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NCT01033552 · ClinicalTrials.gov registry record · Phase 1
Biochemical Correction of Severe EB by Allo HSCT and "Off-the-shelf" MSCs
A Phase 1 study of Epidermolysis Bullosa, sponsored by Masonic Cancer Center, University of Minnesota.
- Completed
- Registry status
- Phase 1
- Development phase
- 32
- Enrollment target
- 1
- Study location
NCT01033552: Completed Phase 1 study of Epidermolysis Bullosa, sponsored by Masonic Cancer Center, University of Minnesota.
NCT01033552 is a Phase 1 study of Epidermolysis Bullosa that has completed, run by Masonic Cancer Center, University of Minnesota. The registered enrollment target is 32 participants, above the 17-participant average among 10 other Epidermolysis Bullosa trials with a reported enrollment target (88% higher). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01033552, a Phase 1 study of Epidermolysis Bullosa, has completed, sponsored by Masonic Cancer Center, University of Minnesota.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 32 participants
- Enrollment target
- 1
- Study location
Study Summary
This is an open-label, single institution, phase II study in patients with epidermolysis bullosa (EB). The underlying hypothesis is that the infusion of bone marrow or umbilical cord blood from a healthy unaffected donor will correct the collagen, laminin, integrin, or plakin deficiency and reduce the skin fragility characteristic of severe forms of EB. A secondary hypothesis is that mesenchymal stem cells from a healthy donor will enhance the safety and efficacy of the allogeneic hematopoietic stem cell transplant as well as serve as a source of renewable cells for the treatment of focal areas of residual blistering.
Primary Outcome
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.
Conditions Studied
Interventions
- DRUG Cyclophosphamide
- DRUG Fludarabine
- DRUG Anti-thymocyte globulin
- DRUG Myeloablative Busulfan
- PROCEDURE Mesenchymal stem cell transplantation
Study Locations (1)
Minnesota
- University of Minnesota Masonic Cancer Center and Medical Center - Minneapolis
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 32 participants |
| Start Date | 2010-01 |
| Est. Completion | 2021-08-12 |
| Phase | Phase 1 |
What the finished NCT01033552 record still lists
NCT01033552 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 32 participants, a relatively small participant target, above the 17-participant average among 10 other Epidermolysis Bullosa trials with a reported enrollment target (88% higher).
The record links to 1 condition, with Epidermolysis Bullosa appearing as the primary indexed condition, and to 5 interventions - of which Cyclophosphamide is the first listed.
NCT01033552 reports a single indexed study location in Minnesota.
Frequently Asked Questions
What is clinical trial NCT01033552 about?
NCT01033552 is a clinical study titled "Biochemical Correction of Severe EB by Allo HSCT and "Off-the-shelf" MSCs". This is an open-label, single institution, phase II study in patients with epidermolysis bullosa (EB). The underlying hypothesis is that the infusion of bone marrow or umbilical cord blood from a healthy unaffected donor will correct the collagen, laminin, integrin, or plakin deficiency and reduce t...
What is the current status of trial NCT01033552?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 32 participants. The study started on 2010-01. Estimated completion is 2021-08-12.
What conditions does trial NCT01033552 study?
This clinical trial studies the following conditions: Epidermolysis Bullosa.
What interventions are being tested in trial NCT01033552?
The interventions under investigation include: Cyclophosphamide (DRUG), Fludarabine (DRUG), Anti-thymocyte globulin (DRUG), Myeloablative Busulfan (DRUG), Mesenchymal stem cell transplantation (PROCEDURE).
Who is sponsoring clinical trial NCT01033552?
This trial is sponsored by Masonic Cancer Center, University of Minnesota, which has 249 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT01033552 being conducted?
This trial has 1 study location across Minnesota. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Epidermolysis Bullosa
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT01033552's enrollment target sits among peer trials
32 2nd of 10 the highest of 10 other Epidermolysis Bullosa trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Epidermolysis Bullosa trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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