Therapeutic Effect of Recombinant Human Growth Hormone (rhGH) on the Myopathy of Cystinosis
Status:
Withdrawn
Trial end date:
2015-11-06
Target enrollment:
Participant gender:
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 the first
12 months. Then, at month 13, if they received GH, they will switch for the next 12
months.
- Participants will take GH as a daily injection. They will be taught how to give the
injections.