MedTrace Logo

Evaluating the Safety and Effectiveness of Bone Marrow Transplants in Children With Sickle Cell Disease (BMT CTN 0601)

NCT00745420 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 30

Last updated 2022-12-09

Study results available
· View outcomes & findings →

Summary

Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited blood disease that can cause organ damage, stroke, and intense pain episodes. A blood stem cell transplant is a treatment option for someone with a severe form of the disease. Prior to undergoing a transplant, people typically receive a conditioning regimen of high doses of chemotherapy and other medications to prepare the body to accept the transplant. A conditioning regimen that uses lower doses of chemotherapy and medications may be safer for transplant recipients. This study will evaluate the safety and effectiveness of blood stem cell transplants, using bone marrow from unrelated donors, in children with severe SCD who receive a reduced intensity conditioning regimen prior to the transplant.

Conditions

Interventions

BIOLOGICAL

Hematopoietic Stem Cell Transplantation

The stem cell transplant preparative regimen is listed below. Day 0 is the day of the transplant. The - sign is the number of days before and the + sign is the number of days after the transplant. * Alemtuzumab: Children weighing 10 kg or more will receive 10 mg, 15 mg, and 20 mg intravenously (IV) on Days -21, -20, and -19, respectively * Fludarabine: 30 mg/m\^2/day IV on Days -8 through -4 * Melphalan: 140 mg/m\^2 IV on Day -3 * Rest on Days -2 and -1 * Transplant occurs on Day 0 * GVHD prophylaxis: Tacrolimus or cyclosporine beginning Day -3, methotrexate (7.5 mg/m2/day) Day 1, 3 and 6 and methylprednisolone/prednisone on Day +7 to +28 followed by a taper if there is no GVHD

Sponsors & Collaborators

  • National Heart, Lung, and Blood Institute (NHLBI)

    collaborator NIH

  • Blood and Marrow Transplant Clinical Trials Network

    collaborator NETWORK

  • National Cancer Institute (NCI)

    collaborator NIH

  • National Marrow Donor Program

    collaborator OTHER

  • Sickle Cell Disease Clinical Research Network

    collaborator UNKNOWN

  • Medical College of Wisconsin

    lead OTHER

Principal Investigators

  • Mary Horowitz, MD · Center for International Blood and Marrow Transplant Research

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Years
Max Age
19 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2008-08-31
Primary Completion
2015-07-31
Completion
2016-09-30

Countries

  • United States

Study Locations

More Related Trials

Entities

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 NCT00745420 on ClinicalTrials.gov