Haploidentical Hematopoietic Stem Cell Transplantation Patients With

Status Completed

Clinical Trial 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.

Clinical Trial 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 Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT00160355
Study type	Interventional
Source	St. Jude Children's Research Hospital
Contact
Status	Completed
Phase	Phase 1
Start date	May 2005
Completion date	February 2009

View Details

See also
Status	Clinical Trial	Phase
Recruiting	`NCT01652092` - Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies	N/A
Completed	`NCT01953016` - Participation in a Research Registry for Immune Disorders
Active, not recruiting	`NCT02333760` - Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome	Phase 1/Phase 2
Terminated	`NCT01319851` - Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation	N/A
Recruiting	`NCT05687474` - Baby Detect : Genomic Newborn Screening
Recruiting	`NCT04371939` - Efficacy and Safety of Romiplostim Versus Eltrombopag in the Treatment of Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome	Phase 2
Completed	`NCT01347346` - Gene Therapy for WAS	Phase 1/Phase 2
Recruiting	`NCT01821781` - Immune Disorder HSCT Protocol	Phase 2
Completed	`NCT01347242` - Gene Therapy for Wiskott-Aldrich Syndrome (WAS)	Phase 1/Phase 2
Completed	`NCT01289847` - A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary Immunodeficiency	Phase 4
Terminated	`NCT00006054` - Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies	N/A
Enrolling by invitation	`NCT03198195` - Post-transplant Cyclophosphamide in Wiskott-Aldrich Syndrome	N/A
Recruiting	`NCT03019809` - A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS Patients	Phase 2
Terminated	`NCT00909363` - Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients	Phase 2
Enrolling by invitation	`NCT01852370` - Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases	Phase 1/Phase 2
Completed	`NCT03513328` - Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation	Phase 1/Phase 2
Active, not recruiting	`NCT01410825` - Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome	Phase 1/Phase 2
Active, not recruiting	`NCT00004341` - Study of Genetic and Molecular Defects in Primary Immunodeficiency Disorders	N/A
Completed	`NCT03399461` - Targeted Literature Review and Subject Interviews in Wiskott-Aldrich Syndrome (WAS)
Active, not recruiting	`NCT03837483` - A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome	Phase 3

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich Syndrome

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms