๐Ÿ›๏ธ Verified NIH Record โ€ข Updated 2026-08-21

High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous Disease (NCT02629120)

๐Ÿ”ต Active, Not Recruiting Phase1 / Phase2 ๐Ÿ’Š Investigational Drug / Biologic Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)

๐Ÿฉบ Plain-English Summary (8th-Grade Level)

Chronic granulomatous disease (CGD) affects white blood cell function. Currently, the only curative treatment is bone marrow transplant to replace the abnormal stem cells with new ones (donor cells) capable of making a normal immune system. Transplant problems include graft versus host disease (GvHD) and graft rejection. With GvHD, donor cells attack the recipient s normal tissue. Researchers want to use preparation drugs and a high cell dose to increase graft success. They want to use 2 immunosuppressive drugs (cyclophosphamide and sirolimus) to lessen the risk of GvHD.

Who can join: Review the eligibility criteria below.

Location: National Institutes of Health Clinical Center โ€” Bethesda, Maryland

Age range: 4 Years to 65 Years

๐Ÿ“‹ "Do I Qualify?" โ€” 1-Minute Self Screener

Instant Checklist

Full eligibility criteria are available on the official NIH protocol. View the criteria โ†—

๐Ÿ“ All Participating Trial Locations (1 Sites)

  • National Institutes of Health Clinical Center โ€” Bethesda, Maryland

๐Ÿ’ฐ Cost, Insurance & Patient Rights

  • 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
  • Routine Patient Care: Under federal law (ACA ยง 2709) and many state statutes, routine care costs are covered during participation.
  • Voluntary Participation: You may withdraw at any time without affecting your standard medical care.

๐Ÿ’ก How to Participate

  1. Check the checklist above to see if you may qualify.
  2. Review the official NIH record below to find the coordinating site.
  3. Ask about the visit schedule, what is covered at no cost, and any travel support.
  4. Confirm with your own doctor before making a decision.
๐Ÿ›๏ธ View Official NIH Record โ†—