Clinical Trial: Pilot Study of Reduced-Intensity Hematopoietic Stem Cell Transplant of DOCK8 Deficiency

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional




Official Title: Related and Unrelated Donor Hematopoietic Stem Cell Transplant of DOCK8 Deficiency

Brief Summary:

Background:

-DOCK8 deficiency is a genetic disorder that affects the immune system and can lead to severe recurrent infections and possible death from infections or certain types of cancers, including blood cancers. A stem cell transplant is a life-saving treatment for this condition. In this study we are evaluating the efficacy and safety of transplant from different donor sources for DOCK8 deficiency. The donors that we are using are matched siblings, matched unrelated donors, and half-matched donors, so called haploidentical related donors, such as as mothers or fathers or half-matched siblings.

Objectives:

-To determine whether transplant of bone marrow cells from different types of donors corrects DOCK8 deficiency.

Eligibility:

  • Donors: Healthy individuals between 4 and 60 years of age who are matched with a recipient.
  • Recipient: Individuals between 5 and 40 years of age who have DOCK8 deficiency, have suffered one or more life-threatening infections, or have had certain viral related cancers of cancer and have a stem cell donor.

Design:

  • All participants will be screened with a physical examination and medical history.
  • DONORS:

    • Donors will donate bone marrow cells or blood stem cells. If donating blood stem cells, donors will receive injections of filgrastim to release stem cells into the blood. After 5 days o