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Wiskott-Aldrich Syndrome clinical trials

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NCT ID: NCT02333760 Active, not recruiting - Clinical trials for Wiskott-Aldrich Syndrome

Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome

WASFUP
Start date: September 2014
Phase: Phase 1/Phase 2
Study type: Interventional

An open follow up study of patients enrolled in the Phase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome and treated with autologous CD34+ cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector.

NCT ID: NCT02064933 Completed - Clinical trials for Wiskott-Aldrich Syndrome

Patients Treated for Wiskott-Aldrich Syndrome (WAS) Since 1990

Start date: February 2, 2014
Phase:
Study type: Observational

Wiskott - Aldrich syndrome (WAS) is a rare serious medical condition that causes problems both with the immune system and with easy bruising and bleeding. The immune abnormalities cause patients with WAS to be very susceptible to infections. Depending on the specific type of primary immune deficiency diseases, there are effective treatments, including antibiotics, cellular therapy and gene therapy, but studies of large numbers of patients are needed to determine the full range of causes, natural history, or the best methods of treatment for long term success. This multicenter study combines retrospective, prospective and cross-sectional analyses of the transplant experiences for patients with WAS who have already received HCT since 1990, or who will undergo Hematopoietic cell transplant (HCT) during the study period. The retrospective and prospective portions of the study will address the impact of a number of pre and post-transplant factors on post-transplant disease correction and ultimate benefit from HCT and the cross-sectional portion of the study will assess the benefit of HCT 2 years post-HCT in consenting surviving patients.

NCT ID: NCT01953016 Completed - Clinical trials for Wiskott-Aldrich Syndrome

Participation in a Research Registry for Immune Disorders

Start date: September 30, 2013
Phase:
Study type: Observational

Background: - People with primary immune deficiency diseases (PIDD) have weak immune systems. This makes it hard for their bodies to fight infection. The Immune Deficiency Foundation has a network to collect data about people with PIDD. It is called the United States Immunodeficiency Network. It will help doctors and scientists better understand these disorders. The goal is to get medical data for everyone with these disorders in the U.S. and Canada. Data will be stored in a registry. Researchers can use it to study if these disorders are increasing. They can also learn how the disorders are diagnosed and treated. Objectives: - To collect data on people with primary immune deficiency disorders. Eligibility: - People who have a PIDD. Design: - Data can be added with no record of personal identity. - Data can be added with identity kept separate. This data will be linked to the registry by a code number. - Data for the registry includes: - Family history - Disease treatment - Disease characteristics - Medical history - Laboratory data

NCT ID: NCT01917708 Completed - Sickle Cell Disease Clinical Trials

Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

Start date: January 2014
Phase: Phase 1
Study type: Interventional

This is a single arm, phase I study to assess the tolerability of abatacept when combined with cyclosporine and mycophenolate mofetil as graft versus host disease prophylaxis in children undergoing unrelated hematopoietic stem cell transplant for serious non-malignant diseases as well as to assess the immunological effects of abatacept. Participants will be followed for 2 years.

NCT ID: NCT01852370 Enrolling by invitation - Clinical trials for Wiskott-Aldrich Syndrome

Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases

BOLT+BMT
Start date: June 20, 2013
Phase: Phase 1/Phase 2
Study type: Interventional

The purpose of this study is to determine whether bilateral orthotopic lung transplantation (BOLT) followed by cadaveric partially-matched hematopoietic stem cell transplantation (HSCT) is safe and effective for patients aged 5-45 years with primary immunodeficiency (PID) and end-stage lung disease.

NCT ID: NCT01821781 Recruiting - Clinical trials for Chronic Granulomatous Disease

Immune Disorder HSCT Protocol

Start date: March 2013
Phase: Phase 2
Study type: Interventional

This study hypothesizes that a reduced intensity immunosuppressive preparative regimen will establish engraftment of donor hematopoietic cells with acceptable early and delayed toxicity in patients with immune function disorders. A regimen that maximizes host immune suppression is expected to reduce graft rejection and optimize donor cell engraftment.

NCT ID: NCT01652092 Recruiting - Clinical trials for Chronic Granulomatous Disease

Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

Start date: September 4, 2012
Phase: N/A
Study type: Interventional

This is a standard of care treatment guideline for allogeneic hematopoetic stem cell transplant (HSCT) in patients with primary immune deficiencies.

NCT ID: NCT01529827 Completed - Clinical trials for Chronic Myelomonocytic Leukemia

Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies

Start date: February 28, 2012
Phase: Phase 2
Study type: Interventional

This phase II trial studies how well giving fludarabine phosphate, melphalan, and low-dose total-body irradiation (TBI) followed by donor peripheral blood stem cell transplant (PBSCT) works in treating patients with hematologic malignancies. Giving chemotherapy drugs such as fludarabine phosphate and melphalan, and low-dose TBI before a donor PBSCT helps stop the growth of cancer and abnormal cells and helps stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from the donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cell from a donor can make an immune response against the body's normal cells. Giving tacrolimus, mycophenolate mofetil (MMF), and methotrexate after transplant may stop this from happening

NCT ID: NCT01515462 Completed - Clinical trials for Wiskott-Aldrich Syndrome (WAS)

Gene Therapy for Wiskott-Aldrich Syndrome

TIGET-WAS
Start date: April 20, 2010
Phase: Phase 1/Phase 2
Study type: Interventional

This is phase I/II protocol to evaluate the safety and efficacy of WAS gene transfer into hematopoietic stem/progenitor cells for the treatment of Wiskott Aldrich Syndrome.

NCT ID: NCT01410825 Active, not recruiting - Clinical trials for Wiskott-Aldrich Syndrome

Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome

Start date: July 2011
Phase: Phase 1/Phase 2
Study type: Interventional

The Wiskott-Aldrich Syndrome (WAS) is an inherited disorder that results in defects of the blood and bone marrow. It affects boys because the genetic mistake is carried on the X chromosome. Normal people have blood cells called platelets that stop bleeding when blood vessels are damaged. Boys with WAS have low numbers of platelets that do not function correctly. Boys with WAS are thus at risk for severe life-threatening bleeding. A normal immune system is made of special blood cells called white blood cells, which protect against infection and also fight certain types of cancer. In WAS, these white blood cells don't work as well as they should, making these boys very susceptible to infections and to a form of blood cancer known as lymphoma. The abnormal white blood cells of patients with WAS also cause diseases such as eczema and arthritis. Although WAS can be mild, severe forms need treatment as early as possible to prevent life-threatening complications due to bleeding, infection and blood cancer. Over the past decade, investigators have developed new treatments based on the investigators knowledge of the defective gene causing WAS. The investigators can now use genes as a type of medicine that will correct the problem in the patient's own bone marrow. The investigators call this process gene transfer. The procedure is very similar to a normal bone marrow transplant, in that the old marrow is killed off using chemotherapy, but is different because the patient's own bone marrow is given back after it is treated by gene transfer. This approach can be used even if the patient does not have any matched donors available and will avoid problems such as GVHD and rejection. The investigators wish to test whether this approach is safe and whether gene transfer will lead to the development of a healthy immune and blood system.