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Therapeutic Effect of Recombinant Human Growth Hormone (rhGH) on the Myopathy of Cystinosis

NCT02124070 · Status: WITHDRAWN · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL

Last updated 2017-10-20

No results posted yet for this study

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.

Conditions

  • Cystinosis
  • Myopathy

Interventions

DRUG

rh Growth Hormone

Sponsors & Collaborators

  • National Human Genome Research Institute (NHGRI)

    lead NIH

Principal Investigators

  • Galina V Nesterova, M.D. · National Human Genome Research Institute (NHGRI)

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Model
CROSSOVER

Eligibility

Min Age
18 Years
Max Age
70 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-03-26
Primary Completion
2015-11-06
Completion
2015-11-06

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02124070 on ClinicalTrials.gov