Efgartigimod for Myasthenia Gravis

(ADAPT-EARLY Trial)

Recruiting at 14 trial locations

Overseen By Sabine Coppieters, MD

Age: 18+

Sex: Any

Trial Phase: Phase 4

Sponsor: argenx

Must be taking: AChEI

Must not be taking: Corticosteroids, Immunosuppressants

Disqualifiers: Thymectomy, MGFA class I or V, others

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

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

Compensation: Varies

Number of Visits: Unknown

Duration: 51 weeks

30 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the effectiveness of a new treatment, efgartigimod PH20 SC, for individuals with Myasthenia Gravis (MG), a condition that causes muscle weakness. It targets adults who have experienced symptoms for less than a year. Participants will receive the treatment for nearly a year to assess symptom improvement. This trial may suit those whose MG symptoms impact daily activities and who have not tried other major treatments. As a Phase 4 trial, this research aims to understand how the already FDA-approved and effective treatment benefits a broader patient population.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but it mentions that participants should not have used systemic corticosteroids or nonsteroidal immunosuppressive therapy for gMG before. If you're on these, you might need to stop.

What is the safety track record for this treatment?

Research has shown that efgartigimod PH20 SC is generally safe for people. Earlier studies found that using this treatment multiple times was both safe and effective. Most side effects were mild and occurred in more than 10% of participants, indicating that while some experienced side effects, they were not serious. The treatment has shown promise in improving symptoms for people with generalized myasthenia gravis (gMG). This supports confidence in its safety for those considering joining the trial. ^{1 2 3 4 5}

Why are researchers enthusiastic about this study treatment?

Efgartigimod PH20 SC is unique because it introduces a new subcutaneous delivery method for treating myasthenia gravis, offering a potentially more convenient and less invasive option compared to intravenous treatments like pyridostigmine, corticosteroids, or IVIG. While most treatments for myasthenia gravis aim to alleviate symptoms by enhancing neuromuscular transmission or suppressing the immune system, efgartigimod works by reducing pathogenic IgG antibodies that contribute to the disease's muscle weakness. Researchers are excited about this treatment because it targets the underlying cause of symptoms more directly and could improve patient quality of life with fewer side effects and greater ease of administration.

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, effectively treats generalized Myasthenia Gravis (gMG). Studies have found that patients experience significant symptom improvements. For example, one study reported a reduction in muscle weakness by 57.5% to 60.3% by the fourth week, as measured by the MG-ADL score. Patients also showed better results on the QMG score, indicating improved muscle strength. Overall, efgartigimod PH20 SC has been well tolerated and effective across multiple treatment cycles. ^{1 3 4 5 6}

Are You a Good Fit for This Trial?

Adults recently diagnosed with generalized Myasthenia Gravis (gMG) can join this study. They should have had muscle weakness symptoms for less than a year, be positive for AChR-Ab antibodies, and either haven't been treated or only used AChEI drugs. Their MG-ADL score must be 5 or higher.

Inclusion Criteria

I have been diagnosed with a moderate to severe form of myasthenia gravis.

I have tested positive for AChR antibodies.

My daily activities are significantly affected by my condition.

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 51 weeks to evaluate clinical outcomes in new-onset generalized myasthenia gravis

51 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4-7 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Efgartigimod PH20 SC

Trial Overview The trial is testing Efgartigimod PH20 SC's effectiveness in treating gMG over a period of 51 weeks. Participants will undergo treatment to see if it improves their condition compared to before the trial started.

How Is the Trial Designed?

Group I: Efgartigimod PH20 SC Experimental Treatment 1 Intervention

Participants receiving efgartigimod PH20 SC

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. 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).

3. 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 ...

4. argenx.com argenx.com/news/2025/press-release-3176635

argenx Presents New Data at AANEM and MGFA ... Improvements in MG-ADL and QMG (Quantitative Myasthenia Gravis score) among patients treated with VYVGART were increasingly pronounced across ...

5. 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.

6. argenxmedical.com argenxmedical.com/content/dam/medical-affairs/congress-posters/nuerology/mgfa/Long-Term%20Safety%20and%20Efficacy%20of%20Subcutaneous%20Efgartigimod%20PH20%20in%20Adult%20Participants%20With%20Generalized%20Myasthenia%20Gravis_%20Interim%20Results%20of%20the%20ADAPT-SC%2B%20Study.pdf

Interim Results of the ADAPT-SC+ Study AEs occurring in >10% of participants receiving efgartigimod PH20 SC in either data cut. Cycle 1 to Cycle 9 was -6.7 (0.97).