Trial · NCT01043640
Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
NCT01043640, Phase 2, Completed, Masonic Cancer Center, University of Minnesota; results first posted 2017-05-12
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders. Trials, record NCT01043640. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01043640
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
- NCT number
- NCT01043640
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 46 participants, actual
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Conditions
Mucopolysaccharidosis, Hurler Syndrome, Hunter Syndrome, Maroteaux-Lamy Syndrome, Sly Syndrome, Alpha Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Adrenoleukodystrophy (ALD), Krabbe Disease, Metachromatic Leukodystrophy (MLD), Sphingolipidoses, Peroxisomal Disorders
- Interventions
- Campath-1H, Cyclophosphamide, Busulfan, Allogeneic stem cell transplantation, Cyclosporine A, Mycophenolate Mofetil
- First posted
- 2010-01-07
- Last updated
- 2018-02-05
- Results first posted
- 2017-05-12
Source ClinicalTrials.gov
Registered outcomes12
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Patients With Donor Derived Engraftment | Day 100 Post Transplant | Donor derived engraftment is defined as 80 percent or greater donor cells in the recipient's bone marrow and blood cells. |
| Secondary | Number of Patients With Grade 0 Graft-Versus-Host Disease (GVHD) | Day 100 Post Transplant | GVHD grading is performed using modified Glucksberg criteria and is as follows: grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4 |
| Secondary | Number of Patients With Grade 1 Graft-Versus-Host Disease (GVHD) | Day 100 Post Transplant | GVHD grading is performed using modified Glucksberg criteria and is as follows: grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4 |
| Secondary | Number of Patients With Grade 2 Graft-Versus-Host Disease (GVHD) | Day 100 Post Transplant | GVHD grading is performed using modified Glucksberg criteria and is as follows: grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4 |
| Secondary | Number of Patients With Grade 3 Graft-Versus-Host Disease (GVHD) | Day 100 Post Transplant | GVHD grading is performed using modified Glucksberg criteria and is as follows: grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4 |
| Secondary | Number of Patients With Grade 4 Graft-Versus-Host Disease (GVHD) | Day 100 Post Transplant | GVHD grading is performed using modified Glucksberg criteria and is as follows: grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4 |
| Secondary | Number of Patients Who Died Peri-Transplant | By Day 100 Post Transplant | Peri-transplant is defined as within 100 days of transplant. |
| Secondary | Donor Cell Chimerism Following Transplant | Day 28 | Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin. |
| Secondary | Donor Cell Chimerism Following Transplant | Day 42 | Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin. |
| Secondary | Donor Cell Chimerism Following Transplant | Day 100 | Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin. |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 registered outcomesOutcomes12
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 Derived Engraftment | Day 100 Post Transplant | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Number of Patients With Grade 0 Graft-Versus-Host Disease (GVHD) | Day 100 Post Transplant | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Number of Patients With Grade 1 Graft-Versus-Host Disease (GVHD) | Day 100 Post Transplant | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Number of Patients With Grade 2 Graft-Versus-Host Disease (GVHD) | Day 100 Post Transplant | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Number of Patients With Grade 3 Graft-Versus-Host Disease (GVHD) | Day 100 Post Transplant | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Number of Patients With Grade 4 Graft-Versus-Host Disease (GVHD) | Day 100 Post Transplant | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Number of Patients Who Died Peri-Transplant | By Day 100 Post Transplant | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Donor Cell Chimerism Following Transplant | Day 28 | percentage of donor cells | MEAN | |
| SECONDARY | Donor Cell Chimerism Following Transplant | Day 42 | percentage of donor cells | MEAN | |
| SECONDARY | Donor Cell Chimerism Following Transplant | Day 100 | percentage of donor cells | MEAN |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 outcomesResults by group12
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 Derived Engraftment | Transplant Patients | 42 | Participants | ||
| Number of Patients With Grade 0 Graft-Versus-Host Disease (GVHD) | Transplant Patients | 32 | Participants | ||
| Number of Patients With Grade 1 Graft-Versus-Host Disease (GVHD) | Transplant Patients | 2 | Participants | ||
| Number of Patients With Grade 2 Graft-Versus-Host Disease (GVHD) | Transplant Patients | 5 | Participants | ||
| Number of Patients With Grade 3 Graft-Versus-Host Disease (GVHD) | Transplant Patients | 2 | Participants | ||
| Number of Patients With Grade 4 Graft-Versus-Host Disease (GVHD) | Transplant Patients | 5 | Participants | ||
| Number of Patients Who Died Peri-Transplant | Transplant Patients | 2 | Participants | ||
| Donor Cell Chimerism Following Transplant | Transplant Patients | 95 | Standard Deviation 9 | percentage of donor cells | |
| Donor Cell Chimerism Following Transplant | Transplant Patients | 93 | Standard Deviation 15 | percentage of donor cells | |
| Donor Cell Chimerism Following Transplant | Transplant Patients | 90 | Standard Deviation 26 | percentage of donor cells |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 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 | 46 | |
| Overall Study | COMPLETED | Transplant Patients | 46 | |
| Overall Study | NOT COMPLETED | Transplant Patients | 0 |
Source ClinicalTrials.gov
Adverse events29
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 | Anorexia | Metabolism and nutrition disorders | Transplant Patients | 7 | 46 | |
| other | Bilirubin Increased | Investigations | Transplant Patients | 16 | 46 | |
| other | Diarrhea | Gastrointestinal disorders | Transplant Patients | 4 | 46 | |
| other | Dyspnea | Respiratory, thoracic and mediastinal disorders | Transplant Patients | 14 | 46 | |
| other | Fever | General disorders | Transplant Patients | 11 | 46 | |
| other | Graft Versus Host Disease | General disorders | Transplant Patients | 9 | 46 | |
| other | Headache | Nervous system disorders | Transplant Patients | 3 | 46 | |
| other | Hemorrhage, Gastrointestinal | Gastrointestinal disorders | Transplant Patients | 8 | 46 | |
| other | Hemorrhage, Genitourinary | Renal and urinary disorders | Transplant Patients | 10 | 46 | |
| other | Hives | Skin and subcutaneous tissue disorders | Transplant Patients | 3 | 46 |
1 to 10 of 29Source ClinicalTrials.gov
See all 29 adverse eventsAdverse 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 | 40 | 46 |
| Total, serious adverse events | Transplant Patients | 8 | 46 |
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/NCT01043640
GET https://conductscience.org/api/trials/v1/NCT01043640/events?page=2