Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich Syndrome

• Status: Completed
• Trial end date: 2009-02-01
• Target enrollment: 0
• Participant gender: Male

Summary

Wiskott - Aldrich syndrome (WAS) is a rare disorder curable only through allogeneic hematopoietic stem cell transplantation. A mismatched family member is an option when no human leukocyte antigen (HLA-immune system type) matched related or matched unrelated donor is available.

This study will evaluate a novel therapeutic strategy for patients with WAS who undergo haploidentical transplantation using a parental donor. To reduce the risk of transplant-related toxicities, participants will receive a reduced intensity chemotherapy and antibody regimen (conditioning treatment). Participants will then receive an infusion of donor stem cells depleted of certain white blood cells called T- and B-lymphocytes. The stem cell depletion processing will be done through the use of the investigational CliniMACS device. A certain number of T-lymphocytes will be added back to the processed stem cell graft prior to infusion into the recipient.

The primary objective of this study is to determine the safety of haploidentical transplantation in WAS patients using this specified conditioning regimen and engineered graft. Safety will be defined in terms of engraftment (meaning how well the graft grows and functions after infusion) and regimen-related toxicity within the first 100 days after transplant.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: St. Jude Children's Research Hospital
• Treatments: Fludarabine; Fludarabine phosphate; Melphalan; Thiotepa

Criteria

Inclusion Criteria:

• Genotypical diagnosis of Wiskott-Aldrich Syndrome.

• Less than 18 years of age at time of transplant.

Must meet two of the eight following clinical criteria:

• Eczema that is refractory to standard therapy.

• Thrombocytopenia as defined by a platelet count < 50,000/mm3.

• Significant risk for or presence of opportunistic infection.

• Autoimmune disease.

• Malignancy or pre-malignant condition.

• Family history as defined as a family member with WAS who died before 10 years of age.

• Does not have a suitable, available 6/6 HLA-matched sibling donor available for donation.

• Does not have a suitable, available 10/10 HLA-allele matched unrelated donor identified through the National Marrow Donor Program (NMDP).

Exclusion Criteria:

If any of the following clinical indicators are met within 45 days prior to transplant, the research participant will not be eligible for the study:

• Symptomatic cardiac disease or evidence of significant cardiac dysfunction by echocardiogram (shortening fraction < 30%).

• Creatinine clearance or Tc 99 less than or equal 40ml/min/1.73 m2.

• SGPT greater than or equal 500 U/L.

• Karnofsky or Lansky Performance Score of < 50.

• Pulmonary function tests: FVC < 50% of predicted value if age appropriate to perform the testing adequately or an O2 saturation less than or equal to 92% on room air at rest.