Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich Syndrome

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Brief Title

Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich Syndrome

Official Title

Haploidentical Hematopoietic Stem Cell Transplantation for Pediatric Patients With Wiskott-Aldrich Syndrome: A Pilot Study

Brief 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

Detailed Description

      Secondary Objectives in this trial include the following:

        -  To estimate the survival of study recipients at one year after infusion of the T- and
           B-lymphocyte depleted stem cell graft.

        -  To assess if the study treatment enables the recipient to generate normal donor-derived
           B-cell numbers and endogenous IgM, IgG, and IgA production, resulting in a
           reduction/elimination of the need for intravenous immunoglobulin infusions.

        -  To determine if the study treatment results in the ability of the research participant
           to generate normal donor-derived T cell response and natural killer (NK) cell numbers
           and function.

        -  To describe the incidence of Epstein-Barr virus-lymphoproliferative disease (EBV-LPD) in
           these transplant recipients.

Study Phase

Phase 1

Study Type


Primary Outcome

To determine safety in regards to engraftment and toxicity within 100 days post-haploidentical T- and B-cell depleted hematopoietic stem cell transplantation for patients with Wiskott-Aldrich syndrome who received a reduced intensity conditioning


Wiskott-Aldrich Syndrome


Hematopoietic stem cell transplantation

Study Arms / Comparison Groups



* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status


Estimated Enrollment


Start Date

May 2005

Completion Date

February 2009

Primary Completion Date

July 2008

Eligibility 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

          -  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




N/A - 18 Years

Accepts Healthy Volunteers



Kimberly Kasow, DO, , 

Location Countries

United States

Location Countries

United States

Administrative Informations



Organization ID


Responsible Party


Study Sponsor

St. Jude Children's Research Hospital

Study Sponsor

Kimberly Kasow, DO, Principal Investigator, St. Jude Children's Research Hospital

Verification Date

February 2009