Epidermolysis BullosaPhase 2NCT02582775
MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCs
A Phase 2 study testing Thymoglobulin, Cyclophosphamide and Fludarabine in people with Epidermolysis Bullosa, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
Trial summary
This is a single-institution, phase II study to determine the event-free survival at 1 year post allogeneic transplant and serial mesenchymal stem cell (MSC) infusions from a related donor (HLA identical, mismatched or haploidentical) or matched unrelated donor for the biochemical correction of severe epidermolysis… Read more about this study
- Who is it for?
- Ages Up to 25 years
- Conditions the study is for
- Epidermolysis Bullosa
- What is being tested?
- Thymoglobulin, Cyclophosphamide and Fludarabine
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 17 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2016to 26 Jul 2023
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis of severe form of EB characterized by collagen, laminin, integrin, keratin or plakin deficiency (by immunofluorescence staining with protein specific antibodies or Western blotting and by mutation analysis).
- Adequate organ function within 4 weeks of study registration defined as:
- Renal: glomerular filtration rate within normal range for age
- Hepatic: Hepatic: bilirubin, AST/ALT, ALP < 5 x upper limit of normal
- Pulmonary: adequate pulmonary function in the opinion of the enrolling investigator
- Cardiac: left ventricular ejection fraction ≥ 45%, normal EKG or approved by Cardiology for transplant
Show all 9Show fewer
- Sexually active participants must agree to use adequate birth control for the during the study period (from before the start of the preparative chemotherapy through 1 year post-transplant)
- Available donor per section 5: targeted MFI < 1,000 (MFI exceeding 1000 must be approved by the PI and treatment team.)
- Voluntary written consent - adult or parent (with information sheet for minors, if applicable) prior to any research related procedures or treatment
Who cannot join
- beta 3 laminin JEB mutants
- Active untreated systemic infection at time of transplantation (including active infection with Aspergillus or other mold within 30 days)
- History of HIV infection
- Evidence of squamous cell carcinoma
- Pregnant or breast feeding. Females of child-bearing potential must have a negative pregnancy test prior to study registration as the agents administered in this study are Pregnancy Category C and D.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Minnesota Masonic Cancer Center and Medical Center
Minneapolis, Minnesota, 55455
About this study
Official title: MT2015-20: Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Cell Transplantation and Serial Donor Mesenchymal Cell Infusions
This is a single-institution, phase II study to determine the event-free survival at 1 year post allogeneic transplant and serial mesenchymal stem cell (MSC) infusions from a related donor (HLA identical, mismatched or haploidentical) or matched unrelated donor for the biochemical correction of severe epidermolysis bullosa (EB).
- Conditions
- Epidermolysis Bullosa
- Being tested
- Thymoglobulin, Cyclophosphamide, Fludarabine, Total Body Irradiation, Bone marrow infusion
- Medical subject headings
- Epidermolysis Bullosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ThymoglobulinDrug
Also called ATG, anti-thymocyte globulin
0.5 mg/kg IV over 6 hours on day -9 and 2 mg/kg IV over 4 hours on day -8 and day -7 with premeds and solumedrol through day -2
CyclophosphamideDrug
Also called Cytoxan
14.5 mg/kg IV over 1 hour day -6 and -5 50 mg/kg IV over 2 hours with mesna 40 mg/kg IV day +2 and +3
FludarabineDrug
Also called fludarabine phosphate, Fludara
30 mg/m2 IV over 60 minutes days -6 through day -2
Total Body IrradiationRadiation
Also called TBI
See arm description for dosing.
Bone marrow infusionProcedure or surgery
Also called HCT
Bone marrow infusion on Day 0
TacrolimusDrug
Also called Prograf
Day +5 through day +100 with goals of 5-10 ug/L (not used for HLA-identical related donors). When used in non-MSD recipients, tapered over 6-8 weeks starting at day +100.
Also called MMF
15 mg/kg IV q8h (based upon actual body weight) with the maximum total daily dose not to exceed 3 grams. Day +5 through day 35
Donor mesenchymal stem cell infusionsBiological
Day 60, 100 and 180 (collected during donor BM harvest for graft)
BusulfanDrug
busulfan IV over 3 hours on days -3 and -2 for HLA-mismatched BM recipients only (Arms F and G)
| Study group | Role | What they receive |
|---|---|---|
| RDEB: HCT plus MSC Arm B | Experimental | Recessive dominant epidermolysis bullosa (RDEB) patients treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (300 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC). Receives: Drug: Thymoglobulin; Drug: Cyclophosphamide; Drug: Fludarabine; Radiation: Total Body Irradiation; Procedure: Bone marrow infusion; Drug: Tacrolimus; Drug: Mycophenolate Mofetil; Biological: Donor mesenchymal stem cell infusions |
| JEB: HCT plus MSC Arm B | Experimental | Junctional epidermolysis bullosa (JEB) patient treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (300 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC). Receives: Drug: Thymoglobulin; Drug: Cyclophosphamide; Drug: Fludarabine; Radiation: Total Body Irradiation; Procedure: Bone marrow infusion; Drug: Tacrolimus; Drug: Mycophenolate Mofetil; Biological: Donor mesenchymal stem cell infusions |
| RDEB: HCT Plus MSC Arm E | Experimental | Recessive dominant epidermolysis bullosa (RDEB) patients treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (400 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC). Receives: Drug: Thymoglobulin; Drug: Cyclophosphamide; Drug: Fludarabine; Radiation: Total Body Irradiation; Procedure: Bone marrow infusion; Drug: Tacrolimus; Drug: Mycophenolate Mofetil; Biological: Donor mesenchymal stem cell infusions |
| HCT with 300 cGy of TBI Arm A | Experimental | Epidermolysis bullosa patients treated per study regimen with chemotherapy and stem cell transplant without mesenchymal stem cell infusions. Receives: Drug: Thymoglobulin; Drug: Cyclophosphamide; Drug: Fludarabine; Radiation: Total Body Irradiation; Procedure: Bone marrow infusion; Drug: Tacrolimus; Drug: Mycophenolate Mofetil; Biological: Donor mesenchymal stem cell infusions |
| Re-Transplant Arm C | Experimental | Epidermolysis bullosa patients treated regardless of original transplant arm with re-transplant using 300 cGy of TBI. Receives: Drug: Thymoglobulin; Drug: Cyclophosphamide; Drug: Fludarabine; Radiation: Total Body Irradiation; Procedure: Bone marrow infusion; Drug: Tacrolimus; Drug: Mycophenolate Mofetil; Biological: Donor mesenchymal stem cell infusions |
| HCT Arm D | Experimental | HLA-matched epidermolysis bullosa patients treated with hematopoietic cell transplant alone using 200 cGY BID of TBI (400 cGy total). Receives: Drug: Thymoglobulin; Drug: Cyclophosphamide; Drug: Fludarabine; Radiation: Total Body Irradiation; Procedure: Bone marrow infusion; Drug: Tacrolimus; Drug: Mycophenolate Mofetil; Biological: Donor mesenchymal stem cell infusions |
| HCT Alone Arm F | Experimental | HLA-mismatched epidermolysis bullosa patients treated with hematopoietic cell transplant alone using 200 cGy BID of TBI (400 cGy total) + addition of low dose busulfan for recipients of HLA-mismatched bone marrow Receives: Drug: Thymoglobulin; Drug: Cyclophosphamide; Drug: Fludarabine; Radiation: Total Body Irradiation; Procedure: Bone marrow infusion; Drug: Tacrolimus; Drug: Mycophenolate Mofetil; Biological: Donor mesenchymal stem cell infusions; Drug: Busulfan |
| HCT plus MSC Arm G | Experimental | HLA-mismatched epidermolysis bullosa patients treated with hematopoietic cell transplant plus serial MSC infusions using 200 cGy BID of TBI (400 cGy total) + addition of low dose busulfan for recipients of HLA-mismatched bone marrow Receives: Drug: Thymoglobulin; Drug: Cyclophosphamide; Drug: Fludarabine; Radiation: Total Body Irradiation; Procedure: Bone marrow infusion; Drug: Tacrolimus; Drug: Mycophenolate Mofetil; Biological: Donor mesenchymal stem cell infusions; Drug: Busulfan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Event-free Survival
An event defined as death or a 50% increase in a patient's IScoreEB from baseline
Measured: 1 year post-transplant
Secondary outcomes (7)
Change of a Patient's iscorEB
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.
Measured: 1 year post-transplant
Transplant-related Mortality
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.
Measured: 180 days post-transplant
Average Change in Quality of Life
Measured by the Lansky or Karnofsky score (10-100). Higher scores indicate a better outcome. Midpoint of change in scores used to indicate median.
Measured: 1 year post-transplant
Average Change in Quality of Life
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.
Measured: 2 years post-transplant
Lymphoid Chimerism
Median hematopoietic cells that are of donor origin per participant.
Measured: Day 28, 60, 100, 180, and year 1 and 2 post-transplant
Myeloid Chimerism
Median hematopoietic cells that are of donor origin per participant.
Measured: Day 28, 60, 100, 180, and year 1 and 2 post-transplant
Average Change of a Patient's iscorEB
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.
Measured: 2 year post-transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Oct 2015 (estimated)
- Study startMar 2016
- Primary completion15 Nov 2022
- Study completion26 Jul 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Epidermolysis BullosaStudies of the same condition, open and under way.
- More trials run by Masonic Cancer Center, University of MinnesotaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jakub Tolar, MD, PhD, Principal investigator, Masonic Cancer Center, University of Minnesota
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02582775
- Sponsor's protocol ID
- 2015LS076
- Status last verified
- Aug 2024
- First submitted
- 16 Oct 2015
- Last updated
- 19 Sep 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Ebens CL, McGrath JA, Tamai K, Hovnanian A, Wagner JE, Riddle MJ, Keene DR, DeFor TE, Tryon R, Chen M, Woodley DT, Hook K, Tolar J. Bone marrow transplant with post-transplant cyclophosphamide for recessive dystrophic epidermolysis bullosa expands the related donor pool and permits tolerance of nonhaematopoietic cellular grafts. Br J Dermatol. 2019 Dec;181(6):1238-1246. doi: 10.1111/bjd.17858. Epub 2019 Jun 28.
PubMed 30843184
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- MT2015-20: Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Cell Transplantation and Serial Donor Mesenchymal Cell Infusions
- ClinicalTrials.gov ID
- NCT02582775
- Acronym / sponsor ID
- 2015LS076
- Conditions
- Epidermolysis Bullosa
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Masonic Cancer Center, University of MinnesotaUniversity, hospital or other
- Study start
- Mar 2016 (actual)
- Primary completion
- 15 Nov 2022 (actual)
- Study completion
- 26 Jul 2023 (actual)
- First posted
- 21 Oct 2015 (estimated)
- Results
- Posted 19 Sep 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a single-institution, phase II study to determine the event-free survival at 1 year post allogeneic transplant and serial mesenchymal stem cell (MSC) infusions from a related donor (HLA identical, mismatched or haploidentical) or matched unrelated donor for the biochemical correction of severe epidermolysis bullosa (EB).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 8
| Arm | Type | Receives |
|---|---|---|
| RDEB: HCT plus MSC Arm B | Experimental | Drug: Thymoglobulin, Drug: Cyclophosphamide, Drug: Fludarabine, Radiation: Total Body Irradiation, Procedure: Bone marrow infusion, Drug: Tacrolimus, Drug: Mycophenolate Mofetil, Biological: Donor mesenchymal stem cell infusionsRecessive dominant epidermolysis bullosa (RDEB) patients treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (300 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC). |
| JEB: HCT plus MSC Arm B | Experimental | Drug: Thymoglobulin, Drug: Cyclophosphamide, Drug: Fludarabine, Radiation: Total Body Irradiation, Procedure: Bone marrow infusion, Drug: Tacrolimus, Drug: Mycophenolate Mofetil, Biological: Donor mesenchymal stem cell infusionsJunctional epidermolysis bullosa (JEB) patient treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (300 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC). |
| RDEB: HCT Plus MSC Arm E | Experimental | Drug: Thymoglobulin, Drug: Cyclophosphamide, Drug: Fludarabine, Radiation: Total Body Irradiation, Procedure: Bone marrow infusion, Drug: Tacrolimus, Drug: Mycophenolate Mofetil, Biological: Donor mesenchymal stem cell infusionsRecessive dominant epidermolysis bullosa (RDEB) patients treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (400 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC). |
| HCT with 300 cGy of TBI Arm A | Experimental | Drug: Thymoglobulin, Drug: Cyclophosphamide, Drug: Fludarabine, Radiation: Total Body Irradiation, Procedure: Bone marrow infusion, Drug: Tacrolimus, Drug: Mycophenolate Mofetil, Biological: Donor mesenchymal stem cell infusionsEpidermolysis bullosa patients treated per study regimen with chemotherapy and stem cell transplant without mesenchymal stem cell infusions. |
| Re-Transplant Arm C | Experimental | Drug: Thymoglobulin, Drug: Cyclophosphamide, Drug: Fludarabine, Radiation: Total Body Irradiation, Procedure: Bone marrow infusion, Drug: Tacrolimus, Drug: Mycophenolate Mofetil, Biological: Donor mesenchymal stem cell infusionsEpidermolysis bullosa patients treated regardless of original transplant arm with re-transplant using 300 cGy of TBI. |
| HCT Arm D | Experimental | Drug: Thymoglobulin, Drug: Cyclophosphamide, Drug: Fludarabine, Radiation: Total Body Irradiation, Procedure: Bone marrow infusion, Drug: Tacrolimus, Drug: Mycophenolate Mofetil, Biological: Donor mesenchymal stem cell infusionsHLA-matched epidermolysis bullosa patients treated with hematopoietic cell transplant alone using 200 cGY BID of TBI (400 cGy total). |
| HCT Alone Arm F | Experimental | Drug: Thymoglobulin, Drug: Cyclophosphamide, Drug: Fludarabine, Radiation: Total Body Irradiation, Procedure: Bone marrow infusion, Drug: Tacrolimus, Drug: Mycophenolate Mofetil, Biological: Donor mesenchymal stem cell infusions, Drug: BusulfanHLA-mismatched epidermolysis bullosa patients treated with hematopoietic cell transplant alone using 200 cGy BID of TBI (400 cGy total) + addition of low dose busulfan for recipients of HLA-mismatched bone marrow |
| HCT plus MSC Arm G | Experimental | Drug: Thymoglobulin, Drug: Cyclophosphamide, Drug: Fludarabine, Radiation: Total Body Irradiation, Procedure: Bone marrow infusion, Drug: Tacrolimus, Drug: Mycophenolate Mofetil, Biological: Donor mesenchymal stem cell infusions, Drug: BusulfanHLA-mismatched epidermolysis bullosa patients treated with hematopoietic cell transplant plus serial MSC infusions using 200 cGy BID of TBI (400 cGy total) + addition of low dose busulfan for recipients of HLA-mismatched bone marrow |
Who may qualify
- Ages
- Up to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (9 listed)
- Diagnosis of severe form of EB characterized by collagen, laminin, integrin, keratin or plakin deficiency (by immunofluorescence staining with protein specific antibodies or Western blotting and by mutation analysis).
- Adequate organ function within 4 weeks of study registration defined as:
- Sexually active participants must agree to use adequate birth control for the during the study period (from before the start of the preparative chemotherapy through 1 year post-transplant)
- Available donor per section 5: targeted MFI < 1,000 (MFI exceeding 1000 must be approved by the PI and treatment team.)
- Voluntary written consent - adult or parent (with information sheet for minors, if applicable) prior to any research related procedures or treatment
Key exclusion criteria (5 listed)
- beta 3 laminin JEB mutants
- Active untreated systemic infection at time of transplantation (including active infection with Aspergillus or other mold within 30 days)
- History of HIV infection
- Evidence of squamous cell carcinoma
- Pregnant or breast feeding. Females of child-bearing potential must have a negative pregnancy test prior to study registration as the agents administered in this study are Pregnancy Category C and D.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ThymoglobulinAlso: ATG, anti-thymocyte globulin0.5 mg/kg IV over 6 hours on day -9 and 2 mg/kg IV over 4 hours on day -8 and day -7 with premeds and solumedrol through day -2 | Drug | HCT Alone Arm F, HCT Arm D, HCT plus MSC Arm G, HCT with 300 cGy of TBI Arm A, JEB: HCT plus MSC Arm B, RDEB: HCT Plus MSC Arm E, RDEB: HCT plus MSC Arm B, Re-Transplant Arm C |
| CyclophosphamideAlso: Cytoxan14.5 mg/kg IV over 1 hour day -6 and -5 50 mg/kg IV over 2 hours with mesna 40 mg/kg IV day +2 and +3 | Drug | HCT Alone Arm F, HCT Arm D, HCT plus MSC Arm G, HCT with 300 cGy of TBI Arm A, JEB: HCT plus MSC Arm B, RDEB: HCT Plus MSC Arm E, RDEB: HCT plus MSC Arm B, Re-Transplant Arm C |
| FludarabineAlso: fludarabine phosphate, Fludara30 mg/m2 IV over 60 minutes days -6 through day -2 | Drug | HCT Alone Arm F, HCT Arm D, HCT plus MSC Arm G, HCT with 300 cGy of TBI Arm A, JEB: HCT plus MSC Arm B, RDEB: HCT Plus MSC Arm E, RDEB: HCT plus MSC Arm B, Re-Transplant Arm C |
| Total Body IrradiationAlso: TBISee arm description for dosing. | Radiation | HCT Alone Arm F, HCT Arm D, HCT plus MSC Arm G, HCT with 300 cGy of TBI Arm A, JEB: HCT plus MSC Arm B, RDEB: HCT Plus MSC Arm E, RDEB: HCT plus MSC Arm B, Re-Transplant Arm C |
| Bone marrow infusionAlso: HCTBone marrow infusion on Day 0 | Procedure or surgery | HCT Alone Arm F, HCT Arm D, HCT plus MSC Arm G, HCT with 300 cGy of TBI Arm A, JEB: HCT plus MSC Arm B, RDEB: HCT Plus MSC Arm E, RDEB: HCT plus MSC Arm B, Re-Transplant Arm C |
| TacrolimusAlso: PrografDay +5 through day +100 with goals of 5-10 ug/L (not used for HLA-identical related donors). When used in non-MSD recipients, tapered over 6-8 weeks starting at day +100. | Drug | HCT Alone Arm F, HCT Arm D, HCT plus MSC Arm G, HCT with 300 cGy of TBI Arm A, JEB: HCT plus MSC Arm B, RDEB: HCT Plus MSC Arm E, RDEB: HCT plus MSC Arm B, Re-Transplant Arm C |
| Mycophenolate MofetilAlso: MMF15 mg/kg IV q8h (based upon actual body weight) with the maximum total daily dose not to exceed 3 grams. Day +5 through day 35 | Drug | HCT Alone Arm F, HCT Arm D, HCT plus MSC Arm G, HCT with 300 cGy of TBI Arm A, JEB: HCT plus MSC Arm B, RDEB: HCT Plus MSC Arm E, RDEB: HCT plus MSC Arm B, Re-Transplant Arm C |
| Donor mesenchymal stem cell infusionsDay 60, 100 and 180 (collected during donor BM harvest for graft) | Biological | HCT Alone Arm F, HCT Arm D, HCT plus MSC Arm G, HCT with 300 cGy of TBI Arm A, JEB: HCT plus MSC Arm B, RDEB: HCT Plus MSC Arm E, RDEB: HCT plus MSC Arm B, Re-Transplant Arm C |
| Busulfanbusulfan IV over 3 hours on days -3 and -2 for HLA-mismatched BM recipients only (Arms F and G) | Drug | HCT Alone Arm F, HCT plus MSC Arm G |
Primary outcome
Event-free Survival
Time frame: 1 year post-transplant
An event defined as death or a 50% increase in a patient's IScoreEB from baseline
7 secondary outcomes
Change of a Patient's iscorEB
Time frame: 1 year post-transplant
Transplant-related Mortality
Time frame: 180 days post-transplant
Average Change in Quality of Life
Time frame: 1 year post-transplant
Average Change in Quality of Life
Time frame: 2 years post-transplant
Lymphoid Chimerism
Time frame: Day 28, 60, 100, 180, and year 1 and 2 post-transplant
Myeloid Chimerism
Time frame: Day 28, 60, 100, 180, and year 1 and 2 post-transplant
Average Change of a Patient's iscorEB
Time frame: 2 year post-transplant
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 30843184Ebens CL, McGrath JA, Tamai K, Hovnanian A, Wagner JE, Riddle MJ, Keene DR, DeFor TE, Tryon R, Chen M, Woodley DT, Hook K, Tolar J. Bone marrow transplant with post-transplant cyclophosphamide for recessive dystrophic epidermolysis bullosa expands the related donor pool and permits tolerance of nonhaematopoietic cellular grafts. Br J Dermatol. 2019 Dec;181(6):1238-1246. doi: 10.1111/bjd.17858. Epub 2019 Jun 28.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jakub Tolar, MD, PhD principal investigator
Masonic Cancer Center, University of Minnesota
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Thymoglobulin, Cyclophosphamide and Fludarabine in people with Epidermolysis Bullosa, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 19 Sep 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Event-free Survival (time frame: 1 year post-transplant). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02582775, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Sep 2024.