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Safety Study of Transvenous Limb Perfusion in Human Muscular Dystrophy
Bu çalışma, kas erimesi hastalığı (musküler distrofi, muscular dystrophy) olan bireylerde, kola veya bacağa damar yoluyla yüksek basınçla serum fizyolojik verilmesine dayanan bir yöntemin (transvenöz ekstremite perfüzyonu) güvenliliğini değerlendirmiştir. Çalışmaya, Duchenne, Becker veya bel-kavşak (limb-girdle) tipi musküler distrofi tanısı olan, ayakta durabilen 21 yaş üzeri bireyler katılabilmiştir. University of North Carolina, Chapel Hill tarafından yürütülen çalışma Faz 1 aşamasında yürütülmüş ve tamamlanmıştır.
Yukarıdaki özet, resmî kaydın sade dile aktarılmış halidir. Orijinal İngilizce başlık referans için gösterilir.
Kriterler ClinicalTrials.gov kaydından orijinal İngilizce haliyle alınmıştır. Uygunluk kararını yalnızca deneyi yürüten ekip verebilir.
Inclusion Criteria: * Diagnosis of Duchenne or Becker muscular dystrophy, as defined by progressive weakness with onset before the age of 21, X-linked inheritance, and reduced dystrophin (less than 3%) on muscle biopsy OR mutation in the dystrophin gene * Diagnosis of limb girdle muscular dystrophy, as defined by progressive weakness with onset before the age of 21, normal dystrophin on muscle biopsy OR proven mutation associated with one of the types of limb girdle dystrophy * Older than 21 years of age and preferably younger 30 years of age * Able to stand, independently or with assistance * Able to communicate with pertinent staff * Able to understand and willingly comply with the requirements of the study Exclusion Criteria: * Confirmed diagnosis of any other muscle disease * Previous compartment syndrome requiring surgical decompression * Previous venous or arterial thrombosis other than superficial venous thrombosis associated with intravenous catheter * Coagulopathy, including known diagnosis of bleeding diathesis, history of excessive bleeding on multiple occasions, or taking anticoagulant or platelet inhibitory medications * Systemic arterial or venous disease (e.g., Raynaud's, aortic coarctation or aneurysm) * Previous injury to selected limb with residual effect other than superficial scarring * Previous vascular surgery to selected limb * Previous compressive neuropathy (e.g., carpal tunnel syndrome in arm, peroneal palsy in leg) * Complex regional pain syndrome or other neurological cause of limb pain * Previous clinical diagnosis of congestive heart failure * Previous echocardiography showing ejection fraction less than 40% or ventricular dilation * Previous chest x-ray showing enlarged cardiac silhouette or pulmonary edema * History of rhabdomyolysis with worsening renal function * Creatinine greater than 1.7 mg/dL * Resting hypoxemia with SaO2 less than 90% on room air * Other significant heart, lung, or kidney disease that would compromise the body's capacity to handle a fluid load * Previous forced vital capacity less than 75% of age and height adjusted norm, in the absence of acute reversible pulmonary disease * Sickle cell disease (sickle cell anemia \[SS\] or sickle hemoglobin C disease \[SC\]) * Pregnant * Non-English speaker