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Myasthenia GravisPhase 2/3NCT05265273

A Study of Nipocalimab in Children Aged 2 to Less Than 18 Years With Generalized Myasthenia Gravis

A Phase 2/3 study testing Nipocalimab in people with Myasthenia Gravis, run by Janssen Research & Development, LLC at 19 sites in 4 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

The purpose of this study is to determine the effect of nipocalimab on total serum immunoglobulin G (IgG) in pediatric participants 2 to less than (<) 18 years of age (globally) and 8 to <18 years of age (for Unites Stated (US) sites only), the safety and tolerability of treatment with nipocalimab in children and… Read more about this study

Who is it for?
Ages 2 to 17 years
Conditions the study is for
Myasthenia Gravis
What is being tested?
Nipocalimab
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
12 participants
Where
19 sitesin 4 countries
Study dates
20 Jul 2022to 2 Jul 2029
Phase
Phase 2/3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 2 to 17 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • Age: For US sites only: 8 to < 18 years
  • Diagnosis of myasthenia gravis (MG) with generalized muscle weakness meeting the clinical criteria for generalized myasthenia gravis (gMG) as defined by the Myasthenia Gravis Foundation of America (MGFA) Clinical Classification Class IIa/b, IIIa/b, or IVa/b at screening
  • Has a positive serologic test for acetylcholine receptor (anti-AChR) antibodies or muscle-specific tyrosine kinase (anti-MuSK) antibodies at screening
  • A participant using herbal, naturopathic, traditional Chinese remedies, ayurvedic or nutritional supplements, or medical marijuana (with a doctor's prescription) is eligible if the use of these medications is acceptable to the Investigator. These remedies must remain at a stable dose and regimen throughout the study
  • Has sufficient venous access to allow drug administration by infusion and blood sampling as per the protocol
  • Participants should have a body weight and body mass index between 5th and 95th percentile for age and sex. Obese participants greater than 95th percentile and underweight participants below 5th percentile may participate following medical clearance
  • A female of childbearing potential must have a negative highly sensitive serum (beta-human chorionic gonadotropin [beta-hCG]) at Screening and a negative urine pregnancy test at Day 1 prior to administration of study intervention

Who cannot join

  • Has a history of severe and/or uncontrolled hepatic (example, viral/alcoholic/ autoimmune hepatitis/ cirrhosis/ and/or metabolic liver disease), gastrointestinal, renal, pulmonary, cardiovascular (including congenital heart diseases), psychiatric, neurological musculoskeletal disorder, any other medical disorder(s) (example, diabetes mellitus), risk factors for thrombosis events (example, a history of venous thromboembolism [VTE] or antiphospholipid syndrome, or a personal or family history of heritable coagulation disorder such as factor V leiden, protein S or protein C deficiency, atrial fibrillation/flutter, major orthopedic surgery or significant trauma that may increase the risk of VTE, is expected to be immobilized for prolonged periods of time), or has clinically significant abnormalities in screening laboratory, that might interfere with participant's full participation in the study, and/ or might jeopardize the safety of the participant or the validity of the study results
  • Has any confirmed or suspected clinical immunodeficiency syndrome not related to treatment of his/her generalized myasthenia gravis (gMG), or has a family history of congenital or hereditary immunodeficiency unless confirmed absent in the participant
  • Has had a thymectomy within 12 months prior to screening, or thymectomy is planned during the Active treatment Phase of the study
  • Has shown a previous severe immediate hypersensitivity reaction, such as anaphylaxis to therapeutic proteins (example, monoclonal antibodies)
  • Has experienced myocardial infarction, unstable ischemic heart disease, or stroke within 12 weeks of screening

Trial locations and contacts

19 sites in 4 countries. Contact the study team or a site near you to ask about taking part.

Study Contact contact

844-434-4210

Participate-In-This-Study1@its.jnj.com

19 sites in 4 countries

United States11 sites, 8 recruiting

  • C.S. Mott Children's Hospital

    Ann Arbor, Michigan, 48109

    Recruiting

  • Children's Hospital Colorado

    Aurora, Colorado, 80045

    Recruiting

  • Penn State Milton S Hershey Medical Ctr

    Hershey, Pennsylvania, 17033

    Recruiting

  • University of Kansas Medical Center

    Lawrence, Kansas, 66045

    Completed

  • Childrens Hospital Los Angeles

    Los Angeles, California, 90027

    Completed

  • Lucile Packard Children's Hospital Stanford

    Palo Alto, California, 94304

    Recruiting

  • Childrens Hospital Of Philadelphia

    Philadelphia, Pennsylvania, 19106

    Recruiting

  • Phoenix Children's Hospital

    Phoenix, Arizona, 85016

    Recruiting

  • University of Pittsburgh Medical Center

    Pittsburgh, Pennsylvania, 15224

    Terminated

  • UCSF Benioff Children's Hospital

    San Francisco, California, 94158

    Recruiting

  • University of South Florida Morsani Center for Advanced Healthcare

    Tampa, Florida, 33613

    Recruiting

Other trials in United States

Japan6 sites, 5 recruiting

  • Nagano Children's Hospital

    Azumino-shi, 399-8288

    Recruiting

  • Chiba University Hospital

    Chiba, 260 8677

    Completed

  • University of Miyazaki Hospital

    Miyazaki, 889-1692

    Recruiting

  • Hyogo College of Medicine Hospital

    Nishinomiya-Shi, 663-8501

    Recruiting

  • Saitama Prefecture Children's Medical Center

    Saitama Shi, 330-8777

    Recruiting

  • Tokyo Women's Medical University Hospital

    Shinjuku-ku, 162-8666

    Recruiting

Other trials in Japan

Netherlands1 site, 1 recruiting

  • Leiden University Medical Center

    Leiden, 2333 ZA

    Recruiting

Other trials in Netherlands

Poland1 site, 1 recruiting

  • Uniwersyteckie Centrum Kliniczne

    Gdansk, 80 211

    Recruiting

Other trials in Poland

About this study

Official title: An Open-Label Uncontrolled Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Activity of Nipocalimab in Children Aged 2 to Less Than 18 Years With Generalized Myasthenia Gravis

The purpose of this study is to determine the effect of nipocalimab on total serum immunoglobulin G (IgG) in pediatric participants 2 to less than (<) 18 years of age (globally) and 8 to <18 years of age (for Unites Stated (US) sites only), the safety and tolerability of treatment with nipocalimab in children and adolescents and to evaluate the pharmacokinetics (PK) of nipocalimab in children and adolescents with generalized myasthenia gravis (gMG) who have an insufficient clinical response to ongoing, stable standard-of-care therapy.

Being tested
Nipocalimab
Keywords
Pediatric
Medical subject headings
Myasthenia Gravis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • NipocalimabDrug

    Also called M281/JNJ-80202135

    Nipocalimab will be administered as an IV infusion.

Study groups in NCT05265273
Study groupRoleWhat they receive
NipocalimabExperimentalParticipants age 2 to less than (<) 18 years of age (globally) and 8 to <18 years of age (for US sites only) will be divided into 2 cohorts as per their age-adolescents 12 to <18 years and children 2 to <12 years and will receive nipocalimab once every two weeks for 24 weeks. After Week 24, all participants will have the option to enroll in long term extension (LTE).
Receives: Drug: Nipocalimab

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change from Baseline in Total Serum Immunoglobulin-G (IgG) Levels

    Change from baseline in total serum IgG levels were reported.

    Measured: Up to 3 years

  2. Number of Participants with Infectious Adverse Events (AEs)

    Number of participants with infectious AEs will be reported. An AE is any untoward medical occurrence in a participant participating in a clinical study that does not necessarily have a causal relationship with the pharmaceutical/biological agent under study.

    Measured: Up to 3 years

  3. Number of Participants with Serious AEs (SAEs)

    Number of participants with SAEs will be reported. A SAE is any AE that results in: death, persistent or significant disability/incapacity, requires inpatient hospitalization or prolongation of existing hospitalization, is life-threatening experience, is a congenital anomaly/birth defect, is suspected transmission of any infectious agent via a medicinal product, is medically important to prevent one of the outcomes listed above.

    Measured: Up to 3 years

  4. Number of Participants with Adverse Events of Special Interests (AESIs)

    Number of participants with AESIs will be reported. Treatment-emergent AEs associated with the following situations are considered an AESI: a) infections that are severe or require intravenous (IV) anti-infective or operative/invasive intervention; b) hypoalbuminemia with albumin less than (<)20 grams per liter (g/L) [<] 2.0 grams per deciliter [g/dL]) c) opportunistic infections and d) Serious and non-serious deep-vein thrombosis (DVT) and/or pulmonary embolism (PE). Any AE occurring at or after the initial administration of study intervention through end of study is treatment emergent.

    Measured: Up to 3 years

  5. Number of Participants with Abnormalities in Clinical Laboratory Tests

    Number of participants with abnormalities in clinical laboratory tests (including chemistry, hematology, coagulation, and urinalysis) will be reported.

    Measured: Up to 3 years

  6. Number of Participants with Abnormalities in Vital Signs

    Number of participants with abnormalities in vital signs including sitting pulse/heart rate, sitting systolic and diastolic blood pressure, and oral temperature (degrees Celsius) will be reported.

    Measured: Up to 3 years

  7. Number of Participants with Abnormalities in Physical Examination

    Number of participants with abnormalities in physical examinations including height, weight, assessments of the skin, head, eyes, ears, nose, throat, neck, thyroid, lungs, heart, abdomen, lymph nodes and extremities will be reported.

    Measured: Up to 3 years

  8. Serum Concentration of Nipocalimab over Time

    Serum samples will be analyzed to determine concentrations of nipocalimab using a validated, specific, and sensitive immunoassay method.

    Measured: Up to 3 years

  9. Clearance (CL) of Nipocalimab

    CL is defined as the volume of serum from which nipocalimab is completely removed per unit time.

    Measured: Up to 3 years

  10. Volume of Distribution (V) of Nipocalimab

    V is defined as the representation of nipocalimab's propensity to either remain in the serum or redistribute to other tissue compartments.

    Measured: Up to 3 years

  11. Half-life (t1/2) of Nipocalimab

    t1/2 is defined as the time it takes for nipocalimab's active substance in the body to reduce by half.

    Measured: Up to 3 years

  12. Steady-state Peak Concentration (Cpeak,ss) of Nipocalimab

    Cpeak,ss is defined as the peak serum concentration of nipocalimab at steady state.

    Measured: Up to 3 years

  13. Steady-state Trough concentration (Ctrough,ss) of Nipocalimab

    Ctrough,ss will be reported. It is defined as the observed serum concentration of nipocalimab just prior to the beginning of a dosing interval at steady state.

    Measured: Up to 3 years

  14. Steady-state Area Under the Curve (AUCss) of Nipocalimab

    AUCss is defined as the area under the curve for nipocalimab at steady state.

    Measured: Up to 3 years

Secondary outcomes (9)

  1. Change from Baseline in Myasthenia Gravis -Activities of Daily Living (MG-ADL) Score

    Change from baseline in MG-ADL score will be reported. The MG-ADL score provides a rapid assessment of the participant's myasthenia gravis (MG) symptom severity. Eight functions (talking, chewing, swallowing, breathing, impairment of ability to brush teeth or comb hair, impairment of ability to arise from a chair, double vision, eyelid droop) are rated on a 4-point scale: 0 (no impairment) to 3 (severe impairment). The total score will be sum of eight function scores and can range from 0 to 24. A higher score indicates greater symptom severity.

    Measured: Up to 3 years

  2. Change in the Quantitative Myasthenia Gravis (QMG) Score

    The QMG score is a standardized quantitative strength assessment comprising 13 components (and is administered by a trained qualified healthcare professional [HCP] eg, physician, physician assistant, nurse practitioner, nurse). The quantitative results of each strength component are mapped to the following 4-point scale: 0 equals to (=) none, 1 = mild, 2 = moderate and 3 = severe. The total score will be sum of 13 components scores and can range from 0 to 39. A higher score indicates greater weakness.

    Measured: Up to 3 years

  3. European Quality of Life 5-Dimension Youth (EQ-5D-Y) Tool Score

    The EQ-5D-Y is a standardized child friendly instrument for use as a measure of health status, primarily designed for self-completion by children and adolescents, or via a proxy version to be completed by the child's caregiver. The EQ-5D-Y descriptive system comprises the following 5 dimensions: Mobility, looking after myself (washing and dressing), usual activities, pain or discomfort and feeling worried or unhappy. Each of the 5 dimensions is divided into 3 levels of perceived problems (Level 1 indicating no problem, Level 2 indicating some problems, Level 3 a lot of problems).

    Measured: Up to 3 years

  4. Neurological Quality of Life (Neuro-QoL) Pediatric Fatigue Score

    The Neuro-QoL pediatric fatigue score will be used to assess the impact of fatigue in participants aged 10 to less than (<) 18 years. The participant will rate each of the 11 items on a 5-point scale. Higher scores indicate greater fatigue.

    Measured: Up to 3 years

  5. Patient Global Impression of Severity (PGI-S) Score

    The PGI-S score will be used to assess the severity of fatigue due to generalized myasthenia gravis (gMG) in participants aged 10 to < 18 years. Participants will be asked to rate their fatigue over the past 7 days using the following 5-point scale: 1 = None, 2 = Mild, 3 = Moderate, 4 = Severe, and 5 = Very severe. Higher scores indicate greater severity of fatigue.

    Measured: Up to 3 years

  6. Patient Global Impression of Change (PGI-C) Score

    The PGI-C score will be used to assess if there has been an improvement or decline in patient-reported fatigue since the beginning of the treatment in participants aged 10 to <18 years. Participants will be asked to rate their current fatigue as compared to when they started the study, using the following 7-point scale: 1 = Much better, 2 = Moderately better, 3 = A little better, 4 = No change, 5 = A little worse, 6 = Moderately worse, and 7 = Much worse. Higher scores indicate greater change in overall fatigue.

    Measured: Up to 3 years

  7. Number of Participants with Anti-Drug Antibodies [ADAs] to Nipocalimab

    Number of participants with ADAs to nipocalimab will be reported.

    Measured: Up to 3 years

  8. Number of Participants with Neutralizing Antibodies (NAbs) to Nipocalimab

    Number of participants with NAbs to nipocalimab will be reported.

    Measured: Up to 3 years

  9. Number of Participants with Vaccine Antibody Titers to Diphtheria or Tetanus

    Number of participants with vaccine antibody titers to diphtheria or tetanus will be reported.

    Measured: Up to 3 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted3 Mar 2022
  2. Study start20 Jul 2022
  3. Primary completion30 Jun 2027 (estimated)
  4. Study completion2 Jul 2029 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2/3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
12 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05265273
Sponsor's protocol ID
CR109137
Other IDs
80202135MYG2001, 2021-002479-20, 2022-502539-21-00
Status last verified
Nov 2026
First submitted
22 Feb 2022
Last updated
4 Nov 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: The data sharing policy of Johnson \& Johnson Innovative Medicine is available at www.innovativemedicine.jnj.com/our-innovation/clinical-trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu
Expanded access
Available

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing Nipocalimab in people with Myasthenia Gravis, run by Janssen Research & Development, LLC at 19 sites in 4 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 4 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 19 sites in United States, Japan, Netherlands, Poland. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Study Contact (844-434-4210, Participate-In-This-Study1@its.jnj.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change from Baseline in Total Serum Immunoglobulin-G (IgG) Levels (time frame: Up to 3 years). The study lists 14 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05265273.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Nov 2026.

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