Actively Recruiting
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
Led by argenx · Updated on 2026-06-04
12
Participants Needed
20
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying efgartigimod PH20 SC in children aged 2 to less than 18 years who have generalized Myasthenia Gravis (gMG). The study aims to understand how the drug is processed by the body, its effects, safety, tolerability, and immune response in this pediatric group. The main goal is to determine the appropriate dose for children using pharmacokinetic and pharmacodynamic data. Participants will receive subcutaneous injections of efgartigimod PH20 SC and be monitored throughout the study, which lasts up to 14 weeks. The study is open-label and uncontrolled, focusing on evaluating the drug's activity and safety. After the follow-up period, eligible participants may join an open-label extension study for continued observation. During the study, researchers will collect blood samples to measure drug levels, immunoglobulin G, and antibodies related to Myasthenia Gravis. They will monitor adverse events, severity of side effects, and changes in disease activity scores suitable for children. Participants will complete assessments of fatigue, quality of life, and global improvement. Safety will be closely observed with regular testing and follow-up visits throughout the study duration.
CONDITIONS
Brief Title
A Study of Efgartigimod PH20 SC in Children Between 2 and Less Than 18 Years of Age With Generalized Myasthenia Gravis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participant or legal representative understands the study and can provide written consent or assent
- Aged 2 to less than 18 years at the time of consent or assent
- Diagnosed with generalized Myasthenia Gravis confirmed by physical exam and positive anti-acetylcholine receptor antibodies
- Unsatisfactory response to immunosuppressants, corticosteroids, or acetylcholinesterase inhibitors but currently on stable treatment for at least 1 month before screening
- Agree to use birth control as required and have negative pregnancy tests if of child-bearing potential
You will not qualify if you...
- Female adolescents of child-bearing potential who are pregnant, breastfeeding, or planning pregnancy during the study
- Worsening muscle weakness due to infection or medication
- No clinical response to plasma exchange treatment
- Received live or live-attenuated vaccine within 4 weeks before screening
- Thymectomy within 3 months before screening or planned during the study
- Known autoimmune diseases or medical conditions interfering with symptom assessment or causing undue risk
- History of cancer unless cured with no recurrence for 3 or more years, except certain skin or in situ cancers
- Active infections including hepatitis B, hepatitis C, HIV, or positive COVID-19 test at screening
- Recent major surgery or medical conditions that could affect study results or safety
- Participation in another interventional clinical trial or prior efgartigimod study within 12 months
- Known allergy to study drug or its ingredients
- History of substance abuse
- Use of certain medications before screening as detailed in the protocol
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 12 weeks
Participants receive subcutaneous injections of Efgartigimod PH20 SC as the investigational treatment for generalized Myasthenia Gravis.
Regular visits during treatment for assessments and injections
Duration - Up to 2 weeks following treatment (total study duration up to 14 weeks)
Participants are monitored for safety and response after the treatment period ends.
Visits for safety assessments and follow-up
Trial Site Locations
Total: 20 locations
1
Carolinas HealthCare System Neurosciences Institute - Neurology
Charlotte, North Carolina, United States, 28207
Actively Recruiting
2
Neurology Rare Disease Center
Denton, Texas, United States, 76208
Actively Recruiting
3
University of Virginia
Charlottesville, Virginia, United States, 22903
Actively Recruiting
4
UZ Gent
Ghent, Belgium, 9000
Actively Recruiting
5
Childrens Hospital of Eastern Ontario
Ottawa, Canada, K1H 8L1
Actively Recruiting
6
Fakultni nemocnice Brno
Brno, Czechia, 613 00
Actively Recruiting
7
Fakultni nemocnice Ostrava
Ostrava, Czechia, 708 00
Actively Recruiting
8
AP-HM- Hôpital de La Timone
Marseille, France, 13385
Actively Recruiting
9
Universitätsklinikum Essen
Essen, Germany, 45147
Actively Recruiting
10
Hadassah Medical Center- Ein Kerem
Jerusalem, Israel, 9112001
Actively Recruiting
11
Schneider Children's Medical Center of Israel
Petah Tikvah, Israel, 4920235
Actively Recruiting
12
Tel Aviv Sourasky Medical Center Ichilov
Tel Aviv, Israel, 6423906
Actively Recruiting
13
Istituto G Gaslini Ospedale Pediatrico IRCCS
Genova, Italy, 16147
Actively Recruiting
14
Leiden University Medical Center
Leiden, Netherlands, 2333 ZA
Actively Recruiting
15
Uniwersyteckie Centrum Kliniczne w Gdansku
Gdansk, Poland, 80-211
Actively Recruiting
16
Neurologia Śląska Centrum Medyczne
Katowice, Poland, 40-689
Actively Recruiting
17
Centralny Szpital Kliniczny - Uniwersyteckie Centrum Kliniczne WUM
Warsaw, Poland, 02-097
Actively Recruiting
18
Hospital Universitari i Politecnic La Fe de Valencia
Valencia, Spain, 46026
Actively Recruiting
19
Hôpital Nestlé
Lausanne, Switzerland, 1011
Actively Recruiting
20
Oxford Children's Hospital
Oxford, United Kingdom, OX3 9DU
Actively Recruiting
Research Team
S
Sabine Coppieters, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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