Trial · NCT00176904
Stem Cell Transplant for Inborn Errors of Metabolism
NCT00176904, Phase 2, Phase 3, Completed, Masonic Cancer Center, University of Minnesota; results first posted 2011-06-10
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Stem Cell Transplant for Inborn Errors of Metabolism. Trials, record NCT00176904. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00176904
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Stem Cell Transplant for Inborn Errors of Metabolism
- NCT number
- NCT00176904
- Status
- Completed
- Phases
- Phase 2, Phase 3
- Enrollment
- 135 participants, actual
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Conditions
Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Gaucher's Disease, Fucosidosis, Wolman Disease, Niemann-Pick Disease, Batten Disease, GM1 Gangliosidosis, Tay Sachs Disease, Sandhoff Disease
- Interventions
- Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin
- First posted
- 2005-09-15
- Last updated
- 2017-12-28
- Results first posted
- 2011-06-10
- Limitations and caveats
Change in Neuropsychometric function data is not available. Data would have been noted as descriptive and patient specific, as well as disease specific; not table format. No formal analysis was intended.
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Overall Survival | 100 Days, 1 Year and 3 Years | Number of patients alive at designated timepoints after transplant. |
| Secondary | Overall Donor Engraftment | Day 100 | Number of patients with full donor chimerism (state in bone marrow transplantation in which bone marrow and host cells exist compatibly without signs of graft-versus-host rejection disease) by Day 100 post-transplant of at least 90%. |
| Secondary | Number of Patients With Grade II-IV Acute Graft-Versus-Host Disease | Day 100 | Number of patients who exhibited acute graft-versus-host disease by Day 100 post transplant. Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted material attacks the transplant recipient's body. Grade I=mild, Grade II=moderate, Grade III=severe, Grade IV=life threatening. |
| Secondary | Number of Patients With Grade III-IV Acute Graft-Versus-Host Disease | Day 100 | Number of patients who exhibited acute graft-versus-host disease by Day 100 post transplant. Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted material attacks the transplant recipient's body. Grade I=mild, Grade II=moderate, Grade III=severe, Grade IV=life threatening. |
| Secondary | Number of Patients With Chronic Graft-Versus-Host Disease | 1 Year Post Transplant | Number of patients who exhibited chronic graft-versus-host disease by 1 Year post transplant. Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted material attacks the transplant recipient's body. Chronic GVHD is an extension of this syndrome. |
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 | Overall Survival | 100 Days, 1 Year and 3 Years | Participants | NUMBER | |
| SECONDARY | Overall Donor Engraftment | Day 100 | 1 Patient not included due to early death (before day 40). | Participants | NUMBER |
| SECONDARY | Number of Patients With Grade II-IV Acute Graft-Versus-Host Disease | Day 100 | Participants | NUMBER | |
| SECONDARY | Number of Patients With Grade III-IV Acute Graft-Versus-Host Disease | Day 100 | Participants | NUMBER | |
| SECONDARY | Number of Patients With Chronic Graft-Versus-Host Disease | 1 Year Post Transplant | Participants | NUMBER |
Source ClinicalTrials.gov
Results by group7
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Overall Survival | Patients Treated With Stem Cell Transplant | Day 100 | 120 | Participants | |
| Overall Survival | Patients Treated With Stem Cell Transplant | 1 Year | 92 | Participants | |
| Overall Survival | Patients Treated With Stem Cell Transplant | 3 Years | 81 | Participants | |
| Overall Donor Engraftment | Patients Treated With Stem Cell Transplant | 123 | Participants | ||
| Number of Patients With Grade II-IV Acute Graft-Versus-Host Disease | Patients Treated With Stem Cell Transplant | 34 | Participants | ||
| Number of Patients With Grade III-IV Acute Graft-Versus-Host Disease | Patients Treated With Stem Cell Transplant | 13 | Participants | ||
| Number of Patients With Chronic Graft-Versus-Host Disease | Patients Treated With Stem Cell Transplant | 13 | 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 | Patients Treated With Stem Cell Transplant | 135 | |
| Overall Study | COMPLETED | Patients Treated With Stem Cell Transplant | 135 | |
| Overall Study | NOT COMPLETED | Patients Treated With Stem Cell Transplant | 0 |
Source ClinicalTrials.gov
Adverse events3
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 |
|---|---|---|---|---|---|---|
| serious | Auto recovery | General disorders | Patients Treated With Stem Cell Transplant | 9 | 135 | 9 |
| serious | Death | General disorders | Patients Treated With Stem Cell Transplant | 54 | 135 | 54 |
| serious | Primary graft failure | Blood and lymphatic system disorders | Patients Treated With Stem Cell Transplant | 2 | 135 | 2 |
Source ClinicalTrials.gov
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Patients Treated With Stem Cell Transplant | ||
| Total, other adverse events | Patients Treated With Stem Cell Transplant | 0 | 135 |
| Total, serious adverse events | Patients Treated With Stem Cell Transplant | 62 | 135 |
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/NCT00176904
GET https://conductscience.org/api/trials/v1/NCT00176904/registered