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

Part of paid clinical trials in Scottsdale, Arizona.

Sponsor
Regeneron Pharmaceuticals
Study ID
NCT05070858
Phase
PHASE3
Status
Active Not Recruiting

Conditions

  • Generalized Myasthenia Gravis

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Pozelimab + Cemdisiran — DRUG
    Subcutaneous administration as described in the protocol
  • Cemdisiran — DRUG
    SC administration as described in the protocol
  • Placebo — OTHER
    SC administration as described in the protocol
  • Pozelimab — DRUG
    SC administration as described in the protocol

Study Details

This study is researching the experimental drugs called pozelimab and cemdisiran, and cemdisiran monotherapy. The study is focused on patients with generalized Myasthenia Gravis (gMG). Myasthenia gravis is a disease that causes weakness and fatigue in muscles in the body because the nerves and muscles are not communicating properly. The aim of the study is to see how effective pozelimab and cemdisiran are when used in combination and when pozelimab and cemdisiran are used alone for patients with gMG. The study is looking at several other research questions, including: * What side effects may happen from taking the study drugs * How the study drugs work inside the body * How much of the study drugs are in the blood at different times * Whether the body makes antibodies against pozelimab and cemdisiran (which could make the drugs less effective or could lead to side effects)

Key Dates

Start date
Dec 14, 2021
Status verified
Feb 2026
Primary completion
Jul 8, 2025
Completion
Nov 11, 2028

Study Design

Enrollment
288 participants (actual)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Group 1
    Placebo in DBTP; Re-randomized to Combination or Cemdisiran in ETP and OLTP
  • Experimental: Group 2
    Combination regimen throughout the study. For participants who have not reached week 108 will receive cemdisiran monotherapy in the OLTP.
  • Experimental: Group 3
    Cemdisiran throughout the study
  • Experimental: Group 4
    Pozelimab monotherapy in DBTP followed by combination in ETP and OLTP. For participants who have not reached week 108 will receive cemdisiran monotherapy in the OLTP.

Primary Outcome Measure

Change in Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score [ Time Frame: From baseline to week 24 ]

Locations (19)

FacilityCityStateZIPSite coordinators
HonorHealth Neurology 2018ScottsdaleArizona85251-
University of California IrvineIrvineCalifornia92697-
University of Southern CaliforniaLos AngelesCalifornia90033-
Colorado Springs Neurological AssociatesColorado SpringsColorado80907-5307-
SFM Clinical Research, LLCBoca RatonFlorida33487-
Diverse Clinical ResearchMiamiFlorida33175-
Aqualane Clinical ResearchNaplesFlorida34105-
Neurological Services of OrlandoOrlandoFlorida32806-
Medsol Clinical Research Center IncPort CharlotteFlorida33952-
University of South Florida Morsani Center for Advanced HealthcareTampaFlorida33612-
NorthShore University Health SystemGlenviewIllinois60026-1301-
St. Elizabeth's HospitalO'FallonIllinois62269-
Northwest Neurology Ltd. - Clinedge - PPDSRolling MeadowsIllinois60080-
Wayne State University School of MedicineDetroitMichigan48201-
Dayton Center for Neurological DisordersCentervilleOhio45459-
University of Cincinnati Gardner Neuroscience InstituteCincinnatiOhio45219-
Penn Medicine University CityPhiladelphiaPennsylvania19104-
National Neuromuscular Research InstituteAustinTexas78759-
Nerve and Muscle Center of TexasHoustonTexas77030-

Find similar trials in Scottsdale, AZ

By research site
HonorHealth Neurology 2018· Scottsdale, AZUniversity of California Irvine· Irvine, CAUniversity of Southern California· Los Angeles, CAColorado Springs Neurological Associates· Colorado Springs, COSFM Clinical Research, LLC· Boca Raton, FLDiverse Clinical Research· Miami, FL

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