A Study to Examine the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Symptomatic Generalized Myasthenia Gravis
- Conditions
- Symptomatic Generalized Myasthenia Gravis
- Registration Number
- JPRN-jRCT2071220059
- Lead Sponsor
- Pavani Rodrigo
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 17
1. Male or female patients >=18 years of age at screening (or >= legal age of adulthood based on local regulations, whichever is older)
2. Patient with documented diagnosis of MG based on medical history and supported by previous evaluations as described in the protocol.
3. Documented prior history of positive serologic test or a positive result during screening of anti-AChR antibodies or anti-LRP4 antibodies.
4. MGFA Clinical Classification Class II to IVa at screening
5. MG-ADL score >=6 at screening. Ocular items should not contribute more than 50% of MG-ADL total score
6. Currently receiving an acetylcholinesterase inhibitor or documented reason for not using acetylcholinesterase inhibitor therapy per investigator
7. Currently receiving an IST for MG, or documented reason why the patient is not taking an IST per investigator
8. If currently receiving an IST, not anticipated to have IST dosage changed before randomization or during DBTP.
9. Other Inclusion Criteria Apply
1. Patients with antibody profile that is only positive for MuSK (MuSK positivity is based on a documented prior history of positive serologic test for antibodies to MuSK or a positive result during screening)
2. History of thymectomy within 12 months prior to screening or planned during the study
3. History of malignant thymoma (patients with stage 1 may be enrolled), or history of cancer within the past 5 years, except for adequately treated basal cell skin cancer, squamous cell skin cancer, or in situ cervical cancer
4. Myasthenic crisis or MGFA Class V within 1 month of screening
5. No documented meningococcal vaccination within 5 years prior to screening visit unless vaccination will be administered during the screening period and prior to initiation of study treatment
6. Known contraindication to meningococcal vaccines (group ACWY conjugate and group B vaccines) as described in the protocol.
7. Patients who require antibiotics for meningococcal prophylaxis and have a
contraindication, warning, or precaution precluding the use of penicillin class and
penicillin-alternative antibiotics planned to be used for prophylaxis, or a history of intolerance leading to the discontinuation of these antibiotics
8. Positive hepatitis B surface antigen or hepatitis C virus RNA during screening.
NOTE: Cases with unclear interpretation should be discussed with the medical monitor
9. History of HIV infection or a positive test at screening per local requirements
10. Other Exclusion Criteria Apply
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Change in MG-ADL total score from baseline to week 24
- Secondary Outcome Measures
Name Time Method