Tundra Space

Tundra Space

Clinical Research Directory

Browse clinical research sites, groups, and studies.

Back to Studies
ACTIVE NOT RECRUITING
NCT01659606
PHASE2

Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita

Sponsor: Boston Children's Hospital

View on ClinicalTrials.gov

Summary

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.

Official title: Radiation- and Alkylator-free Hematopoietic Cell Transplantation for Bone Marrow Failure Due to Dyskeratosis Congenita / Telomere Disease

Key Details

Gender

All

Age Range

30 Days - 65 Years

Study Type

INTERVENTIONAL

Enrollment

40

Start Date

2012-07

Completion Date

2034-12

Last Updated

2026-03-27

Healthy Volunteers

No

Interventions

BIOLOGICAL

alemtuzumab

Conditioning: alemtuzumab 0.2 mg/kg/dose IV/SC x 5 doses

DRUG

Fludarabine

fludarabine 30 mg/m2/dose IV x 6 doses

DRUG

Cyclosporins

DRUG

Mycophenolate mofetil

DRUG

Tacrolimus

Locations (13)

University of Chicago

Chicago, Illinois, United States

Massachusetts General Hospital

Boston, Massachusetts, United States

Boston Children's Hospital (pediatric patients)

Boston, Massachusetts, United States

Dana-Farber Cancer Institute (adult patients)

Boston, Massachusetts, United States

Children's Mercy Hospital Kansas City

Kansas City, Missouri, United States

Hackensack University Medical Center

Hackensack, New Jersey, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Baylor College of Medicine

Houston, Texas, United States

Fred Hutch/University of Washington/Seattle Children's Cancer Consortium

Seattle, Washington, United States

University of Wisconsin Hospital and Clinics

Madison, Wisconsin, United States

Oslo University Hospital

Oslo, Norway

Karolinska University Hospital

Stockholm, Sweden