Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy

NCT01480245 | Terminated Phase 3

• 1 other identifier: 114349
• Study Type: interventional
• Target: 233
• Locations: 24 countries
• Sites: 59

Brief Summary

The purpose of this study is to explore long-term safety, tolerability and efficacy of GSK2402968 in DMD subjects who previously participated in either DMD114117 or DMD114044.

Conditions

Muscular Dystrophies

Keywords

968; Duchenne; DMD; drisapersen

Outcome Measures

Primary Outcomes (1)

• Differences between the 6MWD at baseline and Week 104

  104 weeks

Secondary Outcomes (11)

• Timed Function tests

  104 weeks

• Muscle strength

  104 weeks

• North Star Ambulatory Assessment Scores

  104 weeks

• Creatine kinase Serum concentrations

  104 weeks

• Pulmonary Function

  104 weeks

Study Arms (3)

Continuous Dosing

EXPERIMENTAL

GSK2402968 6mg/kg/week

Drug: GSK2402968

Intermittent Dosing

EXPERIMENTAL

GSK2402968 6mg/kg/week

Drug: GSK2402968

Natural History Observation

NO INTERVENTION

The objective of this arm will be to explore DMD disease progression in a naturalistic setting once discontinuing active treatment

Interventions

GSK2402968 DRUG

6mg/kg/week

Continuous Dosing; Intermittent Dosing

Eligibility Criteria

• Age: 5 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Previous participation in either DMD114117 or DMD114044
• Continued use of glucocorticoids
• Willing and able to comply with all protocol requirements
• Able to give informed consent

You may not qualify if:

• Subject experienced a serious adverse event or who met safety stopping criteria that remains unresolved from DMD114117 or DMD114044, which in opinion of the investigator could have been attributable to study medication and is ongoing,
• Use of anticoagulants, antithrombotics or antiplatelet agents, previous treatment with investigational drugs,except GSK2402968, within 1 month of the first administration of study medication,
• Current or anticipated participation in any investigational clinical studies,
• History of significant medical disorder which may confound the interpretation of either efficacy or safety data e.g. current history of renal or liver disease/impairment, history of inflammatory disease.

Sponsors & Collaborators

• GlaxoSmithKline: lead

Study Sites (59)

GSK Investigational Site

Ciudad Autónoma de Buenos Aires, Buenos Aires, C1125ABD, Argentina

Location

GSK Investigational Site

Westmead, New South Wales, 2145, Australia

Location

GSK Investigational Site

Parkville, Victoria, 3052, Australia

Location

GSK Investigational Site

Ghent, 9000, Belgium

Location

GSK Investigational Site

Leuven, 3000, Belgium

Location

GSK Investigational Site

Curitiba, Paraná, 80250-060, Brazil

Location

GSK Investigational Site

Rio de Janeiro, Rio de Janeiro, 21941-490, Brazil

Location

GSK Investigational Site

Porto Alegre, Rio Grande do Sul, 90035-903, Brazil

Location

GSK Investigational Site

Ribeirão Preto, São Paulo, 14048-900, Brazil

Location

GSK Investigational Site

Santo André, São Paulo, 09060-650, Brazil

Location

GSK Investigational Site

São Paulo, São Paulo, 05403-000, Brazil

Location

GSK Investigational Site

Sofia, 1431, Bulgaria

Location

GSK Investigational Site

Vancouver, British Columbia, V6H 3V4, Canada

Location

GSK Investigational Site

London, Ontario, N6A 5W9, Canada

Location

GSK Investigational Site

Montreal, Quebec, H3T 1C5, Canada

Location

GSK Investigational Site

Temuco, Región de La Araucania, Chile

Location

GSK Investigational Site

Santiago, Región Metro de Santiago, 7500539, Chile

Location

GSK Investigational Site

Santiago, 8330074, Chile

Location

GSK Investigational Site

Brno, 613 00, Czechia

Location

GSK Investigational Site

Prague, Czechia

Location

GSK Investigational Site

Koebenhavn Oe, 2100, Denmark

Location

GSK Investigational Site

Bordeaux, 33076, France

Location

GSK Investigational Site

Lille, 59037, France

Location

GSK Investigational Site

Marseille, 13385, France

Location

GSK Investigational Site

Montpellier, 34295, France

Location

GSK Investigational Site

Nantes, 44093, France

Location

GSK Investigational Site

Paris, 75013, France

Location

GSK Investigational Site

Paris, 75743, France

Location

GSK Investigational Site

Pau, 64046, France

Location

GSK Investigational Site

Toulouse, 31059, France

Location

GSK Investigational Site

Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany

Location

GSK Investigational Site

Munich, Bavaria, 80337, Germany

Location

GSK Investigational Site

Göttingen, Lower Saxony, 37075, Germany

Location

GSK Investigational Site

Essen, North Rhine-Westphalia, 45122, Germany

Location

GSK Investigational Site

Kiel, Schleswig-Holstein, 24105, Germany

Location

GSK Investigational Site

Budapest, 1095, Hungary

Location

GSK Investigational Site

Jerusalem, 91240, Israel

Location

GSK Investigational Site

Ferrara, Emilia-Romagna, 44100, Italy

Location

GSK Investigational Site

Rome, Lazio, 00165, Italy

Location

GSK Investigational Site

Rome, Lazio, 00168, Italy

Location

GSK Investigational Site

Milan, Lombardy, 20122, Italy

Location

GSK Investigational Site

Messina, Sicily, 98125, Italy

Location

GSK Investigational Site

Hyōgo, 650-0017, Japan

Location

GSK Investigational Site

Kumamoto, 860-8556, Japan

Location

GSK Investigational Site

Saitama, 349-0196, Japan

Location

GSK Investigational Site

Tokyo, 187-8551, Japan

Location

GSK Investigational Site

Leiden, 2333 ZA, Netherlands

Location

GSK Investigational Site

Nijmegen, 6525 GC, Netherlands

Location

GSK Investigational Site

Oslo, 0027, Norway

Location

GSK Investigational Site

Warsaw, 02-097, Poland

Location

GSK Investigational Site

Moscow, 125412, Russia

Location

GSK Investigational Site

Seoul, 110-744, South Korea

Location

GSK Investigational Site

Esplugues de Llobregat. Barcelona, 08950, Spain

Location

GSK Investigational Site

Madrid, 28046, Spain

Location

GSK Investigational Site

Valencia, 46026, Spain

Location

GSK Investigational Site

Kaohsiung City, 80708, Taiwan

Location

GSK Investigational Site

Ankara, 06100, Turkey (Türkiye)

Location

GSK Investigational Site

London, WC1N 3JH, United Kingdom

Location

GSK Investigational Site

Newcastle upon Tyne, NE1 4LP, United Kingdom

Location

MeSH Terms

Conditions

Muscular Dystrophies; Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

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

Study Officials

• GSK Clinical Trials

  GlaxoSmithKline

  STUDY DIRECTOR

Study Design

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

Study Record Dates

• First Submitted: November 23, 2011
• First Posted: November 28, 2011
• Study Start: September 1, 2011
• Primary Completion: March 1, 2014
• Study Completion: March 1, 2014
• Last Updated: March 23, 2017

Record last verified: 2017-03

Locations

DK (1); BE (2); HU (1); AU (2); IL (1); BR (6); CA (3); NL (2); CL (3); JP (4); ES (3); TW (1); TU (1); NO (1); CZ (2); RU (1); DE (5); PL (1); AR (1); BU (1); FR (9); IT (5); KR (1); GB (2)