Muscular Dystrophies Clinical Trial
Official title:
A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Adult Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Adult Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)
AOC 1020-CS1 is a first-in-human, 3-part, multi-center, Phase 1/2, randomized, double-blind, placebo-controlled study designed to evaluate safety, tolerability, pharmacokinetics and to explore pharmacodynamics and efficacy of single and multiple-doses of AOC 1020 administered intravenously in adult participants with FSHD Type 1 (FSHD1) and FSHD Type 2 (FSHD2). Part A is a dose titration design which includes a single and multiple dose schedule with 1 cohort. Part B is a single-ascending and multiple-ascending dose design with 2 cohorts. Part C is a parallel, multi-dose cohort design with 1 cohort. For each of Part A, B, and C the patient duration is 12 months as the active treatment period is approximately 9 months followed by a 3-month follow-up period. Once participants have completed active treatment and the follow-up period, they may have the option to participate in a planned open-label extension. If patients do not enroll in the open-label extension, they will be followed through a 6-month safety follow-up period. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Terminated |
NCT01480245 -
Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
|
Phase 3 | |
| Completed |
NCT01153932 -
Phase II Doubleblind Exploratory Study of GSK2402968 in Ambulant Subjects With Duchenne Muscular Dystrophy
|
Phase 2 | |
| Completed |
NCT00027391 -
Study of Albuterol and Oxandrolone in Patients With Facioscapulohumeral Dystrophy (FSHD)
|
N/A | |
| Recruiting |
NCT04392518 -
Telerehabilitation in Proximal Muscle Weakness
|
N/A | |
| Completed |
NCT03851107 -
The Effectiveness of Participation-focused Interventions on Body Functions of Youth With Physical Disabilities
|
N/A | |
| Completed |
NCT01462292 -
A Clinical Study to Assess Two Doses of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD)
|
Phase 2 | |
| Recruiting |
NCT06094205 -
Feasibility of the BrainGate2 Neural Interface System in Persons With Tetraplegia (BG-Speech-02)
|
N/A | |
| Recruiting |
NCT05724173 -
Feasibility of the BrainGate2 Neural Interface System in Persons With Tetraplegia
|
N/A | |
| Recruiting |
NCT03698149 -
ECoG BMI for Motor and Speech Control
|
N/A | |
| Active, not recruiting |
NCT03811301 -
[BrainConnexion] - Neurodevice Phase I Trial
|
N/A | |
| Completed |
NCT05881122 -
Anti-inflammatory Diet Consultation for Those With Neuromuscular Disability
|
N/A | |
| Completed |
NCT04154098 -
Evaluation of a Textile Scapula Orthosis
|
N/A | |
| Recruiting |
NCT05409079 -
Schulze Muscular Dystrophy Ability Clinical Study
|
N/A | |
| Enrolling by invitation |
NCT04009408 -
Expiratory Muscle Strength Training (EMST) in Neuromuscular Disorders
|
N/A | |
| Active, not recruiting |
NCT00674843 -
The Efficacy of Using Far Infrared Radiation to Manage Muscular Dystrophies
|
Phase 1 | |
| Recruiting |
NCT02109692 -
Evaluation of Muscle miRNA as Biomarkers in Dystrophinopathies
|
N/A | |
| Active, not recruiting |
NCT04045158 -
Diaphragm Ultrasound in Neuromuscular Disorders
|
||
| Not yet recruiting |
NCT03508583 -
Turkish Version of The Measure of Processes of Care (MPOC)
|
||
| Terminated |
NCT01803412 -
A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects
|
Phase 3 | |
| Completed |
NCT03406780 -
A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
|
Phase 2 |