Actively Recruiting
A Study of Efgartigimod PH20 SC in Children Between 2 and Less Than 18 Years of Age With Generalized Myasthenia Gravis
Led by argenx · Updated on 2026-04-27
12
Participants Needed
20
Research Sites
117 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official 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 study requirements and can provide written consent or assent
- Aged 2 to less than 18 years at consent/assent
- Diagnosed with generalized myasthenia gravis supported by physical exam and positive anti-acetylcholine receptor antibodies
- Had unsatisfactory response to immunosuppressants, corticosteroids, or acetylcholinesterase inhibitors and is on stable MG therapy
- Stable dose of corticosteroids and/or immunosuppressants for at least 1 month before screening if applicable
- Agrees to use birth control as required and has negative pregnancy tests if of child-bearing potential
You will not qualify if you...
- Female adolescent of child-bearing potential who is pregnant, lactating, or plans pregnancy during the study
- Worsening muscle weakness due to infection or medication
- Lack of clinical response to plasma exchange
- Received live or live-attenuated vaccine within 4 weeks before screening
- Thymectomy within 3 months before screening or plans thymectomy during study
- Known autoimmune disease or medical conditions interfering with MG assessment or increasing risk
- History of malignancy unless cured with no recurrence for 3 or more years, except certain skin and cervical cancers
- Active significant infection unresolved or positive tests for HBV, HCV, HIV
- Positive PCR test for SARS-CoV-2 at screening
- Clinically significant disease, recent major surgery within 3 months, or planned major surgery during study
- Received another study drug within 12 months before screening
- Currently in another interventional clinical study
- Previously participated in an efgartigimod study and received the drug
- Known hypersensitivity to study drug or excipients
- History or current episode of substance abuse
- Use of certain medications before screening as detailed in the protocol
AI-Screening
AI-Powered Screening
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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
CONTACT
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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