MedTrace Logo

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

NCT01659606 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 40

Last updated 2026-03-27

No results posted yet for this study

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.

Conditions

  • Dyskeratosis Congenita
  • Hoyeraal Hreidarsson Syndrome
  • Revesz Syndrome
  • Aplastic 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

Sponsors & Collaborators

  • Dana-Farber Cancer Institute

    collaborator OTHER

  • Children's Hospital Medical Center, Cincinnati

    collaborator OTHER

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

    collaborator UNKNOWN

  • Baylor College of Medicine

    collaborator OTHER

  • Children's Hospital of Philadelphia

    collaborator OTHER

  • University of Wisconsin, Madison

    collaborator OTHER

  • Karolinska University Hospital

    collaborator OTHER

  • Hackensack Meridian Health

    collaborator OTHER

  • Oslo University Hospital

    collaborator OTHER

  • Children's Mercy Hospital Kansas City

    collaborator OTHER

  • University of Chicago

    collaborator OTHER

  • Massachusetts General Hospital

    collaborator OTHER

  • Boston Children's Hospital

    lead OTHER

Principal Investigators

  • Suneet Agarwal, MD, PHD · Boston Children's Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
30 Days
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-07-31
Primary Completion
2026-12-31
Completion
2034-12-31

Countries

  • United States
  • Norway
  • Sweden

Study Locations

More Related Trials

Entities

Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01659606 on ClinicalTrials.gov