Efgartigimod SC for CIDP

(ADHERE+ Trial)

Recruiting at 241 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

229 Participants Needed

Trial Summary

What is the purpose of this trial?

This is the open-label extension study of phase II ARGX-113-1802 to evaluate the long-term safety and efficacy of the subcutaneous formulation of efgartigimod in adults with CIDP. Patients already stabilized on efgartigimod PH20 SC will also have the opportunity to participate in a sub study to explore less frequent dosing of efgartigimod PH20 SC.

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.

What data supports the effectiveness of the drug Efgartigimod SC for CIDP?

Efgartigimod has shown effectiveness in treating generalized myasthenia gravis (a muscle weakness condition) by reducing disease burden and improving muscle strength and quality of life. It has also shown promising results in other autoimmune disorders, suggesting potential benefits for conditions like CIDP (chronic inflammatory demyelinating polyradiculoneuropathy). ^{1 2 3 4 5}

Is Efgartigimod SC generally safe for humans?

There is no specific safety data available for Efgartigimod SC in the provided research articles. ^{6 7 8 9 10}

What makes the drug Efgartigimod SC unique for treating CIDP?

Efgartigimod SC is unique because it is a subcutaneous (under the skin) formulation that targets the neonatal Fc receptor, which helps reduce harmful antibodies in the body. This mechanism is different from other treatments for CIDP, which may not specifically target this receptor. ^{11 12 13 14 15}

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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

Findings from Research

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]

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]

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 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]

References

1. England pubmed.ncbi.nlm.nih.gov

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

2. New Zealand pubmed.ncbi.nlm.nih.gov

Efgartigimod: First Approval. [2022]

3. Germany pubmed.ncbi.nlm.nih.gov

Efgartigimod beyond myasthenia gravis: the role of FcRn-targeting therapies in stiff-person syndrome. [2023]

4. United States pubmed.ncbi.nlm.nih.gov

Randomized phase 2 study of FcRn antagonist efgartigimod in generalized myasthenia gravis. [2020]

5. New Zealand pubmed.ncbi.nlm.nih.gov

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

6. Switzerland 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. [2023]

7. England pubmed.ncbi.nlm.nih.gov

Immune globulin subcutaneous, human - klhw 20% for primary humoral immunodeficiency: an open-label, Phase III study. [2020]

8. New Zealand pubmed.ncbi.nlm.nih.gov

Immune Globulin Subcutaneous (Human) 20% (Hizentra®): A Review in Chronic Inflammatory Demyelinating Polyneuropathy. [2022]

9. England pubmed.ncbi.nlm.nih.gov

Safety and tolerability of subcutaneous immunoglobulin 20% in primary immunodeficiency diseases from two continents. [2020]

10. Netherlands pubmed.ncbi.nlm.nih.gov

Immediate infusion-related adverse reactions to intravenous immunoglobulin in a prospective cohort of 1765 infusions. [2022]

11. England pubmed.ncbi.nlm.nih.gov

Development of a novel Fc fusion protein dual glucagon-like peptide-1 and gastric inhibitory polypeptide receptor agonists. [2023]

12. England pubmed.ncbi.nlm.nih.gov

The SKW 6.4 line of human B lymphocytes specifically binds and responds to vasoactive intestinal peptide. [2018]

13. Netherlands pubmed.ncbi.nlm.nih.gov

Human antibody-dependent cellular cytotoxicity mediated by interferon gamma-activated neutrophils is impaired by vasoactive intestinal peptide. [2019]

14. Netherlands pubmed.ncbi.nlm.nih.gov

Pharmacological effects and lung-binding characteristics of a novel VIP analogue, [R15, 20, 21, L17]-VIP-GRR (IK312532). [2014]

15. England 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. [2022]