Nipocalimab treatment for children with generalized myasthenia gravis
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
This study is testing a new treatment called nipocalimab to see if it can help children with generalized myasthenia gravis who haven't responded well to other therapies.
Quick facts
| Phase | Phase2; Phase3 |
|---|---|
| Study type | Interventional |
| Enrollment | 12 (estimated) |
| Ages | 2 Years to 17 Years |
| Sex | All |
| Sponsor | Janssen Research & Development, LLC Industry-sponsored |
| Drugs / interventions | nipocalimab |
| Locations | 19 sites (Phoenix, Arizona and 18 other locations) |
| Trial ID | NCT05265273 on ClinicalTrials.gov |
What this trial studies
This study evaluates the effects of nipocalimab on serum immunoglobulin G levels in pediatric patients aged 2 to less than 18 years with generalized myasthenia gravis. It aims to assess the safety, tolerability, and pharmacokinetics of nipocalimab in children who have not responded adequately to standard treatments. The study will involve participants from various locations globally, with specific age criteria for U.S. sites. Participants will receive the treatment through infusion while undergoing regular monitoring.
Who should consider this trial
Good fit: Ideal candidates are children aged 2 to less than 18 years diagnosed with generalized myasthenia gravis and showing insufficient response to current therapies.
Not a fit: Patients who do not have a confirmed diagnosis of generalized myasthenia gravis or those who are not within the specified age range may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve the management of generalized myasthenia gravis in children, enhancing their quality of life.
How similar studies have performed: Other studies have shown promise in treating myasthenia gravis with similar approaches, indicating potential for success in this trial.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Phoenix, Arizona and 18 other locations
- Phoenix Children's Hospital — Phoenix, Arizona, United States (Recruiting)
- Childrens Hospital Los Angeles — Los Angeles, California, United States (Recruiting)
- Lucile Packard Children's Hospital Stanford — Palo Alto, California, United States (Recruiting)
- UCSF Benioff Children's Hospital — San Francisco, California, United States (Recruiting)
- Children's Hospital Colorado — Aurora, Colorado, United States (Recruiting)
- University of South Florida Morsani Center for Advanced Healthcare — Tampa, Florida, United States (Recruiting)
- University of Kansas Medical Center — Lawrence, Kansas, United States (Completed)
- C.S. Mott Children's Hospital — Ann Arbor, Michigan, United States (Recruiting)
- Penn State Milton S Hershey Medical Ctr — Hershey, Pennsylvania, United States (Recruiting)
- Childrens Hospital Of Philadelphia — Philadelphia, Pennsylvania, United States (Recruiting)
- University of Pittsburgh Medical Center — Pittsburgh, Pennsylvania, United States (Terminated)
- Nagano Children's Hospital — Azumino-shi, Japan (Recruiting)
- Chiba University Hospital — Chiba, Japan (Completed)
- University of Miyazaki Hospital — Miyazaki, Japan (Recruiting)
- Hyogo College of Medicine Hospital — Nishinomiya-Shi, Japan (Recruiting)
- Saitama Prefecture Children's Medical Center — Saitama Shi, Japan (Recruiting)
- Tokyo Women's Medical University Hospital — Shinjuku-ku, Japan (Recruiting)
- Leiden University Medical Center — Leiden, Netherlands (Recruiting)
- Uniwersyteckie Centrum Kliniczne — Gdansk, Poland (Recruiting)
Study contacts
- Study coordinator: Study Contact
- Email: Participate-In-This-Study1@its.jnj.com
- Phone: 844-434-4210
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.