全身性重症筋無力症の2歳から18歳未満の小児におけるニポカリマブの研究
全身性重症筋無力症の2歳から18歳未満の小児におけるニポカリマブの薬物動態、薬力学、安全性および活性を評価する非盲検非対照多施設研究
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.
調査の概要
研究の種類
入学 (推定)
段階
- フェーズ2
- フェーズ 3
アクセスの拡大
連絡先と場所
研究連絡先
- 名前：Study Contact
- 電話番号：844-434-4210
- メール：Participate-In-This-Study1@its.jnj.com
研究場所
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Arizona
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Phoenix、Arizona、アメリカ、85016
- 募集
- Phoenix Children's Hospital
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California
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Los Angeles、California、アメリカ、90027
- 完了
- Childrens Hospital Los Angeles
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Palo Alto、California、アメリカ、94304
- 募集
- Lucile Packard Children's Hospital Stanford
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San Francisco、California、アメリカ、94158
- 募集
- UCSF Benioff Children's Hospital
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Colorado
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Aurora、Colorado、アメリカ、80045
- 募集
- Children's Hospital Colorado
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Florida
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Tampa、Florida、アメリカ、33613
- 募集
- University of South Florida Morsani Center for Advanced Healthcare
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Kansas
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Lawrence、Kansas、アメリカ、66045
- 完了
- University of Kansas Medical Center
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Michigan
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Ann Arbor、Michigan、アメリカ、48109
- 募集
- C.S. Mott Children's Hospital
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Pennsylvania
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Hershey、Pennsylvania、アメリカ、17033
- 募集
- Penn State Milton S Hershey Medical Ctr
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Philadelphia、Pennsylvania、アメリカ、19106
- 募集
- Childrens Hospital Of Philadelphia
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Pittsburgh、Pennsylvania、アメリカ、15224
- 終了しました
- University of Pittsburgh Medical Center
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Leiden、オランダ、2333 ZA
- 募集
- Leiden University Medical Center
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Gdansk、ポーランド、80 211
- 募集
- Uniwersyteckie Centrum Kliniczne
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Azumino-shi、日本、399-8288
- 募集
- Nagano Children's Hospital
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Chiba、日本、260 8677
- 完了
- Chiba University Hospital
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Miyazaki、日本、889-1692
- 募集
- University of Miyazaki Hospital
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Nishinomiya-Shi、日本、663-8501
- 募集
- Hyogo College of Medicine Hospital
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Saitama Shi、日本、330-8777
- 募集
- Saitama Prefecture Children's Medical Center
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Shinjuku-ku、日本、162-8666
- 募集
- Tokyo Women's Medical University Hospital
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参加基準
適格基準
就学可能な年齢
健康ボランティアの受け入れ
受講資格のある性別
説明
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
研究計画
研究はどのように設計されていますか？
デザインの詳細
- 主な目的：処理
- 割り当て：なし
- 介入モデル：単一グループの割り当て
- マスキング：なし（オープンラベル）
武器と介入
参加者グループ / アーム |
介入・治療 |
|---|---|
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実験的：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.
他の名前：
|
この研究は何を測定していますか？
主要な結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Change from Baseline in Total Serum Immunoglobulin-G (IgG) Levels
時間枠：Up to 3 years
|
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)
時間枠：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)
時間枠：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
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Number of Participants with Adverse Events of Special Interests (AESIs)
時間枠：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
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Number of Participants with Abnormalities in Clinical Laboratory Tests
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：Up to 3 years
|
AUCss is defined as the area under the curve for nipocalimab at steady state. |
Up to 3 years
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
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Change from Baseline in Myasthenia Gravis -Activities of Daily Living (MG-ADL) Score
時間枠：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
時間枠：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
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European Quality of Life 5-Dimension Youth (EQ-5D-Y) Tool Score
時間枠：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
|
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Neurological Quality of Life (Neuro-QoL) Pediatric Fatigue Score
時間枠：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
時間枠：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
|
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Patient Global Impression of Change (PGI-C) Score
時間枠：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
|
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Number of Participants with Anti-Drug Antibodies [ADAs] to Nipocalimab
時間枠：Up to 3 years
|
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
時間枠：Up to 3 years
|
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
時間枠：Up to 3 years
|
Number of participants with vaccine antibody titers to diphtheria or tetanus will be reported. |
Up to 3 years
|
協力者と研究者
捜査官
- スタディディレクター：Janssen Research & Development, LLC Clinical Trial、Janssen Research & Development, LLC
研究記録日
主要日程の研究
研究開始 (実際)
一次修了 (推定)
研究の完了 (推定)
試験登録日
最初に提出
QC基準を満たした最初の提出物
最初の投稿 (実際)
学習記録の更新
投稿された最後の更新 (実際)
QC基準を満たした最後の更新が送信されました
最終確認日
詳しくは
本研究に関する用語
キーワード
追加の関連 MeSH 用語
その他の研究ID番号
- CR109137
- 2021-002479-20 (EudraCT番号)
- 80202135MYG2001 (その他の識別子：Janssen Research & Development, LLC)
- 2022-502539-21-00 (レジストリ識別子：EUCT number)
個々の参加者データ (IPD) の計画
個々の参加者データ (IPD) を共有する予定はありますか?
IPD プランの説明
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
医薬品およびデバイス情報、研究文書
米国FDA規制医薬品の研究
米国FDA規制機器製品の研究
この情報は、Web サイト clinicaltrials.gov から変更なしで直接取得したものです。研究の詳細を変更、削除、または更新するリクエストがある場合は、register@clinicaltrials.gov。 までご連絡ください。 clinicaltrials.gov に変更が加えられるとすぐに、ウェブサイトでも自動的に更新されます。