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Sponsored by: |
St. Jude Children's Research Hospital |
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Information provided by: | St. Jude Children's Research Hospital |
ClinicalTrials.gov Identifier: | NCT00160355 |
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.
Condition | Intervention | Phase |
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Wiskott-Aldrich Syndrome |
Procedure: Hematopoietic stem cell transplantation Device: Miltenyi CliniMACS selection system Drug: Fludarabine, Melphalan, Thiotepa |
Phase I |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety Study |
Official Title: | Haploidentical Hematopoietic Stem Cell Transplantation for Pediatric Patients With Wiskott-Aldrich Syndrome: A Pilot Study |
Estimated Enrollment: | 12 |
Study Start Date: | May 2005 |
Estimated Study Completion Date: | December 2010 |
Estimated Primary Completion Date: | July 2008 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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1 |
Procedure: Hematopoietic stem cell transplantation
To determine the safety in regards to engraftment and toxicity within 100 days of infusing a haploidentical T- and B-cell depleted hematopoietic stem cell graft into patients with Wiskott-Aldrich syndrome who have received a reduced intensity conditioning regimen.
Device: Miltenyi CliniMACS selection system
This system depletes the hematopoietic stem cell graft of T and B lymphocytes.
Drug: Fludarabine, Melphalan, Thiotepa
Participants will receive a reduced intensity conditioning regimen consisting of Fludarabine, Melphalan, Thiotepa, and OKT3 prior to receipt of the haploidentical stem cell graft. Rituximab will be given in an effort to prevent PTLPD. In addition to T-cell depletion of the donor product, cyclosporine will be given for GVHD prophylaxis.
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Secondary Objectives in this trial include the following:
Ages Eligible for Study: | up to 18 Years |
Genders Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Must meet two of the eight following clinical criteria:
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:
Contact: Kimberly Kasow, DO | 1-866-278-5833 | kimberly.kasow@stjude.org |
United States, Tennessee | |
St. Jude Children's Research Hospital | Recruiting |
Memphis, Tennessee, United States, 38105 | |
Contact: Kimberly Kasow, DO 866-278-5833 kimberly.kasow@stjude.org |
Principal Investigator: | Kimberly Kasow, DO | St. Jude Children's Research Hospital |
Principal Investigator: | Kimberly Kasow, DO | St. Jude Children's Research Hospital |
Responsible Party: | St. Jude Children's Research Hospital ( Kimberly Kasow, DO / Principal Investigator ) |
Study ID Numbers: | WASHAP |
Study First Received: | September 8, 2005 |
Last Updated: | December 29, 2008 |
ClinicalTrials.gov Identifier: | NCT00160355 |
Health Authority: | United States: Food and Drug Administration |
Wiskott-Aldrich Syndrome Immunodeficiency Haploidentical transplantation |
Purpura Melphalan Cyclosporine Rituximab Hematologic Diseases Blood Coagulation Disorders Blood Platelet Disorders Fludarabine monophosphate Hemostatic Disorders Cyclosporins Immunologic Deficiency Syndromes |
Purpura, Thrombocytopenic Muromonab-CD3 Thiotepa Thrombocytopathy Wiskott-Aldrich Syndrome Thrombocytopenia Hemorrhagic Disorders Genetic Diseases, Inborn Genetic Diseases, X-Linked Wiskott Aldrich syndrome Fludarabine |
Disease Molecular Mechanisms of Pharmacological Action Immune System Diseases Immunologic Factors Antineoplastic Agents Physiological Effects of Drugs Immunosuppressive Agents Pharmacologic Actions |
Pathologic Processes Blood Coagulation Disorders, Inherited Syndrome Therapeutic Uses Myeloablative Agonists Antineoplastic Agents, Alkylating Alkylating Agents |