CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 6 enrolled
Drug / intervention
Thiotepa--single daily dose +1 moredrug
Likely dose
Thiotepa--single daily dose 5 mg/kgfrom record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT03513328
NCT03513328Phase 2Completed
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PEDS024, Phase I/II Feasibility Study of Busulfan Fludarabine and Thiotepa Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation (HSCT) for Children With Non-Malignant Disorders

University of Florida·interventional·Posted May 1, 2018·Updated Sep 15, 2023

In Brief

A Phase 2 clinical trial evaluating Thiotepa--single daily dose and Thiotepa--escalated dose for Bone Marrow Failure Syndrome and 15 related conditions. Completed, enrolled 6 participants across 1 site.

Detailed 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.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsBone 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
CountriesUnited States
CollaboratorsLive Like Bella Pediatric Cancer Research

Timeline

Phase 2CompletedFinished
20192020202120222023202420252026
First PostedMay 1, 2018
Enrollment StartJun 15, 2018
Primary CompletionFeb 17, 2022
Study CompletionFeb 19, 2023
TodayJul 2, 2026
Enrollment to primary: 3.7 yearsPosted 8.2 years ago

Interventions

Thiotepa--single daily dosedrug

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

Thiotepa--escalated dosedrug

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