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Bone Marrow Transplant

Non-myeloablative conditioning and SCT for Non-malignant Inherited Disorders Sans Donor Match (FHCRC-2032)
HLA-Haploidentical Related Marrow Grafts for the Treatment of Immunodeficiency and Other Nonmalignant Disorders Using Conditioning with Low-Dose Cyclophosphamide, 200 cGy TBI and Fludarabine; Postgrafting Immunosuppression Will Consist of a Single Low Dose of Cyclophosphamide, MMF and Tacrolimus.
Status Conditions Phase Study ID
Recruiting Immunodeficiency Syndromes
Severe Combined Immunodeficiency Syndrome (SCID)
Phase II FHCRC-2032
NCT00358657
Summary

The purpose of this study is to determine the safety of non-myeloablative conditioning and stem cell transplantation for patients with non-malignant inherited disorders who do not have a genetically matched donor. Participants in this study will receive bone marrow donated by a family member whose tissue type is genetically only a partial match. Non-myeloablative conditioning (the chemotherapy and radiation given just before a transplant) uses lower doses of chemotherapy and radiation than conventional conditioning.

Most of the treatment will be given at the Seattle Cancer Care Alliance (SCCA) outpatient clinic. Initial in-hospital treatment will be given at the University of Washington Medical Center or Children?s Hospital and Regional Medical Center.

Participants will be in the study for at least one year, and will come to the SCCA clinic for treatment for about four months (about one month before the transplant and three months after the transplant).

Additional bone marrow or blood samples may be drawn at various time points for up to five years after the transplant.


Investigator
Lauri Burroughs, MD
Location    
Seattle Cancer Care Alliance 800-804-8824  
Eligibility Criteria (must meet the following to participate in this study)
  • Age 54 years or younger. 
  • Non-malignant disease treatable by allogeneic stem cell transplantation.
  • Patient is at high risk of having toxic side effects, or is ineligible for a conventional myeloablative stem cell transplantation,
  • Patient does not have a genetically matched (HLA-matched, related or unrelated) donor.

Other eligibility criteria may apply.

Exclusions (conditions that would prevent participation in this study)
  • Patients with aplastic anemia, Fanconi anemia
  • Patients with HLA-matched (genetically matched) related or unrelated donors
  • Patients with HIV
  • Patients who are female and are pregnant or breastfeeding

Other exclusion criteria may apply.

Last Updated
March 17, 2011
See this trial at ClinicalTrials.gov
Access protocol and consent forms at Fred Hutchinson Cancer Research Center
Disclaimer: We update this information regularly. However, what you read today may not be completely up to date.

Please remember:
  • Talk to your health care providers first before making decisions about your health care.
  • Whether you are eligible for a research study depends on many things. There are specific requirements to be in research studies. These requirements are different for each study.