A Study to Examine the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Symptomatic Generalized Myasthenia Gravis (NIMBLE)

April 5, 2024 updated by: Regeneron Pharmaceuticals

Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Symptomatic Generalized Myasthenia Gravis

The primary objective is:

To evaluate the effect of pozelimab + cemdisiran on daily functioning that is impacted by signs and symptoms in patients with symptomatic generalized myasthenia gravis (gMG)

The secondary objectives of the study are:

  • To evaluate the effect of pozelimab + cemdisiran (ie, combination) and cemdisiran monotherapy on:

    • Clinician-assessed signs of myasthenia gravis (MG) and muscle strength
    • Daily functioning that is impacted by signs and symptoms in patients with symptomatic gMG (cemdisiran monotherapy only).
    • Proportion of patients with improvements in daily function that is impacted by signs and symptoms of MG
    • Proportion of patients that have improvements in clinician-assessed signs of MG and muscle strength
    • Health related quality of life
    • Proportion of patients with minimal MG symptoms
    • Patient- and clinician-reported signs and symptoms of MG
  • To evaluate the safety and tolerability of pozelimab + cemdisiran and cemdisiran monotherapy
  • To assess the concentration of total pozelimab in serum
  • To assess the concentrations of cemdisiran and its metabolites in plasma
  • To assess the immunogenicity of pozelimab
  • To assess the concentration of total C5 in plasma
  • To assess the immunogenicity of cemdisiran
  • To study the effect of pozelimab + cemdisiran and cemdisiran monotherapy on complement activation

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

DBTP- Double blind treatment plan (24 weeks) ETP - Extension treatment plan (28 weeks) OLTP- Open label treatment plan (68 weeks) Off-treatment follow up period (52 weeks)

Study Type

Interventional

Enrollment (Estimated)

235

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Australia
    • New South Wales
      • Sydney, New South Wales, Australia, 2217
        • Recruiting
        • Southern Neurology
    • South Australia
      • Elizabeth Vale, South Australia, Australia, 5112
    • Victoria
      • Fitzroy, Victoria, Australia, 3065
        • Recruiting
        • St Vincents Hospital Melbourne
    • Western Australia
      • Nedlands, Western Australia, Australia, 6009
        • Recruiting
        • Perron Institute for Neurological and Translational Science
        • Contact:
  • Belgium
      • Bruxelles, Belgium, 1070
        • Withdrawn
        • Université Libre de Bruxelles - Hôpital Erasme
    • Antwerpen
      • Edegem, Antwerpen, Belgium, 2650
        • Recruiting
        • UZ Antwerpen
        • Contact:
        • Principal Investigator:
          • Alicia Alonso-Jiménez
    • Hainaut
      • Lodelinsart, Hainaut, Belgium, 6042
        • Withdrawn
        • Chu Charleroi
    • Oost-Vlaanderen
      • Gent, Oost-Vlaanderen, Belgium, 9000
        • Recruiting
        • AZ Sint-Lucas
        • Contact:
        • Principal Investigator:
          • Jan De Bleecker
  • Canada
    • Alberta
      • Edmonton, Alberta, Canada, T6G 2X8
        • Not yet recruiting
        • University of Alberta
    • Ontario
      • London, Ontario, Canada, N6A 5A5
        • Recruiting
        • London Health Sciences Centre
      • Toronto, Ontario, Canada
        • Not yet recruiting
        • Toronto General Hospital
        • Contact:
    • Quebec
      • Montreal, Quebec, Canada, H2X-0J1
        • Withdrawn
        • Centre Hospitalier de l'Universite de Montreal (CHUM)
  • Czechia
    • Jihomoravsky Kraj
      • Brno, Jihomoravsky Kraj, Czechia, 625 00
        • Withdrawn
        • Fakultni nemocnice Brno
    • Moravskoslezský Kraj
      • Ostrava, Moravskoslezský Kraj, Czechia, 708 52
        • Withdrawn
        • Fakultni nemocnice Ostrava
  • Denmark
      • Aarhus N, Denmark, 8200
        • Recruiting
        • Aarhus University Hospital
        • Contact:
      • Copenhagen, Denmark, 3050
        • Recruiting
        • Copenhagen Neuromuscular Clinic at Rigshospitalet
        • Contact:
      • Odense, Denmark, 05000
        • Recruiting
        • Odense Universitetshospital - Infektionsmedicinsk Afdeling
    • Nordjylland
      • Aalborg, Nordjylland, Denmark, 9100
        • Recruiting
        • Aalborg Universitetshospital
        • Contact:
          • Izabella Obal
          • Phone Number: 4597666896
          • Email: anpp@rn.dk
        • Principal Investigator:
          • Izabella Obal
  • France
      • Le Kremlin-Bicêtre, France, 94275
        • Recruiting
        • CHU Bicêtre
      • Paris, France, 75013
        • Recruiting
        • Hopital de la Pitie Salpetriere
        • Contact:
          • Sophie Demeret
          • Phone Number: sophie.demeret@aphp.fr
      • Vandoeuvre Les Nancy, France, 54511
        • Recruiting
        • Centre Hospitalier Regional Universitaire (CHRU) de Nancy
    • Alpes-Maritimes
      • Nice, Alpes-Maritimes, France, 6002
        • Recruiting
        • Centre Hospitalier Universitaire (CHU) de Nice
  • Georgia
      • T'bilisi, Georgia, 0112
        • Recruiting
        • Israeli-Georgian Medical Research Clinic HEALTHYCORE
      • Tbilisi, Georgia, 0114
        • Recruiting
        • Pineo Medical Ecosystem
      • Tbilisi, Georgia, 0186
        • Recruiting
        • Multiprofile Clinic Consilium Medulla
      • Tbilisi, Georgia, 0114
        • Recruiting
        • Ltd New Hospitals
      • Tbilisi, Georgia, 0160
        • Recruiting
        • LTD Academician N. Kipshidze Central University Clinic
  • Germany
      • Berlin, Germany, 10117
        • Recruiting
        • Charité - Universitätsmedizin Berlin
      • Essen, Germany, 45127
        • Recruiting
        • University Hospital Essen, Dep. of Neurology
      • Jena, Germany, 07747
        • Recruiting
        • Universitätsklinikum Jena
    • Bayern
      • Munchen, Bayern, Germany, 80336
        • Not yet recruiting
        • Friedrich-Baur-Institute Dep. of Neurology Klinikum Munchen
    • Nordrhein-Westfalen
      • Munster, Nordrhein-Westfalen, Germany, 48149
    • Sachsen
  • India
      • Bangalore, India, 560002
        • Recruiting
        • Bangalore Medical College and Research Institute (BMCRI) - Victoria Hospital
        • Contact:
      • Chandigarh, India, 160012
        • Recruiting
        • Postgraduate Institute of Medical Education & Research
        • Contact:
      • New Delhi, India, 110029
        • Recruiting
        • All India Institute of Medical Sciences New Delhi
        • Contact:
    • Andhra Pradesh
      • Guntur, Andhra Pradesh, India, 522001
        • Recruiting
        • Government General Hospital, Guntur
        • Contact:
    • Gujarat
      • Surat, Gujarat, India, 395001
        • Recruiting
        • Institute of Neurosciences
    • Hyderabad
      • Panjagutta, Hyderabad, India, 500082
        • Recruiting
        • Nizam's Institute of Medical Sciences (NIMS)
    • Karnataka
      • Bangalore, Karnataka, India, 576104
        • Recruiting
        • Kasturba Medical College (KMC) - Udupi
    • Kerala
      • Thrissur, Kerala, India, 680005
        • Recruiting
        • Jubilee Mission Hospital
    • Kerela
      • Kochi, Kerela, India, 682041
        • Recruiting
        • Amrita Institute of Medical Sciences (AIMS) and Research Centre
        • Contact:
    • Maharashtra
      • Pune, Maharashtra, India, 411004
        • Recruiting
        • Deenanath Mangeshkar Hospital and Research Centre
        • Contact:
    • Mumbai
      • Parel, Mumbai, India, 400012
        • Recruiting
        • Seth G.S. Medical College & K.E.M. Hospital, Mumbai
        • Contact:
    • Punjab
      • Kochi, Punjab, India, 141008
        • Recruiting
        • Polakulath Narayanan Renai Medicity Multi Specialty Hospital, Kochi
      • Ludhiana, Punjab, India, 141008
        • Recruiting
        • Christian Medical College & Hospital
    • Rajasthan
      • Jaipur, Rajasthan, India, 302017
        • Recruiting
        • Apex Hospital
    • Telangana
      • Hyderabad, Telangana, India, 500034
        • Recruiting
        • City Neuro Centre, Hyderabad
        • Contact:
    • Uttar Pradesh
      • Lucknow, Uttar Pradesh, India, 226014
        • Withdrawn
        • Sanjay Gandhi Postgraduate Institute of Medical Sciences (SGPGIMS)
  • Italy
      • Bergamo, Italy, 24127
        • Not yet recruiting
        • Azienda Ospedaliera Papa Giovanni XXIII
      • Naples, Italy, 80131
        • Not yet recruiting
        • AORN Cardarelli Napoli
    • Lazio
      • Roma, Lazio, Italy, 00189
        • Recruiting
        • Azienda Ospedaliera Sant'Andrea
        • Contact:
        • Principal Investigator:
          • Michelangelo Maestri
      • Roma, Lazio, Italy, 00185
        • Recruiting
        • Universita' Degli Studi La Sapienza
        • Contact:
        • Principal Investigator:
          • Maurizio Inghilleri
    • Lombardia
      • Milano, Lombardia, Italy, 20133
        • Recruiting
        • Fondazione IRCCS Di Rilievo Nazionale Istituto Nazionale Neurologico Carlo Besta
        • Contact:
        • Principal Investigator:
          • Renato Mantegazza
      • Pavia, Lombardia, Italy, 27100
        • Not yet recruiting
        • IRCCS Mondino Foundation
    • Toscana
      • Pisa, Toscana, Italy, 56126
        • Recruiting
        • Azienda Ospedaliero Universitaria Pisana
  • Japan
      • Chiba, Japan, 260-8677
        • Recruiting
        • Chiba University Hospital
      • Hiroshima, Japan, 730-8518
        • Recruiting
        • Hiroshima City Hiroshima Citiz
      • Osaka, Japan, 543-8555
        • Recruiting
        • Osaka Red Cross Hospital - Neurology
      • Shimajiri-Gun, Japan, 901-1193
        • Recruiting
        • Okinawa Prefectural Nanbu Medical Center and Children's Medical Center
    • Hyogo
      • Kobe, Hyogo, Japan, 650-0047
        • Recruiting
        • Kobe City Medical Center General Hospital
        • Contact:
          • Michi Kawamoto
          • Phone Number: +80 78 302 4321
    • Koti
      • Nankoku-shi, Koti, Japan, 783-8505
        • Recruiting
        • Kochi Medical School Hospital
    • Okinawa
      • Ginowan-Shi, Okinawa, Japan, 901-2214
        • Recruiting
        • Okinawa National Hospital
    • Saitama
      • Kawagoe, Saitama, Japan, 350-8550
        • Recruiting
        • Saitama Medical University, Saitama Medical Center
    • Tokoyo
      • Kodaira, Tokoyo, Japan, 187-0002
        • Withdrawn
        • Showa General Hospital
    • Tokyo
      • Bunkyo-ku, Tokyo, Japan, 113-8519
        • Recruiting
        • Medical Hospital of Tokyo Medical and Dental University
        • Principal Investigator:
          • Yoichiro Nishida
      • Itabashi-ku, Tokyo, Japan, 173-8610
        • Recruiting
        • Nihon University Itabashi Hospital
      • Shinjuku ku, Tokyo, Japan, 160-0023
        • Recruiting
        • Tokyo Medical University Hospital
    • Yamaguchi
      • Ube, Yamaguchi, Japan, 755-8505
        • Recruiting
        • Yamaguchi University Hospital
  • Korea, Republic of
      • Daegu, Korea, Republic of, 41404
        • Recruiting
        • Kyungpook National University Chilgok Hospital
        • Contact:
        • Principal Investigator:
          • Jin-Sung Park
    • Seoul Teugbyeolsi
      • Seoul, Seoul Teugbyeolsi, Korea, Republic of, 03722
        • Recruiting
        • Samsung Medical Center - PPDS
        • Contact:
        • Principal Investigator:
          • Byoung Joon Kim
  • Poland
    • Malopolskie
      • Kraków, Malopolskie, Poland, 31-505
        • Recruiting
        • Centrum Neurologii Klinicznej, Krakowska Akademia Neurologii
    • Mazovian
      • Warsaw, Mazovian, Poland, 01-684
        • Recruiting
        • NeuroProtect
    • Mazowieckie
      • Warszawa, Mazowieckie, Poland, 02-097
        • Recruiting
        • Uniwersyteckie Centrum Kliniczne WUM, Centralny Szpital Kliniczny
    • Pomorskie
      • Gdansk, Pomorskie, Poland, 80-952
        • Recruiting
        • Gdanski Uniwersytet Medyczny
    • Wielkopolska
      • Poznan, Wielkopolska, Poland, 61-853
        • Recruiting
        • NZOZ Neuro-Kard
    • Wielkopolskie
      • Poznań, Wielkopolskie, Poland, 61-731
        • Recruiting
        • Clinical Research Center Spólka z Ograniczona Odpowiedzialnoscia, Medic-R Spólka Komandytowa
  • Serbia
      • Belgrade, Serbia, 11000
        • Not yet recruiting
        • University Clinical Center of Serbia - PPDS
        • Contact:
      • Nis, Serbia, 700461
        • Not yet recruiting
        • University Clinical Centre Nis
        • Contact:
  • Spain
      • Barcelona, Spain, 08041
        • Recruiting
        • Hospital de la Santa Creu i Sant Pau
        • Contact:
        • Principal Investigator:
          • Elena Cortes Vicente
      • Madrid, Spain, 28046
        • Recruiting
        • Hospital Universitario La Paz - PPDS
        • Contact:
        • Principal Investigator:
          • Francisco Javier Rodriguez de Rivera
      • Valencia, Spain, 46026
        • Recruiting
        • Hospital Universitari i Politecnic La Fe
  • Taiwan
      • Kaohsiung, Taiwan, 833
        • Recruiting
        • Chang Gung Medical Foundation Kaohsiung Chang Gung Memorial Hospital
        • Contact:
        • Principal Investigator:
          • Nai-Wen Tsai
      • Taichung, Taiwan, 404
        • Recruiting
        • China Medical University Hospital
        • Contact:
        • Principal Investigator:
          • Yuh-Cherng Guo
      • Tainan City, Taiwan, 701
        • Recruiting
        • National Cheng Kung University Hospital
        • Contact:
        • Principal Investigator:
          • Yuan Ting Sun
      • Taipei, Taiwan, 111
        • Recruiting
        • Shin Kong Wu Ho Su Memorial Hospital
        • Contact:
        • Principal Investigator:
          • Jiann-Horng Yeh
      • Taoyuan, Taiwan, 333
        • Recruiting
        • Chang Gung memorial hospital
        • Contact:
        • Principal Investigator:
          • Long-Sun Ro
  • Turkey
      • Samsun, Turkey, 55239
        • Recruiting
        • Ondokuz Mayis Universitesi Tip Fakultesi Hastanesi
        • Contact:
        • Principal Investigator:
          • Murat Terzi
      • Trabzon, Turkey, 61080
        • Recruiting
        • Karadeniz Technical University Farabi Hospital
    • Izmir
      • Balcova, Izmir, Turkey, 35340
        • Recruiting
        • Dokuz Eylul University Medical Faculty
    • Van
      • Umuttepe, Van, Turkey, 41380
        • Recruiting
        • Kocaeli University Hospital
        • Contact:
  • United Kingdom
      • Birmingham, United Kingdom, B15 2TH
        • Recruiting
        • University Hospital Birmingham
    • South Yorkshire
      • Sheffield, South Yorkshire, United Kingdom, 511 9NE
        • Recruiting
        • Sheffield Teaching Hospital NHS Foundation Trust
        • Contact:
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85018
    • California
      • Irvine, California, United States, 92868
        • Recruiting
        • University of California, Irvine
      • Los Angeles, California, United States, 90033
        • Recruiting
        • University of Southern California
        • Contact:
        • Principal Investigator:
          • Said Beydoun
    • Colorado
      • Colorado Springs, Colorado, United States, 80907-5307
        • Recruiting
        • Colorado Springs Neurological Associates
        • Contact:
        • Principal Investigator:
          • Laurence Adams
    • Florida
      • Boca Raton, Florida, United States, 33487
        • Recruiting
        • SFM Clinical Research, LLC
        • Contact:
        • Principal Investigator:
          • Marc Feinberg
      • Miami, Florida, United States, 33175
        • Recruiting
        • Diverse Clinical Research
      • Naples, Florida, United States, 34105-8523
      • Orlando, Florida, United States, 32806-6264
        • Recruiting
        • Neurological Services of Orlando
      • Port Charlotte, Florida, United States, 33952
        • Recruiting
        • Medsol Clinical Research Center Inc
      • Tampa, Florida, United States, 33612
        • Recruiting
        • University of South Florida
        • Contact:
        • Principal Investigator:
          • Tuan Vu
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Withdrawn
        • Northwestern University
      • Evanston, Illinois, United States, 60201-3137
        • Recruiting
        • Northshore University Health System
        • Contact:
        • Principal Investigator:
          • Alexandru Barboi
      • O'Fallon, Illinois, United States, 62269
        • Recruiting
        • Prairie Education And Research Cooperative
        • Contact:
        • Principal Investigator:
          • Raghav Govindarajan
      • Rolling Meadows, Illinois, United States, 60008-3154
        • Terminated
        • Northwest Neurology Ltd. - Clinedge - PPDS
    • Michigan
      • Detroit, Michigan, United States, 48201
        • Recruiting
        • Wayne State University School of Medicine
        • Contact:
    • New York
      • New York, New York, United States, 10021
        • Recruiting
        • Weill Cornell Medicine - Peripheral Neuropathy Center
        • Contact:
        • Principal Investigator:
          • Mary Vo
    • North Carolina
      • Charlotte, North Carolina, United States, 28207
        • Withdrawn
        • Atrium Health Neurosciences Institute Charlotte, a facility of Carolinas Medical Center
    • Ohio
      • Centerville, Ohio, United States, 45459
        • Recruiting
        • Dayton Center for Neurological Disorders
      • Cincinnati, Ohio, United States, 45219
        • Recruiting
        • University of Cincinnati Gardner Neuroscience Institute
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • Penn Medicine University City
    • Texas
      • Arlington, Texas, United States, 76015
        • Terminated
        • Texas Institute for Neurological Disorders - Arlington
      • Austin, Texas, United States, 78759
        • Recruiting
        • Austin Neuromuscular Center
        • Contact:
        • Principal Investigator:
          • Yessar Hussain
      • Houston, Texas, United States, 77030
        • Recruiting
        • Nerve and Muscle Center of Texas
        • Contact:
        • Principal Investigator:
          • Aziz Shaibani

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Key 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).

Key Exclusion Criteria:

  1. 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
  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. 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

NOTE: Other protocol-defined Inclusion/ Exclusion Criteria apply

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Group 1
Placebo in DBTP; Re-randomized to Combination or Cemdisiran in ETP and OLTP
Drug: Pozelimab + Cemdisiran
Subcutaneous administration as described in the protocol
Drug: Cemdisiran
SC administration as described in the protocol
Other Names:
  • ALN-CC5
Other: Placebo
SC administration as described in the protocol
Experimental: Group 2
Combination regimen throughout the study
Drug: Pozelimab + Cemdisiran
Subcutaneous administration as described in the protocol
Experimental: Group 3
Cemdisiran throughout the study
Drug: Cemdisiran
SC administration as described in the protocol
Other Names:
  • ALN-CC5
Experimental: Group 4
Pozelimab monotherapy in DBTP followed by combination in ETP and OLTP
Drug: Pozelimab
SC administration as described in the protocol
Other Names:
  • REGN3918

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score
Time Frame: From baseline to week 24
The total MG-ADL score ranges from 0 to 24 points, with higher scores indicating greater functional impairment and disability
From baseline to week 24

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from baseline in Quantitative Myasthenia Gravis (QMG) score
Time Frame: Week 24
QMG total scores range from 0 to 39, with higher scores representing greater impairment
Week 24
Proportion of patients responding on the MG-ADL
Time Frame: From baseline to week 24
≥3-point improvement
From baseline to week 24
Proportion of patients responding on the QMG
Time Frame: From baseline to week 24
≥5-point improvement
From baseline to week 24
Proportion of patients with consistent response on the MG-ADL
Time Frame: From baseline to week 24
At least a 2-point MG-ADL improvement on 2 or more consecutive assessments spanning 4 or more weeks during the DBTP
From baseline to week 24
Proportion of patients with minimal symptom expression (MSE)
Time Frame: Week 24
Score of 0 to 1 on the MG-ADL
Week 24
Change from baseline in the Myasthenia Gravis Composite (MGC) total score
Time Frame: Week 24
MGC score ranges from 0 to 50, with higher score indicating higher impairment
Week 24
Change from baseline in Myasthenia Gravis Quality of Life (MG QOL15r) total score
Time Frame: Week 24
Total score ranges from 0 to 30 points; a higher score represents greater impairment
Week 24
Proportion of patients with improvement point thresholds on MG-ADL
Time Frame: From baseline to week 24
≥2, 4, 5, 6, 7, 8, 9, or 10
From baseline to week 24
Proportion of patients with improvement point thresholds on QMG
Time Frame: From baseline to week 24
≥3, 4, 6, 7, 8, 9, or 10
From baseline to week 24
Incidence and severity of treatment-related adverse events (TEAEs) in patients treated with pozelimab + cemdisiran or placebo
Time Frame: Through week 24
Through week 24
Incidence and severity of serious adverse events (SAEs) in patients treated with pozelimab + cemdisiran or placebo
Time Frame: Through week 24
Through week 24
Incidence and severity of adverse events of special interest (AESIs) in patients treated with pozelimab + cemdisiran or placebo
Time Frame: Through week 24
Through week 24
Concentrations of total pozelimab in serum
Time Frame: Through study duration, approximate 172 weeks
Through study duration, approximate 172 weeks
Concentrations of cemdisiran and its metabolites in plasma
Time Frame: Through study duration, approximate 172 weeks
Through study duration, approximate 172 weeks
Incidence of treatment-emergent anti-drug antibodies (ADAs) to pozelimab over time
Time Frame: Through study duration, approximately 172 weeks
Through study duration, approximately 172 weeks
Incidence of treatment-emergent ADAs to cemdisiran over time
Time Frame: Through study duration, approximate 172 weeks
Through study duration, approximate 172 weeks
Change in CH50 over time
Time Frame: Through study duration, approximately 172 weeks
Through study duration, approximately 172 weeks
Percent change in CH50 over time
Time Frame: Through study duration, approximately 172 weeks
Through study duration, approximately 172 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Regeneron Pharmaceuticals

Investigators

  • Study Director: Clinical Trial Management, Regeneron Pharmaceuticals

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 14, 2021

Primary Completion (Estimated)

August 29, 2025

Study Completion (Estimated)

March 23, 2028

Study Registration Dates

First Submitted

September 27, 2021

First Submitted That Met QC Criteria

September 27, 2021

First Posted (Actual)

October 7, 2021

Study Record Updates

Last Update Posted (Actual)

April 8, 2024

Last Update Submitted That Met QC Criteria

April 5, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • R3918-MG-2018
  • 2020-003272-41 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing

IPD Sharing Time Frame

When Regeneron has received marketing authorization from major health authorities (e.g., FDA, European Medicines Agency (EMA), Pharmaceuticals and Medical Devices Agency (PMDA), etc.) for the product and indication, has made the study results publicly available (e.g., scientific publication, scientific conference, clinical trial registry), has the legal authority to share the data, and has ensured the ability to protect participant privacy.

IPD Sharing Access Criteria

Qualified researchers can submit a proposal for access to individual patient or aggregate level data from a Regeneron-sponsored clinical trial through Vivli. Regeneron's Independent Research Request Evaluation Criteria can be found at: https://www.regeneron.com/sites/default/files/Regeneron-External-Data-Sharing-Policy-and-Independent-Research-Request-Evaluation-Criteria.pdf

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • ICF
  • ANALYTIC_CODE
  • CSR

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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