Trial · NCT00455312
Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA
NCT00455312, Phase 2, Phase 3, Completed, Masonic Cancer Center, University of Minnesota; results first posted 2017-05-31
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA. Trials, record NCT00455312. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00455312
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA
- NCT number
- NCT00455312
- Status
- Completed
- Phases
- Phase 2, Phase 3
- Enrollment
- 36 participants, actual
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Conditions
- Dyskeratosis Congenita, Aplastic Anemia
- Interventions
- Campath 1H, Cyclophosphamide, Fludarabine, Total Body Irradiation, Stem Cell Transplantation, antithymocyte globulin, Methylprednisolone
- First posted
- 2007-04-03
- Last updated
- 2017-12-05
- Results first posted
- 2017-05-31
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Neutrophil Engraftment | Day 100 | 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. |
| Secondary | Incidence of Regimen Related Mortality at 100 Days | 100 days | all deaths without previous relapse or progression |
| Secondary | Incidence of Chronic GVHD | 6 months | Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host. |
| Secondary | Incidence of Chronic GVHD | 1 year | Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host. |
| Secondary | Incidence of Late Secondary Malignancies | 1 Year | Defined as patients who have a secondary malignancy (cancer) occurring. |
| Secondary | Incidence of Grade 2-4 Acute Graft Versus Host Disease (GVHD) | Day 100 | Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host. |
| Secondary | Incidence of Grade 3-4 Acute Graft Versus Host Disease (GVHD) | Day 100 | Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host. |
| Secondary | Overall Survival | Day 100 | Overall survival is defined as time from date of transplant to date of death or censored at the date of last documented contact for patients still alive. |
| Secondary | Overall Survival | 1 Year | Overall survival is defined as time from date of transplant to date of death or censored at the date of last documented contact for patients still alive. |
| Secondary | Incidence of Pulmonary Complications | 6 Months | Defined as patients who exhibit a pulmonary (lung) adverse event. |
Source ClinicalTrials.gov
Outcomes10
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Neutrophil Engraftment | Day 100 | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Incidence of Regimen Related Mortality at 100 Days | 100 days | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Incidence of Chronic GVHD | 6 months | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Incidence of Chronic GVHD | 1 year | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Incidence of Late Secondary Malignancies | 1 Year | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Incidence of Grade 2-4 Acute Graft Versus Host Disease (GVHD) | Day 100 | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Incidence of Grade 3-4 Acute Graft Versus Host Disease (GVHD) | Day 100 | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Overall Survival | Day 100 | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Overall Survival | 1 Year | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Incidence of Pulmonary Complications | 6 Months | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group20
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Neutrophil Engraftment | Patients With DC | 15 | Participants | ||
| Neutrophil Engraftment | Patients With SAA | 20 | Participants | ||
| Incidence of Regimen Related Mortality at 100 Days | Patients With DC | 1 | Participants | ||
| Incidence of Regimen Related Mortality at 100 Days | Patients With SAA | 2 | Participants | ||
| Incidence of Chronic GVHD | Patients With DC | 0 | Participants | ||
| Incidence of Chronic GVHD | Patients With SAA | 0 | Participants | ||
| Incidence of Chronic GVHD | Patients With DC | 1 | Participants | ||
| Incidence of Chronic GVHD | Patients With SAA | 2 | Participants | ||
| Incidence of Late Secondary Malignancies | Patients With DC | 0 | Participants | ||
| Incidence of Late Secondary Malignancies | Patients With SAA | 4 | Participants |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Patients With DC | 15 | |
| Overall Study | STARTED | Patients With SAA | 21 | |
| Overall Study | COMPLETED | Patients With DC | 15 | |
| Overall Study | COMPLETED | Patients With SAA | 20 | |
| Overall Study | NOT COMPLETED | Patients With DC | 0 | |
| Overall Study | NOT COMPLETED | Patients With SAA | 1 |
Source ClinicalTrials.gov
Adverse events152
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Acute Brain Infarction | Nervous system disorders | Patients With DC | 0 | 15 | |
| other | Acute Brain Infarction | Nervous system disorders | Patients With SAA | 1 | 21 | |
| other | Acute Kidney Injury | Renal and urinary disorders | Patients With DC | 0 | 15 | |
| other | Acute Kidney Injury | Renal and urinary disorders | Patients With SAA | 1 | 21 | |
| other | Acute Pancreatitis | Gastrointestinal disorders | Patients With DC | 0 | 15 | |
| other | Acute Pancreatitis | Gastrointestinal disorders | Patients With SAA | 1 | 21 | |
| other | Acute Respiratory Distress Syndrome | Respiratory, thoracic and mediastinal disorders | Patients With DC | 3 | 15 | |
| other | Acute Respiratory Distress Syndrome | Respiratory, thoracic and mediastinal disorders | Patients With SAA | 2 | 21 | |
| other | Aseptic/Avascular Necrosis | Musculoskeletal and connective tissue disorders | Patients With DC | 2 | 15 | |
| other | Aseptic/Avascular Necrosis | Musculoskeletal and connective tissue disorders | Patients With SAA | 1 | 21 |
1 to 10 of 152Source ClinicalTrials.gov
See all 152 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Patients With DC | ||
| Total, all-cause mortality | Patients With SAA | ||
| Total, other adverse events | Patients With DC | 15 | 15 |
| Total, other adverse events | Patients With SAA | 20 | 21 |
| Total, serious adverse events | Patients With DC | 2 | 15 |
| Total, serious adverse events | Patients With SAA | 1 | 21 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 20383216 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons11
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Patients With DC vs Patients With SAA: Incidence of Late Secondary Malignancies | Trial comparisons |
| Patients With DC vs Patients With SAA: Incidence of Chronic GVHD | Trial comparisons |
| Patients With DC vs Patients With SAA: Incidence of Grade 2-4 Acute Graft Versus Host Disease (GVHD) | Trial comparisons |
| Patients With DC vs Patients With SAA: Incidence of Chronic GVHD | Trial comparisons |
| Patients With DC vs Patients With SAA: Incidence of Pulmonary Complications | Trial comparisons |
| Patients With DC vs Patients With SAA: Overall Survival | Trial comparisons |
| Patients With DC vs Patients With SAA: Incidence of Regimen Related Mortality at 100 Days | Trial comparisons |
| Patients With DC vs Patients With SAA: Incidence of Grade 3-4 Acute Graft Versus Host Disease (GVHD) | Trial comparisons |
| Patients With DC vs Patients With SAA: Overall Survival | Trial comparisons |
| Patients With DC vs Patients With SAA: Neutrophil Engraftment | Trial comparisons |
1 to 10 of 11
See all 11 trial comparisonsTests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT00455312
GET https://conductscience.org/api/trials/v1/NCT00455312/events?page=2