Clinical Trials Logo

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

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

See also
  Status Clinical Trial Phase
Completed NCT02227472 - Working Memory and School Readiness in Preschool-Aged Children With Sickle Cell Disease
Recruiting NCT06301893 - Uganda Sickle Surveillance Study (US-3)
Recruiting NCT04398628 - ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
Completed NCT02522104 - Evaluation of the Impact of Renal Function on the Pharmacokinetics of SIKLOS ® (DARH) Phase 4
Recruiting NCT04688411 - An mHealth Strategy to Improve Medication Adherence in Adolescents With Sickle Cell Disease N/A
Terminated NCT03615924 - Effect of Ticagrelor vs. Placebo in the Reduction of Vaso-occlusive Crises in Pediatric Patients With Sickle Cell Disease Phase 3
Not yet recruiting NCT06300723 - Clinical Study of BRL-101 in Severe SCD N/A
Recruiting NCT03937817 - Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants
Completed NCT04134299 - To Assess Safety, Tolerability and Physiological Effects on Structure and Function of AXA4010 in Subjects With Sickle Cell Disease N/A
Completed NCT04917783 - Health Literacy - Neurocognitive Screening in Pediatric SCD N/A
Completed NCT02580565 - Prevalence of Problematic Use of Equimolar Mixture of Oxygen and Nitrous Oxide and Analgesics in the Sickle-cell Disease
Recruiting NCT04754711 - Interest of Nutritional Care of Children With Sickle Cell Disease on Bone Mineral Density and Body Composition N/A
Completed NCT04388241 - Preliminary Feasibility and Efficacy of Behavioral Intervention to Reduce Pain-Related Disability in Pediatric SCD N/A
Recruiting NCT05431088 - A Phase 2/3 Study in Adult and Pediatric Participants With SCD Phase 2/Phase 3
Completed NCT01158794 - Genes Influencing Iron Overload State
Recruiting NCT03027258 - Point-of-Delivery Prenatal Test Results Through mHealth to Improve Birth Outcome N/A
Withdrawn NCT02960503 - Macrolide Therapy to Improve Forced Expiratory Volume in 1 Second in Adults With Sickle Cell Disease Phase 1/Phase 2
Completed NCT02565082 - Evaluation of the Hemostatic Potential in Sickle Cell Disease Patients N/A
Withdrawn NCT02630394 - A Pilot Study of Azithromycin Prophylaxis for Acute Chest Syndrome in Sickle Cell Disease Phase 1
Completed NCT02567695 - A Single-Dose Relative Bioavailability Study Of GBT440 300 mg Capsules in Healthy Subjects Phase 1