A Phase 1/2 interventional study of Efgartigimod in Epidermolysis Bullosa (EB), Epidermolysis Bullosa Acquisita and Recessive Dystrophic Epidermolysis Bullosa, sponsored by M. Peter Marinkovich. Not yet recruiting at 1 site in United States. Open to participants aged 12 Years and older. Per ClinicalTrials.gov, last updated 2026-05-05.
Sponsored by M. Peter Marinkovich · Phase 1/2, Interventional, and Treatment
Help build ClinicalTrials.gg — short surveys, remote interviews or testing, in your language. Take part →
Among 45 studies in Epidermolysis Bullosa Dystrophica with a site in United States — 12 open to participants now, 16 under way, 4 of them phase 3. Epidermolysis Bullosa Dystrophica · United States.
Among the 45 studies in Epidermolysis Bullosa Dystrophica with a site in United States, this one is the 5th largest by planned enrollment of the 32 interventional ones that give a count, is one of 2 that started in 2026, is one of 12 in phase 1/2, reaches primary completion later than 10 of the 15 other live ones, and was the 44th registered.
78 studies on the registry are indexed under Epidermolysis Bullosa Dystrophica; 21 are open to participants now.
Browse Epidermolysis Bullosa Dystrophica studies →12 of the 45 studies in Epidermolysis Bullosa Dystrophica with a site in United States are open now. Recruiting first, the newest first:
Xinnate AB · Sites in United States, France, Greece and 4 more countries
Ages 4+
M. Peter Marinkovich · Sites in United States
Ages 6+
Abeona Therapeutics, Inc · Sites in United States
Krystal Biotech, Inc. · Sites in United States
Ages 6 mo+
Chiesi Farmaceutici S.p.A. · Sites in United States, France, Germany and 3 more countries
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 17 Epidermolysis Bullosa Dystrophica studies recruiting now, 13 (76%) set no upper age limit and 3 (18%) stop at 75 or younger; 15 take children under 18; 2 take healthy volunteers.
This study: 12 Years and older · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
45 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 15 · Phase 2 18 · Phase 3 8
First in 2007; this study’s year is marked. 2 started in 2026.
By drug class, each counting the studies that test any of its medicines:
This study's planned enrollment of 18 is above the median of 9 across 55 interventional studies indexed under Epidermolysis Bullosa Dystrophica.
Median enrollment 8 across the 32 interventional studies, 29 across the 13 observational. Of the 24 that completed or were terminated, 11 (46%) have results posted.
This tab as a report to print or save as PDF → With an account.
29 of 45 have an industry lead sponsor. The 10 most frequent:
M. Peter Marinkovich is the lead sponsor of 2 studies on the registry; 2 are open to participants now.
M. Peter Marinkovich has 2 studies filed under Epidermolysis Bullosa Dystrophica — fewer than ten, so there is no comparison to draw.
4 of the studies in Epidermolysis Bullosa Dystrophica with a site in United States that are under way reach primary completion within a year, 2 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT07016750Phase 3Krystal Biotech, Inc.A Study Comparing KB803 and Matched Placebo in Patients With Dystrophic Epidermolysis Bullosa | 2026-12 |
| NCT06892639Phase 3Castle Creek Biosciences, LLC.Evaluation of D-Fi for the Treatment of Wounds Due to DEB | 2027-09 |
| NCT06834035M. Peter MarinkovichTargeting Collagen VII Antibodies With IV IgG in Dystrophic Epidermolysis Bullosa | 2027-03 |
| NCT06594393Xinnate ABA Phase 2 Study of TCP-25 Gel in Patients With Epidermolysis Bullosa, STEP-study | 2027-06-30 |
Add all 4 to your calendar (.ics) → With an account.
Among the 45 studies in Epidermolysis Bullosa Dystrophica with a site in United States, from their registry updates:
Nothing recorded among them since 2026-09-25.
This tab as a report to print or save as PDF → With an account.
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.
78 studies on the registry are indexed under Epidermolysis Bullosa Dystrophica; 21 are open to participants now.
This study's planned enrollment of 18 is above the median of 9 across 55 interventional studies indexed under Epidermolysis Bullosa Dystrophica.
Browse Epidermolysis Bullosa Dystrophica studies →M. Peter Marinkovich is the lead sponsor of 2 studies on the registry; 2 are open to participants now.
Compared within Epidermolysis Bullosa Dystrophica, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Epidermolysis Bullosa. The counts are of registry records indexed under each term, refreshed daily. How we classify →
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
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.
Female participants:
Exclusion Criteria:
Use of the following EBA treatments:
History of 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:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Karnofsky performance status (KPS)
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Biological: Efgartigimod
Dosage: 10mg/kg Frequency: Once a week Duration: 25 weeks
Also known as: efgartigimod alfa, efgartigimod alfa fcab, VYVGART
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.
Time frame: 26 weeks
Adverse Events and Effects
Occurrence of adverse events and effects.
Time frame: 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.
Time frame: 26 weeks
Start is estimated for Jul 2026; primary completion for Jan 2028, 18 months later.
How these figures are worked out, and which studies are left out of them.
This study is not yet recruiting, as verified in Apr 2026. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Studies of the drug tested here: Efgartigimod alfa
Epidermolysis Bullosa Dystrophica →
M. Peter Marinkovich