Evaluating Long-term Safety of Efgartigimod Administered Intravenously and Efgartigimod PH20 Administered Subcutaneously in Children with Generalized Myasthenia Gravis

Last updated: February 28, 2025
Sponsor: argenx
Overall Status: Active - Enrolling

Phase

2/3

Condition

Polymyositis (Inflammatory Muscle Disease)

Neuropathy

Myasthenia Gravis Generalised

Treatment

Efgartigimod IV or Efgartigimod PH20 SC

Efgartigimod IV

Clinical Study ID

NCT05374590
ARGX-113-2008
2023-507379-23-00
  • Ages 2-18
  • All Genders

Study Summary

The purpose of this trial is to evaluate the long-term safety of efgartigimod IV and efgartigimod PH20 SC administered to participants with gMG in the antecedent studies, ARGX-113-2006 and ARGX-113-2207, respectively.

Eligibility Criteria

Inclusion

Inclusion Criteria:

Participants are eligible to be included in the trial only if all of the following criteria apply:

  1. The participant completed ARGX-113-2006 or ARGX-113-2207, defined as:

  2. The participant reached End of Trial in trial ARGX-113-2006 or End of Study inARGX-113-2207 and agreed to participate in the ARGX-113-2008 trial.

  3. The participant qualifies for retreatment in trial ARGX-113-2006, but cannotcomplete a Treatment Period (TP) and the required Intertreatment Period (IP)visits within the ARGX-113-2006 trial's timeframe.

  4. Either the participant or the participant's legally authorized representative canunderstand the requirements of the trial and provide written informedconsent/assent, and willingness and ability to comply with the trial protocolprocedures.

  5. Contraceptive use for sexually active participants should be consistent with localregulations for those participating in clinical studies. A participant is ofchildbearing potential if, in the opinion of the investigator, he/she isbiologically capable of having children.

  6. Contraceptive requirements for male participants are presented in Section 10.4.2.2.

  7. Female adolescents of childbearing potential (FAOCBP) must have a negativeurine pregnancy test at trial entry (TP1V1 or IP0V1)

Exclusion

Exclusion Criteria:

Participants are excluded from the trial if any of the following criteria apply:

  1. Female adolescents of childbearing potential (FAOCBP): Pregnancy or lactation, orthe participant intends to become pregnant during their participation in the study.

  2. Discontinued early from ARGX-113-2006 or ARGX-113-2207 treatment.

  3. A known hypersensitivity reaction to efgartigimod or any of its excipients.

  4. Any of the following medical conditions:

  5. Clinically significant uncontrolled chronic bacterial, viral, or fungalinfection at study entry not sufficiently resolved in the investigator'sopinion.

  6. Known autoimmune disease or any medical condition that would interfere with anaccurate assessment of clinical symptoms of generalized myasthenia gravis (gMG)or put the participant at undue risk

Study Design

Total Participants: 12
Treatment Group(s): 2
Primary Treatment: Efgartigimod IV or Efgartigimod PH20 SC
Phase: 2/3
Study Start date:
August 18, 2022
Estimated Completion Date:
September 30, 2028

Connect with a study center

  • Investigator site AT430010

    Vienna, 1060
    Austria

    Site Not Available

  • Investigator site BE320021

    Antwerp, 2650
    Belgium

    Active - Recruiting

  • UZ Antwerpen

    Antwerp, 2650
    Belgium

    Site Not Available

  • Universitair Ziekenhuis Antwerpen

    Edegem, 2650
    Belgium

    Site Not Available

  • AP-HM- Hôpital de La Timone

    Marseille, 13385
    France

    Site Not Available

  • Investigator site FR330032

    Marseille, 13005
    France

    Site Not Available

  • Investigator site FR330041

    Paris, 75015
    France

    Site Not Available

  • Investigator site GE9950017

    Tbilisi, 0177
    Georgia

    Site Not Available

  • Investigator site GE9950018

    Tbilisi, 0186
    Georgia

    Active - Recruiting

  • Investigator site DE0490034

    Berlin, 13353
    Germany

    Site Not Available

  • Investigator site DU490032

    Essen, 45147
    Germany

    Site Not Available

  • Investigator site IT390047

    Florence, 50139
    Italy

    Site Not Available

  • Investigator site IT390048

    Genova, 16147
    Italy

    Site Not Available

  • Investigator Site 03 - NL0310001

    Leiden, 2333
    Netherlands

    Active - Recruiting

  • Investigator Site NL0310001

    Leiden, 2333
    Netherlands

    Active - Recruiting

  • Leiden University Medical Center

    Leiden, 2333
    Netherlands

    Active - Recruiting

  • Leids Universitair Medisch Centrum

    Leiden, 2333 ZA
    Netherlands

    Site Not Available

  • Investigator site PL480035

    Gdańsk, 80-952
    Poland

    Site Not Available

  • Uniwersyteckie Centrum Kliniczne w Gdansku

    Gdańsk, 80-211
    Poland

    Site Not Available

  • Investigator Site 02 - PL0480038

    Katowice, 40-123
    Poland

    Active - Recruiting

  • Investigator Site PL0480038

    Katowice, 40-123
    Poland

    Active - Recruiting

  • Wielospecjalistyczna Poradnia Lekarska Synapsis

    Katowice, 40-123
    Poland

    Site Not Available

  • Centralny Szpital Kliniczny - Uniwersyteckie Centrum Kliniczne WUM

    Warszawa, 02-097
    Poland

    Site Not Available

  • Investigator Site 01 - PL0480034

    Warszawa, 02-097
    Poland

    Active - Recruiting

  • Investigator Site PL0480034

    Warszawa, 02-097
    Poland

    Active - Recruiting

  • Uniwersyteckie Centrum Kliniczne WUM, Centralny Szpital Kliniczny

    Warszawa, 02-097
    Poland

    Active - Recruiting

  • Investigator site ES340040

    Barcelona, 08950
    Spain

    Site Not Available

  • Investigator site ES340041

    Valencia, 46026
    Spain

    Site Not Available

  • Great Ormand Street Hospital for Children NHS Foundation Trust - Great Ormond Street Hospital - Pediatric Neurology

    London, WC1N 3JH
    United Kingdom

    Site Not Available

  • Investigator site GB440035

    London, WC1N 3JH
    United Kingdom

    Site Not Available

  • Investigator site UK0440027

    Manchester, M13 9WL
    United Kingdom

    Site Not Available

  • Investigator site GB440024

    Oxford, OX3 0AG
    United Kingdom

    Active - Recruiting

  • Investigator site UK0440024

    Oxford, OX3 9DU
    United Kingdom

    Active - Recruiting

  • Oxford University Hospitals NHS Foundation Trust - John Radcliffe Hospital Children's Hospital

    Oxford, OX3 9DU
    United Kingdom

    Site Not Available

  • Oxford University hospitals NHS Foundation Trust-Oxford Children's Hospital

    Oxford, OX3 9DU
    United Kingdom

    Active - Recruiting

  • Ann and Robert H Lurie Children's Hospital of Chicago - Main Hospital

    Chicago, Illinois 60611
    United States

    Site Not Available

  • Ann and Robert H. Lurie Childrens Hospital of Chicago

    Chicago, Illinois 60611
    United States

    Active - Recruiting

  • Investigator site US10120

    Chicago, Illinois 60611
    United States

    Active - Recruiting

  • Investigator site US10003

    Chapel Hill, North Carolina 27599
    United States

    Site Not Available

  • Investigator site US10142

    Charlottesville, Virginia 22903
    United States

    Active - Recruiting

  • University of Virginia

    Charlottesville, Virginia 22903
    United States

    Active - Recruiting

  • University of Virginia (UVA) Health - Developmental Pediatrics Clinic

    Charlottesville, Virginia 22903
    United States

    Site Not Available

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