Efgartigimod for Myasthenia Gravis

(ADAPTSC+ Trial)

No longer recruiting at 56 trial locations

Overseen By Sabine Coppieters, MD

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: argenx

Disqualifiers: Pregnancy, Uncontrolled infections, Autoimmune diseases, 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: Unknown

Duration: Variable, based on individual needs

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 people with myasthenia gravis, a condition that causes muscle weakness. The study will assess how well participants tolerate the treatment over time and whether it improves their quality of life. Participants will also provide feedback on the ease of using the injection at home. This trial is for individuals who have already participated in previous related studies and are managing the challenges of myasthenia gravis. As a Phase 3 trial, it represents 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 information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.

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

Research has shown that efgartigimod PH20 SC is generally well tolerated by people with generalized myasthenia gravis. Studies indicate that patients who underwent multiple cycles of this treatment experienced it without major safety concerns. One study found that efgartigimod PH20 SC effectively lowered levels of certain proteins in the blood, known as Immunoglobulin G (IgG), which are associated with myasthenia gravis symptoms. This suggests that the treatment is not only effective but also safe for continued use.

Overall, the treatment's safety appears promising based on the available data. Most participants in the studies handled it without significant issues. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Myasthenia Gravis?

Unlike the standard treatments for myasthenia gravis, which often include medications like pyridostigmine, corticosteroids, and immunosuppressants, efgartigimod PH20 SC offers a novel approach. This treatment is unique because it is administered subcutaneously, which could provide more convenience compared to intravenous options. Additionally, efgartigimod works by targeting the neonatal Fc receptor, potentially reducing harmful antibodies in the body more effectively. Researchers are excited about this treatment because it might offer faster relief with fewer side effects, improving the quality of life for patients with myasthenia gravis.

What evidence suggests that efgartigimod PH20 SC might be an effective treatment for Myasthenia Gravis?

Research has shown that efgartigimod PH20 SC, the treatment under study in this trial, yields promising results for treating Myasthenia Gravis. Studies have found that patients experienced an average reduction in symptoms, as measured by the Myasthenia Gravis Activities of Daily Living (MG-ADL) score, with this treatment. Specifically, one study reported an average symptom reduction of 57.5% to 60.3% after four weeks of treatment over several cycles. Additionally, the treatment was well-tolerated, allowing patients to continue using it without major issues. These findings suggest that efgartigimod PH20 SC could effectively manage Myasthenia Gravis symptoms. ^{1 2 3 4 5}

Are You a Good Fit for This Trial?

This trial is for individuals with generalized myasthenia gravis who have previously participated in specific antecedent studies. They must be able to consent, use contraception, and not be pregnant or breastfeeding. Excluded are those allergic to the study drug or its components, recently had major surgery, have serious infections or diseases that could affect results or safety.

Inclusion Criteria

Previously participated in antecedent studies ARGX-113-2001 or ARGX-113-1705 and are eligible for roll over

Women of Child bearing potential (WOCBP) must have a negative urine pregnancy test at baseline before investigational medicinal product (IMP) can be administered

I can sign and follow the study's consent form and rules.

Exclusion Criteria

I had cancer before but have been cancer-free for over 3 years.

I do not have serious health issues or recent major surgery that could risk my safety in the study.

I have had certain skin, cervical, breast, or early-stage prostate cancers but they are treated.

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 1000 mg in 3-week treatment periods, repeated as needed with at least 28 days in between treatment periods

Variable, based on individual needs

Follow-up

Participants are monitored for safety, tolerability, and clinical efficacy, including assessments of immunogenicity and quality of life

Up to 3.5 years

What Are the Treatments Tested in This Trial?

Interventions

• efgartigimod PH20 SC

Trial Overview The trial tests the long-term safety of Efgartigimod PH20 SC (a subcutaneous injection) for treating myasthenia gravis. It looks at how well it works over time, its effects on quality of life and daily activities, and if patients can self-administer the treatment.

How Is the Trial Designed?

Group I: efgartigimod PH20 SC Experimental Treatment 1 Intervention

Patients receiving efgartigimod PH20 subcutaneous (SC) treatment

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. argenxmedical.com argenxmedical.com/content/dam/medical-affairs/congress-posters/nuerology/aan-2025/Long-Term-Safety-and-Efficacy-of-Subcutaneous-Efgartigimod-PH20-in-Adult-Participants-With-Generalized-Myasthenia-Gravis-Interim-Results-of-the-ADAPT-S.pdf

Interim Results of the ADAPT-SC+ Study aData presented represents mean change in MG-ADL score from study baseline maintained as patients move through multiple cycles of efgartigimod ...

2. neurology.org neurology.org/doi/10.1212/WNL.0000000000210678

Interim Results of the ADAPT-SC+ Study (P1-11.005) Conclusions: Treatment with multiple cycles of efgartigimod PH20 SC was well tolerated and efficacious. Disclaimer: Abstracts were not reviewed ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT06909214

NCT06909214 | A Study to Evaluate the Clinical Outcomes ... A Study to Evaluate the Clinical Outcomes of Efgartigimod PH20 SC in Adults With New-onset Generalized Myasthenia Gravis (gMG) (ADAPT-EARLY) · Study Overview.

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11579873/

A phase 3 randomized noninferiority study (ADAPT-SC) ... As of data cutoff in ADAPT-SC+, the maximum mean reduction at week 4 ranged from 57.5% to 60.3% across the 3 treatment cycles (Fig. 3C, Right).

5. argenx.com argenx.com/news/2024/argenx-highlights-data-showing-patient-impact-across-multiple-im

argenx Highlights Data Showing Patient Impact Across ... Real-world gMG data show that at one-year post VYVGART initiation, 55% of patients reduced corticosteroid use by ≥5 mg/day and 42% of patients ...