Efgartigimod SC for CIDP

(ADHERE+ Trial)

Not currently recruiting at 269 trial locations

Overseen By Sabine Coppieters, MD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: argenx

Must be taking: Efgartigimod

Disqualifiers: Pregnancy, Major surgery, 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

Approved in 4 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: Long-term

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial examines the long-term safety and effectiveness of efgartigimod PH20 SC, a potential new drug for CIDP, a condition that causes nerve damage and muscle weakness. The trial evaluates the treatment's efficacy over time and its potential for less frequent dosing. Individuals who participated in a previous efgartigimod study and either completed certain parts or experienced specific changes in their condition may qualify. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, allowing participants to contribute to early-stage findings.

Do I need to stop my current medications for the trial?

The trial protocol does not specify if you need to stop your current medications, but it mentions that some medications might be prohibited. It's best to discuss your current medications with the trial team to see if any are not allowed.

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 safe and well-tolerated. Studies like ADHERE demonstrated its effectiveness in lowering the risk of relapse in people with CIDP and confirmed its good safety record. While side effects can occur, many participants have used this treatment without major safety issues. In previous studies, most people found it well-tolerated, meaning they did not experience serious side effects. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Efgartigimod PH20 SC is unique because it offers a new delivery method for treating Chronic Inflammatory Demyelinating Polyneuropathy (CIDP). Unlike traditional treatments that typically involve intravenous administration, efgartigimod PH20 SC is delivered subcutaneously, which could make it more convenient for patients. Additionally, it targets the neonatal Fc receptor (FcRn) to reduce harmful autoantibodies, which is a different mechanism from standard therapies like corticosteroids or IVIg. Researchers are excited about this treatment's potential to offer a more patient-friendly option with a novel approach to managing CIDP.

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

Research has shown that efgartigimod PH20 SC, the treatment under study in this trial, effectively treats CIDP, a condition affecting the nerves. Studies have found that this treatment can reduce the likelihood of symptom recurrence in responsive patients. Specifically, the ADHERE study demonstrated its ability to provide long-term benefits for CIDP patients. Participants using efgartigimod PH20 SC experienced fewer relapses compared to those taking a placebo. This suggests that efgartigimod PH20 SC could be a promising option for managing CIDP. ^{1 2 3 4 6}

Are You a Good Fit for This Trial?

This trial is for adults with CIDP, an autoimmune disorder affecting nerves. Participants must have been part of a prior efgartigimod study or treatment and deteriorated during that time. They should understand the trial's demands, consent in writing, follow procedures, and use contraception if applicable. Pregnant women or those with serious diseases or recent major surgeries are excluded.

Inclusion Criteria

Your condition got worse during the previous stage of the trial and you can be treated with efgartigimod PH20 SC.

I am a woman who can have children, not pregnant, and using birth control.

I have been treated with efgartigimod PH20 SC or participated in its trials.

Exclusion Criteria

Pregnant, lactating, or intending to become pregnant during the trial

Participation in previous trials or treatment cycles outside specified timeframes

I do not have any serious health issues or recent major surgeries that could affect the trial.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive the subcutaneous formulation of efgartigimod for long-term safety and efficacy evaluation

Long-term

Sub-study

Participants explore less frequent dosing of efgartigimod PH20 SC

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Efgartigimod PH20 SC

Trial Overview The study tests long-term safety and effectiveness of Efgartigimod PH20 SC (a drug given under the skin) in adults with CIDP. It includes patients stabilized on this medication from a previous phase II trial and explores less frequent dosing options within a sub-study.

How Is the Trial Designed?

Group I: efgartigimod PH20 SC Experimental Treatment 1 Intervention

Patients treated with efgartigimod PH20 SC

Efgartigimod PH20 SC is already approved in European Union, United States, Japan, China for the following indications:

Approved in European Union as VYVGART for:

• Generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive

Approved in United States as VYVGART Hytrulo for:

• Generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive
• Chronic inflammatory demyelinating polyneuropathy (CIDP)

Approved in Japan as VYVDURA for:

• Generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive

Approved in China as Efgartigimod alfa injection (subcutaneous injection) for:

• Generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive

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

Published Research Related to This Trial

The study found that the immune globulin subcutaneous, human - klhw 20% solution (IGSC-C 20%) is bioequivalent to the immune globulin injection (IGIV-C 10%), providing similar levels of immunoglobulin G exposure over time, based on a Phase III trial with 53 participants.

IGSC-C 20% was well tolerated, with most adverse events being mild to moderate local infusion site reactions, indicating a safety profile comparable to IGIV-C 10%.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Immune globulin subcutaneous, human - klhw 20% for primary humoral immunodeficiency: an open-label, Phase III study. Sleasman, JW., Lumry, WR., Hussain, I., et al.[2020]

In a pivotal phase III trial, maintenance therapy with subcutaneous immunoglobulin (IgPro20) for 24 weeks significantly reduced relapse rates in patients with chronic inflammatory demyelinating polyneuropathy (CIDP) compared to placebo, demonstrating its efficacy as a treatment option.

IgPro20 was well tolerated with low rates of systemic adverse events, and over half of the patients preferred it over their previous intravenous immunoglobulin (IVIg) therapy, indicating it may be a more convenient and safer alternative for long-term management of CIDP.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Immune Globulin Subcutaneous (Human) 20% (Hizentra®): A Review in Chronic Inflammatory Demyelinating Polyneuropathy. Lamb, YN., Syed, YY., Dhillon, S.[2022]

In a study involving 59 children under 12 years old with primary immunodeficiency diseases or immune thrombocytopenia, Clairyg® 50 mg/mL was found to be well tolerated, with most reported adverse events being mild or moderate, such as headache and vomiting.

Clairyg® effectively controlled serious bacterial infections in patients with primary immunodeficiency and managed bleeding episodes in those with immune thrombocytopenia, demonstrating its safety and efficacy in real-life conditions without new safety concerns identified.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Real-world experience with CLAIRYG® 50 mg/mL (intravenous immunoglobulin) in children under 12 years with primary immunodeficiency or immmune thrombocytopenia: a post-approval safety study. Mahlaoui, N., Fouyssac, F., Mazingue, F., et al.[2023]

In a 2-year study involving 117 patients with primary immunodeficiency diseases receiving regular intravenous immunoglobulin (IVIG) therapy, only 2.15% of infusions resulted in immediate adverse reactions, indicating that IVIG is generally safe.

The most common mild reactions included malaise, headache, and abdominal pain, with severe reactions being rare; however, the risk of adverse reactions was higher with the drug Tegeline® compared to Octagam®.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Immediate infusion-related adverse reactions to intravenous immunoglobulin in a prospective cohort of 1765 infusions. Bichuetti-Silva, DC., Furlan, FP., Nobre, FA., et al.[2022]

In a phase 2 study involving 316 participants with poorly controlled type 2 diabetes, the dual receptor agonist LY3298176 demonstrated significantly better glucose control and weight loss compared to dulaglutide, with a dose-dependent reduction in HbA1c levels.

LY3298176 was generally well-tolerated, with gastrointestinal side effects being the most common but mostly mild to moderate, indicating a favorable safety profile for this new treatment option.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and safety of LY3298176, a novel dual GIP and GLP-1 receptor agonist, in patients with type 2 diabetes: a randomised, placebo-controlled and active comparator-controlled phase 2 trial. Frias, JP., Nauck, MA., Van, J., et al.[2022]

Efgartigimod is a promising new treatment for primary immune thrombocytopenia (ITP) that works by binding to the neonatal Fc receptor (FcRn), which leads to increased degradation of IgG antibodies that cause platelet destruction.

This therapy offers a novel mechanism of action compared to existing treatments, potentially providing an effective option for patients who may not respond well to traditional therapies like corticosteroids or IVIgG.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efgartigimod alfa for the treatment of primary immune thrombocytopenia. Broome, C.[2023]

Efgartigimod, a first-in-class neonatal Fc receptor antagonist, received its first approval in the USA in December 2021 for treating generalized myasthenia gravis in adults who are positive for anti-acetylcholine receptor antibodies.

The drug is also being evaluated for other autoimmune diseases and has been approved in Japan for generalized myasthenia gravis patients regardless of antibody status, indicating its potential broad application in autoimmune conditions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efgartigimod: First Approval. Heo, YA.[2022]

Efgartigimod, a new treatment that reduces pathogenic IgG autoantibodies, showed promising results in three patients with both stiff-person syndrome (SPS) and myasthenia gravis (MG) over a 12-week treatment period, leading to symptom improvement.

This study suggests that efgartigimod could be a potential therapy for SPS and other autoimmune neurological disorders, as it demonstrated efficacy in alleviating symptoms associated with both conditions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efgartigimod beyond myasthenia gravis: the role of FcRn-targeting therapies in stiff-person syndrome. Di Stefano, V., Alonge, P., Rini, N., et al.[2023]

Efgartigimod was found to be safe and well-tolerated in a phase 2 study involving 24 patients with generalized myasthenia gravis, with no serious adverse events reported.

The treatment led to a significant reduction in pathogenic IgG autoantibodies and resulted in a rapid and long-lasting improvement in disease symptoms for 75% of patients, suggesting its potential as an effective therapy for myasthenia gravis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Randomized phase 2 study of FcRn antagonist efgartigimod in generalized myasthenia gravis. Howard, JF., Bril, V., Burns, TM., et al.[2020]

Efgartigimod alfa is the first neonatal Fc receptor antagonist approved for treating generalized myasthenia gravis (gMG), showing significant and rapid improvements in muscle strength and quality of life in a phase 3 trial with a placebo group.

The treatment was generally well tolerated, with most side effects being mild to moderate, indicating a favorable safety profile for patients with gMG.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efgartigimod Alfa in Generalised Myasthenia Gravis: A Profile of Its Use. Heo, YA.[2023]

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39304241/

Safety, tolerability, and efficacy of subcutaneous ... ADHERE showed the efficacy of subcutaneous efgartigimod PH20 in reducing the risk of relapse versus placebo in people with CIDP who responded to treatment.

2. clinicaltrials.gov clinicaltrials.gov/study/NCT04280718

NCT04280718 | A Study to Assess the Long-term Safety ... ... Long-term Safety, Tolerability, and Efficacy of Efgartigimod PH20 SC in Patients With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP). Conditions.

3. argenxmedical.com argenxmedical.com/content/dam/medical-affairs/congress-posters/nuerology/aan-2025/Long-Term-Efficacy-of-Efgartigimod-PH20-SC-in-Patients-With-Chronic-Inflammatory-Demyelinating-Polyneuropathy-Interim-Results-From-the-ADHERE-Study.pdf

Long-Term-Efficacy-of-Efgartigimod-PH20-SC-in-Patients- ... efgartigimod PH20 SC results in long-term clinical efficacy in ... CIDP, chronic inflammatory demyelinating polyneuropathy; PH20 ...

4. neurology.org neurology.org/doi/10.1212/01.wnl.0001050988.41417.05

Efficacy and Safety of Efgartigimod PH20 in Chronic ... The primary endpoints were evidence of clinical improvement (ECI; stage A), the risk of relapse (time to first adjusted Inflammatory Neuropathy ...

5. thelancet.com thelancet.com/journals/laneur/article/PIIS1474-4422(24)00309-0/fulltext

Safety, tolerability, and efficacy of subcutaneous ... Safety, tolerability, and efficacy of subcutaneous efgartigimod in patients with chronic inflammatory demyelinating polyradiculoneuropathy (ADHERE)

6. argenxmedical.com argenxmedical.com/content/dam/medical-affairs/congress-posters/nuerology/cidp/ean-2024/Efficacy%20and%20Safety%20of%20Efgartigimod%20PH20%20in%20Chronic%20Inflammatory%20Demyelinating%20Polyneuropathy%20Results%20of%20ADHERE%20ADHERE+.pdf

Efficacy and Safety of Efgartigimod PH20 in Chronic ... Confirmed Evidence of Clinical Improvement With Efgartigimod in 66.5% of. Participants (Stage A) and 61% Reduced Relapse Risk Compared With ...