Trial · NCT00383448
HSCT for High Risk Inherited Inborn Errors
NCT00383448, Phase 2, Completed, Masonic Cancer Center, University of Minnesota; results first posted 2019-07-11
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. HSCT for High Risk Inherited Inborn Errors. Trials, record NCT00383448. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00383448
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- HSCT for High Risk Inherited Inborn Errors
- NCT number
- NCT00383448
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 38 participants, actual
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Conditions
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Tay Sachs Disease, Sandhoffs Disease, Wolman Disease, I-Cell Disease, Sanfilippo Syndrome, GM1 Gangliosidosis
- Interventions
- Clofarabine, Total body Irradiation, Melphalan, Hematopoietic Stem Cell Transplantation, Alemtuzumab, mycophenylate mofetil, Cyclosporine A, Hydroxyurea
- First posted
- 2006-10-03
- Last updated
- 2019-07-11
- Results first posted
- 2019-07-11
- Limitations and caveats
Because the diseases being treated on this study are rare, our intent was to only report these outcomes in context with other protocols.~The data was collected, but is not interpretable without combining the data with additional studies.
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Patients With Donor Cell Engraftment | Day 100 | Donor Cell Engraftment is defined as the process of transplanted stem cells reproducing new cells. |
| Secondary | Number of Patients Whose Death Was Related to the Transplant | Day 100 | In the field of transplantation, toxicity is high and all deaths without previous relapse or progression are usually considered as related to transplantation. |
| Secondary | Concentrations of Mycophenylate Mofetil (MMF) | Day 3 | MMF levels are to be sent on day +3 to the main laboratory for determinations of MMF kinetics. Data was not collected on this outcome measure and is not available for reporting. |
| Secondary | Number of Patients With Acute Graft Versus Host Disease (GVHD) | Day 100 | Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host. Acute GVHD can occur once the donor's cells have engrafted in the transplant recipient. The symptoms typically appear within weeks after transplant. |
| Secondary | Number of Patients With Chronic Graft Versus Host Disease (GVHD) | 1 year | Graft-Versus-Host Disease is a severe complication created by infusion of donor cells into a foreign host. Chronic GVHD can appear at any time after allogeneic transplant or several years after transplant. |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Patients With Donor Cell Engraftment | Day 100 | One patient was not evaluable due to an early death. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Patients Whose Death Was Related to the Transplant | Day 100 | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Concentrations of Mycophenylate Mofetil (MMF) | Day 3 | Data was not collected on this outcome measure and is not available for reporting. | ||
| SECONDARY | Number of Patients With Acute Graft Versus Host Disease (GVHD) | Day 100 | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Number of Patients With Chronic Graft Versus Host Disease (GVHD) | 1 year | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group4
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Patients With Donor Cell Engraftment | Treated Patients | 26 | Participants | ||
| Number of Patients Whose Death Was Related to the Transplant | Treated Patients | 2 | Participants | ||
| Number of Patients With Acute Graft Versus Host Disease (GVHD) | Treated Patients | 3 | Participants | ||
| Number of Patients With Chronic Graft Versus Host Disease (GVHD) | Treated Patients | 2 | Participants |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Treated Patients | 38 | |
| Overall Study | COMPLETED | Treated Patients | 38 | |
| Overall Study | NOT COMPLETED | Treated Patients | 0 |
Source ClinicalTrials.gov
Adverse events38
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 | Bacterial Infection, Feces | Infections and infestations | Treated Patients | 10 | 38 | |
| other | Bacterial Infection, Blood | Infections and infestations | Treated Patients | 12 | 38 | |
| other | Pneumonia | Infections and infestations | Treated Patients | 14 | 38 | |
| other | Fungal Infection, Skin | Infections and infestations | Treated Patients | 2 | 38 | |
| other | Aseptic/Avascular Necrosis, NOS | General disorders | Treated Patients | 2 | 38 | |
| other | Bloody Stool | Gastrointestinal disorders | Treated Patients | 2 | 38 | |
| other | Capillary Leak | Vascular disorders | Treated Patients | 2 | 38 | |
| other | Cerebral and Cerebellar Atrophy | Nervous system disorders | Treated Patients | 2 | 38 | |
| other | Requires Hemodialysis, NOS | General disorders | Treated Patients | 2 | 38 | |
| other | Hemolytic Anemia | Blood and lymphatic system disorders | Treated Patients | 2 | 38 |
1 to 10 of 38Source ClinicalTrials.gov
See all 38 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Treated Patients | ||
| Total, other adverse events | Treated Patients | 33 | 38 |
| Total, serious adverse events | Treated Patients | 10 | 38 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 21586746 | 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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
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/NCT00383448
GET https://conductscience.org/api/trials/v1/NCT00383448/events?page=2