Calpainopathy clinical trials. org. There is only one clinical and genetic study of CAPN3 from India and none from South India. Clinical trials determine if a new test or treatment for a disease is effective and safe by comparing groups receiving different tests/treatments. Oct 1, 2024 · Our new aim is to establish calpainopathy patient-derived induced pluripotent stem cells (iPSCs) and create a disease model that recapitulates unique CAPN3 variants. Coalition to Cure Calpain 3 provides support for promising research into finding treatments or a cure for limb-girdle muscular dystrophy, type 2A (LGMD2A/R1, a form of calpainopathy). Dec 18, 2024 · * [Though this community letter is specifically about the sarcoglycanopathy clinical programs, we would like to clarify that the JOURNEY study continues to enroll people living with LGMD2A/R1, or calpainopathy, at multiple sites globally. Clinical findings of calpainopathy include the tendency to walk on tiptoe, difficulty in running, scapular winging, waddling gait, laxity of the abdominal muscles, Achilles tendon shortening, and scoliosis. Patients provided informed consent, and experimental procedures were approved by the Central Medical Ethics Committee of Latvia. Sep 21, 2023 · Calpainopathy currently has no cure. These assessments may be used in future clinical trials as tools to determine if investigational drugs are effective. Nov 9, 2021 · Calpainopathy is caused by mutations in the CAPN3 . Jun 2, 2025 · Given the absence of established pharmacological treatments for LGMD, clinical trials are currently underway to evaluate the efficacy and safety of potential drugs in patients with calpainopathy. A total of 72 (male[M]:female [F] = 34:38) genetically confirmed probands from 72 independent . In March 2024, this program obtained authorization to start a phase I/II clinical trial with the new product GNT0007 in France and Italy. IAMRARE. You will be able to choose whether you want to hear about these other studies. May 10, 2005 · Calpainopathy is characterized by symmetric and progressive weakness of proximal limb-girdle muscles. The new study, the LGMD2A/Calpainopathy Registry, creates a platform to bring the Calpainopathy community together and collect patient data that is an Identify people with Calpainopathy who might be willing to take part in other research studies or clinical trials. Calpainopathy is a progressive disease that causes weakness over time in the muscles that affect the upper extremities, including the hip and shoulder girdle muscles [1]. More information may be found on clinicaltrials. Affected individuals typically do not have cardiac involvement or intellectual disability. This observational study will follow LGMD2A/R1 patients over a period of 12 months to establish clinical outcomes assessments that are sensitive to normal disease progression. Observational studies involve recording changes over time among a specific group of people in their natural settings. To identify people with LGMD2A/Calpainopathy who might be willing to take part in other research studies or clinical trials. In March 2024, this program also obtained authorization to start a natural history study in France and Tunisia, to better understand the disease and facilitate the design of future clinical trials. How can you help drive research and speed development of a treatment? If you or your child is living with Calpainopathy, join the registry today at LGMD2A. gov (NCT04475926). vfnh eelu 6e h6rz jal0 ozgg kaad iwkar4 ush4 8sq