An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy

NCT03532542 | Terminated Phase 3 Results DMC FDA Drug

• 2 other identifiers: 4045-3022017-004625-32
• Study Type: interventional
• Target: 171
• Locations: 13 countries
• Sites: 50

Brief Summary

The main objective of this study is to evaluate the safety and tolerability of long-term treatment with casimersen or golodirsen in patients with Duchenne muscular dystrophy (DMD).

Conditions

Duchenne Muscular Dystrophy

Keywords

Duchenne muscular dystrophy; Exon Skipping; DMD; Exon 53; Exon 45; Duchenne

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Treatment-emergent Serious Adverse Events (TESAEs)

  A treatment-emergent adverse event (TEAE) was any untoward medical occurrence in a clinical study participant that did not necessarily have a causal relationship with the study drug. A TEAE could, therefore, be any unfavorable and unintended symptom, sign, disease, condition, or test abnormality that occurred during or after administration of the study drug, whether or not considered related to the study drug. A TESAE was any TEAE that resulted in any of the following outcomes: death, a life-threatening event, required or prolonged inpatient hospitalization, persistent or significant disability/incapacity, or an important medical event (that is, may have jeopardized the participant and may have required medical or surgical intervention to prevent one of the previously mentioned outcomes). A summary of serious and all other non-serious TEAEs regardless of causality is located in the Reported Adverse Events module.

  Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)

Study Arms (2)

Casimersen

EXPERIMENTAL

Patients amenable to exon 45 skipping who have completed a clinical trial evaluating casimersen will receive open-label casimersen intravenous (IV) infusions, weekly, at 30 mg/kg for up to 144 Weeks.

Drug: Casimersen

Golodirsen

EXPERIMENTAL

Patients amenable to exon 53 skipping who have completed a clinical trial evaluating golodirsen will receive open-label golodirsen intravenous (IV) infusions, weekly, at 30 mg/kg for up to 144 Weeks.

Drug: Golodirsen

Interventions

Casimersen DRUG

Casimersen solution for IV infusion

Also known as: SRP-4045, AMONDYS 45

Casimersen

Golodirsen DRUG

Golodirsen solution for IV infusion

Also known as: SRP-4053, VYONDYS 53

Golodirsen

Eligibility Criteria

• Age: 7 Years - 23 Years
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Completed a clinical trial evaluating casimersen or golodirsen, per protocol.
• Is between 7 and 23 years of age, inclusive, at enrollment.

Sponsors & Collaborators

• Sarepta Therapeutics, Inc.: lead

Study Sites (50)

Neuromuscular Research Center

Phoenix, Arizona, 85028, United States

Location

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

University of California Los Angeles

Los Angeles, California, 90095, United States

Location

Stanford Neuroscience Health Center

Palo Alto, California, 94304, United States

Location

Rady Children's Hospital- San Diego

San Diego, California, 92123, United States

Location

UF Health: University of Florida Clinical Research Center

Gainesville, Florida, 32610, United States

Location

NW Florida Clinical Research Group, LLC

Gulf Breeze, Florida, 32561, United States

Location

Rare Disease Research, LLC

Atlanta, Georgia, 30318, United States

Location

Ann and Robert H Lurie Childrens Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

University of Iowa Childrens Hospital

Iowa City, Iowa, 52242, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

Kennedy Krieger Institute

Baltimore, Maryland, 21205, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

St. Louis Children's Hospital

St Louis, Missouri, 63110, United States

Location

Las Vegas Clinic

Las Vegas, Nevada, 89145, United States

Location

University of Rochester Medical Center - Department of Neurology

Rochester, New York, 14642, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Abigail Wexner Research Institute at Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Shriners Hospital for Children

Portland, Oregon, 97239, United States

Location

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, 15224, United States

Location

Children's Medical Center Dallas

Dallas, Texas, 75207, United States

Location

Texas Children's Hospital

Houston, Texas, 77030, United States

Location

University of Utah - PPDS

Salt Lake City, Utah, 84132, United States

Location

Children's Hospital of The King's Daughters

Norfolk, Virginia, 23510, United States

Location

Children's Hospital of Wisconsin, Corporate Center Suite 540

Milwaukee, Wisconsin, 53226, United States

Location

UZ Gent

Ghent, Oost-Vlaanderen, 9000, Belgium

Location

UZ Leuven

Leuven, 3000, Belgium

Location

University Multiprofile Hospital for active treatment Aleksandrovska EAD Clinic of Neurological Diseases

Sofia, 1431, Bulgaria

Location

Alberta Children's Hospital

Calgary, Alberta, T3B6A8, Canada

Location

Children's Hospital of Eastern Ontario

Ottawa, Ontario, K1H 8L1, Canada

Location

Fakultni nemocnice Brno, Klinika detske neurologie LF MU a FN Brno

Brno, 613 00, Czechia

Location

Fakultni nemocnice v Motole

Prague, 150 06, Czechia

Location

Hotel Dieu- CHU Nantes

Nantes, 44093, France

Location

Hôpital Armand Trousseau

Paris, 75013, France

Location

Universitatsklinikum Freiberg

Freiburg im Breisgau, Baden-Wurttemberg, 70106, Germany

Location

Universitatsklinikum Essen

Essen, North Rhine-Westphalia, 45147, Germany

Location

Schneider Children's Medical Center of Israel

Petah Tikva, 4920235, Israel

Location

UOSD Centro Traslazionale di Miologia e Patologie Neurogenerative

Genoa, Liguria, 16147, Italy

Location

Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant' Anna

Cona, 44124, Italy

Location

Fondazione Policlinico Universitario A Gemelli

Milan, 20123, Italy

Location

Samodzielny Publiczny Centralny Szpital Kliniczny

Warsaw, Masovian Voivodeship, 02-097, Poland

Location

Uniwersyteckie Centrum Kliniczne, Klinica Neurologii Rozwojowej

Gdansk, 80-952, Poland

Location

Hospital de La Santa Creu i Sant Pau

Barcelona, Catalonia, 08041, Spain

Location

Hospital Sant Joan de Deu

Barcelona, 8950, Spain

Location

Hospital Universitari i Politecnic La Fe de Valencia

Valencia, 46026, Spain

Location

Provningsenhet Barn, Drottning Silvias Och Ungdomssjukhus

Gothenburg, 41685, Sweden

Location

Leeds General Infirmary, Leeds Teaching Hospitals NHS Trust

Leeds, LS1 3EX, United Kingdom

Location

Alder Hey Children's NHS Foundation Trust

Liverpool, L12 2AP, United Kingdom

Location

Great Ormond Street Hospital (GOSH)

London, WC1N 1EH, United Kingdom

Location

Royal Victoria Infirmary

Newcastle upon Tyne, NE1 4LP, United Kingdom

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

casimersen; golodirsen

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Limitations and Caveats

This study was terminated before all participants had reached the last protocol-defined end-of-study visit. Study termination resulted in participants either being transitioned to a post-trial access program, to another study, or they declined further treatment.

Results Point of Contact

• Title: Medical Director
• Organization: Sarepta Therapeutics, Inc.

SareptAlly@sarepta.com

1-888-727-3782

Study Officials

• Medical Director

  Sarepta Therapeutics, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 10, 2018
• First Posted: May 22, 2018
• Study Start: August 2, 2018
• Primary Completion: July 26, 2023
• Study Completion: July 26, 2023
• Last Updated: September 19, 2024
• Results First Posted: September 19, 2024

Record last verified: 2024-09

Locations

BU (1); US (25); BE (2); PL (2); FR (2); IT (3); ES (3); GB (4); CZ (2); SE (1); DE (2); CA (2); IL (1)