Testing Efgartigimod PH20 SC for generalized myasthenia gravis
Official title: A Study of Efgartigimod PH20 SC in Children Between 2 and Less Than 18 Years of Age With Generalized Myasthenia Gravis
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
- Phase 2
- 1 group
- One site, in Ottawa
- Recruiting
Interventions
- Biological therapy
Efgartigimod PH20 SC
Subcutaneous injections
Canadian Sites (1)
1 of 1 recruiting
- Recruiting
Childrens Hospital of Eastern Ontario
Ottawa
Eligibility Criteria
See who this study is looking for22 criteria
The trial’s own eligibility text, word for word from the registry. Only the trial site can say who takes part.
Inclusion
- +The participant (and/or their legally authorized representative) understands the requirements of the study and is capable of providing written informed consentInformed consentThe process of being told what taking part involves, then choosing freely.Read more →/assent and complying with protocolProtocolThe detailed plan a study must follow.Read more → 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 screeningScreeningThe checks done before joining, to see whether a study fits.Read more →
- +The participant agrees to use birth controlControl groupThe group a new treatment is measured against.Read more → 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
Exclusion
- −Has a known hypersensitivity to study drug or any of its excipients
- −Clinically significant active infection that is not sufficiently resolved in the investigatorPrincipal investigatorThe doctor or researcher responsible for running the study at a site.Read more →'s opinion or positive serum test at screeningScreeningThe checks done before joining, to see whether a study fits.Read more → for active infection with any of the following: Hepatitis B virus (HBV), Hepatitis C virus (HCV), HIV
- −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
- −Has a positive PCR test for SARS-CoV-2 at screening
- −Has received a different study drug in another clinical study within \<12 before screening
- −Is currently participating in another interventionalInterventional studyA study where participants are given something to see what happens.Read more → clinical study
- −Has previously participated in an efgartigimod clinical study and received at least one dose of study drug
- −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 protocolProtocolThe detailed plan a study must follow.Read more →)
- −The complete list of exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → can be found in the protocol.
- −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/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
In plain language
Assembled directly from this trial’s registry record. Every sentence traces to a field below — nothing here is generated or interpreted.
What this study is
This study is testing a treatment for a condition.
Phase 2Phase 2A middle-stage study looking at what a treatment does and watching for side effects.Read more →/3 — a combined study that runs the middle stage and the large comparison stage together.
From the trial registry
Built from these fields:
- designModule.designInfo.primaryPurpose
- designModule.phases
Who receives what
Everyone in this study is in one group and receives Efgartigimod PH20 SC.
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].label
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
How the study is run
There is only one group in this study, so there is no assignment to different treatments.
This study is open-labelOpen-labelEveryone knows which treatment is being given — nothing is hidden.Read more →: everyone knows which treatment is being given, including you and the study team.
From the trial registry
Built from these fields:
- designModule.designInfo.allocation
- designModule.designInfo.maskingInfo.masking
Is there a placebo?
This study does not list a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group.
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
Who the study is looking for
The study lists an age range of 2 years to 17 years.
The study is open to people of any sex.
The study does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
These are the criteria the study lists. Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part.
From the trial registry
Built from these fields:
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How big and how long
The study aims to enrol about 12 people.
The study is currently expected to finish around September 2026.
The main measurement is taken over: Up to 12 weeks.
From the trial registry
Built from these fields:
- designModule.enrollmentInfo.count
- statusModule.completionDateStruct.date
- outcomesModule.primaryOutcomes[].timeFrame
What the study measures
Efgartigimod serum concentrations as input for compartmental, model-driven analysis to determine age and size dependency of Clearance (CL) — measured over Up to 12 weeks.
Efgartigimod serum concentrations as input for compartmental, model-driven analysis to determine age and size dependency of Volume Distribution (Vd) — measured over Up to 12 weeks.
Total G immunoglobulins (IgG) levels as input for pharmacokineticsPharmacokineticsThe study of how the body absorbs, distributes, and clears a treatment.Read more → (PK)/pharmacodynamics (PD) modelling analysis — measured over Up to 12 weeks.
Anti-acetylcholine receptors antibodies (AChR-Ab) as input for pharmacokinetics (PK)/ pharmacodynamics (PD) modelling analysis — measured over Up to 12 weeks.
From the trial registry
Built from these fields:
- outcomesModule.primaryOutcomes[].measure
- outcomesModule.primaryOutcomes[].timeFrame
Source: NCT06392386 on ClinicalTrials.gov. The full registry text is further down this page — this summary never replaces it.
Not medical advice. What do these terms mean?
What is being tested — in plain terms
About Efgartigimod PH20 SCBiological
Subcutaneous injections
From the trial registry — its own words, unedited.
What a biological is here: A treatment made from or by living systems — such as antibodies, vaccines, or cell-based therapies.
Read the full explanation → · in clinical review
Common questions
Answered from this trial’s registry record. Where the record doesn’t say, these answers say so rather than filling the gap.
Am I eligible for this trial?
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part. Concord cannot make that determination, and neither can any tool that has not examined you.
What the study lists: an age range of 2 years to 17 years.
The full inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more → and exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → are published on this page, exactly as the study team wrote them.
The useful next step is to bring this trial to your doctor. Answering a few questions first gives them something concrete to review.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.eligibilityCriteria
Is there a placebo?
This study does not list a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group.
From the trial registry
- armsInterventionsModule.armGroups[].type
Would I know which treatment I am getting?
This study is open-labelOpen-labelEveryone knows which treatment is being given — nothing is hidden.Read more →: everyone knows which treatment is being given, including you and the study team.
There is only one group in this study, so there is no assignment to different treatments.
From the trial registry
- designModule.designInfo.maskingInfo.masking
- designModule.designInfo.allocation
Who can join?
The study lists an age range of 2 years to 17 years.
It is open to people of any sex.
It does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can confirm whether a particular person can take part.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How long would this take?
The study's main measurement is taken over: Up to 12 weeks.
The study as a whole is currently expected to finish around 2026-09-30.
How long any one person takes part can differ from the study length. The study team can tell you what the schedule looks like in practice.
From the trial registry
- outcomesModule.primaryOutcomes[].timeFrame
- statusModule.completionDateStruct.date
How many people are taking part?
The study aims to enrol about 12 people.
From the trial registry
- designModule.enrollmentInfo.count
Where is this happening?
This study lists one location: Ottawa, Canada.
Sites can open and close during a study, so confirm with the team before travelling.
From the trial registry
- trial_locations
About This Trial
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.
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See if this trial could fit youThis page is for informational purposes only and does not constitute medical advice. Clinical trial eligibility can only be determined by the trial site after proper screening. Trial information is sourced from ClinicalTrials.gov and may not reflect the most current status. Always consult your healthcare provider before making decisions about clinical trial participation.