Treatment of Bullous Pemphigoid With Avdoralimab (IPH5401), an Anti-C5aR1 Monoclonal Antibody
New drug tested for severe blistering skin disease
Plain English Summary
Treatment of Bullous Pemphigoid With Avdoralimab (IPH5401), an Anti-C5aR1 Monoclonal Antibody is a Phase 2 clinical trial sponsored by Centre Hospitalier Universitaire de Nice studying Bullous Pemphigoid. This trial tests a new drug called avdoralimab, given alongside strong steroid creams, to see if it's better than steroid creams alone for treating bullous pemphigoid. It is for adults diagnosed with bullous pemphigoid who need strong topical steroids and are hospitalized for treatment. Participants will receive either avdoralimab plus steroid cream or just steroid cream, and will have regular check-ups. The main alternative treatment is strong topical steroids, and sometimes systemic steroids or other immune-suppressing drugs are used for severe cases. The trial aims to enroll 15 participants.
Official Summary
The development of Auto Immune bullous Diseases (AIBD) results from a complex interaction between innate and adaptive immune systems. Bullous pemphigoid (BP), the most frequently encountered AIBD, predominantly affects elderly patients above 70 with an estimated incidence of 21.7 new cases/million/year in France.Interestingly, coversin, an anti-C5a and -leukotriene B4 small molecule, is currently used in a phase IIA clinical trial in BP patients (NCT04035733). However, although overall C5-blocking drugs are potentially interesting, they are likely to interfere with C5a-C5aR2-axis activation as well, a pathway that has recently proved protective in BP 12. The main objective is to investigate the clinical efficacy of an anti-C5aR1 antibody in addition to superpotent topical steroids compared to superpotent topical steroids alone in BP patients at 3 months.It is a case-controlled, randomized, open-labelled, and multicenter phase II clinical trial. Four Dermatologic French centers (Nice, Marseille Nord, Marseille Timone and Montpellier university hospitals) specialized in the in the care of BP patients will be participating in the study. It is expected that forty subjects will be included in this trial. Conversely, a more targeted inhibition of C5a-C5aR1 axis might be more effective in BP, sparing the potentially protective effect of C5a-C5aR2 interaction. Avdoralimab (IPH5401), a specific anti-C5aR1 monoclonal antibody, has already been credited of a good safety profile in the treatment of solid tumors and rheumatoid arthritis. The investigators hypothesize that avdoralimab might be a safe and effective treatment in BP patients
Who Can Participate
Here is what you need to know about eligibility for this trial. Adults aged 18 and older with a confirmed diagnosis of bullous pemphigoid. Patients who require treatment with very strong steroid creams and are currently hospitalized for their condition. Women who have gone through menopause are eligible; pre-menopausal women are not. Individuals with contraindications to topical steroids, or those who have recently used systemic steroids, certain antibiotics, or other specific immune-suppressing drugs, are not eligible. This trial is studying Bullous Pemphigoid, so participants generally need a confirmed diagnosis.
What They're Measuring
Complete clinical remission means that all skin blisters and sores have healed and no new ones have appeared by the end of the 3-month study period. The specific primary outcome measures are: Complete Clinical Remission (At 3 Months). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.
About This Phase
This trial is in Phase 2, which tests whether the treatment actually works against the target condition. Phase 2 trials involve 100-300 patients and continue to monitor safety while evaluating effectiveness. This phase often tests different dosages to find the optimal amount. About 33% of Phase 2 drugs advance to Phase 3. If successful, the treatment will move to large-scale Phase 3 trials needed for FDA approval.
Why This Trial Matters
This trial addresses a need for more effective treatments for bullous pemphigoid, a serious autoimmune skin condition that primarily affects the elderly, by investigating a targeted immune therapy. Phase 2 success would typically lead to larger Phase 3 trials needed for regulatory approval. This research targets Bullous Pemphigoid, where improved treatment options are needed.
Investor Insight
Bullous pemphigoid is a rare but serious condition, and the development of targeted therapies like avdoralimab could represent a significant advancement, potentially capturing a niche market within au Phase 2 trials have approximately a 15-20% chance of eventually gaining FDA approval.
Is This Trial Right for Me?
Ask your doctor about how avdoralimab works and what potential side effects it might have. Understand the schedule of visits and any procedures involved, such as blood tests or skin assessments. Be prepared for daily application of steroid cream and potential weekly injections of avdoralimab. The trial is being conducted at 4 sites. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.
AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.
Study Design
- Study Type: INTERVENTIONAL
- Allocation: RANDOMIZED
- Model: PARALLEL
- Masking: NONE
- Enrollment: 15 participants
Interventions
- DRUG: Avdoralimab (IPH5401) — Preparation of avdoralimab and preparation of the syringes are to be performed aseptically by site pharmacy qualified personnel.
- OTHER: Control — Superpotent topical steroids are the gold standard treatment for BP. All patients will receive 0.05% Clobetasol propionate cream as follows: * Patients of less than 45kg of body weight: 2 tubes of 10g/d * Patients of 45kg and above of body weight: 3 tubes of 10g/d Topical steroids will be applied every day until 15 days after the healing of the last bullous lesion
Primary Outcomes
- Complete Clinical Remission (At 3 Months)
Secondary Outcomes
- The delay (At 3 months)
- Initial Clinical Remission (At 3 months)
Full Eligibility Criteria
Inclusion Criteria: * Male or female * ≥ 18 years of age at the time of signing the informed consent document * Clinical diagnosis of BP confirmed by histology, immunohistochemistry and/or ELISA data * Patient requiring a treatment by superpotent topical steroids * Patients hospitalized for the treatment of their BP * For female, only post-menopaused patients * For male patients included in the study with partners of child bearing potential should agree to use highly effective contraception for the duration of the study and 6 months after the last dose of avdoralimab * Signed informed consent document prior to any study related assessments/procedures being conducted * Patient able to adhere to the study visit schedule and other protocol requirements * Patient registered to the French Social Security Exclusion Criteria: * Patients requiring systemic steroids according to the physician in charge * Contra indication to topical steroid * Use of systemic steroids or any immunosuppressive drugs in the past 4 weeks * Use of doxycycline or minocycline in the past 4 weeks * Use of systemic rituximab or omalizumab or dupilimumab in the past 12 weeks * Use of intravenous immunoglobulmins (IVIG) in the past 4 weeks * Impossibility to come every week to receive the injection * Participants in other clinical therapeutic studies involving a drug that could interfere with the present evaluation * Vulnerable people: pregnant or breast-feeding women, minors, adult under guardianship or deprived of freedom
Trial Locations
- CHU de Nice- Dermatologie, Nice, Alpes-Maritimes, France
- APHM, Timone, Marseille, Bouche Du Rhône, France
- APHM, Hôpital Nord, Marseille, Bouches du rhône, France
- CHU Montpellier, Dermatologie, Montpellier, Herault, France
Frequently Asked Questions
What is clinical trial NCT04563923?
NCT04563923 is a Phase 2 INTERVENTIONAL study titled "Treatment of Bullous Pemphigoid With Avdoralimab (IPH5401), an Anti-C5aR1 Monoclonal Antibody." It is currently completed and is sponsored by Centre Hospitalier Universitaire de Nice. The trial targets enrollment of 15 participants.
What conditions does NCT04563923 study?
This trial investigates treatments for Bullous Pemphigoid. The primary condition under study is Bullous Pemphigoid.
What treatments are being tested in NCT04563923?
The interventions being studied include: Avdoralimab (IPH5401) (DRUG), Control (OTHER). Preparation of avdoralimab and preparation of the syringes are to be performed aseptically by site pharmacy qualified personnel.
What does Phase 2 mean for NCT04563923?
Phase 2 trials test whether the treatment works for the intended condition. They involve 100-300 patients and continue to evaluate safety while measuring effectiveness.
What is the current status of NCT04563923?
This trial is currently "Completed." It started on 2020-10-10. The estimated completion date is 2024-03-31.
Who is sponsoring NCT04563923?
NCT04563923 is sponsored by Centre Hospitalier Universitaire de Nice. The sponsor is responsible for funding, designing, and overseeing the clinical trial.
How many people can participate in NCT04563923?
The trial aims to enroll 15 participants. The trial status is completed.
How is NCT04563923 designed?
This is a interventional study, uses randomized allocation, follows a parallel design, employs none masking.
What are the primary outcomes being measured in NCT04563923?
The primary outcome measures are: Complete Clinical Remission (At 3 Months). These are the main endpoints researchers use to determine whether the treatment is effective.
Where is NCT04563923 being conducted?
This trial is being conducted at 4 sites, including Nice, Alpes-Maritimes; Marseille, Bouche Du Rhône; Marseille, Bouches du rhône; Montpellier, Herault (France).
Where can I find official information about NCT04563923?
The official record for NCT04563923 is available on ClinicalTrials.gov at https://clinicaltrials.gov/study/NCT04563923. This government database provides the most up-to-date and detailed information about the trial.
What is NCT04563923 testing in simple terms?
This trial tests a new drug called avdoralimab, given alongside strong steroid creams, to see if it's better than steroid creams alone for treating bullous pemphigoid. It is for adults diagnosed with bullous pemphigoid who need strong topical steroids and are hospitalized for treatment.
Why is this trial significant?
This trial addresses a need for more effective treatments for bullous pemphigoid, a serious autoimmune skin condition that primarily affects the elderly, by investigating a targeted immune therapy.
What are the potential risks of participating in NCT04563923?
The most common side effects of avdoralimab in other studies have included infusion-related reactions like fever or chills. As with strong topical steroids, there's a risk of skin thinning, stretch marks, or increased susceptibility to skin infections. The study is open-label, meaning both patients and doctors know who is receiving which treatment, which could introduce bias. As with any clinical trial, participants are closely monitored and can withdraw at any time.
Should I consider participating in NCT04563923?
Ask your doctor about how avdoralimab works and what potential side effects it might have. Understand the schedule of visits and any procedures involved, such as blood tests or skin assessments. Be prepared for daily application of steroid cream and potential weekly injections of avdoralimab. Always discuss clinical trial participation with your healthcare provider to determine if it is appropriate for your specific situation.
What does NCT04563923 signal from an investment perspective?
Bullous pemphigoid is a rare but serious condition, and the development of targeted therapies like avdoralimab could represent a significant advancement, potentially capturing a niche market within au This is a Phase 2 trial, which is focused on confirming efficacy before larger pivotal studies.
What happens if the treatment in this trial doesn't work?
Participants will receive either avdoralimab plus steroid cream or just steroid cream, and will have regular check-ups. Participants in clinical trials always have the right to withdraw and pursue alternative treatments. The study team will help transition patients to other available options.
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This analysis is AI-generated and does not constitute medical advice. Always consult your healthcare provider before making decisions about clinical trial participation.