Trial · NCT02582775
MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCs
NCT02582775, Phase 2, Completed, Masonic Cancer Center, University of Minnesota; results first posted 2024-09-19
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCs. Trials, record NCT02582775. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02582775
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCs
- NCT number
- NCT02582775
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 17 participants, actual
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Conditions
- Epidermolysis Bullosa
- Interventions
- Thymoglobulin, Cyclophosphamide, Fludarabine, Total Body Irradiation, Bone marrow infusion, Tacrolimus, Mycophenolate Mofetil, Donor mesenchymal stem cell infusions, Busulfan
- First posted
- 2015-10-21
- Last updated
- 2024-09-19
- Results first posted
- 2024-09-19
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Event-free Survival | 1 year post-transplant | An event defined as death or a 50% increase in a patient's IScoreEB from baseline |
| Secondary | Change of a Patient's iscorEB | 1 year post-transplant | iscorEB surveys are a validated, standard of care tool used to assess disease status in patients with Epidermolysis Bullosa. Measure changes in quality of life (QOL) through pain, itching, and general QOL IScorEB questionnaire. Scores can range from 16 to 112. The QOLS scores are summed so that a higher score indicates higher quality of life. |
| Secondary | Transplant-related Mortality | 180 days post-transplant | Cumulative incidence will be used to estimate the probability of relapse treating non-relapse death as a competing risk and transplant-related mortality conversely treating relapse as a competing risk. |
| Secondary | Average Change in Quality of Life | 1 year post-transplant | Measured by the Lansky or Karnofsky score (10-100). Higher scores indicate a better outcome. Midpoint of change in scores used to indicate median. |
| Secondary | Average Change in Quality of Life | 2 years post-transplant | Measured by the Lansky or Karnofsky score (10-100). The Karnofsky Performance Scale is an assessment tool intended to gauge a patient's functional status and ability to carry out activities of daily living. Higher scores indicate a better outcome. Midpoint of change in scores used to indicate median. |
| Secondary | Lymphoid Chimerism | Day 28, 60, 100, 180, and year 1 and 2 post-transplant | Median hematopoietic cells that are of donor origin per participant. |
| Secondary | Myeloid Chimerism | Day 28, 60, 100, 180, and year 1 and 2 post-transplant | Median hematopoietic cells that are of donor origin per participant. |
| Secondary | Average Change of a Patient's iscorEB | 2 year post-transplant | iscorEB surveys are a validated, standard of care tool used to assess disease status in patients with Epidermolysis Bullosa. Measure percent of changes in quality of life (QOL) through pain, itching, and general QOL IScorEB questionnaire. Scores can range from 16 to 112. The QOLS scores are summed so that a higher score indicates higher quality of life. |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Event-free Survival | 1 year post-transplant | Count of Participants | NUMBER | |
| SECONDARY | Change of a Patient's iscorEB | 1 year post-transplant | units on a scale | MEAN | |
| SECONDARY | Transplant-related Mortality | 180 days post-transplant | Participants | NUMBER | |
| SECONDARY | Average Change in Quality of Life | 1 year post-transplant | Change in score | MEDIAN | |
| SECONDARY | Average Change in Quality of Life | 2 years post-transplant | Participant in JEB: HCT plus MSC Arm B deceased at 2 year post-transplant timepoint. | Change in score | MEDIAN |
| SECONDARY | Lymphoid Chimerism | Day 28, 60, 100, 180, and year 1 and 2 post-transplant | Participants in RDEB: HCT plus MSC arm unable to be analyzed due to missed visits. | Percentage of donor derived cells | MEDIAN |
| SECONDARY | Myeloid Chimerism | Day 28, 60, 100, 180, and year 1 and 2 post-transplant | Participants in RDEB: HCT plus MSC arm unable to be analyzed due to missed visits. | Percentage of donor derived cells | MEDIAN |
| SECONDARY | Average Change of a Patient's iscorEB | 2 year post-transplant | Participant in JEB: HCT plus MSC Arm B deceased at 2 year post-transplant timepoint. | units on a scale | MEAN |
Source ClinicalTrials.gov
Results by group48
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Event-free Survival | RDEB: HCT Plus MSC Arm B | 4 | Count of Participants | ||
| Event-free Survival | RDEB: HCT Plus MSC Arm E | 2 | Count of Participants | ||
| Event-free Survival | JEB: HCT Plus MSC Arm B | 1 | Count of Participants | ||
| Change of a Patient's iscorEB | RDEB: HCT Plus MSC Arm B | -10 | Full Range -10 to 112 | units on a scale | |
| Change of a Patient's iscorEB | RDEB: HCT Plus MSC Arm E | -24 | Full Range -24 to 112 | units on a scale | |
| Change of a Patient's iscorEB | JEB: HCT Plus MSC Arm B | 110 | Full Range 16 to 112 | units on a scale | |
| Transplant-related Mortality | RDEB: HCT Plus MSC Arm B | 0 | Participants | ||
| Transplant-related Mortality | RDEB: HCT Plus MSC Arm E | 0 | Participants | ||
| Transplant-related Mortality | JEB: HCT Plus MSC Arm B | 0 | Participants | ||
| Average Change in Quality of Life | RDEB: HCT Plus MSC Arm B | 10 | Inter-Quartile Range 10 to 100 | Change in score |
1 to 10 of 48Source ClinicalTrials.gov
See all 48 results by groupParticipant flow24
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | RDEB: HCT Plus MSC Arm B | 7 | |
| Overall Study | STARTED | RDEB: HCT Plus MSC Arm E | 9 | |
| Overall Study | STARTED | JEB: HCT Plus MSC Arm B | 1 | |
| Overall Study | STARTED | HCT With 300 cGy of TBI Arm A | 0 | |
| Overall Study | STARTED | Re-Transplant Arm C | 0 | |
| Overall Study | STARTED | HCT Arm D | 0 | |
| Overall Study | STARTED | HCT Alone Arm F | 0 | |
| Overall Study | STARTED | HCT Plus MSC Arm G | 0 | |
| Overall Study | COMPLETED | RDEB: HCT Plus MSC Arm B | 7 | |
| Overall Study | COMPLETED | RDEB: HCT Plus MSC Arm E | 9 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 participant flowAdverse events15
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 | Hepatobiliary disorders - Other, specify | Hepatobiliary disorders | RDEB: HCT Plus MSC Arm B | 0 | 7 | 0 |
| other | Hepatobiliary disorders - Other, specify | Hepatobiliary disorders | RDEB: HCT Plus MSC Arm E | 1 | 9 | 1 |
| other | Hepatobiliary disorders - Other, specify | Hepatobiliary disorders | JEB: HCT Plus MSC Arm B | 1 | 1 | 1 |
| other | Sepsis | Infections and infestations | RDEB: HCT Plus MSC Arm B | 1 | 7 | 1 |
| other | Sepsis | Infections and infestations | RDEB: HCT Plus MSC Arm E | 0 | 9 | 0 |
| other | Sepsis | Infections and infestations | JEB: HCT Plus MSC Arm B | 0 | 1 | 0 |
| other | Nervous system disorders - Other, specify | Nervous system disorders | RDEB: HCT Plus MSC Arm B | 1 | 7 | 1 |
| other | Nervous system disorders - Other, specify | Nervous system disorders | RDEB: HCT Plus MSC Arm E | 0 | 9 | 0 |
| other | Nervous system disorders - Other, specify | Nervous system disorders | JEB: HCT Plus MSC Arm B | 0 | 1 | 0 |
| serious | Other, specify - Veno-occlusive disease | Hepatobiliary disorders | RDEB: HCT Plus MSC Arm B | 2 | 7 | 2 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | RDEB: HCT Plus MSC Arm B | 0 | 7 |
| Total, all-cause mortality | RDEB: HCT Plus MSC Arm E | 1 | 9 |
| Total, all-cause mortality | JEB: HCT Plus MSC Arm B | 1 | 1 |
| Total, other adverse events | RDEB: HCT Plus MSC Arm B | 2 | 7 |
| Total, other adverse events | RDEB: HCT Plus MSC Arm E | 1 | 9 |
| Total, other adverse events | JEB: HCT Plus MSC Arm B | 1 | 1 |
| Total, serious adverse events | RDEB: HCT Plus MSC Arm B | 2 | 7 |
| Total, serious adverse events | RDEB: HCT Plus MSC Arm E | 1 | 9 |
| Total, serious adverse events | JEB: HCT Plus MSC Arm B | 0 | 1 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 30843184 | 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/NCT02582775
GET https://conductscience.org/api/trials/v1/NCT02582775/results?page=2