A study to test how safe pozelimab and cemdisiran combination therapy and cemdisiran alone are and how well they work in adult patients with Generalized Myasthenia Gravis
- Conditions
- Generalised myasthenia gravis
- Registration Number
- 2023-508842-17-00
- Lead Sponsor
- Regeneron Pharmaceuticals Inc.
- Brief Summary
To evaluate the effect of pozelimab + cemdisiran and cemdisiran monotherapy on daily functioning that is impacted by signs and symptoms in patients with symptomatic generalized myasthenia gravis
(gMG)
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Ongoing, recruiting
- Sex
- Not specified
- Target Recruitment
- 118
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
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)
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
History of HIV infection or a positive test at screening per local requirements NOTE: Other protocol-defined Inclusion/ Exclusion Criteria apply
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Change in Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score Change in Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score
- Secondary Outcome Measures
Name Time Method Change from baseline in Quantitative Myasthenia Gravis (QMG) score Change from baseline in Quantitative Myasthenia Gravis (QMG) score
Achievement of a ≥3-point reduction (improvement) in MG-ADL total score Achievement of a ≥3-point reduction (improvement) in MG-ADL total score
Achievement of a ≥5-point reduction (improvement) in QMG total score Achievement of a ≥5-point reduction (improvement) in QMG total score
Achievement of a consistent response on the MG-ADL Achievement of a consistent response on the MG-ADL
Achievement of minimal symptom expression (MSE) Achievement of minimal symptom expression (MSE)
Change from baseline in the Myasthenia Gravis Composite (MGC) total score Change from baseline in the Myasthenia Gravis Composite (MGC) total score
Change from baseline in Myasthenia Gravis Quality of Life (MG QOL15r) total score Change from baseline in Myasthenia Gravis Quality of Life (MG QOL15r) total score
Achievement of a ≥2-, 4-, 5-, 6-, 7-, 8-, 9-, or 10-point reduction on MG-ADL total score Achievement of a ≥2-, 4-, 5-, 6-, 7-, 8-, 9-, or 10-point reduction on MG-ADL total score
Achievement of a ≥3-, 4-, 6-, 7-, 8-, 9-, or 10-point reduction on QMG Achievement of a ≥3-, 4-, 6-, 7-, 8-, 9-, or 10-point reduction on QMG
Incidence and severity of treatment-related adverse events (TEAEs) in patients treated with pozelimab + cemdisiran, cemdisiran monotherapy or placebo Incidence and severity of treatment-related adverse events (TEAEs) in patients treated with pozelimab + cemdisiran, cemdisiran monotherapy or placebo
Incidence and severity of serious adverse events (SAEs) in patients treated with pozelimab + cemdisiran, cemdisiran monotherapy or placebo Incidence and severity of serious adverse events (SAEs) in patients treated with pozelimab + cemdisiran, cemdisiran monotherapy or placebo
Incidence and severity of adverse events of special interest (AESIs) in patients treated with pozelimab + cemdisiran, cemdisiran monotherapy or placebo Incidence and severity of adverse events of special interest (AESIs) in patients treated with pozelimab + cemdisiran, cemdisiran monotherapy or placebo
Concentrations of total pozelimab in serum Concentrations of total pozelimab in serum
Concentrations of total complement component 5 (C5) in plasma Concentrations of total complement component 5 (C5) in plasma
Concentrations of cemdisiran and its metabolites in plasma Concentrations of cemdisiran and its metabolites in plasma
Incidence of treatment-emergent anti-drug antibodies (ADAs) to pozelimab over time Incidence of treatment-emergent anti-drug antibodies (ADAs) to pozelimab over time
Incidence of treatment-emergent ADAs to cemdisiran over time Incidence of treatment-emergent ADAs to cemdisiran over time
Change in total complement hemolysis activity assay (CH50) over time Change in total complement hemolysis activity assay (CH50) over time
Percent change in CH50 over time Percent change in CH50 over time
Trial Locations
- Locations (30)
Hospital Universitario La Paz
🇪🇸Madrid, Spain
Hospital Universitario Y Politecnico La Fe
🇪🇸Valencia, Spain
Hospital De La Santa Creu I Sant Pau
🇪🇸Barcelona, Spain
Odense University Hospital
🇩🇰Odense C, Denmark
Rigshospitalet
🇩🇰Copenhagen Oe, Denmark
Aalborg University Hospital
🇩🇰Aalborg, Denmark
Aarhus Universitetshospital
🇩🇰Aarhus N, Denmark
Hopitaux Universitaires Pitie Salpetriere
🇫🇷Paris, France
CHRU De Nancy
🇫🇷Nancy, France
Centre Hospitalier Universitaire De Nice
🇫🇷Nice, France
Scroll for more (20 remaining)Hospital Universitario La Paz🇪🇸Madrid, SpainFrancisco Javier Rodriguez de RiveraSite contact+34917277444frriveragarrido@salud.madrid.org