Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita (NCT01659606)
Radiation- and Alkylator-free Hematopoietic Cell Transplantation for Bone Marrow Failure Due to Dyskeratosis Congenita / Telomere Disease
🩺 Plain-English Summary (8th-Grade Level)
Dyskeratosis congenita is a disease that affects numerous parts of the body, most typically causing failure of the blood system. Lung disease, liver disease and cancer are other frequent causes of illness and death. Bone marrow transplantation (BMT) can cure the blood system but can make the lung and liver disease and risk of cancer worse, because of DNA damaging agents such as alkylators and radiation that are typically used in the procedure. Based on the biology of DC, we hypothesize that it may be possible to avoid these DNA damaging agents in patients with DC, and still have a successful BMT. In this protocol we will test whether a regimen that avoids DNA alkylators and radiation can permit successful BMT without compromising survival in patients with DC.
Who can join: Review the eligibility criteria below.
Location: University of Chicago — Chicago, Illinois
Age range: 30 Days to 65 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (13 Sites)
- University of Chicago — Chicago, Illinois
- Massachusetts General Hospital — Boston, Massachusetts
- Boston Children's Hospital (pediatric patients) — Boston, Massachusetts
- Dana-Farber Cancer Institute (adult patients) — Boston, Massachusetts
- Children's Mercy Hospital Kansas City — Kansas City, Missouri
- Hackensack University Medical Center — Hackensack, New Jersey
- Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania
- Baylor College of Medicine — Houston, Texas
- Fred Hutch/University of Washington/Seattle Children's Cancer Consortium — Seattle, Washington
- University of Wisconsin Hospital and Clinics — Madison, Wisconsin
- Oslo University Hospital — Oslo,
- Karolinska University Hospital — Stockholm,
💰 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 § 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.
More studies in Dyskeratosis Congenita
- Quercetin Dyskeratosis Congenita (DC)/Telomere Biology Disorders (TBD) — Cincinnati, Ohio
Explore by condition: All Dyskeratosis Congenita clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.