A Study of Efgartigimod PH20 SC in Children Between 2 and Less Than 18 Years of Age With Generalized Myasthenia Gravis

NCT06392386 | Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: ARGX-113-22072023-506159-12-00
• Study Type: interventional
• Target: 12
• Locations: 13 countries
• Sites: 20

Brief Summary

The purpose of this study is to measure the pharmacokinetics (PK), pharmacodynamics (PD), safety, tolerability, and immunogenicity of efgartigimod PH20 SC in pediatric participants with gMG aged 2 to \<18 years. The primary goal is to confirm an appropriate dose of efgartigimod PH20 SC for pediatric patients using PK and PD results from this study. Participants will receive injections of efgartigimod PH20 SC and will be monitored for safety until the end of the study. At the end of the follow-up period, eligible participants may roll over to an open-label extension (OLE) study. The participants will be in the study for up to 14 weeks.

Conditions

Generalized Myasthenia Gravis

Outcome Measures

Primary Outcomes (4)

• Efgartigimod serum concentrations as input for compartmental, model-driven analysis to determine age and size dependency of Clearance (CL)

  Up to 12 weeks

• Efgartigimod serum concentrations as input for compartmental, model-driven analysis to determine age and size dependency of Volume Distribution (Vd)

  Up to 12 weeks

• Total G immunoglobulins (IgG) levels as input for pharmacokinetics (PK)/pharmacodynamics (PD) modelling analysis

  Up to 12 weeks

• Anti-acetylcholine receptors antibodies (AChR-Ab) as input for pharmacokinetics (PK)/ pharmacodynamics (PD) modelling analysis

  Up to 12 weeks

Secondary Outcomes (26)

• Incidence of adverse events (AEs)

  Up to 14 weeks

• Severity of adverse events (AEs)

  Up to 14 weeks

• Incidence of serious adverse events (SAEs)

  Up to 14 weeks

• Severity of serious adverse events (SAEs)

  Up to 14 weeks

• Incidence of adverse events of special interest (AESI)

  Up to 14 weeks

Study Arms (1)

Efgartigimod PH20 SC

EXPERIMENTAL

Participants aged 12 to \<18 years receiving efgartigimod PH20 SC treatment

Biological: Efgartigimod PH20 SC

Interventions

Efgartigimod PH20 SC BIOLOGICAL

Subcutaneous injections

Efgartigimod PH20 SC

Eligibility Criteria

• Age: 2 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• The participant (and/or their legally authorized representative) understands the requirements of the study and is capable of providing written informed consent/assent and complying with protocol requirements
• The participant is aged 2 to \<18 years at the time of informed consent/assent
• The participant has been diagnosed with generalised Myasthenia Gravis that is supported by a physical examination and confirmed seropositivity for anti-acetylcholine receptor antibodies
• The participant has had an unsatisfactory response to immunosuppressants, corticosteroids, or acetylcholinesterase inhibitors but is on stable concomitant MG therapy. If receiving corticosteroids and/or immunosuppressants, must be on a stable dose for ≥1 month before screening
• The participant agrees to use birth control consistent with local regulations and people of child-bearing potential must have a negative blood pregnancy test at screening and a negative urine pregnancy test before receiving the study drug

You may not qualify if:

• Is a female adolescent of child-bearing potential who is pregnant and/or lactating or intends to become pregnant during their participation in the study
• Has worsening muscle weakness secondary to a concurrent infection or as a result of a medication
• Has a documented lack of clinical response to plasma exchange (PLEX)
• Received a live or live-attenuated vaccine within \<4 weeks before screening
• Received a thymectomy within 3 months before screening or is planning to get a thymectomy during their participation in the study
• Has a known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of generalised Myasthenia Gravis or puts the participant at undue risk
• History of malignancy, cancer, unless considered cured by adequate treatment with no evidence of recurrence for ≥3 years. Adequately treated participants with the following cancers can be included at any time: 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
• Clinically significant active infection that is not sufficiently resolved in the investigator's opinion or positive serum test at screening for active infection with any of the following: Hepatitis B virus (HBV), Hepatitis C virus (HCV), HIV
• Has a positive PCR test for SARS-CoV-2 at screening
• Has/had a clinically significant disease, had recent major surgery (within 3 months of screening) or intends to have major surgery during the study, or has/had any other medical condition that, in the investigator's opinion, would confound the results of the study or put the participant at undue risk
• Has received a different study drug in another clinical study within \<12 before screening
• Is currently participating in another interventional clinical study
• Has previously participated in an efgartigimod clinical study and received at least one dose of study drug
• Has a known hypersensitivity to study drug or any of its excipients
• Has a history of or current episode of alcohol, drug, or medication abuse as assessed by the investigator

Sponsors & Collaborators

• argenx: lead

Study Sites (20)

Carolinas HealthCare System Neurosciences Institute - Neurology

Charlotte, North Carolina, 28207, United States

RECRUITING

Neurology Rare Disease Center

Denton, Texas, 76208, United States

RECRUITING

University of Virginia

Charlottesville, Virginia, 22903, United States

RECRUITING

UZ Gent

Ghent, 9000, Belgium

RECRUITING

Childrens Hospital of Eastern Ontario

Ottawa, K1H 8L1, Canada

RECRUITING

Fakultni nemocnice Brno

Brno, 613 00, Czechia

RECRUITING

Fakultni nemocnice Ostrava

Ostrava, 708 00, Czechia

RECRUITING

AP-HM- Hôpital de La Timone

Marseille, 13385, France

RECRUITING

Universitätsklinikum Essen

Essen, 45147, Germany

RECRUITING

Hadassah Medical Center- Ein Kerem

Jerusalem, 9112001, Israel

RECRUITING

Schneider Children's Medical Center of Israel

Petah Tikvah, 4920235, Israel

RECRUITING

Tel Aviv Sourasky Medical Center Ichilov

Tel Aviv, 6423906, Israel

RECRUITING

Istituto G Gaslini Ospedale Pediatrico IRCCS

Genova, 16147, Italy

RECRUITING

Leiden University Medical Center

Leiden, 2333 ZA, Netherlands

RECRUITING

Uniwersyteckie Centrum Kliniczne w Gdansku

Gdansk, 80-211, Poland

RECRUITING

Neurologia Śląska Centrum Medyczne

Katowice, 40-689, Poland

RECRUITING

Centralny Szpital Kliniczny - Uniwersyteckie Centrum Kliniczne WUM

Warsaw, 02-097, Poland

RECRUITING

Hospital Universitari i Politecnic La Fe de Valencia

Valencia, 46026, Spain

RECRUITING

Hôpital Nestlé

Lausanne, 1011, Switzerland

RECRUITING

Oxford Children's Hospital

Oxford, OX3 9DU, United Kingdom

RECRUITING

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

Central Study Contacts

Sabine Coppieters, MD

CONTACT

857-350-4834; clinicaltrials@argenx.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 26, 2024
• First Posted: April 30, 2024
• Study Start: June 28, 2024
• Primary Completion (Estimated): September 30, 2026
• Study Completion (Estimated): September 30, 2026
• Last Updated: April 27, 2026

Record last verified: 2026-02

Locations

IT (1); NL (1); PL (3); ES (1); DE (1); GB (1); US (3); FR (1); CA (1); CH (1); BE (1); IL (3); CZ (2)