Epidermolysis Bullosa Dystrophica, RecessivePhase 1/2EBGraftNCT04186650
Ex Vivo Gene Therapy Clinical Trial for RDEB Using Genetically Corrected Autologous Skin Equivalent Grafts
A Phase 1/2 study testing COL7A1-SIN retroviral vector engineered autologous tissue-engineered skin in people with Epidermolysis Bullosa Dystrophica, Recessive, run by Institut National de la Santé Et de la Recherche Médicale, France at 1 site in France.
Trial summary
This phase I/II clinical trial aims to treat 3 adult subjects with Recessive Dystrophic Epidermolysis Bullosa, expressing residual C7 levels, by genetically corrected autologous skin equivalent grafts on selected areas (up to 300 cm2). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Epidermolysis Bullosa Dystrophica, Recessive
- What is being tested?
- COL7A1-SIN retroviral vector engineered autologous tissue-engineered skin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteFrance
- Study dates
- 10 Jan 2020to 9 Jun 2027
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Clinical and molecular diagnosis of RDEB with confirmed bi-allelic COL7A1 mutations
- Reduced staining of C7 on skin biopsy, measured by immunofluorescence microscopy (IF)
- A reduced number of/or morphologically abnormal anchoring fibrils confirmed by TEM
- Detection of non-collagenous-1 domain (NC-1) of C7 on skin biopsy, measured by immunofluorescence microscopy (IF) and/or Western blot (WB) analysis
- Presence of ≥100cm2 of blistered and/or erosive skin areas including chronic wounds suitable for skin grafting
- Ability to undergo anaesthesia for skin grafting procedures
- Subjects aged 18 years, willing and able to give informed consent
Who cannot join
- Recipients of other investigational medicinal products within 6 months prior to enrolment into this study
- Past medical history of biopsy proven skin malignancy
- Immunotherapy including oral corticosteroids (Prednisolone >1mg/kg) for more than one week (intranasal and topical preparations are permitted) or chemotherapy within 60 days of enrolment into this study
- Known allergy to any of the constituents of the investigational medicinal product (IMP) including Penicillin
- Subjects with BOTH:
- positive serum antibodies to C7 confirmed by ELISA and
Show all 12Show fewer
- positive IIF with binding to the base of salt split skin and/or
- positive Western blot
- Positive results for HIV, Hepatitis BsAg, Hepatitis BcAb, Hepatitis C IgG, HTLV1\&2 or Syphilis serology
- Clinically significant medical, psychological or laboratory abnormalities limiting the ability of the subject to travel to the trial site(s) and to undergo grafting and follow-up procedures, as determined by the Investigator
- Absence of adequate social support
- Subjects who are pregnant, breast-feeding or of child-bearing potential who are neither abstinent nor practicing an acceptable means of contraception when this is in line with the usual and preferred lifestyle of the subject, as determined by the Investigator, for the duration of the trial
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase I/II ex Vivo Gene Therapy Clinical Trial for RDEB Using Autologous Skin Equivalent Grafts Genetically Corrected With a COL7A1-encoding SIN Retroviral Vector
This phase I/II clinical trial aims to treat 3 adult subjects with Recessive Dystrophic Epidermolysis Bullosa, expressing residual C7 levels, by genetically corrected autologous skin equivalent grafts on selected areas (up to 300 cm2).
Read the detailed description
Recessive Dystrophic Epidermolysis Bullosa (RDEB) is a severe orphan genetic disease responsible for skin and mucosal detachments due to a loss of adhesion of the epidermis to the underlying dermis. The disease is caused by loss of function mutations of the COL7A1 encoding type VII collagen (C7) which forms anchoring fibers, which are essential structures for dermal-epidermal adherence. Current treatments are only symptomatic and do not effectively treat or prevent the occurrence of cutaneous and mucosal detachments responsible for local and systemic complications that threaten the vital prognosis.
EBGRAFT is a prospective open-label international monocentric phase I/II clinical trial. It aims to treat 3 adult subjects with RDEB, expressing residual C7 levels, by genetically corrected autologous skin equivalent grafts.
The skin equivalent consists of keratinocytes and fibroblasts from the patient, genetically corrected ex vivo with a secure Self INactivating (SIN) retroviral vector expressing the COL7A1 cDNA under the control of the ubiquitous human promoter EF1a.
Each patient will be grafted sequentially at Necker Hospital in Paris using autologous genetically corrected skin equivalents of approximately 300 cm2 (up to 6 grafts of 50 cm2 each).
The main objective is to evaluate the safety of autologous skin equivalent grafts genetically corrected with a SIN COL7A1 retroviral vector (RV) in adults with RDEB.
The secondary objectives are:
- To evaluate the efficacy of transplanting autologous skin equivalent genetically corrected with RV SIN COL7A1 in adults with RDEB.
- To evaluate the immune response against recombinant type VII collagen (C7).
This clinical trial should evaluate whether the grafting of these genetically corrected autologous skin equivalents is well tolerated and whether they restore normal dermal-epidermal adherence of the grafted areas. The proposed treatment aims to obtain a permanent correction of the grafted areas, allowing skin healing and reducing pain. It has the potential to reduce itching, to prevent the occurrence of blisters and skin detachments, reduce the risk of infections, the duration and cost of care and also the risk of development of squamous cell carcinomas in the grafted areas.
- Being tested
- COL7A1-SIN retroviral vector engineered autologous tissue-engineered skin
- Keywords
- Genetic Therapy, Autologous Skin Graft, SIN retroviral vector, Type VII collagen, COL7A1
- 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.
COL7A1-SIN retroviral vector engineered autologous tissue-engineered skinBiological
Graft of SIN RV-mediated COL7A1 gene-modified autologous skin equivalent
| Study group | Role | What they receive |
|---|---|---|
| Autologous genetically modified tissue-engineered skin graft | Experimental | Graft of SIN RV-mediated COL7A1 gene-modified autologous skin equivalent Receives: Biological: COL7A1-SIN retroviral vector engineered autologous tissue-engineered skin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of grafting SIN RV-mediated COL7A1 gene-modified autologous skin equivalent: Adverse Events (AE), Serious Adverse Events (SAEs), Adverse Reactions (ARs) and Serious Adverse Reactions (SARs)
The primary objective is to evaluate the safety of autologous autologous skin equivalent grafts genetically corrected with a SIN COL7A1 retroviral vector (RV) in adults with RDEB
Primary Endpoints:
Record of Adverse Events (AE), Serious Adverse Events (SAEs), Adverse Reactions (ARs) and Serious Adverse Reactions (SARs).
Measured: Month 12 post grafting.
Secondary outcomes (13)
Change in C7 protein expression
Skin biopsy analysis of grafted skin compared to baseline for :
C7 protein expression by immunofluorescence microscopy (IF) using several specific antibodies
Measured: Month 1, Month 3, Month 6, Month 12 post grafting.
Change in anchoring fibrils number
Count of anchoring fibril (AF) at the dermal-epidermal junction (DEJ) by transmission electron microscopy (TEM) will be performed on skin biopsies and
Measured: Month 1, Month 3, Month 6, Month 12 post grafting.
Change in scar quality: Vancouver Scar Scale (VSS)
Scar quality will be measured using the Vancouver Scar Scale (VSS) and compared to baseline at several time points. Minimum value=0, Maximum value=12. Lower value is better.
Measured: Month 1, Month 2, Month 3, Month 6, Month 12 post grafting.
Changes in blister number over the grafted skin
Blister formation will be monitored and counted by a skilled dermatologist and compared to baseline at several time points.
Measured: Month 1, Month 2, Month 3, Month 6, Month 12 post grafting.
Changes in clinical appearance of grafted skin
Clinical appearance of the grafted skin areas will be assessed by 3D photographic reconstruction using a CANFIELD Vectra H1 device and compared to baseline at several time points.
Measured: Month 1, Month 2, Month 3, Month 6, Month 12 post grafting.
Changes in pruritus of grafted skin
Pruritus will be measured by the 5D pruritus score and compared to baseline at several time points. Minimum value=4, Maximum value=35. Lower value is better.
Measured: Month 1, Month 2, Month 3, Month 6, Month 12 post grafting.
Change in Quality of life: QOLEB questionnaire (Quality of Life for Epidermolysis Bullosa)
Quality of life will be measured using the specific QOLEB questionnaire (Quality of Life for Epidermolysis Bullosa) and compared to baseline at several time points.
Minimum value=0, Maximum value=51. Lower value is better.
Measured: Month 1, Month 2, Month 3, Month 6, Month 12 post grafting.
Change in Birmingham Epidermolysis Score (BEBS)
Change in disease severity will be assessed by the Birmingham Epidermolysis Score (BEBS).
Score will be compared to baseline at several timepoints. Minimum value=0, Maximum value=100. Lower value is better.
Measured: Month 1, Month 2, Month 3, Month 6, Month 12 post grafting.
Change in Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI) - Activity
Change in disease severity will be assessed by the Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI). Score will be compared to baseline at several timepoints. Score measure activity (minimum value=0, maximum value=276). Lower value is better.
Measured: Month 1, Month 2, Month 3, Month 6, Month 12 post grafting.
Change in Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI) - Damage
Change in disease severity will be assessed by the Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI). Score will be compared to baseline at several timepoints. Score measure damage (minimum value=0, maximum value=230). Lower value is better.
Measured: Month 1, Month 2, Month 3, Month 6, Month 12 post grafting.
Change in instrument for Scoring Clinical Outcomes of Research for Epidermolysis Bullosa (iscorEB).
Change in disease severity will be assessed by the instrument for Scoring Clinical Outcomes of Research for Epidermolysis Bullosa (iscorEB). Score will be compared to baseline at several timepoints. Minimum value=0, Maximum value=120. Lower value is better.
Measured: Month 1, Month 2, Month 3, Month 6, Month 12 post grafting.
Evaluation of the humoral immune response against recombinant C7
Detection of circulating anti-C7 antibodies in patient blood by ELISA and/or indirect immunofluorescence (IIF) on split skin at several time points
Measured: Month 1, Month 6, Month 12 post grafting.
Evaluation of the cytotoxic immune response against recombinant C7
Detection of T-cell responses to the full length C7 in patient blood by ELISPOT assay.
Measured: Month 1, Month 6, Month 12 post grafting.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Dec 2019
- Study start10 Jan 2020
- Primary completion9 Jun 2027 (estimated)
- Study completion9 Jun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Epidermolysis Bullosa Dystrophica, RecessiveStudies of the same condition, open and under way.
- More trials run by Institut National de la Santé Et de la Recherche Médicale, FranceThe 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (estimated)
Sponsor and investigators
- Sponsor type
- Other government
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04186650
- Sponsor's protocol ID
- C12-48
- Other IDs
- 2016-002790-35, 2016
- Acronym
- EBGraft
- Status last verified
- Feb 2026
- First submitted
- 25 Nov 2019
- Last updated
- 12 Feb 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Gaucher S, Lwin SM, Titeux M, Abdul-Wahab A, Pironon N, Izmiryan A, Miskinyte S, Ganier C, Duchatelet S, Mellerio JE, Bourrat E, McGrath JA, Hovnanian A. EBGene trial: patient preselection outcomes for the European GENEGRAFT ex vivo phase I/II gene therapy trial for recessive dystrophic epidermolysis bullosa. Br J Dermatol. 2020 Mar;182(3):794-797. doi: 10.1111/bjd.18559. Epub 2019 Nov 27. No abstract available.
PubMed 31557321Titeux M, Pendaries V, Zanta-Boussif MA, Decha A, Pironon N, Tonasso L, Mejia JE, Brice A, Danos O, Hovnanian A. SIN retroviral vectors expressing COL7A1 under human promoters for ex vivo gene therapy of recessive dystrophic epidermolysis bullosa. Mol Ther. 2010 Aug;18(8):1509-18. doi: 10.1038/mt.2010.91. Epub 2010 May 18.
PubMed 20485266Hennig K, Raasch L, Kolbe C, Weidner S, Leisegang M, Uckert W, Titeux M, Hovnanian A, Kuehlcke K, Loew R. HEK293-based production platform for gamma-retroviral (self-inactivating) vectors: application for safe and efficient transfer of COL7A1 cDNA. Hum Gene Ther Clin Dev. 2014 Dec;25(4):218-28. doi: 10.1089/humc.2014.083.
PubMed 25381930
Study at a glance
From the registry record, last updated by the sponsor on 12 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I/II ex Vivo Gene Therapy Clinical Trial for RDEB Using Autologous Skin Equivalent Grafts Genetically Corrected With a COL7A1-encoding SIN Retroviral Vector
- ClinicalTrials.gov ID
- NCT04186650
- Acronym / sponsor ID
- EBGraft / C12-48
- Keywords
- Genetic Therapy, Autologous Skin Graft, SIN retroviral vector, Type VII collagen, COL7A1
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 3 participants (estimated)
- Lead sponsor
- Institut National de la Santé Et de la Recherche Médicale, FranceOther government
- Study start
- 10 Jan 2020 (actual)
- Primary completion
- 9 Jun 2027 (estimated)
- Study completion
- 9 Jun 2027 (estimated)
- First posted
- 5 Dec 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.
This phase I/II clinical trial aims to treat 3 adult subjects with Recessive Dystrophic Epidermolysis Bullosa, expressing residual C7 levels, by genetically corrected autologous skin equivalent grafts on selected areas (up to 300 cm2).
Detailed description
Recessive Dystrophic Epidermolysis Bullosa (RDEB) is a severe orphan genetic disease responsible for skin and mucosal detachments due to a loss of adhesion of the epidermis to the underlying dermis. The disease is caused by loss of function mutations of the COL7A1 encoding type VII collagen (C7) which forms anchoring fibers, which are essential structures for dermal-epidermal adherence. Current treatments are only symptomatic and do not effectively treat or prevent the occurrence of cutaneous and mucosal detachments responsible for local and systemic complications that threaten the vital prognosis.
EBGRAFT is a prospective open-label international monocentric phase I/II clinical trial. It aims to treat 3 adult subjects with RDEB, expressing residual C7 levels, by genetically corrected autologous skin equivalent grafts.
The skin equivalent consists of keratinocytes and fibroblasts from the patient, genetically corrected ex vivo with a secure Self INactivating (SIN) retroviral vector expressing the COL7A1 cDNA under the control of the ubiquitous human promoter EF1a.
Each patient will be grafted sequentially at Necker Hospital in Paris using autologous genetically corrected skin equivalents of approximately 300 cm2 (up to 6 grafts of 50 cm2 each).
The main objective is to evaluate the safety of autologous skin equivalent grafts genetically corrected with a SIN COL7A1 retroviral vector (RV) in adults with RDEB.
The secondary objectives are:
- To evaluate the efficacy of transplanting autologous skin equivalent genetically corrected with RV SIN COL7A1 in adults with RDEB.
- To evaluate the immune response against recombinant type VII collagen (C7).
This clinical trial should evaluate whether the grafting of these genetically corrected autologous skin equivalents is well tolerated and whether they restore normal dermal-epidermal adherence of the grafted areas. The proposed treatment aims to obtain a permanent correction of the grafted areas, allowing skin healing and reducing pain. It has the potential to reduce itching, to prevent the occurrence of blisters and skin detachments, reduce the risk of infections, the duration and cost of care and also the risk of development of squamous cell carcinomas in the grafted areas.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Autologous genetically modified tissue-engineered skin graft | Experimental | Biological: COL7A1-SIN retroviral vector engineered autologous tissue-engineered skinGraft of SIN RV-mediated COL7A1 gene-modified autologous skin equivalent |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Clinical and molecular diagnosis of RDEB with confirmed bi-allelic COL7A1 mutations
- Reduced staining of C7 on skin biopsy, measured by immunofluorescence microscopy (IF)
- A reduced number of/or morphologically abnormal anchoring fibrils confirmed by TEM
- Detection of non-collagenous-1 domain (NC-1) of C7 on skin biopsy, measured by immunofluorescence microscopy (IF) and/or Western blot (WB) analysis
- Presence of ≥100cm2 of blistered and/or erosive skin areas including chronic wounds suitable for skin grafting
Key exclusion criteria (12 listed)
- Recipients of other investigational medicinal products within 6 months prior to enrolment into this study
- Past medical history of biopsy proven skin malignancy
- Immunotherapy including oral corticosteroids (Prednisolone >1mg/kg) for more than one week (intranasal and topical preparations are permitted) or chemotherapy within 60 days of enrolment into this study
- Known allergy to any of the constituents of the investigational medicinal product (IMP) including Penicillin
- Subjects with BOTH:
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 |
|---|---|---|
| COL7A1-SIN retroviral vector engineered autologous tissue-engineered skinGraft of SIN RV-mediated COL7A1 gene-modified autologous skin equivalent | Biological | Autologous genetically modified tissue-engineered skin graft |
Primary outcome
Safety of grafting SIN RV-mediated COL7A1 gene-modified autologous skin equivalent: Adverse Events (AE), Serious Adverse Events (SAEs), Adverse Reactions (ARs) and Serious Adverse Reactions (SARs)
Time frame: Month 12 post grafting.
The primary objective is to evaluate the safety of autologous autologous skin equivalent grafts genetically corrected with a SIN COL7A1 retroviral vector (RV) in adults with RDEB Primary Endpoints: Record of Adverse Events (AE), Serious Adverse Events (SAEs), Adverse Reactions (ARs) and Serious Adverse Reactions (SARs).
13 secondary outcomes
Change in C7 protein expression
Time frame: Month 1, Month 3, Month 6, Month 12 post grafting.
Change in anchoring fibrils number
Time frame: Month 1, Month 3, Month 6, Month 12 post grafting.
Change in scar quality: Vancouver Scar Scale (VSS)
Time frame: Month 1, Month 2, Month 3, Month 6, Month 12 post grafting.
Changes in blister number over the grafted skin
Time frame: Month 1, Month 2, Month 3, Month 6, Month 12 post grafting.
Changes in clinical appearance of grafted skin
Time frame: Month 1, Month 2, Month 3, Month 6, Month 12 post grafting.
Changes in pruritus of grafted skin
Time frame: Month 1, Month 2, Month 3, Month 6, Month 12 post grafting.
Change in Quality of life: QOLEB questionnaire (Quality of Life for Epidermolysis Bullosa)
Time frame: Month 1, Month 2, Month 3, Month 6, Month 12 post grafting.
Change in Birmingham Epidermolysis Score (BEBS)
Time frame: Month 1, Month 2, Month 3, Month 6, Month 12 post grafting.
Change in Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI) - Activity
Time frame: Month 1, Month 2, Month 3, Month 6, Month 12 post grafting.
Change in Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI) - Damage
Time frame: Month 1, Month 2, Month 3, Month 6, Month 12 post grafting.
Change in instrument for Scoring Clinical Outcomes of Research for Epidermolysis Bullosa (iscorEB).
Time frame: Month 1, Month 2, Month 3, Month 6, Month 12 post grafting.
Evaluation of the humoral immune response against recombinant C7
Time frame: Month 1, Month 6, Month 12 post grafting.
Evaluation of the cytotoxic immune response against recombinant C7
Time frame: Month 1, Month 6, Month 12 post grafting.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Feb 2026.
- Background: PubMed 31557321Gaucher S, Lwin SM, Titeux M, Abdul-Wahab A, Pironon N, Izmiryan A, Miskinyte S, Ganier C, Duchatelet S, Mellerio JE, Bourrat E, McGrath JA, Hovnanian A. EBGene trial: patient preselection outcomes for the European GENEGRAFT ex vivo phase I/II gene therapy trial for recessive dystrophic epidermolysis bullosa. Br J Dermatol. 2020 Mar;182(3):794-797. doi: 10.1111/bjd.18559. Epub 2019 Nov 27. No abstract available.
- Background: PubMed 20485266Titeux M, Pendaries V, Zanta-Boussif MA, Decha A, Pironon N, Tonasso L, Mejia JE, Brice A, Danos O, Hovnanian A. SIN retroviral vectors expressing COL7A1 under human promoters for ex vivo gene therapy of recessive dystrophic epidermolysis bullosa. Mol Ther. 2010 Aug;18(8):1509-18. doi: 10.1038/mt.2010.91. Epub 2010 May 18.
- Background: PubMed 25381930Hennig K, Raasch L, Kolbe C, Weidner S, Leisegang M, Uckert W, Titeux M, Hovnanian A, Kuehlcke K, Loew R. HEK293-based production platform for gamma-retroviral (self-inactivating) vectors: application for safe and efficient transfer of COL7A1 cDNA. Hum Gene Ther Clin Dev. 2014 Dec;25(4):218-28. doi: 10.1089/humc.2014.083.
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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 COL7A1-SIN retroviral vector engineered autologous tissue-engineered skin in people with Epidermolysis Bullosa Dystrophica, Recessive, run by Institut National de la Santé Et de la Recherche Médicale, France at 1 site in France.
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 7 inclusion and 12 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: under way, not looking for participants, as of the sponsor's last update on 12 Feb 2026. 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 France. 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?
This study is still under way. Results are not available in the source record yet.
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 of grafting SIN RV-mediated COL7A1 gene-modified autologous skin equivalent: Adverse Events (AE), Serious Adverse Events (SAEs), Adverse Reactions (ARs) and Serious Adverse Reactions (SARs) (time frame: Month 12 post grafting.). 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 NCT04186650, which also lists 3 linked publications.
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 12 Feb 2026.