Clinical Trials List
2021-11-01 - 2029-03-18
Phase III
Recruiting5
ICD-10G70.00
Myasthenia gravis without (acute) exacerbation
ICD-10G70.01
Myasthenia gravis with (acute) exacerbation
ICD-9358.0
Myasthenia gravis
Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Monotherapy in Patients With Symptomatic Generalized Myasthenia Gravis
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Trial Applicant
PAREXEL INTERNATIONAL CO., LTD.
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Sponsor
-
Trial scale
Multi-Regional Multi-Center
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Update
2026/02/01
Investigators and Locations
Co-Principal Investigator
- 林冠佑醫師 Division of Neurology
The Actual Total Number of Participants Enrolled
0 Recruiting
The Actual Total Number of Participants Enrolled
0 Recruiting
Co-Principal Investigator
- 朱俊哲 Division of Neurology
- CHIUNG-MEI CHEN Division of Neurology
- Hong-Chou Kuo Division of Neurology
- 呂榮國 Division of Neurology
- Chin-Chang Huang Division of Neurology
- 張國軒 Division of Neurology
- 謝珮甄 Division of Neurology
The Actual Total Number of Participants Enrolled
0 Recruiting
Co-Principal Investigator
- Ching-Hua Lu Lu Division of Neurology
The Actual Total Number of Participants Enrolled
0 Recruiting
The Actual Total Number of Participants Enrolled
0 Recruiting
Condition/Disease
Objectives
Test Drug
注射液
Active Ingredient
Cemdisiran
Dosage Form
279
Dosage
Endpoints
• Change in the total Myasthenia Gravis-Activities of Daily Living (MG-ADL) score from the baseline period to week 24.
Inclution Criteria
Male or female patients ≥18 years of age at screening (or ≥ legal age of adulthood based on local regulations, whichever is older)
Patient with documented diagnosis of myasthenia gravis (MG) based on medical history and supported by previous evaluations as described in the protocol
Documented prior history of positive serologic test or a positive result during screening of anti-acetylcholine receptor (AChR) antibodies or anti-LRP4 antibodies.
Myasthenia Gravis Foundation of America (MGFA) Clinical Classification Class II to IVa at screening
Myasthenia Gravis-Activities of Daily Living (MG-ADL) score ≥6 at screening. Ocular items should not contribute more than 50% of MG-ADL total score as described in the protocol
Currently receiving an acetylcholinesterase inhibitor or documented reason for not using acetylcholinesterase inhibitor therapy per investigator
Currently receiving an immunosuppressive therapy (IST) for MG, or documented reason why the patient is not taking an IST per investigator
If currently receiving an IST, not anticipated to have IST dosage changed before randomization or during double-blind treatment period (DBTP).
Willing and able to comply with clinic visits and study-related procedures, including completion of the primary series of the meningococcal vaccinations required per protocol
Exclusion Criteria
Patients with antibody profile that is only positive for muscle specific tyrosine kinase (MuSK) (MuSK positivity is based on a documented prior history of positive serologic test for antibodies to MuSK or a positive result during screening
History of thymectomy within 12 months prior to screening or planned during the study
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
Myasthenic crisis or Myasthenia Gravis Foundation of America (MGFA) Class V within 1 month of screening
Not meeting meningococcal vaccination requirements and, at a minimum, documentation of quadrivalent meningococcal vaccination within 5 years prior to randomization and serotype B vaccine (when available) within 3 years prior to randomization as described in the protocol
Known contraindication to meningococcal vaccines (group ACWY conjugate and group B vaccines) as described in the protocol
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
Positive hepatitis B surface antigen or hepatitis C virus ribonucleic acid (RNA) during screening. NOTE: Cases with unclear interpretation should be discussed with the medical monitor
History of HIV infection or a positive test at screening per local requirements
The Estimated Number of Participants
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Taiwan
21 participants
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Global
335 participants
