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Een onderzoek naar nipocalimab bij kinderen van 2 tot jonger dan 18 jaar met gegeneraliseerde myasthenia gravis

2 november 2026 bijgewerkt door: Janssen Research & Development, LLC

Een open-label ongecontroleerde multicenter studie om de farmacokinetiek, farmacodynamiek, veiligheid en activiteit van nipocalimab te evalueren bij kinderen van 2 tot jonger dan 18 jaar met gegeneraliseerde 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.

Studie Overzicht

Toestand

Werving

Conditie

Interventie / Behandeling

Studietype

Ingrijpend

Inschrijving (Geschat)

12

Fase

  • Fase 2
  • Fase 3

Uitgebreide toegang

Tijdelijk niet beschikbaar buiten de klinische proef. Zie uitgebreid toegangsrecord.

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studiecontact

Studie Locaties

  • Japan
      • Azumino-shi, Japan, 399-8288
        • Werving
        • Nagano Children's Hospital
      • Chiba, Japan, 260 8677
        • Voltooid
        • Chiba University Hospital
      • Miyazaki, Japan, 889-1692
        • Werving
        • University of Miyazaki Hospital
      • Nishinomiya-Shi, Japan, 663-8501
        • Werving
        • Hyogo College of Medicine Hospital
      • Saitama Shi, Japan, 330-8777
        • Werving
        • Saitama Prefecture Children's Medical Center
      • Shinjuku-ku, Japan, 162-8666
        • Werving
        • Tokyo Women's Medical University Hospital
  • Nederland
      • Leiden, Nederland, 2333 ZA
        • Werving
        • Leiden University Medical Center
  • Polen
      • Gdansk, Polen, 80 211
        • Werving
        • Uniwersyteckie Centrum Kliniczne
  • Verenigde Staten
    • Arizona
      • Phoenix, Arizona, Verenigde Staten, 85016
        • Werving
        • Phoenix Children's Hospital
    • California
      • Los Angeles, California, Verenigde Staten, 90027
        • Voltooid
        • Childrens Hospital Los Angeles
      • Palo Alto, California, Verenigde Staten, 94304
        • Werving
        • Lucile Packard Children's Hospital Stanford
      • San Francisco, California, Verenigde Staten, 94158
        • Werving
        • UCSF Benioff Children's Hospital
    • Colorado
      • Aurora, Colorado, Verenigde Staten, 80045
        • Werving
        • Children's Hospital Colorado
    • Florida
      • Tampa, Florida, Verenigde Staten, 33613
        • Werving
        • University of South Florida Morsani Center for Advanced Healthcare
    • Kansas
      • Lawrence, Kansas, Verenigde Staten, 66045
        • Voltooid
        • University of Kansas Medical Center
    • Michigan
      • Ann Arbor, Michigan, Verenigde Staten, 48109
        • Werving
        • C.S. Mott Children's Hospital
    • Pennsylvania
      • Hershey, Pennsylvania, Verenigde Staten, 17033
        • Werving
        • Penn State Milton S Hershey Medical Ctr
      • Philadelphia, Pennsylvania, Verenigde Staten, 19106
        • Werving
        • Childrens Hospital Of Philadelphia
      • Pittsburgh, Pennsylvania, Verenigde Staten, 15224
        • Beëindigd
        • University of Pittsburgh Medical Center

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

2 jaar tot 17 jaar (Kind)

Accepteert gezonde vrijwilligers

Nee

Geslachten die in aanmerking komen voor studie

Allemaal

Beschrijving

Key Inclusion Criteria:

  • 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

Key Exclusion Criteria:

  • 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

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Primair doel: Behandeling
  • Toewijzing: NVT
  • Interventioneel model: Opdracht voor een enkele groep
  • Masker: Geen (open label)

Wapens en interventies

Deelnemersgroep / Arm
Interventie / Behandeling
Experimenteel: Nipocalimab

Participants 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).
Geneesmiddel: Nipocalimab

Nipocalimab will be administered as an IV infusion.

Andere namen:
  • M281/JNJ-80202135

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Change from Baseline in Total Serum Immunoglobulin-G (IgG) Levels
Tijdsspanne: Up to 3 years

Change from baseline in total serum IgG levels were reported.
Up to 3 years
Number of Participants with Infectious Adverse Events (AEs)
Tijdsspanne: Up to 3 years

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.
Up to 3 years
Number of Participants with Serious AEs (SAEs)
Tijdsspanne: Up to 3 years

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.
Up to 3 years
Number of Participants with Adverse Events of Special Interests (AESIs)
Tijdsspanne: Up to 3 years

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.
Up to 3 years
Number of Participants with Abnormalities in Clinical Laboratory Tests
Tijdsspanne: Up to 3 years

Number of participants with abnormalities in clinical laboratory tests (including chemistry, hematology, coagulation, and urinalysis) will be reported.
Up to 3 years
Number of Participants with Abnormalities in Vital Signs
Tijdsspanne: Up to 3 years

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.
Up to 3 years
Number of Participants with Abnormalities in Physical Examination
Tijdsspanne: Up to 3 years

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.
Up to 3 years
Serum Concentration of Nipocalimab over Time
Tijdsspanne: Up to 3 years

Serum samples will be analyzed to determine concentrations of nipocalimab using a validated, specific, and sensitive immunoassay method.
Up to 3 years
Clearance (CL) of Nipocalimab
Tijdsspanne: Up to 3 years

CL is defined as the volume of serum from which nipocalimab is completely removed per unit time.
Up to 3 years
Volume of Distribution (V) of Nipocalimab
Tijdsspanne: Up to 3 years

V is defined as the representation of nipocalimab's propensity to either remain in the serum or redistribute to other tissue compartments.
Up to 3 years
Half-life (t1/2) of Nipocalimab
Tijdsspanne: Up to 3 years

t1/2 is defined as the time it takes for nipocalimab's active substance in the body to reduce by half.
Up to 3 years
Steady-state Peak Concentration (Cpeak,ss) of Nipocalimab
Tijdsspanne: Up to 3 years

Cpeak,ss is defined as the peak serum concentration of nipocalimab at steady state.
Up to 3 years
Steady-state Trough concentration (Ctrough,ss) of Nipocalimab
Tijdsspanne: Up to 3 years

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.
Up to 3 years
Steady-state Area Under the Curve (AUCss) of Nipocalimab
Tijdsspanne: Up to 3 years

AUCss is defined as the area under the curve for nipocalimab at steady state.
Up to 3 years

Secundaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Change from Baseline in Myasthenia Gravis -Activities of Daily Living (MG-ADL) Score
Tijdsspanne: Up to 3 years

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.
Up to 3 years
Change in the Quantitative Myasthenia Gravis (QMG) Score
Tijdsspanne: Up to 3 years

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.
Up to 3 years
European Quality of Life 5-Dimension Youth (EQ-5D-Y) Tool Score
Tijdsspanne: Up to 3 years

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).
Up to 3 years
Neurological Quality of Life (Neuro-QoL) Pediatric Fatigue Score
Tijdsspanne: Up to 3 years

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.
Up to 3 years
Patient Global Impression of Severity (PGI-S) Score
Tijdsspanne: Up to 3 years

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.
Up to 3 years
Patient Global Impression of Change (PGI-C) Score
Tijdsspanne: Up to 3 years

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.
Up to 3 years
Number of Participants with Anti-Drug Antibodies [ADAs] to Nipocalimab
Tijdsspanne: Up to 3 years

Number of participants with ADAs to nipocalimab will be reported.
Up to 3 years
Number of Participants with Neutralizing Antibodies (NAbs) to Nipocalimab
Tijdsspanne: Up to 3 years

Number of participants with NAbs to nipocalimab will be reported.
Up to 3 years
Number of Participants with Vaccine Antibody Titers to Diphtheria or Tetanus
Tijdsspanne: Up to 3 years

Number of participants with vaccine antibody titers to diphtheria or tetanus will be reported.
Up to 3 years

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Sponsor

Janssen Research & Development, LLC

Onderzoekers

  • Studie directeur: Janssen Research & Development, LLC Clinical Trial, Janssen Research & Development, LLC

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Werkelijk)

20 juli 2022

Primaire voltooiing (Geschat)

30 juni 2027

Studie voltooiing (Geschat)

2 juli 2029

Studieregistratiedata

Eerst ingediend

22 februari 2022

Eerst ingediend dat voldeed aan de QC-criteria

22 februari 2022

Eerst geplaatst (Werkelijk)

3 maart 2022

Updates van studierecords

Laatste update geplaatst (Werkelijk)

4 november 2026

Laatste update ingediend die voldeed aan QC-criteria

2 november 2026

Laatst geverifieerd

1 november 2026

Meer informatie

Termen gerelateerd aan deze studie

Trefwoorden

Aanvullende relevante MeSH-voorwaarden

Andere studie-ID-nummers

  • CR109137
  • 2021-002479-20 (EudraCT-nummer)
  • 80202135MYG2001 (Andere identificatie: Janssen Research & Development, LLC)
  • 2022-502539-21-00 (Register-ID: EUCT number)

Plan Individuele Deelnemersgegevens (IPD)

Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?

JA

Beschrijving IPD-plan

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

Informatie over medicijnen en apparaten, studiedocumenten

Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel

Ja

Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct

Nee

Deze informatie is zonder wijzigingen rechtstreeks van de website clinicaltrials.gov gehaald. Als u verzoeken heeft om uw onderzoeksgegevens te wijzigen, te verwijderen of bij te werken, neem dan contact op met register@clinicaltrials.gov. Zodra er een wijziging wordt doorgevoerd op clinicaltrials.gov, wordt deze ook automatisch bijgewerkt op onze website .

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