Clinical trial: Safety Study of Transvenous Limb Perfusion in Human Muscular Dystrophy (NCT00873782)

Eligibility criteria:
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
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.
Subject must be at least 21 Years old.


Patient description (medical language): An 8-year-old boy is brought to the clinic by his parents because of weakness and difficulty of standing up from a sitting position. The mother is healthy but had a brother who died in his 20th after being disabled and using wheelchairs in the last few years of his life. Physical examination shows 3/5 lower extremity muscle strength and enlarged calf muscles. The other physical examination and vital signs are unremarkable. Muscle biopsy showed absence of dystrophin protein. The patient is diagnosed with DMD.

Patient description (patient's own words): I brought my  8-year-old son to the doctor's clinic. He was struggling to stand up from a chair for the past few days. My wife is healthy but she was pretty worried since his brother died in his 20s after being disabled and using a wheelchair for the last years of his life. The doctor did an exam which highlighted weakness in the leg muscles and unusually large calf muscles. The rest of the exam was fine, and his vitals were normal. My son did a muscle biopsy, and it showed that the protein important for muscle strength and function is missing. The doctor diagnosed him with Duchenne muscular dystrophy.

Does the patient match the trial criteria? 0