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Haplo T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease

NCT01461837 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 21

Last updated 2025-08-08

No results posted yet for this study

Summary

This study is being done to determine the safety and outcome (long-term control) of a high-dose chemotherapy regimen followed by an infusion of CD34 selected (immune cells) stem cells from a partially matched adult family member donor, called haploidentical stem cell transplantation, in high-risk sickle cell disease patients.

Funding Source - FDA OOPD

Conditions

Interventions

DRUG

CD34 selected T-cell depleted allogeneic SCT

Hydroxyurea (60 mg/kg/day) and azathioprine (3 mg/kg/day) day -59 to day -11; fludarabine (30 mg/m2) Days -17, -16, -15, -14, -13; busulfan (3.2 mg/kg/day) Days -12, -11, -10, -9; thiotepa (10 mg/kg IV) day -8; cyclophosphamide (50 mg/kg) Days -7, -6, -5, -4; TLI on day -3; rabbit ATG (2.0 mg/kg/day) day -5,-4,-3, and -2; Stem Cell infusion day 0

Sponsors & Collaborators

  • UCSF Benioff Children's Hospital Oakland

    collaborator OTHER

  • Medical College of Wisconsin

    collaborator OTHER

  • Washington University School of Medicine

    collaborator OTHER

  • Tufts Medical Center

    collaborator OTHER

  • University of California, San Francisco

    collaborator OTHER

  • University of California, Los Angeles

    collaborator OTHER

  • Miltenyi Biomedicine GmbH

    collaborator INDUSTRY

  • Ann & Robert H Lurie Children's Hospital of Chicago

    collaborator OTHER

  • New York Medical College

    lead OTHER

Principal Investigators

  • Mitchell S Cairo, MD · New York Medical College

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
20 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-01-31
Primary Completion
2025-12-31
Completion
2026-12-31

Countries

  • United States

Study Locations

More Related Trials

Entities

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 NCT01461837 on ClinicalTrials.gov