Not provided
Not provided
Not provided
Not provided
Not provided
The study was formally terminated given that GSK is not submitting an application for regulatory approval for drisapersen in Duchenne Muscular Dystrophy.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Continuous Dosing | Experimental | GSK2402968 6mg/kg/week |
|
| Intermittent Dosing | Experimental | GSK2402968 6mg/kg/week |
|
| 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 |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| GSK2402968 | Drug | 6mg/kg/week |
|
| Measure | Description | Time Frame |
|---|---|---|
| Differences between the 6MWD at baseline and Week 104 | 104 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Timed Function tests | 104 weeks | |
| Muscle strength | 104 weeks | |
| North Star Ambulatory Assessment Scores |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| GSK Clinical Trials | GlaxoSmithKline | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| GSK Investigational Site | Ciudad Autónoma de Buenos Aires | Buenos Aires | C1125ABD | Argentina | ||
| GSK Investigational Site |
| Type | Date | Date Unknown |
|---|---|---|
| Release | Aug 9, 2017 | |
| Reset | Mar 2, 2018 | |
| Release | Apr 2, 2018 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| 104 weeks |
| Creatine kinase Serum concentrations | 104 weeks |
| Pulmonary Function | 104 weeks |
| Pediatric Quality of Life Neuromuscular module | 104weeks |
| Clinician Global Impression of Improvement | 104 weeks |
| Health Utilities Index | 104 weeks |
| Frequency of accidental falls during 6 Minute Walk Distance test | 104 weeks |
| Functional Outcomes Assessment | 104 weeks |
| Time to major disease milestones | 104 weeks |
| Westmead |
| New South Wales |
| 2145 |
| Australia |
| GSK Investigational Site | Parkville | Victoria | 3052 | Australia |
| GSK Investigational Site | Ghent | 9000 | Belgium |
| GSK Investigational Site | Leuven | 3000 | Belgium |
| GSK Investigational Site | Curitiba | Paraná | 80250-060 | Brazil |
| GSK Investigational Site | Rio de Janeiro | Rio de Janeiro | 21941-490 | Brazil |
| GSK Investigational Site | Porto Alegre | Rio Grande do Sul | 90035-903 | Brazil |
| GSK Investigational Site | Ribeirão Preto | São Paulo | 14048-900 | Brazil |
| GSK Investigational Site | Santo André | São Paulo | 09060-650 | Brazil |
| GSK Investigational Site | São Paulo | São Paulo | 05403-000 | Brazil |
| GSK Investigational Site | Sofia | 1431 | Bulgaria |
| GSK Investigational Site | Vancouver | British Columbia | V6H 3V4 | Canada |
| GSK Investigational Site | London | Ontario | N6A 5W9 | Canada |
| GSK Investigational Site | Montreal | Quebec | H3T 1C5 | Canada |
| GSK Investigational Site | Temuco | Región de La Araucania | Chile |
| GSK Investigational Site | Santiago | Región Metro de Santiago | 7500539 | Chile |
| GSK Investigational Site | Santiago | 8330074 | Chile |
| GSK Investigational Site | Brno | 613 00 | Czechia |
| GSK Investigational Site | Prague | Czechia |
| GSK Investigational Site | Koebenhavn Oe | 2100 | Denmark |
| GSK Investigational Site | Bordeaux | 33076 | France |
| GSK Investigational Site | Lille | 59037 | France |
| GSK Investigational Site | Marseille | 13385 | France |
| GSK Investigational Site | Montpellier | 34295 | France |
| GSK Investigational Site | Nantes | 44093 | France |
| GSK Investigational Site | Paris | 75013 | France |
| GSK Investigational Site | Paris | 75743 | France |
| GSK Investigational Site | Pau | 64046 | France |
| GSK Investigational Site | Toulouse | 31059 | France |
| GSK Investigational Site | Freiburg im Breisgau | Baden-Wurttemberg | 79106 | Germany |
| GSK Investigational Site | Munich | Bavaria | 80337 | Germany |
| GSK Investigational Site | Göttingen | Lower Saxony | 37075 | Germany |
| GSK Investigational Site | Essen | North Rhine-Westphalia | 45122 | Germany |
| GSK Investigational Site | Kiel | Schleswig-Holstein | 24105 | Germany |
| GSK Investigational Site | Budapest | 1095 | Hungary |
| GSK Investigational Site | Jerusalem | 91240 | Israel |
| GSK Investigational Site | Ferrara | Emilia-Romagna | 44100 | Italy |
| GSK Investigational Site | Rome | Lazio | 00165 | Italy |
| GSK Investigational Site | Rome | Lazio | 00168 | Italy |
| GSK Investigational Site | Milan | Lombardy | 20122 | Italy |
| GSK Investigational Site | Messina | Sicily | 98125 | Italy |
| GSK Investigational Site | Hyōgo | 650-0017 | Japan |
| GSK Investigational Site | Kumamoto | 860-8556 | Japan |
| GSK Investigational Site | Saitama | 349-0196 | Japan |
| GSK Investigational Site | Tokyo | 187-8551 | Japan |
| GSK Investigational Site | Leiden | 2333 ZA | Netherlands |
| GSK Investigational Site | Nijmegen | 6525 GC | Netherlands |
| GSK Investigational Site | Oslo | 0027 | Norway |
| GSK Investigational Site | Warsaw | 02-097 | Poland |
| GSK Investigational Site | Moscow | 125412 | Russia |
| GSK Investigational Site | Seoul | 110-744 | South Korea |
| GSK Investigational Site | Esplugues de Llobregat. Barcelona | 08950 | Spain |
| GSK Investigational Site | Madrid | 28046 | Spain |
| GSK Investigational Site | Valencia | 46026 | Spain |
| GSK Investigational Site | Kaohsiung City | 80708 | Taiwan |
| GSK Investigational Site | Ankara | 06100 | Turkey (Türkiye) |
| GSK Investigational Site | London | WC1N 3JH | United Kingdom |
| GSK Investigational Site | Newcastle upon Tyne | NE1 4LP | United Kingdom |
| Unrelease | Aug 15, 2018 |
Not provided
| Release Date | Unrelease Date | Unrelease Date Unknown | Reset Date | MCP Release Number |
|---|---|---|---|---|
| Aug 9, 2017 | Mar 2, 2018 | |||
| Apr 2, 2018 | Aug 15, 2018 |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D040181 | Genetic Diseases, X-Linked |
Not provided
Not provided