Clinical Trial: Therapeutic Effect of Recombinant Human Growth Hormone (rhGH) on the Myopathy of Cystinosis
Study Status: Withdrawn
Recruit Status: Withdrawn
Study Type: Interventional
Official Title: Therapeutic Effect of Recombinant Human Growth Hormone (rhGH) on the Myopathy of Cystinosis
Brief Summary:
Background:
-(Degree)ystinosis is an inherited disease. If not treated correctly, it can cause muscle wasting and weakness and kidney damage. Researchers want to learn if growth hormone (GH) can help people with cystinosis.
Objective:
- To learn if GH treatment can slow or reverse muscle wasting and improve muscle strength in people with cystinosis.
Eligibility:
- People 18 and older who are already enrolled in protocol 78-HG-0093.
Design:
- Participants will be admitted to the clinic for eight 3 4 day visits, mostly four months apart.
- At each visit, participants will have a history and physical exam and give urine and blood samples.
- At month 0 or 13, participants will take tests that will be repeated at their 12- or 25-month visit:
- They will have an eye exam, medical consultations, and strength and movement tests.
- They will complete questionnaires.
- They may have tests of heart activity and lung function.
- They will have ultrasound imaging of their arm and hand muscles. They will have a scan of their legs while lying in a magnetic resonance imaging machine (a big metal cylinder). They will have a DEXA bone scan (two X-ray beams measure body composition). They will also swallow barium while X-ray imaging records the throat muscles.
- Participants will be randomly assigned to either receive or not receive GH for
