Trial · NCT00478244
Allogeneic Hematopoietic Stem Cell Transplant For Epidermolysis Bullosa
NCT00478244, Not applicable, Terminated, Masonic Cancer Center, University of Minnesota; results first posted 2013-02-11
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Allogeneic Hematopoietic Stem Cell Transplant For Epidermolysis Bullosa. Trials, record NCT00478244. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00478244
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Allogeneic Hematopoietic Stem Cell Transplant For Epidermolysis Bullosa
- NCT number
- NCT00478244
- Status
- Terminated
- Phases
- Not applicable
- Enrollment
- 7 participants, actual
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Conditions
- Epidermolysis Bullosa
- Interventions
- busulfan, cyclophosphamide, fludarabine phosphate, hematopoietic bone marrow transplantation
- First posted
- 2007-05-24
- Last updated
- 2017-12-28
- Results first posted
- 2013-02-11
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Patients With Detectable Collagen Type VII | Day 100 Post Transplant | Number of patients with epidermolysis bullosa who had collagen type VII. Type VII collagen defects cause recessive dystrophic epidermolysis bullosa (RDEB), a blistering skin disorder often accompanied by epidermal cancers. |
| Secondary | Number of Patients With >70% Donor Chimerism | Days 21, 100, 180, 365 and 730 Post Transplant | Number of patients with donor chimerism - percentage of donor cells in the patient via the peripheral blood or bone marrow. |
| Secondary | Number of Patients With Transplant-Related Mortality | Day 180 Post Transplant | Number of patients who died due to complications of the transplant (includes all deaths without previous relapse or progression). |
| Secondary | Number of Patients With Platelet Engraftment | Day 180 Post Transplant | Number of patients with a platelet count \>5 x 10\^10 cells/liter for 3 consecutive measurements. |
| Secondary | Number of Patients With Acute Graft-Versus-Host Disease (GVHD) | Day 100 Post Transplant | Number of patients with GVHD. Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host. |
| Secondary | Number of Patients With Chronic Graft-Versus-Host Disease (cGVHD) | Day 365 Post Transplant | Number of patients with cGVHD; a severe long-term complication created by infusion of donor cells into a foreign host. |
| Secondary | Overall Survival | 1 year and 2 years Post Transplant | Survival is defined as the number of patients that were alive post transplant. |
| Secondary | Number of Patients With Donor Derived Cells in Skin | Day 90 Post Transplant | Number of patients who had donor skin chimerism - donor cells in the patient's epidermis (a state in bone marrow transplantation in which bone marrow and host cells exist compatibly without signs of graft-versus-host rejection disease). |
| Secondary | Number of Patients With Resistance to Blister Formation | Month 1 through Month 24 Inclusive | Resistance to Blister Formation demonstrated by response to negative pressure. |
| Secondary | Number of Patients With Neutrophil Engraftment | Day 42 Post Transplant | Number of patients with an absolute neutrophil count \>5 x 10\^8 cells/liter for 3 consecutive days. |
Source ClinicalTrials.gov
Outcomes10
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| SECONDARY | Number of Patients With >70% Donor Chimerism | Days 21, 100, 180, 365 and 730 Post Transplant | participants | NUMBER | |
| SECONDARY | Number of Patients With Transplant-Related Mortality | Day 180 Post Transplant | participants | NUMBER | |
| SECONDARY | Number of Patients With Platelet Engraftment | Day 180 Post Transplant | participants | NUMBER | |
| SECONDARY | Number of Patients With Acute Graft-Versus-Host Disease (GVHD) | Day 100 Post Transplant | participants | NUMBER | |
| SECONDARY | Number of Patients With Chronic Graft-Versus-Host Disease (cGVHD) | Day 365 Post Transplant | participants | NUMBER | |
| SECONDARY | Overall Survival | 1 year and 2 years Post Transplant | participants | NUMBER | |
| SECONDARY | Number of Patients With Donor Derived Cells in Skin | Day 90 Post Transplant | participants | NUMBER | |
| SECONDARY | Number of Patients With Resistance to Blister Formation | Month 1 through Month 24 Inclusive | Added blister formation testing later in study; only 2 patients had pre-transplant test. | participants | NUMBER |
| SECONDARY | Number of Patients With Neutrophil Engraftment | Day 42 Post Transplant | participants | NUMBER | |
| PRIMARY | Number of Patients With Detectable Collagen Type VII | Day 100 Post Transplant | participants | NUMBER |
Source ClinicalTrials.gov
Results by group15
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 >70% Donor Chimerism | Evaluable Patients | Day 21 | 6 | participants | |
| Number of Patients With >70% Donor Chimerism | Evaluable Patients | Day 100 | 5 | participants | |
| Number of Patients With >70% Donor Chimerism | Evaluable Patients | Day 180 | 5 | participants | |
| Number of Patients With >70% Donor Chimerism | Evaluable Patients | Day 365 | 5 | participants | |
| Number of Patients With >70% Donor Chimerism | Evaluable Patients | Day 730 | 5 | participants | |
| Number of Patients With Transplant-Related Mortality | Evaluable Patients | 0 | participants | ||
| Number of Patients With Platelet Engraftment | Evaluable Patients | 5 | participants | ||
| Number of Patients With Acute Graft-Versus-Host Disease (GVHD) | Evaluable Patients | 1 | participants | ||
| Number of Patients With Chronic Graft-Versus-Host Disease (cGVHD) | Evaluable Patients | 0 | participants | ||
| Overall Survival | Evaluable Patients | 1 Year Post Transplant | 5 | participants |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Epidermolysis Bullosa (EB) Patients | 7 | |
| Overall Study | COMPLETED | Epidermolysis Bullosa (EB) Patients | 6 | |
| Overall Study | NOT COMPLETED | Epidermolysis Bullosa (EB) Patients | 1 |
Source ClinicalTrials.gov
Adverse events10
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 |
|---|---|---|---|---|---|---|
| serious | Acute respiratory distress syndrome | Respiratory, thoracic and mediastinal disorders | Epidermolysis Bullosa (EB) Patients | 1 | 7 | 1 |
| serious | Bacterial infection | Infections and infestations | Epidermolysis Bullosa (EB) Patients | 6 | 7 | 6 |
| serious | Death | General disorders | Epidermolysis Bullosa (EB) Patients | 1 | 7 | 1 |
| serious | Fungal infection | Infections and infestations | Epidermolysis Bullosa (EB) Patients | 2 | 7 | 2 |
| serious | Grade IV skin toxicity | Skin and subcutaneous tissue disorders | Epidermolysis Bullosa (EB) Patients | 1 | 7 | 1 |
| serious | Graft failure | Blood and lymphatic system disorders | Epidermolysis Bullosa (EB) Patients | 1 | 7 | 1 |
| serious | Hemorrhagic cystitis | Renal and urinary disorders | Epidermolysis Bullosa (EB) Patients | 1 | 7 | 1 |
| serious | Hyperbilirubinemia | Metabolism and nutrition disorders | Epidermolysis Bullosa (EB) Patients | 4 | 7 | 4 |
| serious | Renal insufficiency | Renal and urinary disorders | Epidermolysis Bullosa (EB) Patients | 2 | 7 | 2 |
| serious | Viral infection | Infections and infestations | Epidermolysis Bullosa (EB) Patients | 1 | 7 | 4 |
Source ClinicalTrials.gov
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Epidermolysis Bullosa (EB) Patients | ||
| Total, other adverse events | Epidermolysis Bullosa (EB) Patients | 0 | 7 |
| Total, serious adverse events | Epidermolysis Bullosa (EB) Patients | 6 | 7 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 20818854 | 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/NCT00478244
GET https://conductscience.org/api/trials/v1/NCT00478244/registered