Kidney Transplantation in Patients With Cystinosis
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|ClinicalTrials.gov Identifier: NCT00074516|
Recruitment Status : Completed
First Posted : December 15, 2003
Last Update Posted : October 6, 2017
|First Submitted Date||December 12, 2003|
|First Posted Date||December 15, 2003|
|Last Update Posted Date||October 6, 2017|
|Study Start Date||December 10, 2003|
|Primary Completion Date||Not Provided|
|Current Primary Outcome Measures||Not Provided|
|Original Primary Outcome Measures||Not Provided|
|Change History||Complete list of historical versions of study NCT00074516 on ClinicalTrials.gov Archive Site|
|Current Secondary Outcome Measures||Not Provided|
|Original Secondary Outcome Measures||Not Provided|
|Current Other Pre-specified Outcome Measures||Not Provided|
|Original Other Pre-specified Outcome Measures||Not Provided|
|Brief Title||Kidney Transplantation in Patients With Cystinosis|
|Official Title||Renal Transplantation in Recipients With Nephropathic Cystinosis|
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:
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:
In this study, patients will receive a living donor or cadaveric kidney transplant with steroid free immunosuppression. Graft function, histology and graft and patient survival will be measured. Progression of cystinosis in other organ systems will be monitored. Additional studies on the graft will include transcriptional analysis of inflammatory and immunologic mediators. Serum will be assayed for inflammatory mediators and peripheral white blood cells assayed for immune function. This study will represent the first comprehensive, prospective analysis of kidney transplantation in patients with cystinosis.
|Study Design||Not Provided|
|Target Follow-Up Duration||Not Provided|
|Sampling Method||Not Provided|
|Study Population||Not Provided|
|Study Groups/Cohorts||Not Provided|
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
|Study Completion Date||June 11, 2014|
|Primary Completion Date||Not Provided|
Candidates for a kidney transplant at the National Institutes of Health Clinical Center.
Ability and willingness to provide informed consent (adults greater than or equal to 18.0 years) or assent (children greater than or equal to 7 years and less than 18.0 years).
Willingness to travel to the Clinical Center for protocol samples to be collected and the ability to send samples from a local collection source and mail them overnight.
Inability or unwillingness to comply with protocol, or to provide informed consent. Criteria include a history of noncompliance, circumstances where compliance with protocol requirements is not feasible due to living conditions, travel restrictions, lack of access to urgent medical services, or access to anti-rejection drugs once the study is completed.
Any active malignancy or history of a hematogenous malignancy or lymphoma. Patients with primary, cutaneous basal cell or squamous cell cancers may be enrolled providing the lesions are appropriately treated prior to transplantation.
Significant coagulopathy or requirement for anticoagulation therapy that would contraindicate allograft biopsy.
Any known immunodeficiency syndrome.
Peak panel reactive antibody greater than 20% or historically positive crossmatch due to HLA-specific antibodies.
Prior kidney graft survival less than 3 years or positive T or B cell crossmatch in patients receiving repeat kidney allograft.
Historical or current positive T cell cross match between donor and recipient.
Pregnancy or unwillingness to practice an approved method of birth control. Acceptable methods of birth control may include barrier methods, oral contraceptives, Norplant, Depo-Provera, or partner sterility.
|Ages||7 Years and older (Child, Adult, Older Adult)|
|Accepts Healthy Volunteers||No|
|Contacts||Contact information is only displayed when the study is recruiting subjects|
|Listed Location Countries||United States|
|Removed Location Countries|
|Other Study ID Numbers||040057
|Has Data Monitoring Committee||Not Provided|
|U.S. FDA-regulated Product||Not Provided|
|IPD Sharing Statement||Not Provided|
|Responsible Party||Not Provided|
|Study Sponsor||National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)|
|PRS Account||National Institutes of Health Clinical Center (CC)|
|Verification Date||June 11, 2014|