Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita
Public ClinicalTrials.gov record NCT01659606. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Radiation- and Alkylator-free Hematopoietic Cell Transplantation for Bone Marrow Failure Due to Dyskeratosis Congenita / Telomere Disease
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT01659606
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 40 participants
Conditions and interventions
Interventions
- alemtuzumab Biological
- Fludarabine Drug
- Cyclosporins Drug
- Mycophenolate mofetil Drug
- Tacrolimus Drug
Biological · Drug
Eligibility (public fields only)
- Age range
- 30 Days to 65 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jul 2012 (Actual)
- Primary completion
- Dec 2026 (Estimated)
- Completion
- Dec 2034 (Estimated)
- Last update posted
- Mar 27, 2026
2012 – 2034
United States locations
- U.S. sites
- 11
- U.S. states
- 9
- U.S. cities
- 9
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of Chicago | Chicago | Illinois | 60637 | — |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | — |
| Boston Children's Hospital (pediatric patients) | Boston | Massachusetts | 02115 | — |
| Dana-Farber Cancer Institute (adult patients) | Boston | Massachusetts | 02115 | — |
| Children's Mercy Hospital Kansas City | Kansas City | Missouri | 64108 | — |
| Hackensack University Medical Center | Hackensack | New Jersey | 07601 | — |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| Baylor College of Medicine | Houston | Texas | 77030 | — |
| Fred Hutch/University of Washington/Seattle Children's Cancer Consortium | Seattle | Washington | 98109 | — |
| University of Wisconsin Hospital and Clinics | Madison | Wisconsin | 53792 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 2 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT01659606, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Mar 27, 2026 · Synced Oct 17, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT01659606 live on ClinicalTrials.gov.