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Stem Cell Transplant in Sickle Cell Disease and Thalassemia

NCT00408447 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 53

Last updated 2024-06-13

No results posted yet for this study

Summary

The primary purpose of this study is to see if giving lower doses of chemotherapy (moderately ablative) will result in successful bone marrow replacement without as severe side-effects but with permanent control of the disease. Patients will receive a chemotherapy regimen with busulfan, fludarabine, and alemtuzumab followed by an infusion of stem cells, either from a family-related or cord-blood matched donor.

Conditions

Interventions

DRUG

Busulfan

Busulfan 4 mg/kg/d x 4d

DRUG

Fludarabine

Fludarabine 30 mg/m2/d x 6d

DRUG

Alemtuzumab

Alemtuzumab 2mg/m2 x 1d, 6mg/m2 x 2 d, 20mg/m2 x 2d

PROCEDURE

Allogeneic stem cell transplant

Allogeneic stem cells will be given on day 0 (after chemotherapy conditioning)obtained either from a family donor (first degree relative) or sibling cord blood donor.

Sponsors & Collaborators

Principal Investigators

  • Monica Bhatia, MD · Columbia University

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
1 Month
Max Age
30 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2004-09-30
Primary Completion
2025-02-28
Completion
2025-02-28
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Drugs
Diseases
Companies

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 NCT00408447 on ClinicalTrials.gov