Uno studio su nipocalimab nei bambini di età compresa tra 2 e meno di 18 anni con miastenia grave generalizzata
Uno studio multicentrico non controllato in aperto per valutare la farmacocinetica, la farmacodinamica, la sicurezza e l'attività di nipocalimab nei bambini di età compresa tra 2 e meno di 18 anni con miastenia grave generalizzata
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.
Panoramica dello studio
Tipo di studio
Iscrizione (Stimato)
Fase
- Fase 2
- Fase 3
Accesso esteso
Contatti e Sedi
Contatto studio
- Nome: Study Contact
- Numero di telefono: 844-434-4210
- Email: Participate-In-This-Study1@its.jnj.com
Luoghi di studio
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Azumino-shi, Giappone, 399-8288
- Reclutamento
- Nagano Children's Hospital
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Chiba, Giappone, 260 8677
- Completato
- Chiba University Hospital
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Miyazaki, Giappone, 889-1692
- Reclutamento
- University of Miyazaki Hospital
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Nishinomiya-Shi, Giappone, 663-8501
- Reclutamento
- Hyogo College of Medicine Hospital
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Saitama Shi, Giappone, 330-8777
- Reclutamento
- Saitama Prefecture Children's Medical Center
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Shinjuku-ku, Giappone, 162-8666
- Reclutamento
- Tokyo Women's Medical University Hospital
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Leiden, Olanda, 2333 ZA
- Reclutamento
- Leiden University Medical Center
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Gdansk, Polonia, 80 211
- Reclutamento
- Uniwersyteckie Centrum Kliniczne
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Arizona
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Phoenix, Arizona, Stati Uniti, 85016
- Reclutamento
- Phoenix Children's Hospital
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California
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Los Angeles, California, Stati Uniti, 90027
- Completato
- Childrens Hospital Los Angeles
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Palo Alto, California, Stati Uniti, 94304
- Reclutamento
- Lucile Packard Children's Hospital Stanford
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San Francisco, California, Stati Uniti, 94158
- Reclutamento
- UCSF Benioff Children's Hospital
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Colorado
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Aurora, Colorado, Stati Uniti, 80045
- Reclutamento
- Children's Hospital Colorado
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Florida
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Tampa, Florida, Stati Uniti, 33613
- Reclutamento
- University of South Florida Morsani Center for Advanced Healthcare
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Kansas
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Lawrence, Kansas, Stati Uniti, 66045
- Completato
- University of Kansas Medical Center
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Michigan
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Ann Arbor, Michigan, Stati Uniti, 48109
- Reclutamento
- C.S. Mott Children's Hospital
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Pennsylvania
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Hershey, Pennsylvania, Stati Uniti, 17033
- Reclutamento
- Penn State Milton S Hershey Medical Ctr
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Philadelphia, Pennsylvania, Stati Uniti, 19106
- Reclutamento
- Childrens Hospital Of Philadelphia
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Pittsburgh, Pennsylvania, Stati Uniti, 15224
- Terminato
- University of Pittsburgh Medical Center
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Criteri di partecipazione
Criteri di ammissibilità
Età idonea allo studio
Accetta volontari sani
Sessi ammissibili allo studio
Descrizione
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
Piano di studio
Come è strutturato lo studio?
Dettagli di progettazione
- Scopo principale: Trattamento
- Assegnazione: N / A
- Modello interventistico: Assegnazione di gruppo singolo
- Mascheramento: Nessuno (etichetta aperta)
Armi e interventi
Gruppo di partecipanti / Arm |
Intervento / Trattamento |
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Sperimentale: 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). |
Nipocalimab will be administered as an IV infusion.
Altri nomi:
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Cosa sta misurando lo studio?
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
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Change from Baseline in Total Serum Immunoglobulin-G (IgG) Levels
Lasso di tempo: Up to 3 years
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Change from baseline in total serum IgG levels were reported. |
Up to 3 years
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Number of Participants with Infectious Adverse Events (AEs)
Lasso di tempo: 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
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Number of Participants with Serious AEs (SAEs)
Lasso di tempo: Up to 3 years
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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
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Number of Participants with Adverse Events of Special Interests (AESIs)
Lasso di tempo: Up to 3 years
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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
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Number of Participants with Abnormalities in Clinical Laboratory Tests
Lasso di tempo: Up to 3 years
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Number of participants with abnormalities in clinical laboratory tests (including chemistry, hematology, coagulation, and urinalysis) will be reported. |
Up to 3 years
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Number of Participants with Abnormalities in Vital Signs
Lasso di tempo: Up to 3 years
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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
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Number of Participants with Abnormalities in Physical Examination
Lasso di tempo: Up to 3 years
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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
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Serum Concentration of Nipocalimab over Time
Lasso di tempo: Up to 3 years
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Serum samples will be analyzed to determine concentrations of nipocalimab using a validated, specific, and sensitive immunoassay method. |
Up to 3 years
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Clearance (CL) of Nipocalimab
Lasso di tempo: Up to 3 years
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CL is defined as the volume of serum from which nipocalimab is completely removed per unit time. |
Up to 3 years
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Volume of Distribution (V) of Nipocalimab
Lasso di tempo: Up to 3 years
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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
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Half-life (t1/2) of Nipocalimab
Lasso di tempo: Up to 3 years
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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
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Steady-state Peak Concentration (Cpeak,ss) of Nipocalimab
Lasso di tempo: Up to 3 years
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Cpeak,ss is defined as the peak serum concentration of nipocalimab at steady state. |
Up to 3 years
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Steady-state Trough concentration (Ctrough,ss) of Nipocalimab
Lasso di tempo: Up to 3 years
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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
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Steady-state Area Under the Curve (AUCss) of Nipocalimab
Lasso di tempo: Up to 3 years
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AUCss is defined as the area under the curve for nipocalimab at steady state. |
Up to 3 years
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Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
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Change from Baseline in Myasthenia Gravis -Activities of Daily Living (MG-ADL) Score
Lasso di tempo: Up to 3 years
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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
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Change in the Quantitative Myasthenia Gravis (QMG) Score
Lasso di tempo: Up to 3 years
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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
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European Quality of Life 5-Dimension Youth (EQ-5D-Y) Tool Score
Lasso di tempo: Up to 3 years
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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
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Neurological Quality of Life (Neuro-QoL) Pediatric Fatigue Score
Lasso di tempo: Up to 3 years
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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
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Patient Global Impression of Severity (PGI-S) Score
Lasso di tempo: Up to 3 years
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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
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Patient Global Impression of Change (PGI-C) Score
Lasso di tempo: Up to 3 years
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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
|
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Number of Participants with Anti-Drug Antibodies [ADAs] to Nipocalimab
Lasso di tempo: Up to 3 years
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Number of participants with ADAs to nipocalimab will be reported. |
Up to 3 years
|
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Number of Participants with Neutralizing Antibodies (NAbs) to Nipocalimab
Lasso di tempo: Up to 3 years
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Number of participants with NAbs to nipocalimab will be reported. |
Up to 3 years
|
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Number of Participants with Vaccine Antibody Titers to Diphtheria or Tetanus
Lasso di tempo: Up to 3 years
|
Number of participants with vaccine antibody titers to diphtheria or tetanus will be reported. |
Up to 3 years
|
Collaboratori e investigatori
Investigatori
- Direttore dello studio: Janssen Research & Development, LLC Clinical Trial, Janssen Research & Development, LLC
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Completamento primario (Stimato)
Completamento dello studio (Stimato)
Date di iscrizione allo studio
Primo inviato
Primo inviato che soddisfa i criteri di controllo qualità
Primo Inserito (Effettivo)
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Effettivo)
Ultimo aggiornamento inviato che soddisfa i criteri QC
Ultimo verificato
Maggiori informazioni
Termini relativi a questo studio
Parole chiave
Termini MeSH pertinenti aggiuntivi
- Malattie del sistema nervoso
- Neoplasie per sede
- Neoplasie
- Malattie neuromuscolari
- Malattie autoimmuni
- Malattie del sistema immunitario
- Malattie autoimmuni del sistema nervoso
- Malattie Neurodegenerative
- Sindromi Paraneoplastiche, Sistema Nervoso
- Neoplasie del sistema nervoso
- Sindromi paraneoplastiche
- Malattie della giunzione neuromuscolare
- Miastenia grave
Altri numeri di identificazione dello studio
- CR109137
- 2021-002479-20 (Numero EudraCT)
- 80202135MYG2001 (Altro identificatore: Janssen Research & Development, LLC)
- 2022-502539-21-00 (Identificatore di registro: EUCT number)
Piano per i dati dei singoli partecipanti (IPD)
Hai intenzione di condividere i dati dei singoli partecipanti (IPD)?
Descrizione del piano IPD
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
Informazioni su farmaci e dispositivi, documenti di studio
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