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

• ClinicalTrials.gov Identifier: NCT05403541
• Recruitment Status: Recruiting
• First Posted: June 3, 2022
• Last Update Posted: January 5, 2024
• Sponsor: Immunovant Sciences GmbH
• Information provided by (Responsible Party): Immunovant Sciences GmbH

Tracking Information

• First Submitted Date: May 26, 2022
• First Posted Date: June 3, 2022
• Last Update Posted Date: January 5, 2024
• Actual Study Start Date: June 27, 2022
• Estimated Primary Completion Date: April 2024 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: May 26, 2022)

Change from Baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) score in acetylcholine receptor (AChR) Ab seropositive (AChRAb+) participants [ Time Frame: Baseline (Day 1) to Week 12 ]

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.

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: May 26, 2022)

• Change from Baseline in Quantitative Myasthenia Gravis (QMG) score in AChRAb+ participants [ Time Frame: Baseline (Day 1) to Week 12 ]
  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.
• Change from Baseline in MG-ADL score for AChRAb+ randomized withdrawal participants [ Time Frame: Baseline (Week 12) to Week 24 ]
• Percentage of AChRAb+ participants with greater than equal to (>=) 3-point improvement in QMG score [ Time Frame: Up to Week 12 ]
• Percentage of AChRAb+ participants achieving MG-ADL score of 0 or 1 by Week 12 [ Time Frame: Up to Week 12 ]
• Change from Baseline in MG-ADL score in AChRAB- (AChRAB negative) participants [ Time Frame: Baseline (Day 1) to Week 12 ]
• Percentage of Participants with Treatment Emergent Adverse Events (TEAEs) [ Time Frame: Up to 76 Weeks ]
  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.
• Percentage of Participants With Clinically Significant Changes in Vital Sign Measurements [ Time Frame: Up to 76 Weeks ]
  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.
• Number of Participants with Clinically Significant Changes in Laboratory Results [ Time Frame: Up to 76 Weeks ]
  Blood samples will be collected at specified timepoints for the analysis of laboratory parameters including clinical chemistry, hematology and urinalysis.
• Percentage of participants with clinical laboratory-related TEAEs or treatment emergent laboratory abnormalities. [ Time Frame: Up to 76 Weeks ]

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Phase 3 Study to Assess the Efficacy and Safety of Batoclimab as Induction and Maintenance Therapy in Adult Participants With Generalized Myasthenia Gravis
• Official Title: 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)
• Brief Summary: 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.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 3

Study Design

Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Masking Description:

Sponsor Staff is also masked, so the study is Quadruple-blind.

Primary Purpose: Treatment

• Condition: Generalized Myasthenia Gravis

Intervention

• Drug: Batoclimab 680 mg SC weekly
  Batoclimab is a fully human anti-neonatal fragment crystallizable receptor (FcRn) monoclonal antibody
  Other Name: IMVT-1401
• Drug: Batoclimab 340 mg SC weekly
  Batoclimab is a fully human anti-neonatal fragment crystallizable receptor (FcRn) monoclonal antibody
  Other Name: IMVT-1401
• Drug: Matching Placebo SC
  Placebo
• Drug: Batoclimab 340 mg SC bi-weekly
  Batoclimab is a fully human anti-neonatal fragment crystallizable receptor (FcRn) monoclonal antibody
  Other Name: IMVT-1401

Study Arms

• Experimental: Batoclimab Induction Dose 1 (Period 1)
  Intervention: Drug: Batoclimab 680 mg SC weekly
• Experimental: Batoclimab Induction Dose 2 (Period 1)
  Intervention: Drug: Batoclimab 340 mg SC weekly
• Placebo Comparator: Placebo Induction Dose (Period 1)
  Intervention: Drug: Matching Placebo SC
• Experimental: Batoclimab Maintenance Dose 1 (Period 2)
  Intervention: Drug: Batoclimab 340 mg SC weekly
• Experimental: Batoclimab Maintenance Dose 2 (Period 2)
  Intervention: Drug: Batoclimab 340 mg SC bi-weekly
• Placebo Comparator: Placebo Maintenance Dose (Period 2)
  Intervention: Drug: Matching Placebo SC

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: January 2, 2024): 240
• Original Estimated Enrollment (submitted: May 26, 2022): 210
• Estimated Study Completion Date: April 2025
• Estimated Primary Completion Date: April 2024 (Final data collection date for primary outcome measure)

Eligibility Criteria

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Contacts

Contact: Central Study Contact; 18007970414; clinicaltrials@immunovant.com

• Listed Location Countries: Canada, Georgia, Germany, Hungary, Italy, Japan, Korea, Republic of, Poland, Romania, Serbia, Spain, United States

Administrative Information

• NCT Number: NCT05403541
• Other Study ID Numbers: IMVT-1401-3101
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Immunovant Sciences GmbH
• Original Responsible Party: Same as current
• Current Study Sponsor: Immunovant Sciences GmbH
• Original Study Sponsor: Same as current
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Immunovant Sciences GmbH
• Verification Date: January 2024