A Phase 3, Open-label, Single-arm, Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants (6 to < 18 years of age) with Generalized Myasthenia Gravis (gMG)
- Conditions
- Generalized Myasthenia Gravis (gMG)MedDRA version: 20.0Level: HLTClassification code: 10071942Term: Myasthenia gravis and related conditions Class: 10028395Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- CTIS2022-501882-29-00
- Lead Sponsor
- Alexion Pharmaceuticals Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 12
1.Must be 6 to < 18 years of age at the time of signing the informed consent and body weight = 10 kg., 10. Female participants of childbearing potential and male participants must be willing to follow protocol-specified contraception guidance, 2. Diagnosis of gMG confirmed by a positive serologic test for anti-AChR antibodies (Abs) obtained at Screening and/or during Screening Period and at least 1 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. Demonstrated improvement in MG signs on oral AChIs, as assessed by the Investigator, 3. Must have QMG total score as outlined below: a. Complement inhibitor treatment-naïve participants 12 to < 18 years of age must have QMG total score = 12 at Screening and on Day 1 b. Complement inhibitor treatment-naïve participants 6 to < 12 years of age have no minimum QMG required for inclusion; however, participants must have documented limb weakness in at least 1 limb, 4. Myasthenia Gravis Foundation of America (MGFA) Clinical Classification of Class II to Class IV at Screening, 5. Participants receiving treatment with any of the following must be on a stable dosing regimen of adequate duration prior to Screening and during the Screening Period as follows: a. AZA for = 6 months (180 days) and have been on a stable dose for = 2 months (60 days) prior to Screening b. ISTs (ie, MMF, MTX, CYC, TAC, or CY) for = 3 months (90 days) and have been on a stable dose for = 4 weeks (28 days) prior to Screening c. Chronic IVIg for = 12 months (365 days) and on a stable dose for = 3 months (90 days) prior to Screening, with the frequency and dose expected to remain stable during Screening Period and for at least 18 weeks following the first dose of study intervention (Note: Participants must not have received acute IVIg therapy within 28 days prior to Screening as outlined in Exclusion Criterion 13 of the protocol). d. Oral corticosteroids for = 4 weeks (28 days) prior to Screening e. AChIs for = 2 weeks (14 days) prior to Screening, 6.Eculizumab-experienced participants must have been enrolled and treated with eculizumab in Study ECU-MG-303 for at least 6 months (180 days), must have been on a stable dose for = 2 months (60 days) prior to Screening, and must have benefitted from and tolerated eculizumab in the investigator’s judgment., 7. To reduce the risk of meningococcal infection (Neisseria meningitidis), all participants must be vaccinated against meningococcal infection from serogroups A, C, Y, W135, and B within 3 years prior to, or at least 14 days prior to Day 1, according to national/local guidelines. Participants who do not meet this requirement will be vaccinated against meningococcal infection according to national/local guidelines and will receive prophylactic antibiotics for at least 2 weeks after meningococcal vaccination if Day 1 occurs < 2 weeks after initial vaccination, 8. Must have received vaccination against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae according to current national and local vaccination guidelines, 9. Participant’s legal guardian must be capable of giving written informed consent and the participant must be capable of giving written informed assent (if applicable as determined by the central or local institutional review board [IRB]/independent ethics c
1. Clinical features that, in the opinion of the Investigator, are consistent with MG crisis/exacerbation or Clinical Deterioration during the Screening Period or within: a. = 28 days prior to Screening for complement inhibitor treatment-naïve participants b. = 6 months prior to Screening for eculizumab-experienced participants, 10. Active systemic bacterial, viral, or fungal infection within 14 days prior to Day 1, 11. Presence of fever = 38°C (100.4°F) within 7 days prior to Day 1, 12. Known or suspected history of drug or alcohol abuse or dependence within 1 year prior to the start of the Screening Period, 13. For participants who are not receiving a stable maintenance dose of IVIg, as described in the Inclusion Criteria, use of IVIg (eg, as acute therapy) within 4 weeks prior to first dose of study intervention, 14. Use of PE/PP within = 28 days prior to Day 1, 15. Use of rituximab within = 6 months (180 days) prior to Day 1, 16. Prior use of any experimental C5 antagonist (other than eculizumab) at any time, 17. Participation in another investigational drug or investigational device study (other than Study ECU-MG-303) within 5 half-lives of that investigational product (if known) or within 30 days before initiation of the first dose of study intervention, whichever is longer, 18. Pregnant, breastfeeding, or intending to conceive during the course of the study, 19. Parent or legal guardian is an employee or directly related to an employee of Alexion or the institution/investigational site, 2. Any untreated thymic malignancy, carcinoma, or thymoma. Participants with a history of treated thymic malignancy or carcinoma are eligible for enrollment if they meet the following conditions: a. Treatment completed > 5 years prior to the Screening Visit b. No recurrence within the 5 years prior to the Screening Visit c. No radiological indication of recurrence in a computed tomography (CT) or magnetic resonance imaging (MRI) scan, including administration of IV contrast, performed within 6 months of randomization (Day 1) Participants with a history of treated benign thymoma [= Stage II, according to the Masaoka Staging System (Masaoka, 2010)] are eligible if they meet the following conditions: d. Histopathological or equivalent records confirming the diagnosis of benign thymoma e. Treatment completed > 12 months prior to the Screening Visit f. No known recurrence within the 12 months prior to the Screening Visit g. No radiological indication of recurrence in a CT or MRI scan, including administration of IV contrast, performed within 6 months of randomization (Day 1) h. If adequate records confirming the diagnosis of benign thymoma are not available, the participant must satisfy the eligibility criteria for thymic malignancy or carcinoma stated above, 3. History of thymectomy, thymomectomy, or any thymic surgery within the 12 months prior to Screening, 4. History of hypersensitivity to any ingredient contained in the study intervention, including hypersensitivity to murine proteins, 5. History of N meningitidis infection, 6. Known to be human immunodeficiency virus (HIV) positive, 7. Known medical or psychological condition(s) or risk factor that, in the opinion of the Investigator, might interfere with the participant’s full participation in the study, pose any additional risk for the participant, or confound the assessment of the participant or outcome of the study, 8. History of hospitalization for = 24 hours, for any reason, within the 4 we
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