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Found 469 clinical trials
Prospective Natural History Study of Patients With Myotubular Myopathy and Other CentroNuclear Myopathies (NatHis-CNM)

This is the 2 years extension of the prospective and longitudinal study of the natural history and functional status of patients with myotubular myopathy and other centronuclear (CNM) sponsored

  • 25 Feb, 2022
  • 10 locations
Early Phase Human Drug Trial to Investigate Dynamin 101 (DYN101) in Patients ≥ 16 Years With Centronuclear Myopathies (Unite-CNM)

There are no available treatments aside from supportive care for patients with Centronuclear myopathy (CNM). This trial will assess the safety, tolerability, PK and PD/preliminary efficacy of a

  • 24 Feb, 2022
  • 7 locations
STREAM Trial - Statins in Multimorbid Older Adults Without Cardiovascular Disease (STREAM)

Statins are among the most widely used drugs. While they were found to be effective for primary and secondary prevention of cardiovascular disease (CVD) in middle-aged subjects, their benefits for primary prevention in older adults (aged ≥70 years) without CVD are uncertain, particularly for those with multimorbidity. The aim of …

  • 24 Mar, 2022
Tamoxifen Therapy for Myotubular Myopathy

This is a phase 1 / 2, randomized, double-blinded, single cross-over study, with a washout period between treatment regimens, to test the efficacy and safety of tamoxifen therapy to improve motor and respiratory function in males with XLMTM.

  • 04 Jul, 2021
  • 4 locations
Multi-Center Study of ManNAc for GNE Myopathy (MAGiNE)

GNE myopathy is a rare genetic muscle disease characterized by progressive muscle atrophy and weakness. The disease is caused by mutations in the gene that encodes the enzyme that initiates and

  • 14 May, 2022
  • 2 locations
An Open Label, Long Term Safety Study of REN001 in Primary Mitochondrial Myopathy Patients (Stride Ahead)

This is a long-term safety and tolerability study. REN001 is administered once daily to subjects with PMM who have previously completed Study REN001-201 or participated in Study REN001-101 and withdrew from that study due to the COVID-19 pandemic.

  • 22 Apr, 2022
  • 3 locations
MABs Therapy m.3243A>G Mutation Carriers

combat the myopathy and exercise intolerance which affect >50% of heteroplasmic mtDNA mutation carriers. Myogenic stem cells called mesoangioblasts (MABs), are currently the only myogenic precursors

  • 07 Oct, 2021
  • 1 location
Monotherapy IVIG Gamunex-C for HMG-CoA Reductase Auto-Antibody Positive Necrotizing Myopathy Treatment (The MIGHT Trial)

This is a phase 2, pilot, randomized, placebo-controlled trial of Gamunex-C IVIG as mono-therapy for HMGCoA reductase auto-antibody positive (HMGCR) necrotizing myopathy. The trial will test the

  • 24 Mar, 2022
  • 1 location
Identification of New Markers of Atrial Myopathy in Patients With Embolic Stroke of Undetermined Source (ESUS) From MRI 4D Data

Atrial myopathy is considered to be the underlying cause of a large proportion of embolic strokes of undetermined source. However, the definition of this atrial condition is not well delineated

  • 26 Mar, 2021
  • 3 locations
PReventing EXtubation FAILure Related to Cough

frequently used for patients presenting a chronic neuro-muscular disease affecting their ability to spontaneously clear their airways from an inappropriate bronchial overload. However, the interest of such

  • 02 Jun, 2021
  • 1 location