Trial · NCT00176917
Stem Cell Transplantation for Hurler
NCT00176917, Phase 2, Completed, Masonic Cancer Center, University of Minnesota; results first posted 2009-09-04
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Stem Cell Transplantation for Hurler. Trials, record NCT00176917. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00176917
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Stem Cell Transplantation for Hurler
- NCT number
- NCT00176917
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 41 participants, actual
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Conditions
- Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Mannosidosis, Mucolipidosis Type II (I-cell Disease)
- Interventions
- Stem Cell Transplant, Busulfan, Cyclophosphamide, ATG
- First posted
- 2005-09-15
- Last updated
- 2017-12-28
- Results first posted
- 2009-09-04
- Limitations and caveats
Based on variability in age, diagnosis and condition of these patients, the data on enzyme levels and neuropsych testing is extremely difficult to report.~See Secondary Outcome Measures #3 and #4; no other adverse events were collected.
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Mean Percentage of Donor Cells in Study Population (Chimerism). | at 21 days, 42 days, 60 days, 100 days, 6 months, and 1 year | Donor-derived engraftment determined by restriction fragment length polymorphism (RFLP). |
| Secondary | Number of Patients Surviving on Study | at 100 days, 1 year, and 3 years post transplant | Number of patients surviving (alive) at specified timepoints. |
| Secondary | Number of Patients Who Failed Engraftment. | Day 42 Post Transplant | Toxicity (undesireable effect) of hematologic donor cell engraftment is determined by failure to engraft at Day 42. |
| Secondary | Number of Patients With Grade III-IV Acute Graft-versus-host Disease (aGVHD). | Day 100 Post Transplant | Toxicity (undesireable effect) of this stem cell transplant preparative regimen due to acute graft-versus-host disease. |
Source ClinicalTrials.gov
Outcomes4
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Mean Percentage of Donor Cells in Study Population (Chimerism). | at 21 days, 42 days, 60 days, 100 days, 6 months, and 1 year | Day 21 (24 patients included), Day 42 (15 pts), Day 60 (29 pts), Day 100 (25 pts), 6 Months (18 pts), 1 Year (16 pts). | Percentage | MEAN |
| SECONDARY | Number of Patients Surviving on Study | at 100 days, 1 year, and 3 years post transplant | Day 100 and 1 Year timepoints include all 41 patients. Year 3 includes 36 patients (5 pts not yet at followup timepoint.) | Participants | NUMBER |
| SECONDARY | Number of Patients Who Failed Engraftment. | Day 42 Post Transplant | 1 patient of 41 failed engraftment - per protocol. | Participants | NUMBER |
| SECONDARY | Number of Patients With Grade III-IV Acute Graft-versus-host Disease (aGVHD). | Day 100 Post Transplant | Participants | NUMBER |
Source ClinicalTrials.gov
Results by group11
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Mean Percentage of Donor Cells in Study Population (Chimerism). | Transplant Patients | 21 Days Post Transplant | 85.8 | Standard Deviation 28.4 | Percentage |
| Mean Percentage of Donor Cells in Study Population (Chimerism). | Transplant Patients | 42 Days Post Transplant | 73.2 | Standard Deviation 31.6 | Percentage |
| Mean Percentage of Donor Cells in Study Population (Chimerism). | Transplant Patients | 60 Days Post Transplant | 84.6 | Standard Deviation 27.9 | Percentage |
| Mean Percentage of Donor Cells in Study Population (Chimerism). | Transplant Patients | 100 Days Post Transplant | 81.1 | Standard Deviation 26.8 | Percentage |
| Mean Percentage of Donor Cells in Study Population (Chimerism). | Transplant Patients | 6 Months Post Transplant | 81.6 | Standard Deviation 29.6 | Percentage |
| Mean Percentage of Donor Cells in Study Population (Chimerism). | Transplant Patients | 1 Year Post Transplant | 91.5 | Standard Deviation 18.8 | Percentage |
| Number of Patients Surviving on Study | Transplant Patients | Day 100 Post Transplant | 37 | Participants | |
| Number of Patients Surviving on Study | Transplant Patients | 1 Year Post Transplant | 28 | Participants | |
| Number of Patients Surviving on Study | Transplant Patients | 3 Years Post Transplant | 27 | Participants | |
| Number of Patients Who Failed Engraftment. | Transplant Patients | 1 | Participants |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Transplant Patients | 41 | |
| Overall Study | COMPLETED | Transplant Patients | 41 | |
| Overall Study | NOT COMPLETED | Transplant Patients | 0 |
Source ClinicalTrials.gov
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Transplant Patients | ||
| Total, other adverse events | Transplant Patients | 0 | 41 |
| Total, serious adverse events | Transplant Patients | 0 | 41 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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/NCT00176917
GET https://conductscience.org/api/trials/v1/NCT00176917/registered