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

Generalized Myasthenia Gravis Clinical Trial

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)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05403541
• Other study ID #: IMVT-1401-3101
• Status: Recruiting
• Phase: Phase 3
• Start date: June 27, 2022
• Est. completion date: April 2025

Study information

• Verified date: January 2024
• Source: Immunovant Sciences GmbH
• Contact: Central Study Contact
• Phone: 18007970414
• Email: clinicaltrials[@]immunovant.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 240
• Est. completion date: April 2025
• Est. primary completion date: April 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Generalized Myasthenia Gravis
• Muscle Weakness
• Myasthenia Gravis

Intervention

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

Locations

Country	Name	City	State
Canada	Site Number -2001	Edmonton	Alberta
Canada	Site Number -2004	Montreal	Quebec
Georgia	Site Number - 8005	Tbilisi
Georgia	Site Number -8001	Tbilisi
Georgia	Site Number -8002	Tbilisi
Georgia	Site Number -8003	Tbilisi
Georgia	Site Number -8004	Tbilisi
Germany	Site Number -6504	Leipzig
Germany	Site Number -6502	Würzburg
Hungary	Site Number -7553	Budapest
Hungary	Site Number - 7552	Kistarcsa
Italy	Site Number -6006	Brescia
Italy	Site Number - 6002	Genova
Italy	Site Number - 6003	Milano
Italy	Site Number -6001	Napoli
Italy	Site Number -6005	Roma
Japan	Site Number -4014	Fuchu-shi
Japan	Site Number - 4002	Hanamaki-shi
Japan	Site Number - 4013	Higashimatsushima
Japan	Site Number - 4006	Kagawa
Japan	Site Number - 4009	Kawasaki-shi
Japan	Site Number - 4012	Koriyama-shi
Japan	Site Number - 4007	Koshigaya-shi
Japan	Site Number - 4011	Matsuyama-shi
Japan	Site Number - 4008	Miyagi
Japan	Site Number - 4003	Narita-shi
Japan	Site Number - 4001	Osaka
Japan	Site Number - 4004	Osaka
Japan	Site Number - 4010	Osaka
Japan	Site Number - 4005	Tokyo
Japan	Site Number -4016	Yokohama-shi
Japan	Site Number -4017	Yonago-shi
Korea, Republic of	Site Number -4505	Daegu
Korea, Republic of	Site Number -4501	Seoul
Poland	Site Number -3001	Katowice
Poland	Site Number - 3003	Krakow
Poland	Site Number -3004	Krakow
Poland	Site Number -3002	Kraków
Poland	Site Number - 3008	Lublin
Poland	Site Number -3006	Poznan
Poland	Site Number - 3005	Warszawa
Romania	Site Number -7502	Constanta
Romania	Site Number -7501	Targu Mures
Romania	Site Number -7503	Timisoara
Serbia	Site Number - 9001	Belgrade
Serbia	Site Number - 9002	Niš
Spain	Site Number -3502	Barcelona
Spain	Site Number -3505	Barcelona
Spain	Site Number -3504	Cordoba
Spain	Site Number -3501	Madrid
Spain	Site Number -3503	Madrid
United States	Site Number - 1027	Aurora	Colorado
United States	Site Number -1001	Austin	Texas
United States	Site Number -1017	Boca Raton	Florida
United States	Site Number -1002	Carlsbad	California
United States	Site Number - 1018	Chapel Hill	North Carolina
United States	Site Number - 1030	Charleston	South Carolina
United States	Site Number -1005	Charlottesville	Virginia
United States	Site Number -1007	Clearwater	Florida
United States	Site Number -1004	Cleveland	Ohio
United States	Site Number -1016	Dallas	Texas
United States	Site Number - 1008	Durham	North Carolina
United States	Site Number -1013	East Lansing	Michigan
United States	Site Number -1011	Fairway	Kansas
United States	Site Number -1009	Irvine	California
United States	Site Number -1003	Lexington	Kentucky
United States	Site Number -1010	Maitland	Florida
United States	Site Number -1031	Murray	Utah
United States	Site Number - 1025	New Haven	Connecticut
United States	Site Number -1019	Orlando	Florida
United States	Site Number - 1023	Philadelphia	Pennsylvania
United States	Site Number -1022	Phoenix	Arizona
United States	Site Number - 1028	Port Charlotte	Florida
United States	Site Number -1006	Portland	Oregon
United States	Site Number - 1014	Round Rock	Texas
United States	Site Number -1032	San Francisco	California
United States	Site Number -1029	Scottsdale	Arizona
United States	Site Number - 1015	Tampa	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Immunovant Sciences GmbH

Countries where clinical trial is conducted

United States,  Canada,  Georgia,  Germany,  Hungary,  Italy,  Japan,  Korea, Republic of,  Poland,  Romania,  Serbia,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	Change from Baseline in MG-ADL score for AChRAb+ randomized withdrawal participants		Baseline (Week 12) to Week 24
Secondary	Percentage of AChRAb+ participants with greater than equal to (>=) 3-point improvement in QMG score		Up to Week 12
Secondary	Percentage of AChRAb+ participants achieving MG-ADL score of 0 or 1 by Week 12		Up to Week 12
Secondary	Change from Baseline in MG-ADL score in AChRAB- (AChRAB negative) participants		Baseline (Day 1) to Week 12
Secondary	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
Secondary	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
Secondary	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
Secondary	Percentage of participants with clinical laboratory-related TEAEs or treatment emergent laboratory abnormalities.		Up to 76 Weeks