Clinical Trials Logo
  1. Home
  2. Muscular Dystrophy
  3. NCT05230459

A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)

Muscular Dystrophy Clinical Trial

Official title:
A Two-part Multicenter Study: a Randomized, Double-blind, Placebo-controlled Dose-escalation Safety Phase (Part 1) Followed by Double-blind, Placebo-controlled, Adaptive Phase (Part 2) Study to Evaluate the Safety and Efficacy of AB-1003 in Adult Subjects With LGMD2I/R9 Mutations in the Gene Encoding Fukutin Related Protein (FKRP)

Clinical Trial Summary

The purpose of this study is to evaluate the safety and tolerability of a single intravenous infusion of AB-1003 in adults diagnosed with limb girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). Participants will be treated in sequential, dose-level cohorts. (Part 1)

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05230459
Study type Interventional
Source Asklepios Biopharmaceutical, Inc.
Contact Medical Affairs at AskBio
Phone 919-561-6210
Email AskFirst@askbio.com
Status Recruiting
Phase Phase 1/Phase 2
Start date March 12, 2023
Completion date December 2028
View Details
See also
  Status Clinical Trial Phase
Recruiting NCT01834040 - Study Safety and Efficacy of BMMNC for the Patient With Duchenne Muscular Dystrophy Phase 1/Phase 2
Recruiting NCT01834066 - Study Safety and Efficacy of Bone Marrow Derived Autologous Cells for the Treatment of Muscular Dystrophy. Phase 1/Phase 2
Recruiting NCT00082108 - Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry
Recruiting NCT00138931 - Genetics of Cardiovascular and Neuromuscular Disease
Completed NCT00622453 - Arrhythmias in Myotonic Muscular Dystrophy N/A
Active, not recruiting NCT04038138 - Clinical Trial Readiness Network FSHD France: Prospective 24 Months MRI Study N/A
Completed NCT01451281 - Studying Skeletal Muscle, Heart, and Diaphragm Imaging in Boys With Duchenne Muscular Dystrophy
Not yet recruiting NCT05470478 - iBCI Optimization for Veterans With Paralysis N/A
Completed NCT04154098 - Evaluation of a Textile Scapula Orthosis N/A
Terminated NCT02653833 - The Study of Skeletal Muscle Blood Flow in Becker Muscular Dystrophy Early Phase 1
Not yet recruiting NCT06363526 - Effectiveness of 5-week Digital Respiratory Practice in a Group of Children With Duchenne Muscular Dystrophy and Becker Muscular Dystrophy. N/A
Completed NCT01990976 - Study of Morphology and Functional Magnetic Resonance Imaging (MRI) Muscle Patients With Muscular Dystrophy Type FSHD Benefiting a Physical Training Introduced. N/A
Completed NCT01393444 - ECoG Direct Brain Interface for Individuals With Upper Limb Paralysis N/A
Recruiting NCT00912041 - BrainGate2: Feasibility Study of an Intracortical Neural Interface System for Persons With Tetraplegia N/A
Completed NCT00866112 - A Randomized Exercise Trial for Wheelchair Users N/A
Completed NCT01882400 - Assessment of Response to Treatment of Osteoporosis With Oral Bisphosphonates in Patients With Muscular Dystrophy Phase 4
Recruiting NCT05726591 - Evaluating Long-term Use of a Pediatric Robotic Exoskeleton (P.REX/Agilik) to Improve Gait in Children With Movement Disorders N/A
Completed NCT02815878 - Enhance Wellness for Individuals With Long-Term Physical Disabilities N/A
Completed NCT04035967 - Investigation of Parents' Anxiety Level and Health Related Quality of Life in Different Types of Physical Disabilities
Not yet recruiting NCT06290713 - Vasodilator and Exercise Study for DMD (VASO-REx) Phase 2