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Found 8 clinical trials
Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome

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

  • 25 views
  • 24 Jan, 2021
  • 2 locations
Efficacy and Safety of Romiplostim Versus Eltrombopag in the Treatment of Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome

thrombocytopenia in patients with Wiskott-Aldrich syndrome

  • 0 views
  • 24 Jan, 2021
  • 1 location
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

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

griscelli syndrome
bone marrow transplant
chediak-higashi syndrome
busulfan
bone marrow procedure
  • 265 views
  • 27 Jan, 2021
  • 1 location
Immune Disorder HSCT Protocol

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.

preparative regimen
hsct
immune disorder
immunosuppression
treatment regimen
  • 123 views
  • 24 Jan, 2021
  • 2 locations
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation

In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum effective dose required for reliable engraftment for subjects undergoing hematopoietic stem cell transplantation for …

malignant disease
follicle-stimulating hormone
thrombocytopenia
busulfan
thiotepa
  • 176 views
  • 24 Jan, 2021
  • 1 location
Fludarabine Phosphate Cyclophosphamide Total Body Irradiation and Donor Stem Cell Transplant in Treating Patients With Blood Cancer

This phase II trial studies how well fludarabine phosphate, cyclophosphamide, total body irradiation, and donor stem cell transplant work in treating patients with blood cancer. Drugs used in chemotherapy, such as fludarabine phosphate and cyclophosphamide, work in different ways to stop the growth of cancer cells, either by killing the …

autograft
secondary myelodysplastic syndrome
cytogenetic abnormalities
b cell lymphoma
calcineurin inhibitor
  • 134 views
  • 24 Jan, 2021
  • 1 location
Participation in a Research Registry for Immune Disorders

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 …

primary immunodeficiency
undernutrition
  • 113 views
  • 28 Apr, 2021
  • 1 location
Studies of Skin Microbes in Healthy People and in People With Skin Conditions

syndrome), WAS (Wiskott-Aldrich syndrome), or DOCK8 immunodeficiency because they frequently have skin problems similar to AD. Eligible participants undergo the following tests and procedures:

immune disorder
rash
eczema
blood test
skin disorder
  • 208 views
  • 01 May, 2021
  • 1 location