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Generalized Myasthenia GravisPhase 2/3ADAPT Jr SCNCT06392386

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

A Phase 2/3 study testing Efgartigimod PH20 SC in people with Generalized Myasthenia Gravis, run by argenx at 22 sites in 14 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial 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. Read more about this study

Who is it for?
Ages 2 to 17 years
Conditions the study is for
Generalized Myasthenia Gravis
What is being tested?
Efgartigimod PH20 SC
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
12 participants
Where
22 sitesin 14 countries
Study dates
28 Jun 2024to Sep 2027
Phase
Phase 2/3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 2 to 17 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • 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

Who cannot join

  • 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
Show all 17Show fewer
  • 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
  • Use of some medications before screening (more information is found in the protocol)
  • The complete list of exclusion criteria can be found in the protocol.

Trial locations and contacts

22 sites in 14 countries. Contact the study team or a site near you to ask about taking part.

Sabine Coppieters, MD contact

857-350-4834

clinicaltrials@argenx.com

22 sites in 14 countries

United States3 sites
  • Carolinas HealthCare System Neurosciences Institute - Neurology

    Charlotte, North Carolina, 28207

    Active, not recruiting
  • University of Virginia

    Charlottesville, Virginia, 22903

    Active, not recruiting
  • Neurology Rare Disease Center

    Denton, Texas, 76208

    Active, not recruiting

Other trials in United States

Israel3 sites, 3 recruiting
  • Hadassah Medical Center- Ein Kerem

    Jerusalem, 9112001

    Talya Dor-Wollman, MD, 857-350-4834, clinicaltrials@argenx.com

    Recruiting
  • Schneider Children's Medical Center of Israel

    Petah Tikvah, 4920235

    Yoram Nevo, MD, 857-350-4834, clinicaltrials@argenx.com

    Recruiting
  • Tel Aviv Sourasky Medical Center Ichilov

    Tel Aviv, 6423906

    Liora Sagi, MD, 857-350-4834, clinicaltrials@argenx.com

    Recruiting

Other trials in Israel

Poland3 sites, 3 recruiting
  • Uniwersyteckie Centrum Kliniczne w Gdansku

    Gdansk, 80-211

    Maria Mazurkiewicz-Beldzinska, MD, +48583492390, mmazur@gumed.edu.pl

    Recruiting
  • Neurologia Śląska Centrum Medyczne

    Katowice, 40-689

    Marek Smilowski, MD, +48500282494, marek.smilowski2@gmail.com

    Recruiting
  • Centralny Szpital Kliniczny - Uniwersyteckie Centrum Kliniczne WUM

    Warsaw, 02-097

    Anna Kostera-Pruszczyk, MD, +48225992858, anna.kostera-pruszczyk@wum.edu.pl

    Recruiting

Other trials in Poland

Australia2 sites, 2 recruiting
  • Royal Children's Hospital Melbourne

    Melbourne, New South Wales, 3052

    Eppie Yiu, MD, 61 41 2599077, eppie.yiu@rch.org.au

    Recruiting
  • Sydney Children's Hospital

    Randwick, 2031

    Michelle Farrar, MD, +61293821549, michelle.farrar@health.nsw.gov.au

    Recruiting

Other trials in Australia

Czechia2 sites, 2 recruiting
  • Fakultni nemocnice Brno

    Brno, 613 00

    Ondrej Havlin, MD, +42532234581, havlin.ondrej@fnbrno.cz

    Recruiting
  • Fakultni nemocnice Ostrava

    Ostrava, 708 00

    Hana Medricka, MD, +420597373612, hana.medricka@fno.cz

    Recruiting

Other trials in Czechia

Belgium1 site, 1 recruiting
  • UZ Gent

    Ghent, 9000

    Nicolas Deconinck, MD, +32473966619, nicolas.deconinck@huderf.be

    Recruiting

Other trials in Belgium

Canada1 site, 1 recruiting
  • Childrens Hospital of Eastern Ontario

    Ottawa, K1H 8L1

    Hugh McMillan, MD, 857-350-4834, clinicaltrials@argenx.com

    Recruiting

Other trials in Canada

France1 site, 1 recruiting
  • AP-HM- Hôpital de La Timone

    Marseille, 13385

    Cecile Halbert, MD, +33491385605, cecile.halbert@ap-hm.fr

    Recruiting

Other trials in France

Germany1 site, 1 recruiting
  • Universitätsklinikum Essen

    Essen, 45147

    Adela Della Marina, MD, +492017233618, adela.dellamarina@uk-essen.de

    Recruiting

Other trials in Germany

Italy1 site, 1 recruiting
  • Istituto G Gaslini Ospedale Pediatrico IRCCS

    Genova, 16147

    Chiara Fiorillo, MD, +3901056363566, chiarafiorillo@edu.unige.it

    Recruiting

Other trials in Italy

Netherlands1 site, 1 recruiting
  • Leiden University Medical Center

    Leiden, 2333 ZA

    Erik Harmen Niks, MD, +310715262197, e.h.niks@lumc.nl

    Recruiting

Other trials in Netherlands

Spain1 site, 1 recruiting
  • Hospital Universitari i Politecnic La Fe de Valencia

    Valencia, 46026

    Teresa Sevilla Mantecón, MD, +34961244000, sevilla_ter@gva.es

    Recruiting

Other trials in Spain

Switzerland1 site, 1 recruiting
  • Hôpital Nestlé

    Lausanne, 1011

    David Jacquier, MD, 857-350-4834, clinicaltrials@argenx.com

    Recruiting

Other trials in Switzerland

United Kingdom1 site, 1 recruiting
  • Oxford Children's Hospital

    Oxford, OX3 9DU

    Sithara Ramdas, MD, 857-350-4834, clinicaltrials@argenx.com

    Recruiting

Other trials in United Kingdom

About this study

Official title: An Open-label, Uncontrolled Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Activity of Efgartigimod PH20 SC in Participants From 2 to Less Than 18 Years of Age With Generalized Myasthenia Gravis

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.

Being tested
Efgartigimod PH20 SC
Medical subject headings
Myasthenia Gravis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Efgartigimod PH20 SCBiological

    Subcutaneous injections

Study groups in NCT06392386
Study groupRoleWhat they receive
Efgartigimod PH20 SCExperimentalParticipants aged 12 to <18 years receiving efgartigimod PH20 SC treatment
Receives: Biological: Efgartigimod PH20 SC

What is being measured

The primary outcome is the main question the study is designed to answer.

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

    Measured: Up to 12 weeks

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

    Measured: Up to 12 weeks

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

    Measured: Up to 12 weeks

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

    Measured: Up to 12 weeks

Secondary outcomes (26)
  1. Incidence of adverse events (AEs)

    Measured: Up to 14 weeks

  2. Severity of adverse events (AEs)

    Measured: Up to 14 weeks

  3. Incidence of serious adverse events (SAEs)

    Measured: Up to 14 weeks

  4. Severity of serious adverse events (SAEs)

    Measured: Up to 14 weeks

  5. Incidence of adverse events of special interest (AESI)

    Measured: Up to 14 weeks

  6. Severity of adverse events of special interest (AESI)

    Measured: Up to 14 weeks

  7. Efgartigimod serum concentrations

    Measured: Up to 12 weeks

  8. Absolute values of total Immunoglobulin G (IgG) from blood samples

    Measured: Up to 12 weeks

  9. Change from baseline values of total Immunoglobulin G (IgG) from blood samples

    Measured: Up to 12 weeks

  10. Percentage change from baseline values of total Immunoglobulin G (IgG) from blood samples

    Measured: Up to 12 weeks

  11. Absolute values of anti-acetylcholine receptor antibodies (AChR-Ab) from blood samples

    Measured: Up to 12 weeks

  12. Change from baseline values of anti-acetylcholine receptor antibodies (AChR-Ab) from blood samples

    Measured: Up to 12 weeks

  13. Percentage change from baseline values of anti-acetylcholine receptor antibodies (AChR-Ab) from blood samples

    Measured: Up to 12 weeks

  14. Incidence of anti-drug antibodies (ADAs) against efgartigimod in serum samples

    Measured: Up to 12 weeks

  15. Prevalence of anti-drug antibodies (ADAs) against efgartigimod in serum samples

    Measured: Up to 12 weeks

  16. Incidence of antibodies against rHuPH20 in serum samples

    Measured: Up to 12 weeks

  17. Prevalence of antibodies against rHuPH20 in serum samples

    Measured: Up to 12 weeks

  18. Absolute value of total Myasthenia Gravis Activity of Daily Living (MG-ADL) score, appropriate for pediatric use

    Minimum value: 0 (no impairment); Maximum value: 24 (highest impairment)

    Measured: Up to 12 weeks

  19. Change from baseline of total Myasthenia Gravis Activity of Daily Living (MG-ADL) score, appropriate for pediatric use

    Minimum value: 0 (no impairment); Maximum value: 24 (highest impairment)

    Measured: Up to 12 weeks

  20. Absolute value of Quantitative Myasthenia Gravis (QMG) score

    Minimum value: 0 (no impairment); Maximum value: 39 (most severe impairment)

    Measured: Up to 12 weeks

  21. Change from baseline of Quantitative Myasthenia Gravis (QMG) score

    Minimum value: 0 (no impairment); Maximum value: 39 (most severe impairment)

    Measured: Up to 12 weeks

  22. Absolute value of EuroQoL 5 Dimensions Youth (EQ-5D-Y) score

    Measured: Up to 12 weeks

  23. Change from baseline value of Neuro-QoL Pediatric Fatigue Score

    Measured: Up to 12 weeks

  24. Change from baseline value of EuroQoL 5 Dimensions Youth (EQ-5D-Y) score

    Measured: Up to 12 weeks

  25. Change from baseline value of Clinical Global Impression of Improvement (CGI-I)

    Measured: Up to 12 weeks

  26. Changes in protective antibody titers to vaccines

    Measured: Up to 12 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted30 Apr 2024
  2. Study start28 Jun 2024
  3. Primary completionSep 2027 (estimated)
  4. Study completionSep 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2/3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
12 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06392386
Sponsor's protocol ID
ARGX-113-2207
Other IDs
2023-506159-12-00, 2023
Acronym
ADAPT Jr SC
Status last verified
Sep 2026
First submitted
26 Apr 2024
Last updated
28 Sep 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing Efgartigimod PH20 SC in people with Generalized Myasthenia Gravis, run by argenx at 22 sites in 14 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 28 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 22 sites in United States, Israel, Poland, Australia, Czechia and 9 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Sabine Coppieters, MD (857-350-4834, clinicaltrials@argenx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Efgartigimod serum concentrations as input for compartmental, model-driven analysis to determine age and size dependency of Clearance (CL) (time frame: Up to 12 weeks). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Sep 2026.

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