Myotonic Muscular Dystrophy Clinical Trial
Official title:
Myotonic Dystrophy:Muscle Wasting and Altered Metabolism
OBJECTIVES: I. Examine the interrelationships between muscle wasting (phenotype), the degree
of myotonic dystrophy (DM) gene expression (genotype) in patients with DM.
II. Characterize the insulin resistance in these patients. III. Assess the glucose uptake in
the leg and forearm tissues of these patients.
IV. Determine the stability of the DM gene lesion in muscles over a 5-10 year period.
PROTOCOL OUTLINE: Patients are placed on a meatless diet 3 days prior to study entry.
During the first 5-day hospital stay, patients receive an oral glucose tolerance test, an
intravenous glucose tolerance test, and an intravenous infusion of insulin and glucose
(dextrose) to determine the degree of insulin resistance. Patients also receive dual x-ray
absorptiometry (DEXA) scan and total body potassium count to measure muscle mass. Patients
undergo strength testing and physical fitness screening. A needle biopsy is performed to
investigate the genetic alterations associated with this disease.
During the second 3-day hospital stay, patients receive an intravenous infusion of insulin,
stable isotopic glucose, and stable isotopic glycerol.
During the third 3-day hospital stay, a catheter is placed in the femoral artery, femoral
vein, and in each arm. Patients receive an infusion of stable isotopic glucose, stable
isotopic phenylalanine, and insulin. Measurements of the balance of amino acids and glucose
across the forearm and leg are completed. Green dye is infused to measure blood flow in the
leg.
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Observational Model: Case Control, Time Perspective: Prospective
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Recruiting |
NCT00082108 -
Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry
|
||
| Completed |
NCT05027269 -
Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients
|
Phase 1/Phase 2 |