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Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation

NCT03513328 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2023-09-15

Study results available
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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.

Conditions

  • Bone Marrow Failure Syndrome
  • Thalassemia
  • Sickle Cell Disease
  • Diamond Blackfan Anemia
  • Acquired Neutropenia in Newborn
  • Acquired Anemia Hemolytic
  • Acquired Thrombocytopenia
  • Hemophagocytic Lymphohistiocytoses
  • Wiskott-Aldrich Syndrome
  • Chronic Granulomatous Disease
  • Common Variable Immunodeficiency
  • X-linked Lymphoproliferative Disease
  • Severe Combined Immunodeficiency
  • Hurler Syndrome
  • Mannosidosis
  • Adrenoleukodystrophy

Interventions

DRUG

Thiotepa--single daily dose

Conditioning regimen for hematopoietic stem-cell transplant. Single daily IV dose of Thiotepa at 5 mg/kg.

DRUG

Thiotepa--escalated dose

Twice daily IV dose of Thiotepa at 5 mg/kg, twelve hours apart, 10mg/kg total.

Sponsors & Collaborators

  • Live Like Bella Pediatric Cancer Research

    collaborator OTHER

  • University of Florida

    lead OTHER

Principal Investigators

  • Biljana Horn, MD · University of Florida

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
3 Months
Max Age
39 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-06-15
Primary Completion
2022-02-17
Completion
2023-02-19
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03513328 on ClinicalTrials.gov