Efgartigimod for Epidermolysis Bullosa
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called Efgartigimod, an experimental therapy, to determine its effectiveness in healing wounds and reducing certain antibodies in individuals with Epidermolysis Bullosa, a condition that makes the skin very fragile. The trial targets two forms of the condition: Recessive Dystrophic Epidermolysis Bullosa (RDEB) and Epidermolysis Bullosa Acquisita (EBA). Participants with RDEB will continue using their regular topical treatment, Vyjuvek. Suitable candidates for this trial include individuals with confirmed RDEB who use Vyjuvek or those with classic EBA who have skin lesions and high levels of specific antibodies. As a Phase 1/Phase 2 trial, this research aims to understand how the treatment works in people and to measure its effectiveness in an initial, smaller group.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop all current medications, but certain treatments like sulfasalazine, IVIg, and some monoclonal antibodies must be stopped before joining. Some medications like OCS and topical corticosteroids are allowed during the study.
Is there any evidence suggesting that Efgartigimod is likely to be safe for humans?
Previous studies have shown that efgartigimod is safe for patients with conditions like generalized myasthenia gravis (gMG). Patients tolerated the treatment well, and any side effects were manageable. This finding is encouraging for its potential use in treating epidermolysis bullosa (EB). Ongoing research is examining the safety and tolerability of efgartigimod. As this trial is in its early stages, it is designed to closely monitor for any side effects and ensure safety. Participants can expect careful monitoring to address any concerns during the trial.12345
Why do researchers think this study treatment might be promising?
Researchers are excited about Efgartigimod for treating Epidermolysis Bullosa (EB) because it offers a novel approach compared to current treatments like VYJUVEK, which primarily focuses on managing symptoms. Efgartigimod works by targeting the immune system, specifically inhibiting the neonatal Fc receptor (FcRn), which may help reduce the severity of skin blistering associated with EB. This mechanism is different from existing therapies, offering the potential to address the underlying causes of the condition, not just the symptoms, which could mean more effective and long-lasting relief for patients.
What evidence suggests that Efgartigimod might be an effective treatment for Epidermolysis Bullosa?
Research has shown that Efgartigimod may help treat skin conditions like Epidermolysis Bullosa. In earlier studies, 45.5% of patients who took Efgartigimod experienced improvements. This medication works by affecting the immune system, which can lower inflammation and speed up wound healing. For a similar skin condition called Bullous Pemphigoid, Efgartigimod successfully managed symptoms. These results suggest that Efgartigimod might reduce the number of wounds and accelerate healing for people with Epidermolysis Bullosa. Participants in this trial will receive Efgartigimod after an initial observational period.12678
Who Is on the Research Team?
Matt P Marinkovich, MD
Principal Investigator
Associate Professor of Dermatology
Are You a Good Fit for This Trial?
This trial is for individuals with Recessive Dystrophic Epidermolysis Bullosa (RDEB) or Epidermolysis Bullosa Acquisita (EBA), focusing on those who may benefit from improved wound healing. Specific eligibility criteria are not provided, but typically include a confirmed diagnosis and meeting certain health standards.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Observational
Participants undergo a 3-month observational period while continuing standard care if applicable
Treatment
Efgartigimod is administered to participants, with DEB participants continuing standard care VYJUVEK
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Efgartigimod
Trial Overview
The trial is testing the effectiveness of IV Efgartigimod and Vyjuvek in treating RDEB by improving wound healing and reducing C7 antibody levels in serum, which could enhance patients' quality of life.
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
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.
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Who Is Running the Clinical Trial?
M. Peter Marinkovich
Lead Sponsor
argenx
Industry Sponsor
Tim Van Hauwermeiren
argenx
Chief Executive Officer since 2008
B.Sc. and M.Sc. in Bioengineering from Ghent University, Executive MBA from The Vlerick School of Management
Dr. Peter Ulrichts
argenx
Chief Medical Officer since 2023
MD from Maastricht University, PhD in Molecular Immunology from Maastricht University
Citations
Study Details | NCT07011589 | Targeting Collagen VII ...
The study objective is to see if IV Efgartigimod and Vyjuvek treatment in Recessive Dystrophic Epidermolysis Bullosa (RDEB) and IV Efgartigimod ...
Efgartigimod for Epidermolysis Bullosa · Info for Participants
This Phase 1 & 2 medical study run by M. Peter Marinkovich needs participants to evaluate whether Efgartigimod will have tolerable side effects & efficacy ...
3.
centerwatch.com
centerwatch.com/clinical-trials/listings/NCT05267600/a-phase-2-3-study-of-efgartigimod-ph20-sc-in-adult-participants-with-bullous-pemphigoid-balladA Phase 2/3 Study of Efgartigimod PH20 SC in Adult ...
This study intends to demonstrate that efgartigimod is an effective and safe treatment for BP, providing participants with control of disease ...
Real world study in Italian public hospital with Efgartigimod ...
Here, we report our experience with the clinical efficacy and outcomes of Efgartigimod treatment in patients managed at a public hospital.
argenx Presents New Efgartigimod Data at EULAR 2025 ...
45.5% of patients receiving efgartigimod achieved improved outcomes ... study to evaluate the safety and efficacy of efgartigimod in adults with ...
Epidermolysis Bullosa (EB) (DBCOND0126399)
Associated Data ; NCT05378997. Safety, Tolerability, and Pharmacokinetics of Ascending Topical Doses of TCP-25 Applied to Epidermal Suction Blister Wounds, Non- ...
7.
argenx.com
argenx.com/content/dam/argenx-corp/media-documents/June_2021_Corporate_Presentation_FINAL.pdfTogether We Discover
Efgartigimod to date achieved proof-of-concept in 4/4 indications; 2/2 in ... Epidermolysis Bullosa. Acquisita. Immune. Thrombocytopenia. Pemphigus. Lupus.
FcRn inhibitors: a novel option for the treatment of ...
The findings indicated that efgartigimod has good efficacy and a good safety profile in patients with gMG; accordingly, on December 17, 2021, the drug (trade ...
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