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A Reduced Toxicity Allogeneic Unrelated Donor Stem Cell Transplantation (SCT) for Severe Sickle Cell Disease

NCT01279616 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 8

Last updated 2019-04-03

Study results available
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Summary

Majority of patients who are eligible for allogeneic HSCT for cure of severe sickle cell disease lack a matched family donor. This study aims for cure of sickle cell disease by performing unrelated donor (outside family) allogeneic HSCT. Donors or unrelated cord blood units will be selected from the NMDP database. It is designed to estimate the safety of a novel reduced toxicity, yet an immunosuppressive and myeloablative preparative regimen. This is meant for patients \<21 years old who have severe complications from sickle cell and do not have matched sibling donors in the family to undergo stem cell transplant. Patients will undergo transplant using unrelated donor stem cells after receiving the protocol therapy. They will be followed for 1 year to monitor for engraftment of donor cells and complications like graft versus host disease (GVHD), infections and death.

Conditions

Interventions

DRUG

Fludarabine monophosphate

180 mg/m2 over 6 days.

DRUG

Rituximab

375 mg/m2 on day -13 and day -3

DRUG

Busulfan

AUC 1000-1200 microM.mt

DRUG

ATG

2.5 mg/kg for 3 days

DRUG

Cyclophosphamide

50 mg/kg on day +3

DRUG

Mycophenolate mofetil

15 mg/kg q 8 hours

DRUG

Tacrolimus

0.03 mg/kg /d

Sponsors & Collaborators

  • Nationwide Children's Hospital

    lead OTHER

Principal Investigators

  • Sandeep Soni, MD · Nationwide Children's Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
21 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2010-09-30
Primary Completion
2015-01-31
Completion
2015-01-31

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs
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 NCT01279616 on ClinicalTrials.gov