Phase 3 Study to Assess the Efficacy and Safety of Batoclimab as Induction and Maintenance Therapy in Adult Participants With Generalized Myasthenia Gravis

A Phase 3, Multi-center, Randomized, Quadruple-blind, Placebo-controlled Study to Assess the Efficacy and Safety of Batoclimab as Induction and Maintenance Therapy in Adult Participants With Generalized Myasthenia Gravis (gMG)

The purpose of this 4-period study is to confirm the efficacy and safety of batoclimab in participants with gMG. In Period 1, participants will be randomized 1:1:1 to receive batoclimab 680 milligrams (mg) subcutaneously (SC) once a week (QW) or 340 mg SC QW or placebo. The primary efficacy endpoint will be assessed by change in the myasthenia gravis activities of daily living (MG- ADL) score in acetylcholine receptor antibody seropositive (AChRAb+) participants. In Period 2, participants previously treated with batoclimab will be re-randomized to stay on batoclimab (340 mg SC QW or 340 mg SC every two weeks) or receive placebo treatment. The secondary endpoint of maintenance of efficacy will be assessed by change in the MG- ADL score in AChRAb+ participants. Participants demonstrating a response to batoclimab during either Period 1 or 2 may enter the long-term extension (Period 3). Participants who complete Period 3 are eligible to participate in Period 4 (Optional Long-Term extension) according to their treatment assignment in Period 3.

Study Overview

• Status: Recruiting
• Conditions: Generalized Myasthenia Gravis
• Intervention / Treatment: Drug: Batoclimab 680 mg SC weekly; Drug: Batoclimab 340 mg SC weekly; Drug: Matching Placebo SC; Drug: Batoclimab 340 mg SC bi-weekly

• Study Type: Interventional
• Enrollment (Estimated): 240
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Central Study Contact; Phone Number: 18007970414; Email: clinicaltrials@immunovant.com

Study Locations

• Canada
  • Alberta
    • Edmonton, Alberta, Canada, T6G 2B7
      • Recruiting
      • Site Number -2001
  • Quebec
    • Montreal, Quebec, Canada, H3A 2B4
      • Recruiting
      • Site Number -2004
• Georgia
  • Tbilisi, Georgia, 00114
    • Recruiting
    • Site Number -8002
  • Tbilisi, Georgia, 00114
    • Recruiting
    • Site Number -8004
  • Tbilisi, Georgia, 00160
    • Recruiting
    • Site Number -8001
  • Tbilisi, Georgia, 00186
    • Recruiting
    • Site Number -8003
  • Tbilisi, Georgia, 0179
    • Recruiting
    • Site Number - 8005
• Germany
  • Leipzig, Germany, 04103
    • Recruiting
    • Site Number -6504
  • Würzburg, Germany, 97074
    • Recruiting
    • Site Number -6502
• Hungary
  • Budapest, Hungary, 1082
    • Recruiting
    • Site Number -7553
  • Kistarcsa, Hungary, 2143
    • Recruiting
    • Site Number - 7552
• Italy
  • Brescia, Italy, 25123
    • Recruiting
    • Site Number -6006
  • Genova, Italy, 16132
    • Recruiting
    • Site Number - 6002
  • Milano, Italy, 20133
    • Recruiting
    • Site Number - 6003
  • Napoli, Italy, 80131
    • Recruiting
    • Site Number -6001
  • Roma, Italy, 00189
    • Recruiting
    • Site Number -6005
• Japan
  • Fuchu-shi, Japan, 183-0042
    • Recruiting
    • Site Number -4014
  • Hanamaki-shi, Japan, 025-0082
    • Recruiting
    • Site Number - 4002
  • Higashimatsushima, Japan, 355-0005
    • Recruiting
    • Site Number - 4013
  • Kagawa, Japan, 761-0793
    • Recruiting
    • Site Number - 4006
  • Kawasaki-shi, Japan, 216-8511
    • Recruiting
    • Site Number - 4009
  • Koriyama-shi, Japan, 963-8563
    • Recruiting
    • Site Number - 4012
  • Koshigaya-shi, Japan, 343-8555
    • Recruiting
    • Site Number - 4007
  • Matsuyama-shi, Japan, 791-8026
    • Recruiting
    • Site Number - 4011
  • Miyagi, Japan, 983-8520
    • Recruiting
    • Site Number - 4008
  • Narita-shi, Japan, 286-8520
    • Recruiting
    • Site Number - 4003
  • Osaka, Japan, 534-0021
    • Recruiting
    • Site Number - 4010
  • Osaka, Japan, 565-0871
    • Recruiting
    • Site Number - 4004
  • Osaka, Japan, 589-8511
    • Recruiting
    • Site Number - 4001
  • Tokyo, Japan, 160-0023
    • Recruiting
    • Site Number - 4005
  • Yokohama-shi, Japan, 222-0036
    • Recruiting
    • Site Number -4016
  • Yonago-shi, Japan, 683-8504
    • Recruiting
    • Site Number -4017
• Korea, Republic of
  • Daegu, Korea, Republic of, 41944
    • Recruiting
    • Site Number -4505
  • Seoul, Korea, Republic of, 06351
    • Recruiting
    • Site Number -4501
• Poland
  • Katowice, Poland, 40-123
    • Recruiting
    • Site Number -3001
  • Krakow, Poland, 31-324
    • Recruiting
    • Site Number -3004
  • Krakow, Poland, 31-503
    • Recruiting
    • Site Number - 3003
  • Kraków, Poland, 31-505
    • Recruiting
    • Site Number -3002
  • Lublin, Poland, 20-701
    • Recruiting
    • Site Number - 3008
  • Poznań, Poland, 61-731
    • Recruiting
    • Site Number -3006
  • Warszawa, Poland, 01-684
    • Recruiting
    • Site Number - 3005
• Romania
  • Constanta, Romania, 900628
    • Recruiting
    • Site Number -7502
  • Targu Mures, Romania, 540136
    • Recruiting
    • Site Number -7501
  • Timişoara, Romania, 300723
    • Recruiting
    • Site Number -7503
• Serbia
  • Belgrade, Serbia, 11000
    • Recruiting
    • Site Number - 9001
  • Niš, Serbia, 1800
    • Recruiting
    • Site Number - 9002
• Spain
  • Barcelona, Spain, 08195
    • Recruiting
    • Site Number -3502
  • Barcelona, Spain, 8041
    • Recruiting
    • Site Number -3505
  • Cordoba, Spain, 14011
    • Recruiting
    • Site Number -3504
  • Madrid, Spain, 28034
    • Recruiting
    • Site Number -3501
  • Madrid, Spain, 28046
    • Recruiting
    • Site Number -3503
• United States
  • Arizona
    • Phoenix, Arizona, United States, 85028
      • Recruiting
      • Site Number -1022
    • Scottsdale, Arizona, United States, 85251
      • Recruiting
      • Site Number -1029
  • California
    • Carlsbad, California, United States, 92011
      • Recruiting
      • Site Number -1002
    • Irvine, California, United States, 92697
      • Recruiting
      • Site Number -1009
    • San Francisco, California, United States, 94143
      • Recruiting
      • Site Number -1032
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Recruiting
      • Site Number - 1027
  • Connecticut
    • New Haven, Connecticut, United States, 06519
      • Recruiting
      • Site Number - 1025
  • Florida
    • Boca Raton, Florida, United States, 33487
      • Recruiting
      • Site Number -1017
    • Clearwater, Florida, United States, 33761
      • Recruiting
      • Site Number -1007
    • Maitland, Florida, United States, 32751
      • Recruiting
      • Site Number -1010
    • Orlando, Florida, United States, 32806
      • Recruiting
      • Site Number -1019
    • Port Charlotte, Florida, United States, 33952
      • Recruiting
      • Site Number - 1028
    • Tampa, Florida, United States, 33612
      • Recruiting
      • Site Number - 1015
  • Kansas
    • Fairway, Kansas, United States, 66205
      • Recruiting
      • Site Number -1011
  • Kentucky
    • Lexington, Kentucky, United States, 40536
      • Recruiting
      • Site Number -1003
  • Michigan
    • East Lansing, Michigan, United States, 48824
      • Recruiting
      • Site Number -1013
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27599
      • Recruiting
      • Site Number - 1018
    • Durham, North Carolina, United States, 27710
      • Recruiting
      • Site Number - 1008
  • Ohio
    • Cleveland, Ohio, United States, 44195
      • Recruiting
      • Site Number -1004
  • Oregon
    • Portland, Oregon, United States, 97239
      • Recruiting
      • Site Number -1006
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Recruiting
      • Site Number - 1023
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Recruiting
      • Site Number - 1030
  • Texas
    • Austin, Texas, United States, 78759
      • Recruiting
      • Site Number -1001
    • Dallas, Texas, United States, 75206
      • Recruiting
      • Site Number -1016
    • Round Rock, Texas, United States, 78681
      • Recruiting
      • Site Number - 1014
  • Utah
    • Murray, Utah, United States, 84107
      • Recruiting
      • Site Number -1031
  • Virginia
    • Charlottesville, Virginia, United States, 22908
      • Recruiting
      • Site Number -1005

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Are ≥ 18 years of age at the Screening Visit.
2. Have mild to severe gMG by Myasthenia Gravis Foundation of America (MGFA) classification Class II, III, or IVa at the Screening Visit.
3. Have a QMG score ≥ 11 at the Screening and Baseline Visits.
4. Have a MG-ADL score of ≥ 5 at the Screening and Baseline Visits.
5. Additional inclusion criteria are defined in the protocol.

Exclusion Criteria:

1. Have experienced myasthenic crisis within 3 months of the Screening Visit.
2. Have had a thymectomy performed < 6 months prior to the Screening Visit or have a planned thymectomy during the study period.
3. Have any active or untreated malignant thymoma.
4. Have received any agent or therapy (exclusive of those identified within inclusion criteria) with immunosuppressive properties (e.g., stem cell therapy, chemotherapies) within the past year.
5. Have used anti-FcRn treatment within 3 months prior to the Screening Visit or have a documented history of non-response to prior anti-FcRn treatment.
6. Additional exclusion criteria are defined in the protocol.

Study Plan

How is the study designed?

Design Details

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

Number of Arms

6

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Batoclimab Induction Dose 1 (Period 1)	Drug: Batoclimab 680 mg SC weekly; Batoclimab is a fully human anti-neonatal fragment crystallizable receptor (FcRn) monoclonal antibody; Other Names: IMVT-1401
Experimental: Batoclimab Induction Dose 2 (Period 1)	Drug: Batoclimab 340 mg SC weekly; Batoclimab is a fully human anti-neonatal fragment crystallizable receptor (FcRn) monoclonal antibody; Other Names: IMVT-1401
Placebo Comparator: Placebo Induction Dose (Period 1)	Drug: Matching Placebo SC; Placebo
Experimental: Batoclimab Maintenance Dose 1 (Period 2)	Drug: Batoclimab 340 mg SC weekly; Batoclimab is a fully human anti-neonatal fragment crystallizable receptor (FcRn) monoclonal antibody; Other Names: IMVT-1401
Experimental: Batoclimab Maintenance Dose 2 (Period 2)	Drug: Batoclimab 340 mg SC bi-weekly; Batoclimab is a fully human anti-neonatal fragment crystallizable receptor (FcRn) monoclonal antibody; Other Names: IMVT-1401
Placebo Comparator: Placebo Maintenance Dose (Period 2)	Drug: Matching Placebo SC; Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from Baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) score in acetylcholine receptor (AChR) Ab seropositive (AChRAb+) participants	MG-ADL is an 8-item, participant-reported questionnaire that assesses gMG symptoms and their effects on activities of daily living. Each item is assessed on a 4-point scale where a score of 0 represents normal function and a score of 3 represents loss of ability to perform that function. Total score ranges from 0 to 24, with higher scores indicating greater functional impairment and disability.	Baseline (Day 1) to Week 12

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from Baseline in Quantitative Myasthenia Gravis (QMG) score in AChRAb+ participants	QMG is clinician-reported assessment to evaluate muscle weakness in participants with MG. The QMG consists of 13 items ranging from 0 to 3 with 3 being the most severe. Total score ranges from 0 to 39, with higher scores representing greater impairment.	Baseline (Day 1) to Week 12
Change from Baseline in MG-ADL score for AChRAb+ randomized withdrawal participants		Baseline (Week 12) to Week 24
Percentage of AChRAb+ participants with greater than equal to (>=) 3-point improvement in QMG score		Up to Week 12
Percentage of AChRAb+ participants achieving MG-ADL score of 0 or 1 by Week 12		Up to Week 12
Change from Baseline in MG-ADL score in AChRAB- (AChRAB negative) participants		Baseline (Day 1) to Week 12
Percentage of Participants with Treatment Emergent Adverse Events (TEAEs)	An adverse event (AE) is defined as any untoward medical occurrence in a participant who has either been administered a study drug or has undergone study procedures.	Up to 76 Weeks
Percentage of Participants With Clinically Significant Changes in Vital Sign Measurements	Vital signs, including systolic and diastolic blood pressures, pulse rate, respiratory rate, and temperature will be obtained and recorded at specified timepoints. All vital sign measures will be obtained with the participant in the supine position and having rested for at least 5 minutes.	Up to 76 Weeks
Number of Participants with Clinically Significant Changes in Laboratory Results	Blood samples will be collected at specified timepoints for the analysis of laboratory parameters including clinical chemistry, hematology and urinalysis.	Up to 76 Weeks
Percentage of participants with clinical laboratory-related TEAEs or treatment emergent laboratory abnormalities.		Up to 76 Weeks

Collaborators and Investigators

• Sponsor: Immunovant Sciences GmbH

Study record dates

Study Major Dates

• Study Start (Actual): June 27, 2022
• Primary Completion (Estimated): April 1, 2024
• Study Completion (Estimated): April 1, 2025

Study Registration Dates

• First Submitted: May 26, 2022
• First Submitted That Met QC Criteria: May 26, 2022
• First Posted (Actual): June 3, 2022

Study Record Updates

• Last Update Posted (Actual): January 5, 2024
• Last Update Submitted That Met QC Criteria: January 2, 2024
• Last Verified: January 1, 2024

More Information

Terms related to this study

• Keywords: IMVT-1401; Myasthenia Gravis; monoclonal antibody; batoclimab; acetylcholine receptor; RVT-1401
• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Immune System Diseases; Neoplasms; Autoimmune Diseases of the Nervous System; Autoimmune Diseases; Neoplasms by Site; Neurologic Manifestations; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Neuromuscular Manifestations; Nervous System Neoplasms; Paraneoplastic Syndromes, Nervous System; Paraneoplastic Syndromes; Neuromuscular Junction Diseases; Muscle Weakness; Myasthenia Gravis
• Other Study ID Numbers: IMVT-1401-3101

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No