A study to examine the efficacy and safety of pozelimab and cemdisiran combination therapy in patients with symptomatic Generalized Myasthenia Gravis

Phase 1

• Conditions: Generalized Myasthenia Gravis; MedDRA version: 21.1Level: PTClassification code 10028417Term: Myasthenia gravisSystem Organ Class: 10029205 - Nervous system disorders; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2020-003272-41-IT
• Lead Sponsor: REGENERON PHARMACEUTICALS, INC.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-10-19
• Last Update Posted: 2 March 2022

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 210

Inclusion Criteria

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 myasthenia gravis (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-acetylcholine receptor (AChR) antibodies or
anti-LRP4 antibodies.
4. Myasthenia Gravis Foundation of America (MGFA) Clinical
Classification Class II to IVa at screening
5. 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
6. Currently receiving an acetylcholinesterase inhibitor or documented
reason for not using acetylcholinesterase inhibitor therapy per
investigator
7. Currently receiving an immunosuppressive therapy (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 double-blind treatment period
(DBTP).
NOTE: Other Inclusion Criteria apply
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 200
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 10

Exclusion Criteria

1. Patients with a positive serologic test for antibodies to muscle specific
tyrosine kinase (MuSK) 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 Myasthenia Gravis Foundation of America (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 ribonucleic
acid (RNA) during screening.
9. History of HIV infection
NOTE: Other Exclusion Criteria apply

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified