Induction of Stable Chimerism for Sickle Cell Anemia

July 28, 2016 updated by: National Heart, Lung, and Blood Institute (NHLBI)
To investigate a modified hematopoeitic cell transplantation (HCT) procedure for sickle cell disease that significantly reduces the toxicity of HCT, yet retains its therapeutic benefit.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

BACKGROUND:

Hematopoietic cell transplantation (HCT) has curative potential for individuals with sickle cell disease. While the results of conventional HCT have been good, this treatment carries risks of significant short- term and longterm toxicities. For this reason, HCT has been reserved for children who have experienced severe symptoms that predict a poor outcome. Of interest, some patients developed stable donor-host hematopoietic chimerism after conventional HCT. Due to a natural enrichment of donor erythrocytes in the blood, those who developed stable chimerism had a significant clinical benefit, even when there was a minority of donor cells. These observations have paralleled efforts to develop less-toxic, non-myeloablative preparative regiments for transplantation, proved first in a canine model of transplantation, and subsequently translated successfully in a clinical trial for older adults with hematological malignancies.

DESIGN NARRATIVE:

Multicenter open-label phase I-II study in 30 children with sickle cell disease that combines a non-myeloablative pre-transplant hematopoietic cell transplantation (HCT) therapy with modulated post-grafting immunosuppression to control host-versus-graft and graft-versus-host reactions. The approach relies on the ability to establish and maintain donor-host chimerism. The primary study endpoint is stable donor cell engraftment; secondary endpoints measure the impact of therapy on sickle cell-related symptoms and end-organ damage (disease-free survival, patient survival, graft-versus-host disease, complications etc). The trial will be conducted within the existing network of Comprehensive Sickle Cell Centers, and will be centrally coordinated by the Sickle Cell Coordinating Center.

Study Type

Interventional

Phase

  • Phase 2

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 100 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

No eligibility criteria

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Masking: None (Open Label)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

National Heart, Lung, and Blood Institute (NHLBI)

Investigators

  • Mark Walters, UCSF Benioff Children's Hospital Oakland

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

August 1, 2001

Primary Completion (Actual)

July 1, 2007

Study Completion (Actual)

July 1, 2007

Study Registration Dates

First Submitted

January 10, 2002

First Submitted That Met QC Criteria

January 10, 2002

First Posted (Estimate)

January 11, 2002

Study Record Updates

Last Update Posted (Estimate)

July 29, 2016

Last Update Submitted That Met QC Criteria

July 28, 2016

Last Verified

January 1, 2008

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 142
  • U01HL068091 (U.S. NIH Grant/Contract)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Anemia, Sickle Cell

Clinical Trials on Hematopoietic Stem Cell Transplantation

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Netherlands

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe