JSP191-Based Conditioning

A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplantation With JSP191-Based Conditioning in Participants With GATA2 Deficiency

What's the purpose of the trial?

Background: People with GATA2 deficiency have a mutation on the GATA2 gene. This gene affects immune function. People with this disease are prone to serious infections; in time, they may develop blood cancers. A hematopoietic stem cell (HSC) transplant can cure GATA2 deficiency, but using stem cells donated by other people can cause serious side effects. Objective: To test a new drug (JSP191) to see if it can make HSC transplants safer. Eligibility: People aged 6 to 70 years who have GATA2 deficiency. Design: Participants will be screened. They will have a physical exam, with blood and urine tests. They will have tests of their heart and lung function. They may have a bone marrow biopsy: Their hip will be numbed; a large needle will be inserted to draw out tissue from inside the pelvis. Participants will have a central venous catheter placed in a vein of the neck or chest. This will be used to draw blood and administer drugs. JSP191 will be given through the catheter about 11 days before the transplant. This is part of conditioning: preparing the body to receive the new stem cells. Conditioning also includes other medications and total body irradiation. Donor stem cells will be administered through the catheter. Participants will receive other approved drugs to help prevent side effects. Participants will stay in the hospital from the beginning of the conditioning until several weeks after the transplant. They will remain in the local area for 100 days after discharge; they will come to the clinic at least once a week during this time. Follow-up visits will continue for 3 years....
Trial status

Accepting patients

Phase
Phase 2
Enrollment
40
Last Updated
3 weeks ago
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Participating Centers

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Experimental Treatments

Learn more about the experimental treatments being evaluated in this clinical trial.

  • Allogeneic Stem Cell Transplant is a type of stem cell transplant that utilizes a donor's pre-collected stem cells to rescue the bone marrow from the toxic effects of very high dose chemotherapy.
  • Briquilimab is an anti-CD117 monoclonal antibody that is being studied for use in several different indications.
  • Cyclophosphamide is a chemotherapy agent used in the treatment of cancer.
  • Fludarabine is a chemotherapy medication that may be used in the treatment of many different cancers.
  • Mycophenolate Mofetil is an immunosuppressive medication used to help prevent rejection in patients who have had a transplant.
  • Tacrolimus is an immunosuppressive medication used to help prevent rejection in patients who have had a transplant.

Arms / Cohorts

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Not currently accepting

Arm A

Not currently accepting

Arm B

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