Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT00455312 · ClinicalTrials.gov registry record · Phase 2
Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA
A Phase 2 study, sponsored by Masonic Cancer Center, University of Minnesota.
- Completed
- Registry status
- Phase 2
- Development phase
- 36
- Enrollment target
NCT00455312: Completed Phase 2 study, sponsored by Masonic Cancer Center, University of Minnesota.
NCT00455312 is a Phase 2 study that has completed, run by Masonic Cancer Center, University of Minnesota. The registered enrollment target is 36 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (73% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00455312, a Phase 2 study, has completed, sponsored by Masonic Cancer Center, University of Minnesota.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 36 participants
- Enrollment target
Study Summary
Transplantation with stem cells is a standard therapy in many centers around the world. Previous experience with stem cell transplantation therapy for leukemias, lymphomas, other cancers, aplastic anemia and other non-malignant diseases, has led to prolonged disease-free survival or cure for some patients. However, the high doses of pre-transplant radiation and chemotherapy drugs used, and the type of drugs used, often cause many side effects that are intolerable for some patients. Slow recovery of blood counts is a frequent complication of high dose pre-transplant regimens, resulting in a longer period of risk for bleeding and infection plus a longer time in the hospital. Recent studies have shown that using lower doses of radiation and chemotherapy (ones that do not completely kill all of the patient's bone marrow cells) before blood or bone marrow transplant, may be a better treatment for high risk patients, such as those with Dyskeratosis Congenita (DC) or Severe Aplastic Anemia(SAA). These low dose transplants may result in shorter periods of low blood counts, and blood counts that do not go as low as with traditional pre-transplant radiation and chemotherapy. Furthermore, in patients with Dyskeratosis Congenita or SAA, the stem cell transplant will replace the blood forming cells with healthy cells. It has recently been shown that healthy marrow can take and grow after transplantation which uses doses of chemotherapy and radiation that are much lower than that given to patients with leukemia. While high doses of chemotherapy and radiation may be necessary to get rid of leukemia, this may not be important to patients with Dyskeratosis Congenita or SAA. The purpose of this research is to see if this lower dose chemotherapy and radiation regimen followed by transplant is a safe and effective treatment for patients with Dyskeratosis Congenita or SAA.
Primary Outcome
Defined as an absolute neutrophil count (ANC) \>5 x 10\^8/L (first of three consecutive laboratory measurements on different days) with at least 10% donor cells by day 100. Demonstrate sustained engraftment after a fludarabine based preparative regimen in patients with dyskeratosis congenita followed by hematopoietic cell transplantation.
Interventions
- DRUG Cyclophosphamide
- DRUG Fludarabine
- PROCEDURE Stem Cell Transplantation
- PROCEDURE Total Body Irradiation
- DRUG Campath 1H
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 36 participants |
| Start Date | 2007-08 |
| Est. Completion | 2016-06 |
| Phase | Phase 2 |
What the finished NCT00455312 record still lists
NCT00455312 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 36 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (73% lower).
The record links to 0 conditions, and to 5 interventions - of which Cyclophosphamide is the first listed.
NCT00455312 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00455312 about?
NCT00455312 is a clinical study titled "Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA". Transplantation with stem cells is a standard therapy in many centers around the world. Previous experience with stem cell transplantation therapy for leukemias, lymphomas, other cancers, aplastic anemia and other non-malignant diseases, has led to prolonged disease-free survival or cure for some pa...
What is the current status of trial NCT00455312?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 36 participants. The study started on 2007-08. Estimated completion is 2016-06.
What interventions are being tested in trial NCT00455312?
The interventions under investigation include: Cyclophosphamide (DRUG), Fludarabine (DRUG), Stem Cell Transplantation (PROCEDURE), Total Body Irradiation (PROCEDURE), Campath 1H (DRUG).
Who is sponsoring clinical trial NCT00455312?
This trial is sponsored by Masonic Cancer Center, University of Minnesota, which has 249 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00455312's enrollment target sits among peer trials
36 1496th of 2000 higher than 477 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT00929006 · 36 participants · Early Phase 1
Acute Progesterone Suppression of Wake vs. Sleep Luteinizing Hormone Pulse Frequency in Pubertal Girls With and Without Hyperandrogenism
- NCT01891318 · 36 participants · Phase 1
Neoadjuvant Radiosurgery for Resectable Brain Metastases: Phase I/II Study
- NCT02291848 · 36 participants · Phase 1
Tumor-Associated Antigen-Specific Cytotoxic T-Lymphocytes for Multiple Myeloma
- NCT02366819 · 36 participants · Phase 4
Genetic Analysis-Guided Irinotecan Hydrochloride Dosing of mFOLFIRINOX in Treating Patients With Locally Advanced Gastroesophageal or Stomach Cancer
Similar registry start date
- NCT05988463 · started 2027-01-01 · Phase 1
Dose-Escalation Study of Artesunate Patients With IPF
- NCT07219706 · started 2026-12-10 · NA
Dietary Fiber and Time Restricted Eating
- NCT02831335 · started 2026-12-01
Technical Development of Multi-Parametric Renocerebral MRI
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia