NCT00074516

Kidney Transplantation in Patients With Cystinosis

Completed Last updated 6 October 2017

What this trial tests

trial in Cystinosis in 11 participants. Completed in 11 June 2014.

Timeline

10 December 2003

11 June 2014

Quick facts

Lead sponsor	National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Status	Completed
Study type	OBSERVATIONAL
Enrollment	11
Start date	10 December 2003
Estimated completion	11 June 2014
Sites	1 location across United States

Conditions studied

• Cystinosis — all drugs for Cystinosis →

Sponsor

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Who can join

7 and older, any sex, with Cystinosis. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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:

1. Kidney transplant: Patients undergo kidney transplant surgery under general anesthesia.
2. 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.
3. 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 Whole blood is withdrawn through a catheter in an arm vein or through the central line and directed into a machine that separates the blood components by spinning. The white cells are removed and the red cells and plasma are returned to the body.
4. Immunosuppressive medication following transplantation

* Adults receive thymoglobulin at the time of the transplant and for 3 days after surgery; mycophenolate mofetil daily after the transplant; tacrolimus twice a day once the kidney is working well; and sirolimus daily.
* Children receive daclizumab the day of the transplant, day 4 after surgery, and at weeks 2, 4, 6, 8, 11, 15, 19, and 23, and mycophenolate mofetil daily after the transplant.
5. Follow-up visits: After discharge from the hospital, patients return to the Clinical Center for follow-up at 6 months, at 1 year, and then yearly for 5 years. A physical examination is done the first four visits, and blood and urine samples are collected at every visit. Kidney biopsies (removal of a small amount of kidney tissue through a thin needle) are done at 6 months, 1, 3, and 5 years after the transplant. The biopsied tissue is examined to evaluate how well the kidney is responding to the immunosuppression medicines, to determine whether more or less medication is needed, and to evaluate how the patient is responding to the donor kidney.
6. Routine laboratory tests, coordinated by the patient's local physician, are done 2 to 3 times a week for the first 2 to 3 months after transplantation, then weekly for several more months, and at least monthly for life.

Publications & conference data

No peer-reviewed publications indexed yet for this trial. Completed trials usually publish results within 12-18 months.