Trial · NCT01626092
Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders
NCT01626092, Not applicable, Completed, Masonic Cancer Center, University of Minnesota; results first posted 2016-10-11
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders. Trials, record NCT01626092. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01626092
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders
- NCT number
- NCT01626092
- Status
- Completed
- Phases
- Not applicable
- Enrollment
- 3 participants, actual
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Conditions
- Lysosomal Storage Disease, Peroxisomal Disorder
- Interventions
- Campath-1H, Clofarabine, Melphalan, Total Body Irradiation with Marrow Boosting, Hematopoietic stem cell transplantation, Cyclosporine A, Mycophenolate mofetil
- First posted
- 2012-06-22
- Last updated
- 2017-12-05
- Results first posted
- 2016-10-11
Source ClinicalTrials.gov
Registered outcomes3
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Donor (Allogeneic) Hematopoietic Engraftment | Day 100 Following Hematopoietic Cell Transplant (HCT) | Number of patients who achieve hematopoietic engraftment - assessment of nucleated peripheral blood cells for donor (allogeneic) chimerism following this reduced-intensity HCT. |
| Secondary | Transplant-Related Mortality | Day 100 following HCT | Incidence of death due to complications of HCT following this reduced-intensity conditioning regimen. |
| Secondary | Neurologic Outcomes | Changes from Baseline, Days 30, 60, 100, Year 1, Year 2, Year 3 Following HCT | Depending upon underlying primary disease, a combination of evaluative tools (e.g. brain magnetic resonance imaging (MRI), clinical neurologic exam, neuropsychologic testing, electromyography) will be applied for assessment of neurologic function and how it may be affected by this reduced-intensity HCT regimen. |
Source ClinicalTrials.gov
Outcomes3
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Donor (Allogeneic) Hematopoietic Engraftment | Day 100 Following Hematopoietic Cell Transplant (HCT) | participants | NUMBER | |
| SECONDARY | Transplant-Related Mortality | Day 100 following HCT | participants | NUMBER | |
| SECONDARY | Neurologic Outcomes | Changes from Baseline, Days 30, 60, 100, Year 1, Year 2, Year 3 Following HCT | None of the 3 patients enrolled in the study were evaluable for this outcome. Two had a repeat transplant and one was lost to follow-up. |
Source ClinicalTrials.gov
Results by group2
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Donor (Allogeneic) Hematopoietic Engraftment | Intent-To-Treat Patients | 1 | participants | ||
| Transplant-Related Mortality | Intent-To-Treat Patients | 0 | 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 | Intent-To-Treat Patients | 3 | |
| Overall Study | COMPLETED | Intent-To-Treat Patients | 3 | |
| Overall Study | NOT COMPLETED | Intent-To-Treat Patients | 0 |
Source ClinicalTrials.gov
Adverse events32
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 | Hearing loss | Ear and labyrinth disorders | Intent-To-Treat Patients | 1 | 3 | |
| other | Decreased vision | Eye disorders | Intent-To-Treat Patients | 2 | 3 | |
| other | Abdominal pain | Gastrointestinal disorders | Intent-To-Treat Patients | 1 | 3 | |
| other | Nausea | Gastrointestinal disorders | Intent-To-Treat Patients | 3 | 3 | |
| other | Vomiting | Gastrointestinal disorders | Intent-To-Treat Patients | 2 | 3 | |
| other | Mucositis | Gastrointestinal disorders | Intent-To-Treat Patients | 3 | 3 | |
| other | Acid reflux | Gastrointestinal disorders | Intent-To-Treat Patients | 1 | 3 | |
| other | Loose stools | Gastrointestinal disorders | Intent-To-Treat Patients | 1 | 3 | |
| other | Fever | General disorders | Intent-To-Treat Patients | 2 | 3 | |
| other | Positive blood culture - Staphylococcus aureus | Infections and infestations | Intent-To-Treat Patients | 1 | 3 |
1 to 10 of 32Source ClinicalTrials.gov
See all 32 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Intent-To-Treat Patients | ||
| Total, other adverse events | Intent-To-Treat Patients | 3 | 3 |
| Total, serious adverse events | Intent-To-Treat Patients | 0 | 3 |
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/NCT01626092
GET https://conductscience.org/api/trials/v1/NCT01626092/events?page=2