Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell

Status Completed

Clinical Trial Summary

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 non-malignant disease.

Clinical Trial Description

Hematopoietic stem cell transplantation is the only curative choice for a number of inherited bone marrow failure syndromes, hemoglobinopathies, metabolic disorders and primary immune deficiencies. While survival of these patients is typically better than survival of patients with malignancies, toxicities of conditioning regimens and failure of engraftment remain challenges. Most children with non-malignant disorders present with normocellular or even hypercellular bone marrow, posing a barrier to engraftment and requiring intensive conditioning. Commonly used backbone of busulfan and fludarabine, although well tolerated, results in variable engraftment, in particular with mismatched unrelated donors and cord blood recipients. 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) in order to determine the minimum effective dose required for reliable engraftment. Subjects are stratified in groups A and B based the risk of graft failure. ;

Study Design

Related Conditions & MeSH terms

Acquired Anemia Hemolytic
Acquired Neutropenia in Newborn
Acquired Thrombocytopenia
Adrenoleukodystrophy
Anemia
Anemia, Diamond-Blackfan
Anemia, Sickle Cell
Bone Marrow Failure Disorders
Bone Marrow Failure Syndrome
Chronic Granulomatous Disease
Common Variable Immunodeficiency
Diamond Blackfan Anemia
Granulomatous Disease, Chronic
Hemophagocytic Lymphohistiocytoses
Hurler Syndrome
Immunologic Deficiency Syndromes
Lymphohistiocytosis, Hemophagocytic
Lymphoproliferative Disorders
Mannosidosis
Mucopolysaccharidosis I
Neutropenia
Pancytopenia
Severe Combined Immunodeficiency
Sickle Cell Disease
Syndrome
Thalassemia
Thrombocytopenia
Wiskott-Aldrich Syndrome
X-linked Lymphoproliferative Disease

Clinical Trial Details

NCT number	NCT03513328
Study type	Interventional
Source	University of Florida
Contact
Status	Completed
Phase	Phase 1/Phase 2
Start date	June 15, 2018
Completion date	February 19, 2023

View Details

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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.

Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms