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

PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

40

Participants

Timeline

Start Date

July 31, 2012

Primary Completion Date

July 31, 2025

Study Completion Date

December 31, 2034

Conditions
Dyskeratosis CongenitaHoyeraal Hreidarsson SyndromeRevesz SyndromeAplastic Anemia
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

Trial Locations (16)

19104

Children's Hospital of Philadelphia, Philadelphia

27705

Duke University Medical Center, Pediatric BMT, Durham

45229

Cincinnati Children's Hospital Medical Center, Cincinnati

53792

University of Wisconsin Hospital and Clinics, Madison

55905

Mayo Clinic, Rochester

60637

University of Chicago, Chicago

64108

Children's Mercy Hospital Kansas City, Kansas City

77030

Baylor College of Medicine, Houston

90027

Children's Hospital Los Angeles, Los Angeles

98109

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

02114

Massachusetts General Hospital, Boston

02115

Boston Children's Hospital (pediatric patients), Boston

Dana-Farber Cancer Institute (adult patients), Boston

07601

Hackensack University Medical Center, Hackensack

Unknown

Oslo University Hospital, Oslo

Karolinska University Hospital, Stockholm

Sponsors
All Listed Sponsors
collaborator

Dana-Farber Cancer Institute

OTHER

collaborator

Children's Hospital Medical Center, Cincinnati

OTHER

collaborator

Children's Hospital Los Angeles

OTHER

collaborator

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

UNKNOWN

collaborator

Baylor College of Medicine

OTHER

collaborator

Children's Hospital of Philadelphia

OTHER

collaborator

University of Wisconsin, Madison

OTHER

collaborator

Karolinska University Hospital

OTHER

collaborator

Hackensack Meridian Health

OTHER

collaborator

Duke University

OTHER

collaborator

Oslo University Hospital

OTHER

collaborator

Children's Mercy Hospital Kansas City

OTHER

collaborator

Mayo Clinic

OTHER

collaborator

University of Chicago

OTHER

collaborator

Massachusetts General Hospital

OTHER

lead

Boston Children's Hospital

OTHER

NCT01659606 - Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita | Biotech Hunter | Biotech Hunter