Clinical Trial: Kidney Transplantation in Patients With Cystinosis
Study Status: Completed
Recruit Status: Completed
Study Type: Observational
Official Title: Renal Transplantation in Recipients With Nephropathic Cystinosis
Brief Summary:
This study will test the effectiveness of a combination of anti-rejection medicines in preventing complications typically seen in cystinosis patients undergoing kidney transplants. Cystinosis is a rare disease affecting children that causes growth retardation and kidney failure. Kidney transplant is the standard treatment for kidney failure in these patients, followed by immunosuppression to prevent organ rejection. The standard drug regimen for immunosuppression includes steroids, which can lead to other serious complications. This study will look at combination therapy that does not include steroids.
Patients 7 years of age and older with cystinosis who are candidates for a kidney transplant at the National Institutes of Health Clinical Center may be eligible for this 5-year study. Candidates will be screened with a medical history, physical examination, and blood tests.
Participants will undergo the following tests and procedures:
- Kidney transplant: Patients undergo kidney transplant surgery under general anesthesia.
- Central line placement: A large intravenous catheter (plastic tube, or IV line) is placed in a vein in the chest or neck under local anesthesia before the transplant surgery. The line remains in place for some time during the hospitalization to administer immunosuppressive medications, antibiotics, and blood, if needed. The line is also used to collect blood samples.
- Leukapheresis: This procedure for collecting white blood cells is done before the transplant. The cells are studied to evaluate the patient's immune system. Whole blood is withdrawn through a catheter in an arm vein or through the central line and directed into a machine that separates the blood
Detailed Summary:
Cystinosis is an autosomal recessive disorder due to impaired cystine transport across the lysosomal membrane, resulting in abnormal cystine accumulation throughout the body. Fanconi syndrome manifests in the first year of life, and end-stage renal failure occurs at ~10 years of age, requiring dialysis or kidney transplantation. Oral cysteamine therapy delays but does not always prevent the need for a renal allograft in cystinosis, which accounts for ~3% of all pediatric transplants in the United States. With improvements in immunosuppression, short-term graft and patient survival is quite good for cystinosis patients, although not as good as for patients with ESRD due to structural defects. Long-term graft survival can be affected by the non-renal complications of cystinosis, which persist after renal transplantation.
The aim of this study is to establish standards for the transplantation of kidneys into patients with nephropathic cystinosis using steroid-sparing immunosuppression. Our goals are to:
- Analyze the long and short term outcome of cystinosis patients following living and cadaveric kidney transplantation using steroid-free immunosuppression;
- Determine a consistent clinical approach to recipients with cystinosis in the immediate post-operative period, specifically regarding fluid and electrolyte management, retention of native kidneys, and timing of the re-initiation of cystinosis therapy after transplantation;
- Assess the immunologic impact of cystinosis by monitoring immune responses using a number of in vitro assays.
In this study, patients will receive a living donor or cadaveric kidney transplant with steroid free immunosuppression. Graft function, hist
Sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Current Primary Outcome:
Original Primary Outcome:
Current Secondary Outcome:
Original Secondary Outcome:
Information By: National Institutes of Health Clinical Center (CC)
Dates:
Date Received: December 12, 2003
Date Started: December 10, 2003
Date Completion: June 11, 2014
Last Updated: April 19, 2017
Last Verified: June 11, 2014
