Efgartigimod for Bullous Pemphigoid

(BALLAD+ Trial)

No longer recruiting at 66 trial locations

Overseen By Sabine s Coppieters, MD

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: argenx

Disqualifiers: Significant disease, Recent surgery, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 13

Duration: 48 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called efgartigimod PH20 SC to determine its safety and effectiveness for individuals with moderate-to-severe bullous pemphigoid (BP), a skin condition that causes painful blisters. Participants who completed a previous study can join this trial to continue receiving the treatment. The trial includes a treatment phase where participants receive the medication and an observation phase for those not currently receiving it. Individuals with moderate-to-severe BP who have finished the earlier study might be suitable candidates. As a Phase 3 trial, this is the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.

Do I have to stop taking my current medications for the trial?

The trial does not specify if you need to stop your current medications. However, oral or topical corticosteroids can be used at the investigator's discretion, so it's best to discuss your current medications with the study team.

Is there any evidence suggesting that efgartigimod PH20 SC is likely to be safe for humans?

Research shows that efgartigimod PH20 SC is generally safe. In earlier studies, such as one called ADDRESS, participants who received efgartigimod SC had a good safety record, with most experiencing no serious side effects. Another study on long-term safety found the treatment safe for individuals with bullous pemphigoid, a skin condition that causes blisters. Administered as an injection under the skin, this treatment has proven safe for long-term use. While all medications can have some side effects, evidence suggests that efgartigimod PH20 SC is generally safe. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for bullous pemphigoid?

Unlike the standard of care for bullous pemphigoid, which often involves prolonged use of corticosteroids like Prednisone, efgartigimod PH20 SC acts differently by targeting the neonatal Fc receptor (FcRn). This unique mechanism helps reduce the levels of pathogenic antibodies thought to play a role in the disease. Researchers are excited about efgartigimod because it offers the potential for more effective management of symptoms with fewer side effects compared to long-term steroid use. Plus, the subcutaneous delivery method makes it a more convenient option for patients.

What evidence suggests that efgartigimod PH20 SC might be an effective treatment for bullous pemphigoid?

Research shows that efgartigimod PH20 SC might be a helpful treatment for bullous pemphigoid, a skin condition that causes painful blisters. In earlier studies, patients with similar conditions experienced significant improvement with this treatment. Participants in this trial will receive efgartigimod PH20 SC combined with Prednisone. Efgartigimod has shown promise in reducing the need for steroids and helping patients reach remission, significantly improving their symptoms. Early data suggests it helps control the disease, providing relief for those affected. These findings offer hope that efgartigimod could effectively manage bullous pemphigoid symptoms. ^{1 3 4 6 7}

Are You a Good Fit for This Trial?

Adults with Bullous Pemphigoid who completed a previous study (ARGX-113-2009) can join. They must agree to use contraception, be able to consent, and not have any health issues or recent surgeries that could risk their safety or skew the study's results.

Inclusion Criteria

Agrees to use contraceptive measures consistent with local regulations and the following: Women of childbearing potential must have a negative urine pregnancy test at baseline before receiving IMP and must use one of the contraception methods described in the protocol from signing the ICF until the last dose of IMP

Is capable of providing signed informed consent and complying with protocol requirements

You have finished the week 36 check-up for the ARGX-113-2009 study.

Exclusion Criteria

I have no major surgeries planned and no health issues that would risk my safety in the study.

I stopped taking a trial drug due to side effects and it was not beneficial for me.

You are allergic to the study medication or any of its ingredients.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive efgartigimod PH20 SC for up to 48 weeks

48 weeks

5 initial visits, then every 4 weeks

Observation

Participants not receiving efgartigimod PH20 SC are monitored with visits every 8 weeks

Variable, depending on participant status

Every 8 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

8 weeks

What Are the Treatments Tested in This Trial?

Interventions

• efgartigimod PH20 SC
• Prednisone

Trial Overview The trial is testing efgartigimod PH20 SC as a long-term treatment for Bullous Pemphigoid. It aims to control symptoms and achieve remission while reducing reliance on oral steroids like Prednisone.

How Is the Trial Designed?

Group I: efgartigimod PH20 SC Experimental Treatment 2 Interventions

participants receiving efgartigimod PH20 SC on top of Prednisone

Who Is Running the Clinical Trial?

argenx

Lead Sponsor

Trials

76

Recruited

11,500+

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

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05267600

NCT05267600 | A Phase 2/3 Study of Efgartigimod PH20 ... An interim analysis will be performed during part A (on data obtained through week 26 for all Part A participants) to assess the primary endpoint and several ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT05681481

NCT05681481 | A Phase 3 Study to Evaluate the Long- ... The purpose of this study is to evaluate the safety of efgartigimod PH20 SC over a longer period of time in adult participants with moderate-to-severe ...

3. argenx.com argenx.com/news/2023/argenx-reports-topline-results-address-study-efgartigimod-sc-pemphigus

argenx Reports Topline Results from ADDRESS Study of ... The ADDRESS results show the proportion of PV patients achieving the primary endpoint of complete remission on a minimal dose of steroids (CRmin) ...

4. ctv.veeva.com ctv.veeva.com/study/a-phase-2-3-study-of-efgartigimod-ph20-sc-in-adult-participants-with-bullous-pemphigoid-ballad

A 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 activity (CDA) ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10866767/

Advancements in Bullous Pemphigoid Treatment A Phase 2/3 Study of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid (BALLAD). 2023. Available at: https://classic ...

6. clinicaltrials.eu clinicaltrials.eu/trial/study-on-long-term-safety-and-effects-of-efgartigimod-ph20-sc-for-adults-with-bullous-pemphigoid/

Study on Long-term Safety and Effects of Efgartigimod ... This study examines the long-term safety and effects of efgartigimod PH20 SC, an under-the-skin injection, for the treatment of Bullous ...

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11652471/

Pharmacokinetics, Pharmacodynamics, and Safety of ... We present the pharmacokinetic, pharmacodynamic, and safety of IV and SC PH20 efgartigimod in healthy Chinese participants.