Generalized Myasthenia GravisPhase 2/3ADAPT JrNCT04833894
Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis
A Phase 2/3 study testing Efgartigimod IV in people with Generalized Myasthenia Gravis, run by argenx at 25 sites in 12 countries.
Trial summary
The purpose of this trial is to investigate the PK, PD, safety, and activity of efgartigimod IV in children and adolescents aged from 2 to less than 18 years of age with gMG. Trial details include: The maximum trial duration for each individual participant will be approximately 28 weeks The treatment duration will be… Read more about this study
- Who is it for?
- Ages 2 to 18 years
- Conditions the study is for
- Generalized Myasthenia Gravis
- What is being tested?
- Efgartigimod IV
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 25 sitesin 12 countries
- Study dates
- 26 Oct 2021to Mar 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 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Ability of the participant and/or his/her legally authorized representative to understand the requirements of the trial and provide written informed consent/assent, if applicable (including consent/assent for the use and disclosure of research-related health information), willingness and ability to comply with the trial protocol procedures (including attending the required trial visits).
- Male or female participants between 2 to less than 18 years of age at the time of providing informed consent/assent. Age groups are enrolled in a staggered fashion respectively: 6 participants in the 12 to less than 18 years of age group followed by 6 participants in the 2 to less than 12 years of age group at the time of providing informed consent/assent.
- Diagnosed with Generalized Myasthenia Gravis (gMG) with confirmed documentation
- Meeting the clinical criteria as defined by the Myasthenia Gravis Foundation of America (MGFA) class II, III, and IVa.
- Eligible participants should have an unsatisfactory response (efficacy and/or safety) to immunosuppressants, steroids or acetylcholinesterase (AChE) inhibitors and should be on stable concomitant gMG therapy of adequate duration before screening.
- Positive serologic test for acetylcholine receptor (anti-AChR) antibodies at screening (for younger participants (<15kg) historical values can be used).
Show all 9Show fewer
- Contraceptive use should be consistent with local regulations regarding the methods of contraception for those participating in clinical trials. A subject is of childbearing potential if, in the opinion of the investigator, he/she is biologically capable of having children and is sexually active.
- Male participants: Male participants must agree to not donate sperm from of providing informed consent/assent until they have completed the trial.
- Female participants: Female adolescents of childbearing potential must have a negative serum pregnancy test at screening and a negative urine pregnancy test at baseline before investigational medicinal product (IMP) can be administered.
Who cannot join
- Participants with MGFA class I, IVb, and V.
- Female adolescents of childbearing potential: Pregnancy or lactation, or the participant intends to become pregnant during the trial or within 90 days after the last dose of IMP.
- Has any of the following medical conditions:
- Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection at screening.
- Any other known autoimmune disease that, in the opinion of the investigator, would interfere with an accurate assessment of clinical symptoms of myasthenia gravis or put the participant at undue risk.
- History of malignancy unless deemed cured by adequate treatment with no evidence of recurrence for ≥3 years before the first administration of IMP. Participants with the following cancers can be included at any time: Adequately treated 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
Show all 18Show fewer
- Clinical evidence of other significant serious diseases, or have had a recent major surgery, or who have any other condition that, in the opinion of the investigator, could confound the results of the trial or put the participant at undue risk
- Worsening muscle weakness secondary to concurrent infections or medications (aminoglycosides, fluoro-quinolones, beta-blockers, etc).
- A documented lack of clinical response to plasma exchange (PLEX).
- Received a live or live-attenuated vaccine fewer than 28 days before screening. Receiving an inactivated, subunit, polysaccharide, or conjugate vaccine any time before screening is not exclusionary.
- Received a thymectomy <3 months before screening or 1 is planned to be performed during the trial period.
- The following results from these diagnostic assessments will be considered exclusionary:
- Positive serum test at screening for an active viral infection with any of the following conditions: Hepatitis B virus (HBV) that is indicative of an acute or chronic infection; Hepatitis C virus (HCV) based on HCV antibody assay; Positive HIV serology at screening; Positive nasopharyngeal swab polymerase chain reaction (PCR) test for severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) at screening.
- Using the following prior or concomitant therapies: Use of an investigational product within 3 months or 5 half-lives (whichever is longer) before the first dose of IMP, Use of any monoclonal antibody within the 6 months before the first dose of IMP, Use of intravenous immunoglobulin (IVIg), administered subcutaneously or intramuscularly, or PLEX within 4 weeks before screening.
- Total immunoglobulin (IgG) levels <6 g/L below the lower limit of normal (LLN) according to the reference ranges of the central laboratory for participant by sex and age at screening.
- A known hypersensitivity reaction to efgartigimod or any of its excipients.
- Current participation in another interventional clinical trial or previous participation in an efgartigimod trial with at least 1 dose of IMP received.
- History (within 12 months of screening) of current alcohol, drug, or medication abuse as assessed by the investigator.
Trial locations and contacts
25 sites in 12 countries.
25 sites in 12 countries
United States3 sites
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599
University of Virginia (UVA) Health - Developmental Pediatrics Clinic
Charlottesville, Virginia, 22903
Ann and Robert H Lurie Children's Hospital of Chicago - Main Hospital
Chicago, Illinois, 60611
Italy3 sites
Azienda Ospedaliera Universitaria Policlinico Consorziale Di Bari
Bari, 70124
Azienda Ospedaliero Universitaria A. Meyer
Florence, 50139
Ospedale Giannina Gaslini
Genova, 16147
Poland3 sites
Uniwersyteckie Centrum Kliniczne
Gdansk, Woj. Pomorskie, 80-952
Wielospecjalistyczna Poradnia Lekarska Synapsis
Katowice, Woj. Slaskie, 40-123
Centralny Szpital Kliniczny - Uniwersyteckie Centrum Kliniczne WUM
Warsaw, 02-097
United Kingdom3 sites
Great Ormand Street Hospital for Children NHS Foundation Trust - Great Ormond Street Hospital
London, WC1N 3JH
Manchester University NHS Foundation Trust - Royal Manchester Children's Hospital
Manchester, M13 9WL
Oxford University Hospitals NHS Foundation Trust - John Radcliffe Hospital Children's Hospital
Oxford, OX3 9DU
Canada2 sites
Alberta Childrens Hospital
Calgary, T3B 6A8
British Columbia Children's Hospital
Vancouver, V6H 3N1
France2 sites
AP-HM - Hopital de la Timone
Marseille, 13385
Assistance Publique Hopitaux de Paris (AP-HP) - Hopital Necker-Enfants Malades
Paris, 75015
Georgia2 sites
Vian - M. Iashvili Children's Central Hospital
Tbilisi, 121
Tbilisi State Medical University - Givi Zhvania Pediatric Academic Clinic
Tbilisi, 159
Germany2 sites
Charite Universitaetsmedizin Berlin - Campus Virchow-Klinikum - Sozialpadiatrisches Zentrum
Berlin, 13353
Universitätsklinikum Essen
Essen, 45147
Spain2 sites
Hospital Sant Joan de Deu
Esplugues de Llobregat, 08950
Hospital Universitari i Politecnic La Fe de Valencia
Valencia, 46026
About this study
Official title: Open-label Uncontrolled Trial to Evaluate Pharmacokinetics, Pharmacodynamics, Safety, and Activity of Efgartigimod in Children From 2 to Less Than 18 Years of Age With Generalized Myasthenia Gravis
The purpose of this trial is to investigate the PK, PD, safety, and activity of efgartigimod IV in children and adolescents aged from 2 to less than 18 years of age with gMG.
Trial details include:
- The maximum trial duration for each individual participant will be approximately 28 weeks
- The treatment duration will be 8 weeks for the dose-confirmatory part (Part A) and 18 weeks for the treatment response-confirmatory part (Part B)
- Conditions
- Generalized Myasthenia Gravis
- Being tested
- Efgartigimod IV
- 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 IVBiological
Intravenous infusion of Efgartigimod
| Study group | Role | What they receive |
|---|---|---|
| Efgartigimod | Experimental | Patients receiving efgartigimod intravenous (IV) treatment Receives: Biological: Efgartigimod IV |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efgartigimod concentrations as input for compartmental, model-driven analysis to determine (age and size dependency of) Clearance (CL)
Blood samples will be collected from each participant for measurement of serum concentrations of efgartigimod
Measured: up to 26 weeks
Efgartigimod concentrations as input for compartmental, model-driven analysis to determine (age and size dependency of) Volume of Distribution (Vd)
Blood samples will be collected from each participant for measurement of serum concentrations of efgartigimod
Measured: up to 26 weeks
Total Immunoglobulin G (IgG) levels as input for pharmacokinetics (PK) and pharmacodynamics (PD) modeling analysis
Total Immunoglobulin G levels will be measured from blood samples
Measured: up to 26 weeks
Anti-acetylcholine receptors antibodies (AChR-Ab) as input for pharmacokinetics (PK) and pharmacodynamics (PD) modeling analysis
Total Immunoglobulin G (IgG) levels will be measured from blood samples
Measured: up to 26 weeks
Secondary outcomes (19)
Incidence and severity of adverse events (AEs), serious adverse events (SAEs) and adverse events of special interest (AESIs)
Measured: up to 28 weeks
Efgartigimod serum concentrations from blood samples
Measured: up to 26 weeks
Absolute values of levels of total Immunoglobulin G (IgG) from blood samples
Measured: up to 26 weeks
Change from baseline of levels of total Immunoglobulin G (IgG) from blood samples
Measured: up to 26 weeks
Percentage change from baseline of total Immunoglobulin G (IgG) from blood samples
Measured: up to 26 weeks
Absolute values of anti-acetylcholine receptor antibodies (AChR-Ab) from blood samples
Measured: up to 26 weeks
Change from baseline of anti-acetylcholine receptor antibodies (AChR-Ab) from blood samples
Measured: up to 26 weeks
Percentage change from baseline of anti-acetylcholine receptor antibodies (AChR-Ab) from blood samples
Measured: up to 26 weeks
Incidence of anti-drug antibodies (ADAs) against efgartigimod in serum samples
Measured: up to 28 weeks
Prevalence of anti-drug antibodies (ADAs) against efgartigimod in serum samples
Measured: up to 28 weeks
Absolute values of total Myasthenia Gravis Activity of Daily Living (MG-ADL) score. Total score can range from 0 to 24, with higher total scores indicating more impairment.
Measured: up to 26 weeks
Change from baseline of total Myasthenia Gravis Activity of Daily Living (MG-ADL) score. Total score can range from 0 to 24, with higher total scores indicating more impairment.
Measured: up to 26 weeks
Absolute values of total Quantitative Myasthenia Gravis Score (QMG score). The total possible score is 39, where higher total scores indicate more severe impairments.
Measured: up to 26 weeks
Change from baseline of total Myasthenia Gravis Score (QMG score). The total possible score is 39, where higher total scores indicate more severe impairments.
Measured: up to 26 weeks
Absolute values of total score EuroQoL 5 Dimensions Youth (EQ-5D-Y)
Description of the participant's health state is done by digits for 5 dimensions combined in a 5-digit number. A unique health state is defined by combining 1 level from each of the 5 dimensions. Each state is referred to in terms of a 5-digit code, whereas code 11111 would indicate no problems in any of the 5 dimensions and 33333 would indicate worst problems in any of the 5 dimensions.
Measured: up to 26 weeks
Change from baseline of total score EuroQoL 5 Dimensions Youth (EQ-5D-Y)
Description of the participant's health state is done by digits for 5 dimensions combined in a 5-digit number. A unique health state is defined by combining 1 level from each of the 5 dimensions. Each state is referred to in terms of a 5-digit code, whereas code 11111 would indicate no problems and 33333 would indicate worst problems in any of the 5 dimensions.
Measured: up to 26 weeks
Values of Neurological Quality of Life (Neuro-QoL) pediatric fatigue questionnaire
Measured: up to 26 weeks
Change from baseline of Neurological Quality of Life (Neuro-QoL) pediatric fatigue questionnaire
Measured: up to 26 weeks
Change in protective antibody titers to vaccines received before or during the trial from blood samples
Measured: up to 28 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Apr 2021
- Study start26 Oct 2021
- Primary completionMar 2027 (estimated)
- Study completionMar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Generalized Myasthenia GravisStudies of the same condition, open and under way.
- More trials run by argenxThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- argenx
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04833894
- Sponsor's protocol ID
- ARGX-113-2006
- Other IDs
- 2024-513854-31-00
- Acronym
- ADAPT Jr
- Status last verified
- Feb 2026
- First submitted
- 16 Mar 2021
- Last updated
- 29 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 29 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open-label Uncontrolled Trial to Evaluate Pharmacokinetics, Pharmacodynamics, Safety, and Activity of Efgartigimod in Children From 2 to Less Than 18 Years of Age With Generalized Myasthenia Gravis
- ClinicalTrials.gov ID
- NCT04833894
- Acronym / sponsor ID
- ADAPT Jr / ARGX-113-2006
- Conditions
- Generalized Myasthenia Gravis
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 15 participants (actual)
- Lead sponsor
- argenxIndustry (pharma, biotech, devices)
- Study start
- 26 Oct 2021 (actual)
- Primary completion
- Mar 2027 (estimated)
- Study completion
- Mar 2027 (estimated)
- First posted
- 6 Apr 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this trial is to investigate the PK, PD, safety, and activity of efgartigimod IV in children and adolescents aged from 2 to less than 18 years of age with gMG.
Trial details include:
- The maximum trial duration for each individual participant will be approximately 28 weeks
- The treatment duration will be 8 weeks for the dose-confirmatory part (Part A) and 18 weeks for the treatment response-confirmatory part (Part B)
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Efgartigimod | Experimental | Biological: Efgartigimod IVPatients receiving efgartigimod intravenous (IV) treatment |
Who may qualify
- Ages
- 2 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (9 listed)
- Ability of the participant and/or his/her legally authorized representative to understand the requirements of the trial and provide written informed consent/assent, if applicable (including consent/assent for the use and disclosure of research-related health information), willingness and ability to comply with the trial protocol procedures (including attending the required trial visits).
- Male or female participants between 2 to less than 18 years of age at the time of providing informed consent/assent. Age groups are enrolled in a staggered fashion respectively: 6 participants in the 12 to less than 18 years of age group followed by 6 participants in the 2 to less than 12 years of age group at the time of providing informed consent/assent.
- Diagnosed with Generalized Myasthenia Gravis (gMG) with confirmed documentation
- Meeting the clinical criteria as defined by the Myasthenia Gravis Foundation of America (MGFA) class II, III, and IVa.
- Eligible participants should have an unsatisfactory response (efficacy and/or safety) to immunosuppressants, steroids or acetylcholinesterase (AChE) inhibitors and should be on stable concomitant gMG therapy of adequate duration before screening.
Key exclusion criteria (18 listed)
- Participants with MGFA class I, IVb, and V.
- Female adolescents of childbearing potential: Pregnancy or lactation, or the participant intends to become pregnant during the trial or within 90 days after the last dose of IMP.
- Has any of the following medical conditions:
- Worsening muscle weakness secondary to concurrent infections or medications (aminoglycosides, fluoro-quinolones, beta-blockers, etc).
- A documented lack of clinical response to plasma exchange (PLEX).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Efgartigimod IVIntravenous infusion of Efgartigimod | Biological | Efgartigimod |
Primary outcome
Efgartigimod concentrations as input for compartmental, model-driven analysis to determine (age and size dependency of) Clearance (CL)
Time frame: up to 26 weeks
Blood samples will be collected from each participant for measurement of serum concentrations of efgartigimod
Efgartigimod concentrations as input for compartmental, model-driven analysis to determine (age and size dependency of) Volume of Distribution (Vd)
Time frame: up to 26 weeks
Blood samples will be collected from each participant for measurement of serum concentrations of efgartigimod
Total Immunoglobulin G (IgG) levels as input for pharmacokinetics (PK) and pharmacodynamics (PD) modeling analysis
Time frame: up to 26 weeks
Total Immunoglobulin G levels will be measured from blood samples
Anti-acetylcholine receptors antibodies (AChR-Ab) as input for pharmacokinetics (PK) and pharmacodynamics (PD) modeling analysis
Time frame: up to 26 weeks
Total Immunoglobulin G (IgG) levels will be measured from blood samples
19 secondary outcomes
Incidence and severity of adverse events (AEs), serious adverse events (SAEs) and adverse events of special interest (AESIs)
Time frame: up to 28 weeks
Efgartigimod serum concentrations from blood samples
Time frame: up to 26 weeks
Absolute values of levels of total Immunoglobulin G (IgG) from blood samples
Time frame: up to 26 weeks
Change from baseline of levels of total Immunoglobulin G (IgG) from blood samples
Time frame: up to 26 weeks
Percentage change from baseline of total Immunoglobulin G (IgG) from blood samples
Time frame: up to 26 weeks
Absolute values of anti-acetylcholine receptor antibodies (AChR-Ab) from blood samples
Time frame: up to 26 weeks
Change from baseline of anti-acetylcholine receptor antibodies (AChR-Ab) from blood samples
Time frame: up to 26 weeks
Percentage change from baseline of anti-acetylcholine receptor antibodies (AChR-Ab) from blood samples
Time frame: up to 26 weeks
Incidence of anti-drug antibodies (ADAs) against efgartigimod in serum samples
Time frame: up to 28 weeks
Prevalence of anti-drug antibodies (ADAs) against efgartigimod in serum samples
Time frame: up to 28 weeks
Absolute values of total Myasthenia Gravis Activity of Daily Living (MG-ADL) score. Total score can range from 0 to 24, with higher total scores indicating more impairment.
Time frame: up to 26 weeks
Change from baseline of total Myasthenia Gravis Activity of Daily Living (MG-ADL) score. Total score can range from 0 to 24, with higher total scores indicating more impairment.
Time frame: up to 26 weeks
Absolute values of total Quantitative Myasthenia Gravis Score (QMG score). The total possible score is 39, where higher total scores indicate more severe impairments.
Time frame: up to 26 weeks
Change from baseline of total Myasthenia Gravis Score (QMG score). The total possible score is 39, where higher total scores indicate more severe impairments.
Time frame: up to 26 weeks
Absolute values of total score EuroQoL 5 Dimensions Youth (EQ-5D-Y)
Time frame: up to 26 weeks
Change from baseline of total score EuroQoL 5 Dimensions Youth (EQ-5D-Y)
Time frame: up to 26 weeks
Values of Neurological Quality of Life (Neuro-QoL) pediatric fatigue questionnaire
Time frame: up to 26 weeks
Change from baseline of Neurological Quality of Life (Neuro-QoL) pediatric fatigue questionnaire
Time frame: up to 26 weeks
Change in protective antibody titers to vaccines received before or during the trial from blood samples
Time frame: up to 28 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Oct 2026.
- Individual participant dataSharing planned: Undecided
Related trials for Generalized Myasthenia Gravis
Active, not recruitingPhase 3
An Extension Study Evaluating 6-week Treatment Cycles of Rozanolixizumab in Pediatric Study Participants With Generalized Myasthenia Gravis
Generalized Myasthenia Gravis
RecruitingPhase 2/3
A Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis
Generalized Myasthenia Gravis
Recruiting
Holistic Assessment and Remote Digital MONitoring of mYasthenia Gravis Via MyoSense 360
Myasthenia Gravis, Generalized
Not yet recruitingEarly Phase 1
A Clinical Study to Evaluate the Safety and Efficacy of GT802 Injection in the Treatment of Refractory Generalized Myasthenia Gravis
Generalized Myasthenia Gravis (gMG)
RecruitingPhase 2
ADAPT Forward 2 - ISA2 - a Study to Evaluate the Safety, Tolerability and Efficacy of Empasiprubart IV Monotherapy in Participants With AChR-Ab Seropositive Generalized Myasthenia Gravis
AChR-Ab Seropositive Generalized Myasthenia Gravis, Myasthenia Gravis (MG)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
25 sites in 12 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Efgartigimod IV in people with Generalized Myasthenia Gravis, run by argenx at 25 sites in 12 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 9 inclusion and 18 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: under way, not looking for participants, as of the sponsor's last update on 29 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 25 sites in United States, Italy, Poland, United Kingdom, Canada and 7 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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Efgartigimod concentrations as input for compartmental, model-driven analysis to determine (age and size dependency of) Clearance (CL) (time frame: up to 26 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 29 Oct 2026.