Epidermolysis Bullosa (EB)Phase 1/2NCT07011589
Targeting Collagen VII Antibodies in Bullous Diseases Using Efgartigimod IV (VYVGART)
A Phase 1/2 study testing Efgartigimod in people with Epidermolysis Bullosa (EB) and Epidermolysis Bullosa Acquisita, run by M. Peter Marinkovich at 1 site in United States.
Trial summary
The study objective is to see if IV Efgartigimod and Vyjuvek treatment in Recessive Dystrophic Epidermolysis Bullosa (RDEB) and IV Efgartigimod treatment in Epidermolysis Bullosa Acquisita (EBA) improves wound healing and affects the levels of C7 antibody levels in serum. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Epidermolysis Bullosa (EB), Epidermolysis Bullosa Acquisita, Recessive Dystrophic Epidermolysis Bullosa
- What is being tested?
- Efgartigimod
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2026to Jan 2028
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- For DEB patients (aged 12 years or older): DEB confirmed with mutation analysis and correlated by phenotype, and treatment of at least 1 wound treated with topical gene therapy (VYJUVEK). Presence of C7 antibodies above normal cutoff on ELISA.
- For (classic) EBA patients (aged 18 years or older): EBA confirmed with positive histopathology (DIF), C7 antibodies above normal cutoff on ELISA, and having at least 1 skin lesion.
- The participant has a Karnofsky performance status of at least 60% at screening.
- Contraceptive use by reproductive male and female patients should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies and:
- Male participants:
- Must agree to use an acceptable method of contraception and not donate sperm from the time that the ICF is signed until they have received their last dose of IMP.
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- Female participants:
- Women of childbearing potential must have a negative serum pregnancy test at screening and a negative urine pregnancy test at week 1 / baseline before study intervention can be administered. Subsequent urine pregnancy tests are to be completed at week 6, week 11, week 16, week 21, and week 26 / EoS.
- WOCBP must agree to use a highly effective or acceptable contraception method until at least 90 days after they receive their last dose of IMP.
Who cannot join
- Linear IgA dermatosis-like EBA or other autoimmune blistering diseases (including but not limited to pemphigus vulgaris, bullous pemphigoid, mucous membrane pemphigoid).
- Use of the following EBA treatments:
- sulfasalazine, IVIg, subcutaneous administration of immunoglobulin (SCIg), immunoadsorption or plasma exchange within 2 weeks of the screening visit, tetracyclines with or without nicotinamide at doses higher than the recommended daily allowance (RDA)/dietary reference intake (DRI) within 2 weeks of the screening visit.
- any monoclonal antibody (including rituximab or another anti-CD20 biologic) within 6 months of the screening visit.
- complementary therapies-such as traditional Chinese medicines, herbs, or procedures (e.g., acupuncture)-within 4 weeks (or 5 half-lives) of the screening visit, if the investigator determines that such therapies may interfere with the study's efficacy assessments and/or potentially risk the safety of the participant.
- The use of the following EBA treatments is permitted throughout the study: OCS, topical corticosteroids, conventional immunosuppressants (e.g., azathioprine, cyclophosphamide, methotrexate, mycophenolate, or mofetil), and dapsone.
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- Moderate to severe renal insufficiency.
- Known contraindication to OCS therapy.
- Clinically significant uncontrolled active or chronic, bacterial, viral, or fungal infection at screening
- Medical instability limiting ability to travel to the Investigative Center
- Diseases or conditions that could interfere with the assessment of safety and efficacy of the study treatment and compliance of the subject with study visits/procedures, as determined by the investigator.
- Subjects actively receiving chemotherapy or immunotherapy at screening
- Active drug or alcohol addiction as determined by the investigator.
- Pregnant or nursing women
- History of malignancy unless deemed cured by adequate treatment with no evidence of recurrence for ≥3 years before the first administration of the IMP. Participants with the following cancers can be included at any time, provided they are adequately treated prior to screening:
- Basal cell or squamous cell skin cancer
- Carcinoma in situ of the cervix
- Carcinoma in situ of the breast
- Incidental histological finding of prostate cancer (TNM stage T1a or T1b)
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Clinical Research Coordinator contact
United States1 site
Stanford University
Redwood City, California, 94163
Clinical Research Coordinator, 650-723-3439, cimino@stanford.edu
Clinical Research Coordinator, 6507214902, kunju@stanford.edu
About this study
The study objective is to see if IV Efgartigimod and Vyjuvek treatment in Recessive Dystrophic Epidermolysis Bullosa (RDEB) and IV Efgartigimod treatment in Epidermolysis Bullosa Acquisita (EBA) improves wound healing and affects the levels of C7 antibody levels in serum.
Fewer wounds, more rapidly healing wounds, and decreased C7 antibodies could improve quality of life.
- Conditions
- Epidermolysis Bullosa (EB), Epidermolysis Bullosa Acquisita, Recessive Dystrophic Epidermolysis Bullosa, Dystrophic Epidermolysis Bullosa
- Being tested
- Efgartigimod
- Keywords
- epidermolysis bullosa, epidermolysis bullosa acquisita, dystrophic epidermolysis bullosa, recessive dystrophic epidermolysis bullosa, recessive, dystrophic, epidermolysis, bullosa, acquisita, collagven 7, c7, Col7, EB, EBA, DEB, RDEB, antibodies, Collagen VII, VII, IV IgG
- Medical subject headings
- Epidermolysis Bullosa, Epidermolysis Bullosa Acquisita, Epidermolysis Bullosa Dystrophica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EfgartigimodBiological
Also called efgartigimod alfa, efgartigimod alfa fcab, VYVGART
Dosage: 10mg/kg Frequency: Once a week Duration: 25 weeks
| Study group | Role | What they receive |
|---|---|---|
| Efgartigimod | Experimental | There is one arm of the study. First, each participant undergoes a 3-month observational period. If the participant has DEB, they will continue their standard of care VYJUVEK as prescribed. After the observational period concludes, the participant enters the treatment period, during which Efgartigimod is administered. DEB participants will continue their standard of care VYJUVEK as prescribed. Receives: Biological: Efgartigimod |
What is being measured
The primary outcome is the main question the study is designed to answer.
Reduction in serum C7 antibody levels
The proportion of patients who exhibit reduction in serum C7 antibody levels at week 26 as compared to week 1.
Measured: 26 weeks
Adverse Events and Effects
Occurrence of adverse events and effects.
Measured: 38 weeks
The Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI) improvement
The overall improvement of EB symptoms at week 26 as compared to week 1, measured by percentage change of a participant's EBDASI score (overall total score, total activity score, and total damage score). The EBDASI is scored in the range of 0 - 506, with a lower score corresponding to mild disease and higher score corresponding to more severe disease.
Measured: 26 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jun 2025
- Study startJul 2026 (estimated)
- Primary completionJan 2028 (estimated)
- Study completionJan 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Epidermolysis Bullosa (EB)Studies 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
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- M. Peter Marinkovich
- Sponsor type
- University, hospital or other
- Collaborators
- argenx
- Responsible party
- Sponsor-investigator, M. Peter Marinkovich, Stanford University
- Investigators
- Matt P Marinkovich, MD, Principal investigator, Associate Professor of Dermatology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07011589
- Sponsor's protocol ID
- 74784
- Status last verified
- Apr 2026
- First submitted
- 5 Jun 2025
- Last updated
- 5 May 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Targeting Collagen VII Antibodies in Bullous Diseases Using Efgartigimod IV (VYVGART)
- ClinicalTrials.gov ID
- NCT07011589
- Acronym / sponsor ID
- 74784
- Conditions
- Epidermolysis Bullosa (EB), Epidermolysis Bullosa Acquisita, Recessive Dystrophic Epidermolysis Bullosa, Dystrophic Epidermolysis Bullosa
- Keywords
- epidermolysis bullosa, epidermolysis bullosa acquisita, dystrophic epidermolysis bullosa, recessive dystrophic epidermolysis bullosa, recessive, dystrophic, epidermolysis, bullosa
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- M. Peter MarinkovichUniversity, hospital or other
- Collaborators
- argenx
- Study start
- Jul 2026 (estimated)
- Primary completion
- Jan 2028 (estimated)
- Study completion
- Jan 2028 (estimated)
- First posted
- 9 Jun 2025 (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.
The study objective is to see if IV Efgartigimod and Vyjuvek treatment in Recessive Dystrophic Epidermolysis Bullosa (RDEB) and IV Efgartigimod treatment in Epidermolysis Bullosa Acquisita (EBA) improves wound healing and affects the levels of C7 antibody levels in serum.
Fewer wounds, more rapidly healing wounds, and decreased C7 antibodies could improve quality of life.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Efgartigimod | Experimental | Biological: EfgartigimodThere is one arm of the study. First, each participant undergoes a 3-month observational period. If the participant has DEB, they will continue their standard of care VYJUVEK as prescribed. After the observational period concludes, the participant enters the treatment period, during which Efgartigimod is administered. DEB participants will continue their standard of care VYJUVEK as prescribed. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- For DEB patients (aged 12 years or older): DEB confirmed with mutation analysis and correlated by phenotype, and treatment of at least 1 wound treated with topical gene therapy (VYJUVEK). Presence of C7 antibodies above normal cutoff on ELISA.
- For (classic) EBA patients (aged 18 years or older): EBA confirmed with positive histopathology (DIF), C7 antibodies above normal cutoff on ELISA, and having at least 1 skin lesion.
- The participant has a Karnofsky performance status of at least 60% at screening.
- Contraceptive use by reproductive male and female patients should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies and:
Key exclusion criteria (19 listed)
- Linear IgA dermatosis-like EBA or other autoimmune blistering diseases (including but not limited to pemphigus vulgaris, bullous pemphigoid, mucous membrane pemphigoid).
- Use of the following EBA treatments:
- Moderate to severe renal insufficiency.
- Known contraindication to OCS therapy.
- Clinically significant uncontrolled active or chronic, bacterial, viral, or fungal infection at screening
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 |
|---|---|---|
| EfgartigimodAlso: efgartigimod alfa, efgartigimod alfa fcab, VYVGARTDosage: 10mg/kg Frequency: Once a week Duration: 25 weeks | Biological | Efgartigimod |
Primary outcome
Reduction in serum C7 antibody levels
Time frame: 26 weeks
The proportion of patients who exhibit reduction in serum C7 antibody levels at week 26 as compared to week 1.
Adverse Events and Effects
Time frame: 38 weeks
Occurrence of adverse events and effects.
The Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI) improvement
Time frame: 26 weeks
The overall improvement of EB symptoms at week 26 as compared to week 1, measured by percentage change of a participant's EBDASI score (overall total score, total activity score, and total damage score). The EBDASI is scored in the range of 0 - 506, with a lower score corresponding to mild disease and higher score corresponding to more severe disease.
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 5 May 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.
Clinical Research Coordinator contact
Matt P Marinkovich, MD principal investigator
Associate Professor of Dermatology
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 Efgartigimod in people with Epidermolysis Bullosa (EB) and Epidermolysis Bullosa Acquisita, run by M. Peter Marinkovich 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 19 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: opening soon, as of the sponsor's last update on 5 May 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 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 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?
Contact Kunju Clinical Research Coordinator, PhD (650-721-4902, kunju@stanford.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Reduction in serum C7 antibody levels (time frame: 26 weeks). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 5 May 2026.