Epidermolysis Bullosa Dystrophica, RecessivePhase 1/2NCT04153630
Safety Study and Preliminary Efficacy of Infusion Haploidentical Mesenchymal Stem Cells Derived From Bone Marrow for Treating Recessive Dystrophic Epidermolysis Bullosa
A Phase 1/2 study testing mesenchymal stem cells derived from bone marrow (BM-MSCs) in people with Epidermolysis Bullosa Dystrophica, Recessive, run by Instituto de Investigación Hospital Universitario La Paz at 1 site in Spain.
Trial summary
Phase I / II pilot clinical trial, to evaluate the safety and preliminary efficacy of the systemic infusion of mesenchymal stem cells derived from bone marrow (BM-MSCs) from a haploidentical donor to improve the healing process and / or the mucocutaneous fragility phenotype associated with EBDR. Read more about this study
- Who is it for?
- Ages 12 months to 18 years
- Conditions the study is for
- Epidermolysis Bullosa Dystrophica, Recessive
- What is being tested?
- mesenchymal stem cells derived from bone marrow (BM-MSCs)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteSpain
- Study dates
- 17 May 2018to Mar 2021
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
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 12 months to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Male and female patients ≥ 12 months and ≤ 18 years of age at the time of inclusion in the study.
- Patients with a clinical, molecular and genetic diagnosis of EBDR.
- Patients with presence of the NC-1 domain of type VII collagen, in skin biopsies and/or Western-Blot, detected with a battery of specific antibodies.
- Patients with a haploidentical donor.
- Subjects with a severity score> 20 according to "The Birmingham Epidermolysis Bullosa Severity Score".
- Minor subjects whose representative / legal guardian has voluntarily signed the informed consent before the first intervention of the study.
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- In the case of mature minors (12-17 years of age), in addition to the consent signed by the legal guardian, the consent of the minor will be obtained.
- Women with reproductive capacity must have a negative pregnancy test at the time of inclusion and must agree to use highly effective contraceptive methods (diaphragms plus spermicide or male condom plus spermicide, oral contraceptive combined with a second method contraceptive implant, contraceptive injectable, permanent intrauterine device, sexual abstinence or partner with vasectomy) during their participation in the study until 30 days after the last visit.
- Males should agree to use a double-barrier contraceptive method (condom plus spermicide or diaphragm plus spermicide) during their participation in the study and up to 30 days after the last dose of the study drug, or the male patient or his Female partners must be surgically sterilized or the female partner must be postmenopausal.
- The patient must be able to attend all study visits and comply with all study procedures.
Who cannot join
- Subjects who for medical reasons can not be moved to the University Hospital La Paz in Madrid.
- Subjects who have received immunotherapy including oral corticosteroids (> 15 mg / day) for more than 1 week (excluding inhaled and ophthalmic preparations) or chemotherapy 8 days prior to inclusion in the study. The inclusion of the patient is understood from the signing of the informed consent.
- Subjects with a known allergy to any of the components of the investigational product (including penicillin and streptomycin), or who can not receive treatment with antihistamines and/or corticosteroids.
- Subjects with signs of active systemic infection at the time of inclusion in the study. In any case, according to the researcher's criteria, the patient can be reevaluated for inclusion in the study after improvement of the infectious pathology.
- Subjects with a history or signs of malignancy, including cutaneous squamous cell carcinoma.
- Subjects with circulating anti-C7 antibodies and anti-C7 antibodies deposited in the dermo-epidermal junction detected in skin biopsies by indirect immunofluorescence.
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- Pregnant women at the time of inclusion or women of childbearing age who do not practice abstinence or employ acceptable means of contraception, as determined by the investigator during the trial.
- Biochemical abnormalities at the time of inclusion: albumin <2.5 g / dL, Hemoglobin <7.5 g / dL.
- Subjects to whom other investigational drugs have been administered in the 90 days prior to the treatment phase.
- Subjects who are unable to understand the information sheet and unable to sign the informed consent.
Trial locations and contacts
1 site in 1 country.
About this study
Phase I / II pilot clinical trial, to evaluate the safety and preliminary efficacy of the systemic infusion of mesenchymal stem cells derived from bone marrow (BM-MSCs) from a haploidentical donor to improve the healing process and / or the mucocutaneous fragility phenotype associated with EBDR.
Read the detailed description
The Main Objective is to evaluate the safety and therapeutic efficacy of haploidentical MSCs derived from bone marrow administered by intravenous injection for the treatment of patients with RDBS. The assessment of the symptomatic improvement of the treated patients will be made regarding the baseline situation and the response to treatment at the biochemical, histological and molecular level.
Secondary Objectives:
Describe the clinical and molecular phenotype of the mucocutaneous involvement of patients, including the characterization of the mutations responsible for the disease.
Study drug: Allogenic mesenchymal cells (haploidentical) derived from bone marrow and expanded.
Method of administration: Systemic / Intravenous Administration dose: 2-3x10e6 BM-MSC / Kg. Weekly dose for three consecutive weeks
Follow-up period: 12 months after the infusion. However, patients will be monitored outside the clinical trial over a 5-year period
- Being tested
- mesenchymal stem cells derived from bone marrow (BM-MSCs)
- Medical subject headings
- Epidermolysis Bullosa Dystrophica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
mesenchymal stem cells derived from bone marrow (BM-MSCs)Biological
Procedure: Haploidentical MSCs derived from bone marrow administered by intravenous injection with a dose of 2-3x106 cells / Kg
| Study group | Role | What they receive |
|---|---|---|
| Haploidentical MSCs derived from bone marrow | Experimental | Haploidentical MSCs derived from bone marrow administered by intravenous injection with a dose of 2-3x106 cells / Kg Receives: Biological: mesenchymal stem cells derived from bone marrow (BM-MSCs) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety evaluation: Incidence of Treatment-Emergent Adverse Events as assessed by protocol.
To evaluate the safety of haploidentical MSCs derived from bone marrow administered by intravenous injection with a dose of 2-3x106 cells / Kg in 3 infusions separated by 21 days each for the treatment of patients with RDEB: All adverse events will be registered for 1 year from first infusion of cells as assessed by grade: mild, moderate or severe (according to protocol)
Measured: 1 year after infusion
Secondary outcomes (17)
Cutaneous mechanical resistance
Cutaneous mechanical resistance measured using a negative pressure cutaneous suction device from Electronic Diversities (NP-2 model).
Measured: 2 year after infusion
Skin surface affected
Percentage of skin surface affected in patients with RDEB
Measured: 2 year after infusion
Number of blisters
Quantification of the number of blisters in patient with RDEB
Measured: 2 year after infusion
Non-specific general markers of systemic inflammation: white blood cell count
Biomarkers evaluated are white blood cell count (109/L)
Measured: 2 year after infusion
Non-specific general markers of systemic inflammation: Negative acute phase reactant (albumin)
Biomarkers evaluated are negative acute phase reactants: albumin (g/dl)
Measured: 2 year after infusion
Non-specific general markers of systemic inflammation: Negative acute phase reactants (pre-albumin, transferrin and retinol-binding protein)
Biomarkers evaluated are negative acute phase reactants: pre-albumin, transferrin, and retinol-binding protein (mg/dl)
Measured: 2 year after infusion
Non-specific general markers of systemic inflammation: Positive acute phase reactants (c-reactive protein and fibrinogen)
Biomarkers evaluated are positive acute phase reactants: C reactive protein and fibrinogen (mg/dl)
Measured: 2 year after infusion
Non-specific general markers of systemic inflammation: Positive acute phase reactants (ferritin)
Biomarkers evaluated are positive acute phase reactants: ferritin (ng/ml)
Measured: 2 year after infusion
Severity index according to "The Birmingham Epidermolysis Bullosa Severity Score" before and after treatment
The Birmingham Epidermolysis Bullosa Severity Score evaluated the severity of disease before and after treatment. Eleven items were scored: area of damaged skin, involvement of nails, mouth, eyes, larynx and oesophagus, scarring of hands, skin cancer, chronic wounds, alopecia and nutritional compromise (giving a maximum score of 100, where 0 = better outcome and 100 = worse outcome)
Measured: 2 year after infusion
Severity index according to "The Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI) before and after treatment
The Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI) used to classify patients according to disease severity and clinical response in three types: mild, moderate and severe (obtained by the analysis of 12 skin sites in addition to the scalp, mucous membranes, nails and other epithelized surfaces - total activity (of 276) and damage (of 230) combine to give an overall score of 506; where 0 = better outcome and 506 = worse outcome).
Measured: 2 year after infusion
Expression of Collagen VII (C7) in skin biopsy
Expression of Collagen VII (C7) in skin biopsy will be analyzed by immunofluorescence with antibodies specific for the NC1 domain of collagen VII.
Measured: 2 year after infusion
Analysis of anchoring fibrils
Analysis of anchoring fibrils by electron microscopy in skin biopsy after treatment
Measured: 2 year after infusion
Variation of pain respect to baseline status: Visual Analog Scale
Variation of pain with respect to baseline that will be assessed using the Visual Analogy Scale (VAS) in all visits after the first infusion, used different type of VAS scale depends on age of subject. Range was 1 to 10, where 10 are considered a worse outcome.
Measured: 2 year after infusion
Modification in itching perceived
Assessment of the change in the itching perceived respect baseline status will be assessed by Leuven Itch Scale or Itch Man Scale, depends on the age of the subject. These scales are the instrument to evaluate itching perceived in patients with pruritus origin different. There subscale in 6 domains: frequency, duration, severity, distress, impact, and area: Itch frequency score: scale from 0 to 100. (Maximum score 100, itching all the time - is worse outcome) Itch severity score: scale from 0 to 100 (Maximum score 100, maximum severity =worse outcome) Itch distress score: scale from 0 to 100 (Maximum score 100, maximum distress = worse outcome) Itch consequences score: scale from 0 to 100 (Maximum score 100, maximum consequences = worse outcome) Itch Surface area score: Scale from 0 to 100 (Maximum score is 100, the entire body = worse outcome).
Measured: 2 year after infusion
Change in quality of life of patient: European Quality of Life-5 Dimensions-5 level (EUROQL-5D)
Assessment of the change in quality of life: survey of specific assessment of the quality of life (European Quality of Life-5 Dimensions-5 level; EUROQL-5D). The descriptive system comprises five dimensions: mobility, self-care, habitual activities, pain/discomfort and anxiety/depression. Each dimension has 5 levels of gravity, where a value of 1 is better outcome and 3 is worse outcome. Also, there are a 2nd part included a VAS scale, where 0 = worse outcome and 100 =better outcome.
Measured: 2 year after infusion
Evaluation of circulating anti-C7 antibodies
Analysis of circulating anti-C7 antibodies determined by ELISA before and after treatment.
Measured: 2 year after infusion
Change in general condition of the patient
Evaluation of the changes produced in the patient before and after the study treatment, assessing according to the set of tests performed on the patient during the same; indicating it through a Likert scale, from 1-4, where 4 is the most serious evaluation and 1 is the slightest.
Measured: 2 year after infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Nov 2019
- Study start17 May 2018
- Primary completionMar 2021 (estimated)
- Study completionMar 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Epidermolysis Bullosa Dystrophica, RecessiveStudies of the same condition, open and under way.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Instituto de Investigación Hospital Universitario La Paz
- Sponsor type
- University, hospital or other
- Collaborators
- Universidad Carlos III de Madrid, St John's Institute of Dermatology Kings College London, Instituto de Salud Carlos III, DEBRA, CIBER Enfermedades raras
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04153630
- Sponsor's protocol ID
- MesenSistem-EB
- Status last verified
- Apr 2019
- First submitted
- 6 Aug 2019
- Last updated
- 8 Nov 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Maseda R, Arriba MC, Martinez-Santamaria L, Jimenez E, Herraiz-Gil S, Illera N, Quintana-Castanedo L, Garcia M, Suarez-Sancho S, Yanez R, Perez-Conde I, Carretero M, Martinez-Queipo M, de Paz R, Leon C, Jimenez-Yuste V, Borobia AM, Vicente A, Lwin SM, McGrath JA, Fernandez-Santos ME, Butta N, Sacedon R, Del Rio M, de Lucas R, Escamez MJ. MesenSistem-EB: systemic haploidentical mesenchymal stem cell therapy in recessive dystrophic epidermolysis bullosa associated with clinical benefits and correlated with MCP1 and sCD40L dynamics. Front Immunol. 2026 May 5;17:1789537. doi: 10.3389/fimmu.2026.1789537. eCollection 2026.
PubMed 42164509
Study at a glance
From the registry record, last updated by the sponsor on 8 Nov 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety Study and Preliminary Efficacy of Infusion Haploidentical Mesenchymal Stem Cells Derived From Bone Marrow for Treating Recessive Dystrophic Epidermolysis Bullosa
- ClinicalTrials.gov ID
- NCT04153630
- Acronym / sponsor ID
- MesenSistem-EB
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Instituto de Investigación Hospital Universitario La PazUniversity, hospital or other
- Collaborators
- Universidad Carlos III de Madrid, St John's Institute of Dermatology Kings College London, Instituto de Salud Carlos III, DEBRA, CIBER Enfermedades raras
- Study start
- 17 May 2018 (actual)
- Primary completion
- Mar 2021 (estimated)
- Study completion
- Mar 2021 (estimated)
- First posted
- 6 Nov 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Phase I / II pilot clinical trial, to evaluate the safety and preliminary efficacy of the systemic infusion of mesenchymal stem cells derived from bone marrow (BM-MSCs) from a haploidentical donor to improve the healing process and / or the mucocutaneous fragility phenotype associated with EBDR.
Detailed description
The Main Objective is to evaluate the safety and therapeutic efficacy of haploidentical MSCs derived from bone marrow administered by intravenous injection for the treatment of patients with RDBS. The assessment of the symptomatic improvement of the treated patients will be made regarding the baseline situation and the response to treatment at the biochemical, histological and molecular level.
Secondary Objectives:
Describe the clinical and molecular phenotype of the mucocutaneous involvement of patients, including the characterization of the mutations responsible for the disease.
Study drug: Allogenic mesenchymal cells (haploidentical) derived from bone marrow and expanded.
Method of administration: Systemic / Intravenous Administration dose: 2-3x10e6 BM-MSC / Kg. Weekly dose for three consecutive weeks
Follow-up period: 12 months after the infusion. However, patients will be monitored outside the clinical trial over a 5-year period
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Haploidentical MSCs derived from bone marrow | Experimental | Biological: mesenchymal stem cells derived from bone marrow (BM-MSCs)Haploidentical MSCs derived from bone marrow administered by intravenous injection with a dose of 2-3x106 cells / Kg |
Who may qualify
- Ages
- 12 months to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (10 listed)
- Male and female patients ≥ 12 months and ≤ 18 years of age at the time of inclusion in the study.
- Patients with a clinical, molecular and genetic diagnosis of EBDR.
- Patients with presence of the NC-1 domain of type VII collagen, in skin biopsies and/or Western-Blot, detected with a battery of specific antibodies.
- Patients with a haploidentical donor.
- Subjects with a severity score> 20 according to "The Birmingham Epidermolysis Bullosa Severity Score".
Key exclusion criteria (10 listed)
- Subjects who for medical reasons can not be moved to the University Hospital La Paz in Madrid.
- Subjects who have received immunotherapy including oral corticosteroids (> 15 mg / day) for more than 1 week (excluding inhaled and ophthalmic preparations) or chemotherapy 8 days prior to inclusion in the study. The inclusion of the patient is understood from the signing of the informed consent.
- Subjects with a known allergy to any of the components of the investigational product (including penicillin and streptomycin), or who can not receive treatment with antihistamines and/or corticosteroids.
- Subjects with signs of active systemic infection at the time of inclusion in the study. In any case, according to the researcher's criteria, the patient can be reevaluated for inclusion in the study after improvement of the infectious pathology.
- Subjects with a history or signs of malignancy, including cutaneous squamous cell carcinoma.
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 |
|---|---|---|
| mesenchymal stem cells derived from bone marrow (BM-MSCs)Procedure: Haploidentical MSCs derived from bone marrow administered by intravenous injection with a dose of 2-3x106 cells / Kg | Biological | Haploidentical MSCs derived from bone marrow |
Primary outcome
Safety evaluation: Incidence of Treatment-Emergent Adverse Events as assessed by protocol.
Time frame: 1 year after infusion
To evaluate the safety of haploidentical MSCs derived from bone marrow administered by intravenous injection with a dose of 2-3x106 cells / Kg in 3 infusions separated by 21 days each for the treatment of patients with RDEB: All adverse events will be registered for 1 year from first infusion of cells as assessed by grade: mild, moderate or severe (according to protocol)
17 secondary outcomes
Cutaneous mechanical resistance
Time frame: 2 year after infusion
Skin surface affected
Time frame: 2 year after infusion
Number of blisters
Time frame: 2 year after infusion
Non-specific general markers of systemic inflammation: white blood cell count
Time frame: 2 year after infusion
Non-specific general markers of systemic inflammation: Negative acute phase reactant (albumin)
Time frame: 2 year after infusion
Non-specific general markers of systemic inflammation: Negative acute phase reactants (pre-albumin, transferrin and retinol-binding protein)
Time frame: 2 year after infusion
Non-specific general markers of systemic inflammation: Positive acute phase reactants (c-reactive protein and fibrinogen)
Time frame: 2 year after infusion
Non-specific general markers of systemic inflammation: Positive acute phase reactants (ferritin)
Time frame: 2 year after infusion
Severity index according to "The Birmingham Epidermolysis Bullosa Severity Score" before and after treatment
Time frame: 2 year after infusion
Severity index according to "The Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI) before and after treatment
Time frame: 2 year after infusion
Expression of Collagen VII (C7) in skin biopsy
Time frame: 2 year after infusion
Analysis of anchoring fibrils
Time frame: 2 year after infusion
Variation of pain respect to baseline status: Visual Analog Scale
Time frame: 2 year after infusion
Modification in itching perceived
Time frame: 2 year after infusion
Change in quality of life of patient: European Quality of Life-5 Dimensions-5 level (EUROQL-5D)
Time frame: 2 year after infusion
Evaluation of circulating anti-C7 antibodies
Time frame: 2 year after infusion
Change in general condition of the patient
Time frame: 2 year after infusion
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Nov 2019.
- Derived from this study: PubMed 42164509Maseda R, Arriba MC, Martinez-Santamaria L, Jimenez E, Herraiz-Gil S, Illera N, Quintana-Castanedo L, Garcia M, Suarez-Sancho S, Yanez R, Perez-Conde I, Carretero M, Martinez-Queipo M, de Paz R, Leon C, Jimenez-Yuste V, Borobia AM, Vicente A, Lwin SM, McGrath JA, Fernandez-Santos ME, Butta N, Sacedon R, Del Rio M, de Lucas R, Escamez MJ. MesenSistem-EB: systemic haploidentical mesenchymal stem cell therapy in recessive dystrophic epidermolysis bullosa associated with clinical benefits and correlated with MCP1 and sCD40L dynamics. Front Immunol. 2026 May 5;17:1789537. doi: 10.3389/fimmu.2026.1789537. eCollection 2026.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing mesenchymal stem cells derived from bone marrow (BM-MSCs) in people with Epidermolysis Bullosa Dystrophica, Recessive, run by Instituto de Investigación Hospital Universitario La Paz at 1 site in Spain.
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 10 inclusion and 10 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: status not confirmed, as of the sponsor's last update on 8 Nov 2019. 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 Spain. 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Results are not available in the source record.
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?
Safety evaluation: Incidence of Treatment-Emergent Adverse Events as assessed by protocol. (time frame: 1 year after infusion). 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 NCT04153630, 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 8 Nov 2019.