Active not recruitingPHASE2INTERVENTIONAL

Early Human Leukocyte Antigen (HLA) Matched Sibling Hematopoietic Stem Cell Transplantation

This research study is looking for children aged 2 to 12 with sickle cell disease who have a healthy brother or sister who can donate bone marrow. The study aims to see how safe and effective a specific type of bone marrow transplant is for these children, including those with different levels of sickle cell disease. They will receive special medicines before the transplant to prepare their body. After the transplant, children will stay in hospital for a few weeks and then have close check-ups for about a year. The study hopes to understand the long-term effects of this treatment, such as how it affects their brain, kidneys, and overall well-being. The goal is to improve future care for children with sickle cell disease.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Emory University

Enrolment target

Start

22 Mar 2019

Estimated completion

01 Nov 2026

Sickle Cell Disease

What is this study about?

This study is looking at a type of bone marrow transplant for young children with sickle cell disease. Bone marrow transplant involves replacing unhealthy blood-forming cells in a patient with healthy cells from a donor. In this study, the donor must be a healthy brother or sister who is a very good match for the patient.

Sickle cell disease can cause a lot of health problems, even for children who seem relatively well. This study is exploring if getting a bone marrow transplant at a younger age could prevent some of these serious problems as they get older. The doctors want to find out if this specific transplant method is safe and works well for children with different levels of sickle cell disease, including those who aren't severely affected yet.

The researchers will also be carefully tracking children for a long time after the transplant. They want to understand how this treatment affects important things like brain health, kidney function, and overall quality of life in the years following the transplant. This information will help doctors know if this treatment can lead to better health outcomes for children with sickle cell disease in the long run.

Key takeaways

This study is for children aged 2-12 with sickle cell disease.
A healthy sibling (under 13) must be able to donate bone marrow.
It tests a specific type of bone marrow transplant.
The transplant involves special medicines and a hospital stay.
Long-term health and quality of life are being closely monitored.
The aim is to improve future treatment for sickle cell disease.

Who may be eligible?

This study is looking for children who are between 2 and 12 years old and have sickle cell disease. They also need to have a healthy brother or sister who is under 13 years old and a very close match for a bone marrow donation.

Your child's sickle cell disease needs to be causing some health problems. This could include things like having had a stroke, changes seen on brain scans, needing frequent hospital visits for pain, or having recurring chest problems. The study is open to children with both more severe and less severe forms of sickle cell disease.

Before taking part, your child and their potential sibling donor will have health checks to make sure they are suitable for the transplant.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Is your child between 2 and 12 years old?
Does your child have sickle cell disease?
Does your child have a healthy brother or sister who is under 13 and a very good bone marrow match?
Has your child had any health issues related to their sickle cell disease (e.g., pain, stroke, chest problems)?
Is your child healthy enough for a transplant, based on initial checks?

Answer every question to see your result.

What does participation involve?

If your child qualifies for this study, they will have check-ups over 2 to 3 months to make sure they are healthy enough for the transplant. Once cleared, your child will go into the hospital. For about three weeks before the transplant, they will receive special medicines (including chemotherapy) to prepare their body to accept the donor cells.

After these preparations, your child will receive the bone marrow transplant. They will then stay in the hospital for another 4 to 6 weeks for close monitoring and recovery. Once they leave the hospital, they will have frequent appointments at the clinic for 6 to 12 months. After that, they will continue to have yearly check-ups for many years. The study will collect information about your child's health for two years after the transplant.

Potential risks and benefits

Potential benefits could include stopping the progression of sickle cell disease symptoms and improving your child's long-term health and quality of life. However, bone marrow transplants carry risks, such as side effects from the medicines, infections, and the possibility of the body rejecting the new cells. There's also a chance the transplant may not work. You have the right to withdraw your child from the study at any time without affecting their regular medical care.

Locations (15)

Children's Hospital of Alabama
Verified postcode
Birmingham, United States
Phoenix Children's
Verified postcode
Phoenix, United States
Yale University, Yale Cancer Center
Verified postcode
New Haven, United States
Children's National Hospital
Verified postcode
Washington D.C., United States
Children's Healthcare of Altanta
Verified postcode
Atlanta, United States
University of Chicago
Verified postcode
Chicago, United States
Riley Children's Health/Indiana University
Verified postcode
Indianapolis, United States
Dana-Farber Cancer Institute/Boston Children's Hospital
Verified postcode
Boston, United States
Washington University School of Medicine
Verified postcode
St Louis, United States
Hackensack University Medical Center
Verified postcode
Hackensack, United States
University of North Carolina Medical Center
Verified postcode
Chapel Hill, United States
Atrium Health Levine Cancer Institute
Verified postcode
Charlotte, United States

Common questions

What is sickle cell disease?

Sickle cell disease is an inherited blood condition where red blood cells are shaped like a 'C' or sickle. These cells can block blood flow, causing pain and other serious problems.

What is a bone marrow transplant?

A bone marrow transplant replaces unhealthy blood-forming cells in your child's body with healthy cells from a donor. In this study, the donor is a healthy brother or sister.

What are 'conditioning medicines'?

These are special medicines, including chemotherapy, given before the transplant to prepare your child's body to accept the new healthy cells and prevent them from rejecting the donor's cells.

How long will my child be in the hospital?

Your child will be in the hospital for about three weeks before the transplant and then for another 4 to 6 weeks after the transplant.

Will my child still need check-ups after the study ends?

Yes, even after the study collects data for two years, your child will continue to have yearly check-ups for many years by doctors who understand sickle cell and transplants.