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Trial Title:
A Study of Efgartigimod PH20 SC in Children Between 2 and Less Than 18 Years of Age with Generalized Myasthenia Gravis
NCT ID:
NCT06392386
Condition:
Generalized Myasthenia Gravis
Conditions: Official terms:
Myasthenia Gravis
Muscle Weakness
Study type:
Interventional
Study phase:
Phase 2/Phase 3
Overall status:
Recruiting
Study design:
Allocation:
N/A
Intervention model:
Single Group Assignment
Primary purpose:
Treatment
Masking:
None (Open Label)
Intervention:
Intervention type:
Biological
Intervention name:
Efgartigimod PH20 SC
Description:
Subcutaneous injections
Arm group label:
Efgartigimod PH20 SC
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.
Criteria for 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.
Gender:
All
Minimum age:
2 Years
Maximum age:
17 Years
Healthy volunteers:
No
Locations:
Facility:
Name:
Neurology Rare Disease Center
Address:
City:
Denton
Zip:
76208
Country:
United States
Status:
Recruiting
Contact:
Last name:
Diana Castro, MD
Phone:
857-350-4834
Email:
clinicaltrials@argenx.com
Facility:
Name:
Neurologia Śląska Centrum Medyczne
Address:
City:
Katowice
Zip:
40-689
Country:
Poland
Status:
Recruiting
Contact:
Last name:
Marek Smilowski, MD
Phone:
857-350-4834
Email:
clinicaltrials@argenx.com
Facility:
Name:
Hospital Universitari i Politecnic La Fe de Valencia
Address:
City:
Valencia
Zip:
46026
Country:
Spain
Status:
Recruiting
Contact:
Last name:
Teresa Sevilla Mantecón, MD
Phone:
857-350-4834
Email:
clinicaltrials@argenx.com
Start date:
June 28, 2024
Completion date:
September 30, 2026
Lead sponsor:
Agency:
argenx
Agency class:
Industry
Source:
argenx
Record processing date:
ClinicalTrials.gov processed this data on November 12, 2024
Source: ClinicalTrials.gov page:
https://clinicaltrials.gov/ct2/show/NCT06392386
