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

NCT04735432 | Completed Phase 3 Results DMC FDA Drug

• 1 other identifier: ARGX-113-2001
• Study Type: interventional
• Target: 110
• Locations: 11 countries
• Sites: 47

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)

Conditions

Generalized Myasthenia Gravis

Outcome Measures

Primary Outcomes (1)

• Percent Change From Baseline in Total IgG Levels at Day 29 (mITT Analysis Set)

  ANCOVA Analysis of Percent Change From Baseline in Total IgG Level at Day 29 (ie, 7 days after the fourth IV or SC administration).

  From week 0 to week 4

Secondary Outcomes (13)

• Percent Change From Baseline in Total IgG Levels Over Time (mITT Analysis Set)

  From baseline to week 10

• Percent Change From Baseline in AChR-Ab Levels Over Time in AChR- Ab Positive Patients (mITT Analysis Set)

  From baseline to week 10

• Percent Change From Baseline in IgG Subtype Levels Over Time (mITT Analysis Set)

  Baseline to week 10

• AUEC of the Percent Change From Baseline in Total IgG Level (mITT Analysis Set)

  From baseline to week 10

• Еfgartigimod IV and PH20 SC Serum Pharmacokinetic Parameter Ctrough

  From Week 1 to Week 4.

Study Arms (2)

efgartigimod PH20 SC

EXPERIMENTAL

Patients receiving efgartigimod PH20 subcutaneous (SC) treatment

Biological: efgartigimod PH20 SC

efgartigimod

EXPERIMENTAL

Patients receiving efgartigimod intravenous (IV) treatment

Biological: efgartigimod IV

Interventions

efgartigimod PH20 SC BIOLOGICAL

Subcutaneous injection with efgartigimod PH20 SC

efgartigimod PH20 SC

efgartigimod IV BIOLOGICAL

Intravenous infusion of efgartigimod

efgartigimod

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• At least 18 years of age at the time of signing the informed consent form.
• Diagnosed with generalized Myasthenia Gravis (gMG) with confirmed documentation and supported by at least 1 of the following:
• History of abnormal neuromuscular transmission demonstrated by single fiber electromyography or repetitive nerve stimulation
• History of positive edrophonium chloride test
• Demonstrated improvement in Myasthenia Gravis (MG) signs upon treatment with oral acetylcholinesterase (AChE) inhibitors as assessed by the treating physician
• Meeting the clinical criteria as defined by the Myasthenia Gravis Foundation of America (MGFA) class II, III, IVa, or IVb

You may not qualify if:

• Are pregnant or lactating, or intend to become pregnant during the study or within 90 days after the last dose of Investigational Medicinal Product.
• Has any of the following medical conditions:
• Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection at screening
• 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.
• 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).
• 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.

Sponsors & Collaborators

• argenx: lead

Study Sites (47)

Investigator site 2 - US0010032

Carlsbad, California, 92011, United States

Location

Investigator Site 41 - US0010004

Orange, California, 92868, United States

Location

Investigator site 2 - US0010108

Boca Raton, Florida, 33428, United States

Location

Investigator site 1 - US0010110

Port Charlotte, Florida, 33952, United States

Location

Investigator Site 27 - US0010006

Tampa, Florida, 41076, United States

Location

Investigator Site 47 - US0010113

Augusta, Georgia, 30912, United States

Location

Investigator Site 42 - US0010015

Kansas City, Kansas, 66205, United States

Location

Investigator Site 11 - US0010111

Amherst, New York, 14226, United States

Location

Investigator Site 40 - US0010003

Chapel Hill, North Carolina, 27514, United States

Location

Investigator Site 38 - US0010077

Durham, North Carolina, 27710, United States

Location

Investigator Site 43 - 0010019

Cleveland, Ohio, 44195, United States

Location

Investigator site 4 - US0010008

Cordova, Tennessee, 38018, United States

Location

Investigator Site 28 - US0010066

Austin, Texas, 78756, United States

Location

Investigator Site 46 - US0010009

Texas City, Texas, 78229, United States

Location

Investigator site 5 - BE0320007

Ghent, East-Flanders, 9000, Belgium

Location

Investigator site 13 - GE9950002

Tbilisi, 0112, Georgia

Location

Investigator site 12 - GE9950001

Tbilisi, 0114, Georgia

Location

Investigator site 14 - GE9950003

Tbilisi, 0114, Georgia

Location

Investigator Site 44 - GE9950004

Tbilisi, 0160, Georgia

Location

Investigator Site 45 - GE9950016

Tbilisi, 0160, Georgia

Location

Investigator Site 30 - DE490006

Berlin, 10117, Germany

Location

Investigator Site 29 - DE490009

Münster, 48149, Germany

Location

Investigator site 15 - HU0360013

Budapest, 1082, Hungary

Location

Investigator Site 16 - HU0360020

Debrecen, 4032, Hungary

Location

Investigator Site 17 - IT0390003

Milan, 20133, Italy

Location

Investigator Site 39 - IT0390008

Roma, 00189, Italy

Location

Investigator Site 31 - JP0810055

Sapporo, Hokkaido, 063-0005, Japan

Location

Investigator Site 18 - JP0810002

Chiba, 260-8677, Japan

Location

Investigator site 6 - JPN0810004

Hanamaki, 025-0082, Japan

Location

Investigator Site 33 - JP0810058

Hiroshima, 0810058, Japan

Location

Investigator Site 19 - JP0810007

Osaka, 565-0871, Japan

Location

Investigator Site 34 - JP0810005

Sendai, 983-8520, Japan

Location

Investigator Site 32 - JP0810059

Tokyo, 143-8541, Japan

Location

Investigator Site 20 - JP0810009

Tokyo, 160-0023, Japan

Location

Investigator Site 7 - NL0310001

Leiden, 2333, Netherlands

Location

Investigator Site 21 - PL0480001

Gdansk, 80-952, Poland

Location

Investigator Site 8 - PL0480007

Katowice, 40-123, Poland

Location

Investigator Site 9 - PL0480024

Krakow, 31-2002, Poland

Location

Investigator Site 22 - PL0480005

Krakow, 31-505, Poland

Location

Investigator Site 23 - PL0480018

Lublin, 20-093, Poland

Location

Investigator Site 24 - PL0480022

Warsaw, 02-097, Poland

Location

Investigator Site 35 - RU0070002

Novosibirsk, 630087, Russia

Location

Investigator Site 36 - RU0070014

Saint Petersburg, 194354, Russia

Location

Investigator Site 37 - ES0340021

Barcelona, 08035, Spain

Location

Investigator Site 26 - ES0340038

Barcelona, 08041, Spain

Location

Investigator Site 25 - ES0340002

Madrid, 28046, Spain

Location

Investigator Site 10 - ES0340039

Valencia, 46026, Spain

Location

MeSH Terms

Conditions

Myasthenia Gravis

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Limitations and Caveats

None reported.

Results Point of Contact

• Title: Regulatory Manager
• Organization: argenx BV

regulatory@argenx.com

+32 9 310 34 00

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 26, 2021
• First Posted: February 3, 2021
• Study Start: February 5, 2021
• Primary Completion: November 2, 2021
• Study Completion: December 13, 2021
• Last Updated: February 28, 2023
• Results First Posted: February 28, 2023

Record last verified: 2023-01

Data Sharing

• IPD Sharing: Will not share

Locations

GE (5); JP (8); IT (2); HU (2); NL (1); PL (6); DE (2); US (14); ES (4); RU (2); BE (1)