Investigator: Leo H Wang MD PhD
Category: Research - Clinical
Clinical study to determine whether MRI can be used as a noninvasive measure of the immune response and T-cell infiltration in FSHD muscle
We propose a clinical trial to test the safety and tolerability of immunosuppressive medications in FSHD patients. Our hypothesis, based on the work of Frisullo et al. and Tasca et al. suggesting that STIR+ muscle represent inflammatory changes, is that treatment with immunosuppressive medications will decrease the STIR+ changes. Tawil, et al. conducted a pilot trial of prednisone in eight FSHD patients in a 12-week open-label therapeutic trial. There was no improvement in muscle strength as assessed by manual motor testing or maximum voluntary isometric contraction testing. However, the primary outcome measure was assessed over a short duration for a chronic disease.
The study will be a non-randomized, open-label, single-group trial to assess the safety and tolerability of treating FSHD patients for 12 weeks with tacrolimus and prednisone. Safety will be determined by physical examination, vital signs, serum hematological and metabolic profiles, adverse events and serious adverse events. Tolerability will be assessed by compliance to the medication regimen and monitoring adverse events. We plan to enroll six subjects at UW. Some of the patients will have enrolled in our Wellstone study and we will have longitudinal MRIs.
Adapting MRI as a clinical outcome measure for a facioscapulohumeral muscular dystrophy trial of prednisone and tacrolimus: case report
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