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Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich Syndrome
This study has been completed.
Study NCT00160355   Information provided by St. Jude Children's Research Hospital
First Received: September 8, 2005   Last Updated: February 12, 2009   History of Changes

September 8, 2005
February 12, 2009
May 2005
July 2008   (final data collection date for primary outcome measure)
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 [ Time Frame: March 2010 ] [ Designated as safety issue: Yes ]
  • To determine the safety and toxicity factors involved in giving a mismatched stem cell transplant which has been depleted of T-cells and B-cells to participants with
  • Wiskott-Aldrich syndrome.
Complete list of historical versions of study NCT00160355 on ClinicalTrials.gov Archive Site
 
To study the participant’s ability, following transplantation, to produce T and B-cells which function properly.
 
Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich Syndrome
Haploidentical Hematopoietic Stem Cell Transplantation for Pediatric Patients With Wiskott-Aldrich Syndrome: A Pilot Study

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.

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.
Phase I
Interventional
Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety Study
Wiskott-Aldrich Syndrome
  • Procedure: Hematopoietic stem cell transplantation
  • Device: Miltenyi CliniMACS selection system
  • Drug: Fludarabine, Melphalan, Thiotepa
 
 

*   Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
 
Completed
12
February 2009
July 2008   (final data collection date for primary outcome measure)

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.
Male
up to 18 Years
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT00160355
Kimberly Kasow, DO / Principal Investigator, St. Jude Children's Research Hospital
WASHAP
St. Jude Children's Research Hospital
 
Principal Investigator: Kimberly Kasow, DO St. Jude Children's Research Hospital
Principal Investigator: Kimberly Kasow, DO St. Jude Children's Research Hospital
St. Jude Children's Research Hospital
February 2009

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP