Long-term Safety of Efgartigimod for Myasthenia Gravis
(ADAPT Jr + Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests the long-term safety of efgartigimod, an immunotherapy for individuals with generalized myasthenia gravis (gMG), a condition that causes muscle weakness. Participants will receive efgartigimod either through an IV or under the skin to assess its ongoing safety and effectiveness. The study is open to those who have already participated in specific previous studies related to this treatment. Individuals who completed those studies and are willing to adhere to the trial's guidelines may be suitable candidates. As a Phase 2, Phase 3 trial, this study evaluates the treatment's effectiveness in an initial group and represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking therapy.
Do I need to stop my current medications to join the trial?
The trial information does not specify whether 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 this trial's treatments are likely to be safe?
Research has shown that efgartigimod, available in both IV (intravenous) and PH20 SC (under the skin) forms, is generally safe for individuals with generalized myasthenia gravis (gMG). Earlier studies identified respiratory infections, headaches, and urinary tract infections as the most common side effects, which are already associated with efgartigimod use.
For the IV form, no new safety issues have emerged with long-term use, though some studies mention potential kidney or metabolic problems over time. The under-the-skin form has also demonstrated long-term safety and consistent effectiveness in improving symptoms.
Both treatments are generally well-tolerated, but like any medication, they may pose some risk of side effects. Discussing potential risks and benefits with a healthcare professional before starting any new treatment is important.12345Why are researchers excited about this trial's treatments?
Efgartigimod is unique because it offers a novel approach to treating myasthenia gravis by targeting the neonatal Fc receptor (FcRn). Most current treatments for this condition, like acetylcholinesterase inhibitors, immunosuppressants, or corticosteroids, work by either increasing communication at the neuromuscular junction or broadly suppressing the immune system. Efgartigimod, on the other hand, specifically reduces pathogenic antibodies by interfering with FcRn, potentially leading to fewer side effects and a more targeted action. Researchers are excited about this treatment because it promises a more precise method of managing symptoms, which could result in better long-term outcomes for patients.
What evidence suggests that efgartigimod is effective for myasthenia gravis?
Research shows that efgartigimod, available in both IV (intravenous) and SC (subcutaneous) forms, holds promise for people with generalized Myasthenia Gravis (gMG). In this trial, participants will receive either Efgartigimod IV or Efgartigimod PH20 SC. Studies have found that patients experience significant improvements in their ability to perform daily activities, as measured by the MG-ADL scale, within just a few weeks of starting treatment. Additionally, long-term data up to 170 weeks indicate that efgartigimod remains effective over time. This treatment targets and reduces specific antibodies that contribute to gMG symptoms. Overall, these findings suggest that efgartigimod could effectively manage gMG.16789
Are You a Good Fit for This Trial?
This trial is for children who have a muscle weakness condition called generalized myasthenia gravis and were part of an earlier study (ARGX-113-2006). They must not plan to donate sperm or become pregnant, agree to use contraception, and understand the trial's requirements. Those with hypersensitivity to efgartigimod, recent live vaccines, other autoimmune diseases, or uncontrolled infections cannot join.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive Efgartigimod IV or Efgartigimod PH20 SC treatment
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Efgartigimod IV
Efgartigimod IV is already approved in United States, European Union for the following indications:
- Generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive
- Generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive
- Generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive
- Chronic inflammatory demyelinating polyneuropathy (CIDP)
Find a Clinic Near You
Who Is Running the Clinical Trial?
argenx
Lead 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