Haploidentical HCT for Primary Immunodeficiency
What You Need to Know Before You Apply
What is the purpose of this trial?
This research is being done to learn if a new type of haploidentical transplantation using TCR alpha beta and CD19 depleted stem cell graft from the donor is safe and effective to treat the patient's underlying condition. This study will use stem cells obtained via peripheral blood or bone marrow from parent or other half-matched family member donor. These will be processed through a special device called CliniMACS, which is considered investigational.
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 haploidentical hematopoietic cell transplantation generally safe for humans?
Haploidentical hematopoietic cell transplantation (HaploHCT) has been associated with challenges like graft rejection and graft-versus-host disease (GvHD, where the donor cells attack the recipient's body), but recent advances have improved safety and outcomes. Techniques like T-cell depletion and post-transplant cyclophosphamide have been developed to reduce these risks, making the procedure safer over time.12345
How is Haploidentical Hematopoietic Cell Transplantation different from other treatments for primary immunodeficiency?
Haploidentical Hematopoietic Cell Transplantation is unique because it allows for the use of stem cells from a partially matched family donor, making it more accessible for patients who do not have a fully matched donor. This treatment has become the preferred option for many severe primary immunodeficiencies due to the availability of haploidentical donors and its success in reconstituting the immune system.23678
What data supports the effectiveness of the treatment Haploidentical Hematopoietic Cell Transplantation for Primary Immunodeficiency?
Who Is on the Research Team?
Deepak Chellapandian, MD
Principal Investigator
Johns Hopkins All Children's Hospital
Are You a Good Fit for This Trial?
This trial is for children with primary immunodeficiency or inherited metabolic disorders. Eligible participants include those with various immune system issues, autoimmune diseases, and certain metabolic conditions that could benefit from a stem cell transplant. Pregnant individuals, patients with HIV or uncontrolled infections, prior organ transplants, or severe graft-versus-host disease are excluded.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Transplantation
Participants undergo TCR alpha beta and CD19 depleted stem cell transplantation using CliniMACS
Engraftment Monitoring
Monitoring of donor engraftment and chimerism, including platelet and neutrophil engraftment
Follow-up
Participants are monitored for safety, effectiveness, and complications such as GvHD and infections
What Are the Treatments Tested in This Trial?
Interventions
- Haploidentical Hematopoietic Cell Transplantation
Haploidentical Hematopoietic Cell Transplantation is already approved in United States, European Union for the following indications:
Find a Clinic Near You
Who Is Running the Clinical Trial?
Johns Hopkins All Children's Hospital
Lead Sponsor