Clinical trial NCT06392386: A Study of Efgartigimod PH20 SC in Children Between 2 and Less Than 18 Years of Age with Generalized Myasthenia Gravis
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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
PHASE3
Conditions: Generalized Myasthenia Gravis
Lead sponsor: argenx
Sponsor class: INDUSTRY
First posted: 2024-04-30
Enrollment: 12
Sex: ALL
Minimum age (years): 2
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

* 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
* 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.

Primary outcomes:
measure: Efgartigimod serum concentrations as input for compartmental, model-driven analysis to determine age and size dependency of Clearance (CL)
timeFrame: Up to 12 weeks
measure: Efgartigimod serum concentrations as input for compartmental, model-driven analysis to determine age and size dependency of Volume Distribution (Vd)
timeFrame: Up to 12 weeks
measure: Total G immunoglobulins (IgG) levels as input for pharmacokinetics (PK)/pharmacodynamics (PD) modelling analysis
timeFrame: Up to 12 weeks
measure: Anti-acetylcholine receptors antibodies (AChR-Ab) as input for pharmacokinetics (PK)/ pharmacodynamics (PD) modelling analysis
timeFrame: Up to 12 weeks
Secondary outcomes:
measure: Incidence of adverse events (AEs)
timeFrame: Up to 14 weeks
measure: Severity of adverse events (AEs)
timeFrame: Up to 14 weeks
measure: Incidence of serious adverse events (SAEs)
timeFrame: Up to 14 weeks
measure: Severity of serious adverse events (SAEs)
timeFrame: Up to 14 weeks
measure: Incidence of adverse events of special interest (AESI)
timeFrame: Up to 14 weeks
measure: Severity of adverse events of special interest (AESI)
timeFrame: Up to 14 weeks
measure: Efgartigimod serum concentrations
timeFrame: Up to 12 weeks
measure: Absolute values of total Immunoglobulin G (IgG) from blood samples
timeFrame: Up to 12 weeks
measure: Change from baseline values of total Immunoglobulin G (IgG) from blood samples
timeFrame: Up to 12 weeks
measure: Percentage change from baseline values of total Immunoglobulin G (IgG) from blood samples
timeFrame: Up to 12 weeks
measure: Absolute values of anti-acetylcholine receptor antibodies (AChR-Ab) from blood samples
timeFrame: Up to 12 weeks
measure: Change from baseline values of anti-acetylcholine receptor antibodies (AChR-Ab) from blood samples
timeFrame: Up to 12 weeks
measure: Percentage change from baseline values of anti-acetylcholine receptor antibodies (AChR-Ab) from blood samples
timeFrame: Up to 12 weeks
measure: Incidence of anti-drug antibodies (ADAs) against efgartigimod in serum samples
timeFrame: Up to 12 weeks
measure: Prevalence of anti-drug antibodies (ADAs) against efgartigimod in serum samples
timeFrame: Up to 12 weeks
measure: Incidence of antibodies against rHuPH20 in serum samples
timeFrame: Up to 12 weeks
measure: Prevalence of antibodies against rHuPH20 in serum samples
timeFrame: Up to 12 weeks
measure: Absolute value of total Myasthenia Gravis Activity of Daily Living (MG-ADL) score, appropriate for pediatric use
timeFrame: Up to 12 weeks
measure: Change from baseline of total Myasthenia Gravis Activity of Daily Living (MG-ADL) score, appropriate for pediatric use
timeFrame: Up to 12 weeks
measure: Absolute value of Quantitative Myasthenia Gravis (QMG) score
timeFrame: Up to 12 weeks
measure: Change from baseline of Quantitative Myasthenia Gravis (QMG) score
timeFrame: Up to 12 weeks
measure: Absolute value of EuroQoL 5 Dimensions Youth (EQ-5D-Y) score
timeFrame: Up to 12 weeks
measure: Change from baseline value of Neuro-QoL Pediatric Fatigue Score
timeFrame: Up to 12 weeks
measure: Change from baseline value of EuroQoL 5 Dimensions Youth (EQ-5D-Y) score
timeFrame: Up to 12 weeks
measure: Change from baseline value of Clinical Global Impression of Improvement (CGI-I)
timeFrame: Up to 12 weeks
measure: Changes in protective antibody titers to vaccines
timeFrame: Up to 12 weeks
Other outcomes:
Locations:
city: Denton
country: United States
facility: Neurology Rare Disease Center
state: Texas
city: Gent
country: Belgium
facility: UZ Gent
city: Marseille
country: France
facility: AP-HM- Hôpital de La Timone
city: Petah Tikvah
country: Israel
facility: Schneider Children's Medical Center of Israel
city: Tel Aviv
country: Israel
facility: Tel Aviv Sourasky Medical Center Ichilov
city: Genova
country: Italy
facility: Istituto G Gaslini Ospedale Pediatrico IRCCS
city: Leiden
country: Netherlands
facility: Leiden University Medical Center
city: Katowice
country: Poland
facility: Neurologia Śląska Centrum Medyczne
city: Valencia
country: Spain
facility: Hospital Universitari i Politecnic La Fe de Valencia
Study officials:
References: