

Royal Brisbane And Women's Hospital

Herston, Queensland, Australia

RECRUITING

Alfred Health

Melbourne, Victoria, 3004, Australia

RECRUITING

Nucleus Network Brisbane Clinic

Herston, Australia

RECRUITING

Mater Hospital Brisbane

South Brisbane, Australia

RECRUITING

Universitair Ziekenhuis Gent

Ghent, Belgium

RECRUITING

UZ Leuven

Leuven, Belgium

RECRUITING

University Of Calgary

Calgary, Canada

RECRUITING

University Of Alberta

Edmonton, Canada

RECRUITING

McGill University

Montreal, Canada

RECRUITING

University Hospital of Angers

Angers, France

RECRUITING

Centre Hospitalier Universitaire De Bordeaux

Bordeaux, France

RECRUITING

Centre Hospitalier Universitaire De Lille

Lille, France

RECRUITING

CHU de Nice - Hôpital Pasteur 2

Nice, France

RECRUITING

Assistance Publique Hôpitaux De Paris

Paris, France

RECRUITING

Klinikum der Universität München AöR

Munich, Germany

RECRUITING

Deutsches Zentrum für Neurodegenerative Erkrankungen e.V.

Ulm, Germany

RECRUITING

Fondazione Serena Onlus - Centro Clinico Nemo Brescia

Gussago, Italy

RECRUITING

Centro Clinico Nemo (Milan)

Milan, Italy

RECRUITING

Azienda Ospedaliera Universitaria Pisana (AOUP)

Pisa, Italy

RECRUITING

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Roma, Italy

RECRUITING

New Zealand Clinical Research Christchurch

Christchurch, 8011, New Zealand

RECRUITING

Hospital Universitario Torrecárdenas

Almería, Spain

RECRUITING

Hospital Universitari Vall D Hebron

Barcelona, Spain

RECRUITING

Hospital Universitario Infanta Sofia

Madrid, Spain

RECRUITING

China Medical University Hospital

Taichung, 40447, Taiwan

RECRUITING

National Taiwan University Hospital

Taipei, Taiwan

RECRUITING

Taipei Veterans General Hospital

Taipei, Taiwan

RECRUITING

Songklanagarind Hospital

Hat Yai, Changwat Songkhla, 90110, Thailand

RECRUITING

Siriraj Hospital

Bangkok, 10700, Thailand

RECRUITING

Lampang Hospital

Lampang, 52000, Thailand

RECRUITING

The National Hospital for Neurology and Neurosurgery, NHS Foundation Trust

London, United Kingdom

RECRUITING

Nottingham University Hospitals NHS Trust

Nottingham, United Kingdom

RECRUITING

Southampton General Hospital, NHS Foundation Trust

Southampton, United Kingdom

RECRUITING

MeSH Terms

Conditions

Myotonic Dystrophy

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Myotonic Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Medical Director

  Sarepta Therapeutics, Inc.

  STUDY DIRECTOR

Central Study Contacts

Sarepta Therapeutics Inc. For Clinical Trial Information, Select Option 4

CONTACT

1-888-SAREPTA (1-888-727-3782); SareptAlly@sarepta.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 14, 2023
• First Posted: November 18, 2023
• Study Start: March 4, 2024
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: August 11, 2026

Record last verified: 2026-08

Data Sharing

• IPD Sharing: Will not share

Locations

NZ (1); TH (3); TW (3); AU (6); CA (3); DE (2); BE (2); FR (5); IT (4); ES (3); GB (3)