Stem Cell Transplant for Sickle Cell Disease
(HaploSCD Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment for sickle cell disease using a stem cell transplant called CD34 selected T-cell depleted allogeneic SCT. The goal is to determine if high-dose chemotherapy followed by a transplant of immune cells from a family member can safely control sickle cell disease long-term. The trial seeks participants with sickle cell disease who experience serious complications such as strokes, repeated chest problems, or frequent pain episodes. Participants must have a family member who can donate cells and does not have severe sickle cell disease. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to advancing treatment options for sickle cell disease.
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
Is there any evidence suggesting that this treatment is likely to be safe for humans?
Research has shown that using CD34 selected T-cell depleted stem cells can be promising for people with sickle cell disease. In past studies, about 5.4% of patients experienced graft failure, meaning the transplanted cells didn't grow. Graft rejection, where the body attacks the transplanted cells, occurred in about 7.5% of patients.
One study found that a year after treatment, 83% of patients were still alive, although two individuals died from infections. These findings suggest that while risks exist, many patients respond well to this treatment.
Other reports indicate that the safety of this type of stem cell transplant has improved over time. It is considered a potential cure for sickle cell disease, but discussing possible risks with a doctor is important.12345Why do researchers think this study treatment might be promising for sickle cell disease?
Unlike the standard treatments for sickle cell disease, such as hydroxyurea or blood transfusions, the stem cell transplant under study offers a potentially curative approach. This treatment utilizes CD34 selected T-cell depleted allogeneic stem cell transplantation, which involves replacing the patient’s defective blood-forming cells with healthy ones from a donor. Researchers are excited about this method because it specifically targets the root cause of the disease, offering the potential to eliminate sickle cell symptoms entirely, rather than just managing them. This approach is groundbreaking as it aims to provide long-term solutions rather than short-term relief.
What evidence suggests that this stem cell transplant is effective for sickle cell disease?
Research has shown that a specific type of stem cell transplant, which involves removing certain T-cells, can effectively treat severe sickle cell disease. In this trial, participants will receive the CD34-selected T-cell depleted allogeneic stem cell transplant. Studies have found that recipients of this transplant have a high survival rate of 90% to 100%. The risk of transplant failure is about 10%. The chance of developing acute graft-versus-host disease (GVHD), where donor cells attack the body, ranges from 0% to 10%. Chronic GVHD, a longer-lasting form, occurs in about 20% of cases. These findings suggest that this treatment could help control the disease over the long term.12367
Who Is on the Research Team?
Mitchell S Cairo, MD
Principal Investigator
New York Medical College
Are You a Good Fit for This Trial?
This trial is for high-risk sickle cell disease patients who've had multiple painful events, adequate organ function, and specific complications like stroke or acute chest syndrome. They need a partially matched family donor and can't be pregnant, breastfeeding, non-compliant with medical care, have significant liver fibrosis/cirrhosis or previous stem cell transplants.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Chemotherapy
Participants receive a high-dose chemotherapy regimen as part of the conditioning process
Stem Cell Transplantation
Infusion of CD34 selected T-cell depleted allogeneic stem cells from a haploidentical donor
Follow-up
Participants are monitored for safety, effectiveness, and long-term outcomes such as donor chimerism and organ function
What Are the Treatments Tested in This Trial?
Interventions
- CD34 selected T-cell depleted allogeneic SCT
Find a Clinic Near You
Who Is Running the Clinical Trial?
New York Medical College
Lead Sponsor
Miltenyi Biomedicine GmbH
Industry Sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Collaborator
Washington University School of Medicine
Collaborator
UCSF Benioff Children's Hospital Oakland
Collaborator
Medical College of Wisconsin
Collaborator
Tufts Medical Center
Collaborator
University of California, Los Angeles
Collaborator
University of California, San Francisco
Collaborator