Efgartigimod for Low Platelet Count

(ADVANCE SC+ Trial)

Recruiting at 82 trial locations

Overseen By Antonio Guglietta, MD

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: argenx

Must not be taking: Anti-CD20, Romiplostim, Monoclonal antibodies, others

Disqualifiers: Pregnancy, Hypersensitivity, Other investigational drugs, 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

173 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing a new injection treatment for adults whose immune systems destroy their own platelets. The treatment aims to calm the immune system to stop it from attacking these important blood cells. Romiplostim promotes platelet production and has been used in the treatment of this condition.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop your current medications, but you cannot continue or start certain medications like anti-CD20 therapy, romiplostim, or monoclonal antibodies during the trial.

What data supports the effectiveness of the drug efgartigimod PH20 SC for low platelet count?

Research shows that efgartigimod, when used in patients with primary immune thrombocytopenia (a condition causing low platelet counts), led to increased platelet counts in 46% of patients compared to 25% with a placebo. This suggests that efgartigimod can effectively help increase platelet levels in people with this condition. ^{1 2 3 4 5}

Is efgartigimod safe for humans?

Efgartigimod has been shown to be well tolerated and has a favorable safety profile in studies involving patients with primary immune thrombocytopenia, a condition characterized by low platelet counts. ^{1 2 3 6 7}

How is the drug efgartigimod different from other treatments for low platelet count?

Efgartigimod is unique because it works by blocking the neonatal Fc receptor (FcRn), which leads to the breakdown of IgG antibodies that contribute to low platelet counts. This mechanism is different from other treatments like corticosteroids or IVIgG, which have different targets and methods of action. ^{1 2 3 8 9}

Eligibility Criteria

This trial is for adults with primary immune thrombocytopenia who completed a previous 24-week study and can commit to another 52 weeks. They must understand the trial, consent to procedures, attend visits, use contraception if applicable, and not be pregnant or breastfeeding.

Inclusion Criteria

Ability to understand the requirements of the additional 52-week treatment period of the trial, to provide written informed consent (including consent for the use and disclosure of research-related health information), and to comply with the trial protocol procedures (including required trial visits)

Ability to understand the requirements of the trial and provide written informed consent (including consent for the use and disclosure of research-related health information), willing and able to comply with the trial protocol procedures (including attending the required trial visits)

Agree to use contraceptives consistent with local regulations and have a negative urine pregnancy test at baseline before receiving IMP (applicable to female participants of childbearing potential)

Exclusion Criteria

Pregnant or lactating females and those who intend to become pregnant during the trial or within 90 days after last dose of efgartigimod PH20 SC

I am not taking any restricted medications like anti-CD20 therapy or live vaccines.

Use of any other investigational drug or participation in any other investigational trial

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Patients receive efgartigimod PH20 SC treatment

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants may continue to receive the study drug long-term

Treatment Details

Interventions

• efgartigimod PH20 SC

Trial Overview The safety and effectiveness of efgartigimod PH20 SC (a subcutaneous injection) are being tested in patients with low platelet counts due to primary immune thrombocytopenia over an additional year following a prior study.

Participant Groups

Group I: efgartigimod PH20 SC Experimental Treatment 1 Intervention

Patients receiving efgartigimod PH20 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

Findings from Research

In a Phase 2 study involving 38 patients with primary immune thrombocytopenia (ITP), efgartigimod, an FcRn antagonist, was well tolerated and showed a favorable safety profile, consistent with earlier Phase 1 data.

Efgartigimod treatment led to a significant reduction in total IgG levels and clinically relevant increases in platelet counts, with 46% of patients achieving a platelet count of ≥50 × 10^9/L compared to 25% on placebo, indicating its potential as a novel treatment for ITP.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Phase 2 study of efgartigimod, a novel FcRn antagonist, in adult patients with primary immune thrombocytopenia. Newland, AC., Sánchez-González, B., Rejtő, L., et al.[2021]

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]

In a phase 3 study involving 131 adults with chronic primary immune thrombocytopenia, efgartigimod significantly improved sustained platelet count responses compared to placebo, with 22% of patients achieving the primary endpoint versus 5% in the placebo group.

Efgartigimod was well tolerated, with most adverse events being mild to moderate, indicating a favorable safety profile for patients who had previously undergone multiple treatments for their condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and safety of the neonatal Fc receptor inhibitor efgartigimod in adults with primary immune thrombocytopenia (ADVANCE IV): a multicentre, randomised, placebo-controlled, phase 3 trial. Broome, CM., McDonald, V., Miyakawa, Y., et al.[2023]

New generation thrombopoietic growth factors, like romiplostim and eltrombopag, effectively increase platelet counts in patients with immune thrombocytopenic purpura (ITP), showing response rates above 70-80% even in those who have failed previous treatments.

These agents work by increasing platelet production rather than reducing consumption, but their long-term safety is not fully understood, leading to recommendations for their use primarily in refractory cases or before splenectomy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Chronic immune thrombocytopenic purpura. New agents. Rodeghiero, F., Ruggeri, M.[2016]

In a 52-week study involving 234 adults with immune thrombocytopenia, romiplostim significantly increased the rate of platelet response compared to standard care, with a response rate 2.3 times higher (P<0.001).

Patients receiving romiplostim experienced fewer treatment failures and required splenectomy less often (11% vs. 30% for standard care, P<0.001), along with lower rates of bleeding events and improved quality of life.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Romiplostim or standard of care in patients with immune thrombocytopenia. Kuter, DJ., Rummel, M., Boccia, R., et al.[2016]

AMG 531 was generally well tolerated in a phase I study with 30 healthy Japanese men, showing safety profiles similar to placebo and only mild treatment-related adverse events reported.

The study demonstrated that AMG 531 effectively increased platelet counts, with significant increases observed in 4 out of 8 subjects at 1 microg/kg and 7 out of 8 at 2 microg/kg, indicating its potential efficacy for treating chronic immune thrombocytopenic purpura.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pharmacodynamics and pharmacokinetics of AMG 531, a thrombopoiesis-stimulating peptibody, in healthy Japanese subjects: a randomized, placebo-controlled study. Kumagai, Y., Fujita, T., Ozaki, M., et al.[2016]

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]

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]

References

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

Phase 2 study of efgartigimod, a novel FcRn antagonist, in adult patients with primary immune thrombocytopenia. [2021]

2. England pubmed.ncbi.nlm.nih.gov

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

3. England pubmed.ncbi.nlm.nih.gov

Efficacy and safety of the neonatal Fc receptor inhibitor efgartigimod in adults with primary immune thrombocytopenia (ADVANCE IV): a multicentre, randomised, placebo-controlled, phase 3 trial. [2023]

4. Germany pubmed.ncbi.nlm.nih.gov

Chronic immune thrombocytopenic purpura. New agents. [2016]

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

Romiplostim or standard of care in patients with immune thrombocytopenia. [2016]

6. England pubmed.ncbi.nlm.nih.gov

An open-label, unit dose-finding study of AMG 531, a novel thrombopoiesis-stimulating peptibody, in patients with immune thrombocytopenic purpura. [2016]

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

Pharmacodynamics and pharmacokinetics of AMG 531, a thrombopoiesis-stimulating peptibody, in healthy Japanese subjects: a randomized, placebo-controlled study. [2016]

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

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

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

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