Status:
ENROLLING_BY_INVITATION
Clinical, Immunological, Morphological and Genetic Characteristics of Patients With Dysferlinopathy (LGMD R2) in the RF
Lead Sponsor:
Artgen Biotech
Collaborating Sponsors:
Genotarget
Conditions:
Dysferlinopathy
Miyoshi Myopathy
Eligibility:
All Genders
18-85 years
Brief Summary
To evaluate specific characteristics of phenotype, immune status, molecular and genetic as well as morphological characteristics of adult patients with limb-girdle muscular dystrophy R2 in various reg...
Detailed Description
A single-center, cohort clinical study. Subjects of both sexes aged 18 to 65 inclusive with genetically confirmed diagnosis of limb-girdle muscular dystrophy type R2, who have signed the written infor...
Eligibility Criteria
Inclusion
- 18 to 85 (inclusive) years-old subjects of both sexes;
- A signed written informed consent form;
- Genetically confirmed diagnosis of limb-girdle muscular dystrophy (type 2B) (a case group)
Exclusion
- A subject who is an investigator, study assistant, study coordinator and a member of the other personnel indirectly or directly associated with the conduct of the study;
- Acute medical conditions associated with visceral dysfunction, life-threatening conditions which occurred less than 6 months prior to enrollment into the study such as acute cardiac, renal, hepatic insufficiency, myocardial infarction or an acute cerebrovascular accident (stroke) as well as infectious diseases;
- Excessive alcohol consumption (\> 20 g/day).
Key Trial Info
Start Date :
January 15 2020
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
July 1 2027
Estimated Enrollment :
100 Patients enrolled
Trial Details
Trial ID
NCT04824040
Start Date
January 15 2020
End Date
July 1 2027
Last Update
June 27 2025
Active Locations (1)
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1
Human Stem Cells Institute
Moscow, Russia, 119333