At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Efgartigimod PH20 SC (biological)
- How long the study runs
- Study runs about 39 months (dates as stated)
- About the drug or intervention
- Efgartigimod PH20 SC — biological: Subcutaneous injections
- Patient visit burden
- Not specified by the sponsor
- Type of study
- Testing a treatment
- Ages
- 2 Years to 17 Years
- Who
- All
- Number of participants
- 12
- Started
- 2024-06-28
- Last checked
- 2026-09
Plain English Summary
What is this study?
- • Testing a new treatment for generalized myasthenia gravis
- • Phase2/Phase3 - 12 participants
- • 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
Who can take part?
- • Ages 2 Years to 17 Years
- • Diagnosed with generalized myasthenia gravis
Where?
- • Oxford - Oxford Children's Hospital
This is a simplified summary. Always discuss with your doctor before making any decisions.
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.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 2 Years - 17 Years
- Who can join: All genders
Biomarkers mentioned
Treatment history
Treatments you must have had:
- ✓ a negative blood pregnancy test at screening and a negative urine pregnancy test
What the study is looking for
- ✓The participant is aged 2 to \<18 years at the time of agreement to take part/assent
- ✓The participant has been diagnosed with generalised Myasthenia Gravis that is supported by a physical examination...
Who cannot take part
- ✗Is a female adolescent of child-bearing potential who is pregnant and/or lactating or intends to become pregnant...
- ✗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...
See the full criteria
Where Is This Study? (1 UK site)
Oxford Children's Hospital
Oxford OX3 9DU, United Kingdom
How to Get in Touch
Sabine Coppieters, MD
Sponsor contactCONTACT
