Trial · NCT01033552
Biochemical Correction of Severe EB by Allo HSCT and "Off-the-shelf" MSCs
NCT01033552, Phase 1, Phase 2, Completed, Masonic Cancer Center, University of Minnesota; results first posted 2024-04-03
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Biochemical Correction of Severe EB by Allo HSCT and "Off-the-shelf" MSCs. Trials, record NCT01033552. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01033552
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Biochemical Correction of Severe EB by Allo HSCT and "Off-the-shelf" MSCs
- NCT number
- NCT01033552
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 32 participants, actual
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Conditions
- Epidermolysis Bullosa
- Interventions
Cyclophosphamide, Fludarabine, Anti-thymocyte globulin, Myeloablative Busulfan, Mesenchymal stem cell transplantation, Total body irradiation, Bone marrow or umbilical cord blood (UCG) stem cell transplantation
- First posted
- 2009-12-16
- Last updated
- 2024-04-03
- Results first posted
- 2024-04-03
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants With Event-free Survival | 1 year and 2 Years Post-transplant | Event-free survival rate, with an event defined as death or failure to have a demonstrable increase in collagen, laminin, intergrin, keratin or plakin deposition. Assessed at follow up appointments through questionnaire and patient samples. |
| Secondary | Percentage of Participants Transplant-related Mortality (TRM) | 180 Days Post Transplant | Incidence of transplant-related mortality (TRM) |
| Secondary | Average Biochemical Improvement | 1 Year Post-Transplant | Pattern of biochemical improvement measured by cumulative increase in protein expression and related structural and physical changes |
| Secondary | Measure Patients Quality of Life Using a Questionnaire | Pretreatment and 1 year | Health quality of life questionnaire as compared to pretreatment results. Scores can range from 0 to 100. The QOLS scores are summed so that a higher score indicates higher quality of life. |
| Secondary | Durability of HSC Donor Engraftment in the Skin | 100 Days | Incidence of HSC donor engraftment in the skin |
| Secondary | Probability of Survival | 1 Year | Surviving patients one year after engraftment |
| Secondary | Percentage of Participants Who Experienced Acute GVHD | 100 Days | Incidence of acute GCHD |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants With Event-free Survival | 1 year and 2 Years Post-transplant | Percentage of participants | NUMBER | |
| SECONDARY | Percentage of Participants Transplant-related Mortality (TRM) | 180 Days Post Transplant | Percentage of participants | NUMBER | |
| SECONDARY | Average Biochemical Improvement | 1 Year Post-Transplant | Percentage of participants | NUMBER | |
| SECONDARY | Measure Patients Quality of Life Using a Questionnaire | Pretreatment and 1 year | Participants unnable to complete quality of life questionnaires at various time points. | Scores on a scale | MEDIAN |
| SECONDARY | Durability of HSC Donor Engraftment in the Skin | 100 Days | Percentage of Incidence | NUMBER | |
| SECONDARY | Probability of Survival | 1 Year | Percentage of participants | NUMBER | |
| SECONDARY | Percentage of Participants Who Experienced Acute GVHD | 100 Days | Percentage of participants | NUMBER |
Source ClinicalTrials.gov
Results by group33
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants With Event-free Survival | RDEB Mac | 1 year | 67 | 95% Confidence Interval 19 to 90 | Percentage of participants |
| Percentage of Participants With Event-free Survival | RDEB RIC | 1 year | 80 | 95% Confidence Interval 50 to 93 | Percentage of participants |
| Percentage of Participants With Event-free Survival | JEB MAC | 1 year | 50 | 95% Confidence Interval 1 to 91 | Percentage of participants |
| Percentage of Participants With Event-free Survival | JEB RIC | 1 year | 29 | 95% Confidence Interval 4 to 61 | Percentage of participants |
| Percentage of Participants With Event-free Survival | RDEB Mac | 2 year | 44 | 95% Confidence Interval 7 to 78 | Percentage of participants |
| Percentage of Participants With Event-free Survival | RDEB RIC | 2 year | 80 | 95% Confidence Interval 50 to 93 | Percentage of participants |
| Percentage of Participants With Event-free Survival | JEB MAC | 2 year | 50 | 95% Confidence Interval 1 to 91 | Percentage of participants |
| Percentage of Participants With Event-free Survival | JEB RIC | 2 year | 14 | 95% Confidence Interval 1 to 46 | Percentage of participants |
| Percentage of Participants Transplant-related Mortality (TRM) | RDEB Mac | 43 | 95% Confidence Interval 11 to 75 | Percentage of participants | |
| Percentage of Participants Transplant-related Mortality (TRM) | RDEB RIC | 19 | 95% Confidence Interval 2 to 36 | Percentage of participants |
1 to 10 of 33Source ClinicalTrials.gov
See all 33 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | RDEB Mac | 7 | |
| Overall Study | STARTED | RDEB RIC | 16 | |
| Overall Study | STARTED | JEB MAC | 2 | |
| Overall Study | STARTED | JEB RIC | 7 | |
| Overall Study | COMPLETED | RDEB Mac | 7 | |
| Overall Study | COMPLETED | RDEB RIC | 16 | |
| Overall Study | COMPLETED | JEB MAC | 2 | |
| Overall Study | COMPLETED | JEB RIC | 7 | |
| Overall Study | NOT COMPLETED | RDEB Mac | 0 | |
| Overall Study | NOT COMPLETED | RDEB RIC | 0 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events200
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 | Dyspnea | Respiratory, thoracic and mediastinal disorders | RDEB Mac | 2 | 7 | 2 |
| other | Dyspnea | Respiratory, thoracic and mediastinal disorders | RDEB RIC | 0 | 16 | 0 |
| other | Dyspnea | Respiratory, thoracic and mediastinal disorders | JEB MAC | 1 | 2 | 1 |
| other | Dyspnea | Respiratory, thoracic and mediastinal disorders | JEB RIC | 1 | 7 | 1 |
| other | Hypoxia | General disorders | RDEB Mac | 0 | 7 | 0 |
| other | Hypoxia | General disorders | RDEB RIC | 0 | 16 | 0 |
| other | Hypoxia | General disorders | JEB MAC | 0 | 2 | 0 |
| other | Hypoxia | General disorders | JEB RIC | 1 | 7 | 1 |
| other | Febrile neutropenia | Blood and lymphatic system disorders | RDEB Mac | 6 | 7 | 13 |
| other | Febrile neutropenia | Blood and lymphatic system disorders | RDEB RIC | 2 | 16 | 2 |
1 to 10 of 200Source ClinicalTrials.gov
See all 200 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | RDEB Mac | 4 | 7 |
| Total, all-cause mortality | RDEB RIC | 3 | 16 |
| Total, all-cause mortality | JEB MAC | 1 | 2 |
| Total, all-cause mortality | JEB RIC | 6 | 7 |
| Total, other adverse events | RDEB Mac | 7 | 7 |
| Total, other adverse events | RDEB RIC | 16 | 16 |
| Total, other adverse events | JEB MAC | 2 | 2 |
| Total, other adverse events | JEB RIC | 7 | 7 |
| Total, serious adverse events | RDEB Mac | 7 | 7 |
| Total, serious adverse events | RDEB RIC | 16 | 16 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers0
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/NCT01033552
GET https://conductscience.org/api/trials/v1/NCT01033552/results?page=2