Efgartigimod in IVIG Dependent Myasthenia Gravis Patients

NCT06765161 | Recruiting Phase 3 DMC

• 1 other identifier: ESR-CA-NEU-96
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

This study is an open label, single center, prospective, 26 weeks study with descriptive analysis where IVIG is replaced by efgartigimod therapy. MG-ADL and MGQOL evaluations will occur weekly throughout the study to week 26.

Conditions

Myasthaenia Gravis

Keywords

Myasthenia gravis; Vyvgart; IVIG

Outcome Measures

Primary Outcomes (1)

• MG-ADL

  The percentage of patients who do not experience, at any time during the study, a 2 or more-point deterioration from their averaged baseline MG-ADL score that is sustained for a period of 4 or more consecutive weeks. (stable or improved patients)

  a sustained decrease persistent for at least 4 consecutive weeks over the 26 week period

Secondary Outcomes (3)

• QMG

  those who experience a sustained decrease for 4 consecutive weeks during the 26 week period

• MGQOL

  during the whole study period of 26 weeks

• incidence of patients who are still receiving efgartigimod by the end of the 26 weeks

  for the duration of the 26 week study

Study Arms (1)

treatment

EXPERIMENTAL

Efgartigimod infusion

Drug: efgartigimod administration

Interventions

efgartigimod administration DRUG

efgartigimod infusion

Also known as: Vyvgart

treatment

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• \. Signed informed consent. 2. Age 18-80 years 3. Acetylcholine receptor antibody positive, myasthenia gravis patients, with stable disease for the past four or more months. Stable disease is defined as no change in dosage or interval in IVIG treatments and without any significant change in clinical status.
• \. No modification or addition of NSISTs in the past six months 5. No modification or addition in corticosteroid therapy for the past three months 6. Myasthenia Gravis diagnosis was supported by abnormal neurotransmission test or history of improvement with AChE inhibitors.
• \. Receiving chronic regular IVIG treatments for myasthenia gravis for the past year or more.

You may not qualify if:

• \. Patients with previous rituxan or eculizumab treatment or plasma exchange within the past six months 2. Patients with previous thymectomy within the past 3months 3. Patients that have active Hepatitis B, are seropositive for Hepatitis C or HIV or have latent, untreated or active TB or any other significant active infection 4. Patients that have at screening a serum IgG less than 6.0gm/L or a history of chronic hypogammaglobulinemia from any cause.
• \. Patients that are pregnant or considering becoming pregnant in the next 6 months.
• \. Patients with severe renal impairment (eGFR less than 30ml/min) 7. Patients who in the opinion of the investigator should not participate in the study.

Sponsors & Collaborators

• Clinique Neuro-Outaouais: lead
• argenx: collaborator

Study Sites (1)

Clinique Neuro-Outaouais

Gatineau, Quebec, J8Y1W2, Canada

RECRUITING

Related Publications (6)

• Burns TM, Sadjadi R, Utsugisawa K, Gwathmey KG, Joshi A, Jones S, Bril V, Barnett C, Guptill JT, Sanders DB, Hobson-Webb L, Juel VC, Massey J, Gable KL, Silvestri NJ, Wolfe G, Cutter G, Nagane Y, Murai H, Masuda M, Farrugia ME, Carmichael C, Birnbaum S, Hogrel JY, Nafissi S, Fatehi F, Ou C, Liu W, Conaway M. International clinimetric evaluation of the MG-QOL15, resulting in slight revision and subsequent validation of the MG-QOL15r. Muscle Nerve. 2016 Dec;54(6):1015-1022. doi: 10.1002/mus.25198. Epub 2016 Nov 7.

  PMID: 27220659 BACKGROUND

• Barnett C, Katzberg H, Nabavi M, Bril V. The quantitative myasthenia gravis score: comparison with clinical, electrophysiological, and laboratory markers. J Clin Neuromuscul Dis. 2012 Jun;13(4):201-5. doi: 10.1097/CND.0b013e31824619d5.

  PMID: 22622164 BACKGROUND

• Howard JF Jr, Bril V, Vu T, Karam C, Peric S, De Bleecker JL, Murai H, Meisel A, Beydoun SR, Pasnoor M, Guglietta A, Van Hoorick B, Steeland S, T'joen C, Utsugisawa K, Verschuuren J, Mantegazza R; ADAPT+ Study Group. Long-term safety, tolerability, and efficacy of efgartigimod (ADAPT+): interim results from a phase 3 open-label extension study in participants with generalized myasthenia gravis. Front Neurol. 2024 Jan 17;14:1284444. doi: 10.3389/fneur.2023.1284444. eCollection 2023.

  PMID: 38318236 BACKGROUND

• Wolfe GI, Herbelin L, Nations SP, Foster B, Bryan WW, Barohn RJ. Myasthenia gravis activities of daily living profile. Neurology. 1999 Apr 22;52(7):1487-9. doi: 10.1212/wnl.52.7.1487.

  PMID: 10227640 BACKGROUND

• Howard JF Jr, Bril V, Vu T, Karam C, Peric S, Margania T, Murai H, Bilinska M, Shakarishvili R, Smilowski M, Guglietta A, Ulrichts P, Vangeneugden T, Utsugisawa K, Verschuuren J, Mantegazza R; ADAPT Investigator Study Group. Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo-controlled, phase 3 trial. Lancet Neurol. 2021 Jul;20(7):526-536. doi: 10.1016/S1474-4422(21)00159-9.

  PMID: 34146511 BACKGROUND

• Sesarman A, Vidarsson G, Sitaru C. The neonatal Fc receptor as therapeutic target in IgG-mediated autoimmune diseases. Cell Mol Life Sci. 2010 Aug;67(15):2533-50. doi: 10.1007/s00018-010-0318-6. Epub 2010 Mar 9.

  PMID: 20217455 BACKGROUND

MeSH Terms

Conditions

Myasthenia Gravis

Interventions

efgartigimod alfa

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

Study Officials

• François Jacques, Doctor

  Clinique Neuro-Outaouais

  PRINCIPAL INVESTIGATOR

Central Study Contacts

François Jacques, Doctor

CONTACT

819-777-2500; francois.jacques@neuro-outaouais.ca

Victorine Sikati Foko, Nurse

CONTACT

8197772500

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Neurologist

Model Details: Open label

Study Record Dates

• First Submitted: January 3, 2025
• First Posted: January 9, 2025
• Study Start: February 6, 2025
• Primary Completion (Estimated): January 6, 2027
• Study Completion (Estimated): January 6, 2027
• Last Updated: July 29, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1)