Evaluating the Pharmacodynamic Noninferiority of Efgartigimod PH20 SC Administered Subcutaneously as Compared to Efgartigimod Administered Intravenously in Patients With Generalized Myasthenia Gravis

NCT ID: NCT04735432

Last Updated: 2023-02-28

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 110 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-02-05
• Study Completion Date: 2021-12-13

Brief Summary

The purpose of this study is to investigate the Pharmacodynamics (PD), Pharmacokinetics (PK), safety, tolerability, immunogenicity, and clinical efficacy of efgartigimod coformulated with recombinant human hyaluronidase PH20 (rHuPH20) as compared to efgartigimod IV infused in patients with generalized myasthenia gravis (gMG). The study duration is approximately 12 weeks. After screening, patients will be randomized to receive either efgartigimod infusions or efgartigimod PH20 subcutaneously (SC)

Detailed Description

Main objective of the trial: To demonstrate that the pharmacodynamic (PD) effect of injections of 1000 mg efgartigimod PH20 SC (efgartigimod co-formulated with recombinant humanhyaluronidase PH20 for subcutaneous administration), administered once weekly for 4 administrations, is NI (noninferior) to IV infusions of efgartigimod (efgartigimod formulation for intravenous infusion) at a dose of 10 mg/kg administered once weekly for 4 administrations.

Secondary objectives: To compare the PD effect of efgartigimod PH20 SC and efgartigimod IV over time; To evaluate the pharmacokinetics (PK) of efgartigimod PH20 SC and efgartigimod IV; To evaluate the safety, tolerability, and immunogenicity of efgartigimod PH20 SC and efgartigimod IV; To evaluate the clinical efficacy of efgartigimod PH20 SC and efgartigimod IV.

Conditions

Generalized Myasthenia Gravis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

efgartigimod PH20 SC

Patients receiving efgartigimod PH20 subcutaneous (SC) treatment

Group Type: EXPERIMENTAL

efgartigimod PH20 SC

Intervention Type: BIOLOGICAL

Subcutaneous injection with efgartigimod PH20 SC

efgartigimod

Patients receiving efgartigimod intravenous (IV) treatment

Group Type: EXPERIMENTAL

efgartigimod IV

Intervention Type: BIOLOGICAL

Intravenous infusion of efgartigimod

Interventions

efgartigimod PH20 SC

Subcutaneous injection with efgartigimod PH20 SC

Intervention Type: BIOLOGICAL

efgartigimod IV

Intravenous infusion of efgartigimod

Intervention Type: BIOLOGICAL

Eligibility Criteria

Inclusion Criteria

Bullet list of each inclusion criterium:

1. Must be capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.

2. At least 18 years of age at the time of signing the informed consent form.

3. Diagnosed with generalized Myasthenia Gravis (gMG) with confirmed documentation and supported by at least 1 of the following:

1. History of abnormal neuromuscular transmission demonstrated by single fiber electromyography or repetitive nerve stimulation

2. History of positive edrophonium chloride test

3. Demonstrated improvement in Myasthenia Gravis (MG) signs upon treatment with oral acetylcholinesterase (AChE) inhibitors as assessed by the treating physician

4. Meeting the clinical criteria as defined by the Myasthenia Gravis Foundation of America (MGFA) class II, III, IVa, or IVb

Exclusion Criteria

Bullet list of each exclusion criterium:

1. Are pregnant or lactating, or intend to become pregnant during the study or within 90 days after the last dose of Investigational Medicinal Product.

2. Has any of the following medical conditions:

1. Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection at screening

2. Any other known autoimmune disease that, in the opinion of the investigator, would interfere with an accurate assessment of clinical symptoms of myasthenia gravis or put the participant at undue risk.

3. History of malignancy unless deemed cured by adequate treatment with no evidence of reoccurrence for ≥3 years before the first administration of the IMP. Participants with the following cancers can be included at any time:

• adequately treated basal cell or squamous cell skin cancer
• carcinoma in situ of the cervix
• carcinoma in situ of the breast
• incidental histological findings of prostate cancer (TNM Classification of Malignant Tumors stage T1a or T1b).

4. Clinical evidence of other significant serious diseases, or the participant has had a recent major surgery, or who have any other condition that, in the opinion of the investigator, could confound the results of the study or put the participant at undue risk.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

argenx

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Investigator site 2 - US0010032

Carlsbad, California, United States

Investigator Site 41 - US0010004

Orange, California, United States

Investigator site 2 - US0010108

Boca Raton, Florida, United States

Investigator site 1 - US0010110

Port Charlotte, Florida, United States

Investigator Site 27 - US0010006

Tampa, Florida, United States

Investigator Site 47 - US0010113

Augusta, Georgia, United States

Investigator Site 42 - US0010015

Kansas City, Kansas, United States

Investigator Site 11 - US0010111

Amherst, New York, United States

Investigator Site 40 - US0010003

Chapel Hill, North Carolina, United States

Investigator Site 38 - US0010077

Durham, North Carolina, United States

Investigator Site 43 - 0010019

Cleveland, Ohio, United States

Investigator site 4 - US0010008

Cordova, Tennessee, United States

Investigator Site 28 - US0010066

Austin, Texas, United States

Investigator Site 46 - US0010009

Texas City, Texas, United States

Investigator site 5 - BE0320007

Ghent, East-Flanders, Belgium

Investigator site 13 - GE9950002

Tbilisi, , Georgia

Investigator site 12 - GE9950001

Tbilisi, , Georgia

Investigator site 14 - GE9950003

Tbilisi, , Georgia

Investigator Site 44 - GE9950004

Tbilisi, , Georgia

Investigator Site 45 - GE9950016

Tbilisi, , Georgia

Investigator Site 30 - DE490006

Berlin, , Germany

Investigator Site 29 - DE490009

Münster, , Germany

Investigator site 15 - HU0360013

Budapest, , Hungary

Investigator Site 16 - HU0360020

Debrecen, , Hungary

Investigator Site 17 - IT0390003

Milan, , Italy

Investigator Site 39 - IT0390008

Roma, , Italy

Investigator Site 31 - JP0810055

Sapporo, Hokkaido, Japan

Investigator Site 18 - JP0810002

Chiba, , Japan

Investigator site 6 - JPN0810004

Hanamaki, , Japan

Investigator Site 33 - JP0810058

Hiroshima, , Japan

Investigator Site 19 - JP0810007

Osaka, , Japan

Investigator Site 34 - JP0810005

Sendai, , Japan

Investigator Site 32 - JP0810059

Tokyo, , Japan

Investigator Site 20 - JP0810009

Tokyo, , Japan

Investigator Site 7 - NL0310001

Leiden, , Netherlands

Investigator Site 21 - PL0480001

Gdansk, , Poland

Investigator Site 8 - PL0480007

Katowice, , Poland

Investigator Site 9 - PL0480024

Krakow, , Poland

Investigator Site 22 - PL0480005

Krakow, , Poland

Investigator Site 23 - PL0480018

Lublin, , Poland

Investigator Site 24 - PL0480022

Warsaw, , Poland

Investigator Site 35 - RU0070002

Novosibirsk, , Russia

Investigator Site 36 - RU0070014

Saint Petersburg, , Russia

Investigator Site 37 - ES0340021

Barcelona, , Spain

Investigator Site 26 - ES0340038

Barcelona, , Spain

Investigator Site 25 - ES0340002

Madrid, , Spain

Investigator Site 10 - ES0340039

Valencia, , Spain

Countries

United States; Belgium; Georgia; Germany; Hungary; Italy; Japan; Netherlands; Poland; Russia; Spain

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

ARGX-113-2001

Identifier Type: -

Identifier Source: org_study_id