AN OPEN-LABEL, MULTICENTER STUDY TO EVALUATE THE EFFICACY, SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF ECULIZUMAB IN PEDIATRIC PATIENTS WITH REFRACTORY GENERALIZED MYASTHENIA GRAVIS
- Conditions
- Generalized Myasthenia Gravis (gMG)muscle weakness disease10003816
- Registration Number
- NL-OMON54896
- Lead Sponsor
- Alexion Pharmaceuticals
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Withdrawn
- Sex
- Not specified
- Target Recruitment
- 2
* 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:
a. History of abnormal neuromuscular transmission test demonstrated by
single-fiber electromyography or repetitive nerve stimulation, or
b. History of positive anticholinesterase test (eg, edrophonium chloride or
neostigmine test), or
c. 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:
a. Failed treatment * 1 year with at least 1 IST
b. Require maintenance PE or IVIg to control symptoms
c. 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.
* 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, as
described in the Inclusion Criteria, 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
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Change from Baseline in the QMG total score over time regardless of rescue<br /><br>treatment.</p><br>
- Secondary Outcome Measures
Name Time Method