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Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia

NCT01005576 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 21

Last updated 2017-12-13

Study results available
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Summary

This study is being done to determine if blood cell transplants, with either bone marrow or cord blood from unrelated donors, are effective in children with severe thalassemia and if this treatment approach has acceptable risks and side effects.

This study includes a preparative regimen with Hydroxyurea, Alemtuzumab, Fludarabine, Thiotepa and Melphalan that provides intense host immunosuppression without myeloablation. The primary hypothesis is that this regimen will promote stable engraftment of unrelated donor hematopoietic cells, support normal erythropoiesis, and result in an event free survival of \> 75% of children with thalassemia major.

Conditions

  • Severe Thalassemia

Interventions

DRUG

Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan

Days -50 to -21: Hydroxyurea 30mg/kg po Day -22: Alemtuzumab 3mg IV Day -21: Alemtuzumab 10mg IV Day -20: Alemtuzumab 15mg IV Day -19: Alemtuzumab 20mg IV Day -8: Fludarabine 30mg/m2 IV Day -7: Fludarabine 30mg/m2 IV Day -6: Fludarabine 30mg/m2 IV Day -5: Fludarabine 30mg/m2 IV Day -4: Fludarabine 30mg/m2 IV Day -4: Thiotepa 8mg/kg IV Day -3: Melphalan 140mg/m2 IV Day 0: Stem cell infusion

Sponsors & Collaborators

  • Carelon Research

    collaborator OTHER

  • Pediatric Blood and Marrow Transplant Consortium

    collaborator OTHER

  • Washington University School of Medicine

    lead OTHER

Principal Investigators

  • Shalini Shenoy, MD · Washington University School of Medicine

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Year
Max Age
16 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2010-01-31
Primary Completion
2014-04-30
Completion
2014-07-31

Countries

  • United States

Study Locations

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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 NCT01005576 on ClinicalTrials.gov