An Open-Label, Multicenter Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Eculizumab in Pediatric Patients with Refractory Generalized Myasthenia Gravis (gMG)
- Conditions
- Refractory Generalized Myasthenia GravisMedDRA version: 21.1Level: PTClassification code 10028417Term: Myasthenia gravisSystem Organ Class: 10029205 - Nervous system disordersTherapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2016-001384-37-DE
- Lead Sponsor
- Alexion Pharmaceuticals, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 11
? Male or female pediatric patients 6 to < 18 years of age at time of assent/consent
? Diagnosis of MG confirmed by positive serologic test for anti-AChR-Ab at Screening, and one of the following: history of abnormal neuromuscular transmission test demonstrated by single-fiber electromyography or repetitive nerve stimulation, or history of positive anticholinesterase test, or patient demonstrated improvement in MG signs on oral AChIs, as assessed by the Investigator
? Presence of refractory gMG, defined as patients with gMG who have one or more of the following: failed treatment = 1 year with at least 1 IST, require maintenance PE or IVIg to control symptoms, in the opinion of the Investigator MG poses a significant functional burden despite current MG treatment.
? All MG-specific treatment has been administered at a stable dosing regimen of adequate duration prior to Screening
? Myasthenia Gravis Foundation of America (MGFA) Clinical Classification of Class II to IV at Screening
? In patients aged 12 to 18 years, QMG total score = 12 at Screening; in patients aged 6 to 11 years, no minimum QMG is required for inclusion; however, patients must have documented limb weakness in at least one limb.
? Patients must be vaccinated against meningococcal infections within the 3 years prior to, or at the time of, initiating study drug. Patients who initiate study drug treatment less than 2 weeks after receiving a meningococcal vaccine must receive treatment with appropriate prophylactic antibiotics until 2 weeks after vaccination.
? Documented vaccination against H influenzae and S pneumoniae infections prior to dosing as per local and country specific immunization guidelines for the appropriate age group.
Are the trial subjects under 18? yes
Number of subjects for this age range: 17
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
? Any active or untreated thymoma. History of thymic carcinoma or thymic malignancy unless deemed cured by adequate treatment with no evidence of recurrence for =5 years before Screening.
? History of thymectomy within 12 months prior to Screening.
? Weakness only affecting ocular or periocular muscles (MGFA Class I).
? Myasthenia Gravis crisis or impending crisis at or during Screening (MGFA Class V).
? Any unresolved acute, or chronic, systemic bacterial or other infection, which is clinically significant in the opinion of the Investigator and has not been treated with appropriate antibiotics.
? Unresolved meningococcal infection
? Patients who are under 15 kg and are receiving maintenance
IVIg.
? For patients who are not receiving a stable maintenance dose of IVIg, use of IVIg (eg, as rescue therapy) within 4 weeks prior to first dose
? Use of PE within 4 weeks prior to first dose
? Use of rituximab within 6 months prior to first dose
? Hypersensitivity to murine proteins or to one of the excipients of eculizumab
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method