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NCT01659606 · ClinicalTrials.gov registry record · Phase 2
Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita
A Phase 2 study of Aplastic Anemia and Dyskeratosis Congenita, sponsored by Boston Children's Hospital.
- Active
- Registry status
- Phase 2
- Development phase
- 40
- Enrollment target
- 14
- Study locations
NCT01659606 is a Phase 2 study of Aplastic Anemia and Dyskeratosis Congenita that is active but no longer recruiting, run by Boston Children's Hospital. The registered enrollment target is 40 participants, roughly in line with the 41-participant average among 23 other Aplastic Anemia trials with a reported enrollment target. The trial reports 14 study locations across 11 states.
The verdict
NCT01659606, a Phase 2 study of Aplastic Anemia and Dyskeratosis Congenita, is active but no longer recruiting, sponsored by Boston Children's Hospital.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 2
- Development phase
- 40 participants
- Enrollment target
- 14
- Study locations
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 Studied
Interventions
- DRUG Fludarabine
- DRUG Tacrolimus
- DRUG Mycophenolate mofetil
- BIOLOGICAL alemtuzumab
- DRUG Cyclosporins
Study Locations (14)
Massachusetts
- Massachusetts General Hospital - Boston
- Boston Children's Hospital (pediatric patients) - Boston
- Dana-Farber Cancer Institute (adult patients) - Boston
Other
- Oslo University Hospital - Oslo
- Karolinska University Hospital - Stockholm
California
- Children's Hospital Los Angeles - Los Angeles
Illinois
- University of Chicago - Chicago
Missouri
- Children's Mercy Hospital Kansas City - Kansas City
New Jersey
- Hackensack University Medical Center - Hackensack
Ohio
- Cincinnati Children's Hospital Medical Center - Cincinnati
Pennsylvania
- Children's Hospital of Philadelphia - Philadelphia
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 40 participants |
| Start Date | 2012-07 |
| Est. Completion | 2034-12 |
| Phase | Phase 2 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT01659606
The ClinicalTrials.gov registry entry for NCT01659606 describes a study currently listed as active not recruiting, categorized as Phase 2. The registered enrollment target is 40 participants, a figure that helps gauge the scale of data the investigators plan to collect, roughly in line with the 41-participant average among 23 other Aplastic Anemia trials with a reported enrollment target. The listed sponsor is Boston Children's Hospital, which has 462 total studies on file at ClinicalTrials.gov.
The record links to 4 conditions, with Aplastic Anemia appearing as the primary indexed condition, and to 5 interventions - of which Fludarabine is the first listed.
NCT01659606 reports 14 study locations spanning 11 distinct geographic areas - top geographies include Massachusetts, Other, California.
Frequently Asked Questions
What is clinical trial NCT01659606 about?
NCT01659606 is a clinical study titled "Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita". 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 an...
What is the current status of trial NCT01659606?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 40 participants. The study started on 2012-07. Estimated completion is 2034-12.
What conditions does trial NCT01659606 study?
This clinical trial studies the following conditions: Aplastic Anemia, Dyskeratosis Congenita, Hoyeraal-Hreidarsson Syndrome, Revesz Syndrome.
What interventions are being tested in trial NCT01659606?
The interventions under investigation include: Fludarabine (DRUG), Tacrolimus (DRUG), Mycophenolate mofetil (DRUG), alemtuzumab (BIOLOGICAL), Cyclosporins (DRUG).
Who is sponsoring clinical trial NCT01659606?
This trial is sponsored by Boston Children's Hospital, which has 462 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT01659606 being conducted?
This trial has 14 study locations across California, Illinois, Massachusetts, Missouri, New Jersey. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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