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

NCT05403541 | Active Not Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: IMVT-1401-3101
• Study Type: interventional
• Target: 240
• Locations: 16 countries
• Sites: 93

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.

Conditions

Generalized Myasthenia Gravis

Keywords

monoclonal antibody; batoclimab; IMVT-1401; Myasthenia Gravis; acetylcholine receptor; RVT-1401

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (9)

• Change from Baseline in Quantitative Myasthenia Gravis (QMG) score in AChRAb+ participants

  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

Study Arms (6)

Batoclimab Induction Dose 1 (Period 1)

EXPERIMENTAL

Drug: Batoclimab 680 mg SC weekly

Batoclimab Induction Dose 2 (Period 1)

EXPERIMENTAL

Drug: Batoclimab 340 mg SC weekly

Placebo Induction Dose (Period 1)

PLACEBO COMPARATOR

Drug: Matching Placebo SC

Batoclimab Maintenance Dose 1 (Period 2)

EXPERIMENTAL

Drug: Batoclimab 340 mg SC weekly

Batoclimab Maintenance Dose 2 (Period 2)

EXPERIMENTAL

Drug: Batoclimab 340 mg SC bi-weekly

Placebo Maintenance Dose (Period 2)

PLACEBO COMPARATOR

Drug: Matching Placebo SC

Interventions

Batoclimab 680 mg SC weekly DRUG

Batoclimab is a fully human anti-neonatal fragment crystallizable receptor (FcRn) monoclonal antibody

Also known as: IMVT-1401

Batoclimab Induction Dose 1 (Period 1)

Batoclimab 340 mg SC weekly DRUG

Batoclimab is a fully human anti-neonatal fragment crystallizable receptor (FcRn) monoclonal antibody

Also known as: IMVT-1401

Batoclimab Induction Dose 2 (Period 1); Batoclimab Maintenance Dose 1 (Period 2)

Matching Placebo SC DRUG

Placebo

Placebo Induction Dose (Period 1); Placebo Maintenance Dose (Period 2)

Batoclimab 340 mg SC bi-weekly DRUG

Batoclimab is a fully human anti-neonatal fragment crystallizable receptor (FcRn) monoclonal antibody

Also known as: IMVT-1401

Batoclimab Maintenance Dose 2 (Period 2)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Are ≥ 18 years of age at the Screening Visit.
• Have mild to severe gMG by Myasthenia Gravis Foundation of America (MGFA) classification Class II, III, or IVa at the Screening Visit.
• Have a QMG score ≥ 11 at the Screening and Baseline Visits.
• Have a MG-ADL score of ≥ 5 at the Screening and Baseline Visits.

You may not qualify if:

• Have experienced myasthenic crisis within 3 months of the Screening Visit.
• Have had a thymectomy performed \< 6 months prior to the Screening Visit or have a planned thymectomy during the study period.
• Have any active or untreated malignant thymoma.
• 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.

Sponsors & Collaborators

• Immunovant Sciences GmbH: lead

Study Sites (93)

Site Number -1022

Phoenix, Arizona, 85028, United States

Location

Site Number -1029

Scottsdale, Arizona, 85251, United States

Location

Site Number -1002

Carlsbad, California, 92011, United States

Location

Site Number -1009

Irvine, California, 92697, United States

Location

Site Number - 1012

Olive View, California, 91342, United States

Location

Site Number -1032

San Francisco, California, 94143, United States

Location

Site Number - 1027

Aurora, Colorado, 80045, United States

Location

Site Number - 1025

New Haven, Connecticut, 06519, United States

Location

Site Number -1007

Clearwater, Florida, 33761, United States

Location

Site Number -1010

Maitland, Florida, 32751, United States

Location

Site Number - 1020

Miami, Florida, 33136, United States

Location

Site Number - 1019

Orlando, Florida, 32806, United States

Location

Site Number - 1028

Port Charlotte, Florida, 33952, United States

Location

Site Number - 1015

Tampa, Florida, 33612, United States

Location

Site Number -1011

Fairway, Kansas, 66205, United States

Location

Site Number -1003

Lexington, Kentucky, 40536, United States

Location

Site Number -1013

East Lansing, Michigan, 48824, United States

Location

Site Number - 1024

Minneapolis, Minnesota, 55414, United States

Location

Site Number - 1021

Buffalo, New York, 14203, United States

Location

Site Number - 1018

Chapel Hill, North Carolina, 27599, United States

Location

Site Number - 1008

Durham, North Carolina, 27710, United States

Location

Site Number -1004

Cleveland, Ohio, 44195, United States

Location

Site Number -1006

Portland, Oregon, 97239, United States

Location

Site Number - 1023

Philadelphia, Pennsylvania, 19104, United States

Location

Site Number - 1026

Memphis, Tennessee, 38120, United States

Location

Site Number -1001

Austin, Texas, 78759, United States

Location

Site Number -1016

Dallas, Texas, 75206, United States

Location

Site Number - 1034

Houston, Texas, 77030, United States

Location

Site Number - 1014

Round Rock, Texas, 78681, United States

Location

Site Number -1005

Charlottesville, Virginia, 22908, United States

Location

Site Number - 5002

Buenos Aires, 4190, Argentina

Location

Site Number - 5001

Buenos Aires, C1428AQK, Argentina

Location

Site Number - 5003

Rosario, S2000, Argentina

Location

Site Number - 5501

Ribeirão Preto, 14051-140, Brazil

Location

Site Number- 5503

Rio de Janeiro, 22640-100, Brazil

Location

Site Number - 2003

Vancouver, British Columbia, V5Z 1M9, Canada

Location

Site Number - 2002

Toronto, Ontario, M5G 2C4, Canada

Location

Site Number - 2004

Montreal, Quebec, H3A 2B4, Canada

Location

Site Number -8002

Tbilisi, 00114, Georgia

Location

Site Number -8004

Tbilisi, 00114, Georgia

Location

Site Number -8001

Tbilisi, 00160, Georgia

Location

Site Number -8003

Tbilisi, 00186, Georgia

Location

Site Number - 8005

Tbilisi, 0179, Georgia

Location

Site Number - 6507

Berlin, 10117, Germany

Location

Site Number - 6505

Gummersbach, 51643, Germany

Location

Site Number - 6503

Jena, 7747, Germany

Location

Site Number -6504

Leipzig, 04103, Germany

Location

Site Number -6502

Würzburg, 97074, Germany

Location

Site Number -7553

Budapest, 1082, Hungary

Location

Site Number - 7552

Kistarcsa, 2143, Hungary

Location

Site Number - 6004

Bergamo, 24127, Italy

Location

Site Number -6006

Brescia, 25123, Italy

Location

Site Number - 6002

Genova, 16132, Italy

Location

Site Number - 6003

Milan, 20133, Italy

Location

Site Number - 6007

Naples, 80131, Italy

Location

Site Number -6001

Naples, 80131, Italy

Location

Site Number - 4020

Asahikawa-shi, 078-8510, Japan

Location

Site Number - 4014

Fuchu-shi, 183-0042, Japan

Location

Site Number - 4002

Hanamaki-shi, 025-0082, Japan

Location

Site Number - 4013

Higashimatsushima, 355-0005, Japan

Location

Site Number - 4009

Kawasaki-shi, 216-8511, Japan

Location

Site Number - 4012

Koriyama-shi, 963-8563, Japan

Location

Site Number - 4007

Koshigaya-shi, 343-8555, Japan

Location

Site Number - 4011

Matsuyama, 791-8026, Japan

Location

Site Number - 4008

Miyagi, 983-8520, Japan

Location

Site Number - 4003

Narita-shi, 286-8520, Japan

Location

Site Number - 4010

Osaka, 534-0021, Japan

Location

Site Number - 4001

Osaka, 589-8511, Japan

Location

Site Number - 4005

Tokyo, 160-0023, Japan

Location

Site Number - 4016

Yokohama, 222-0036, Japan

Location

Site Number - 4019

Yokohama, 230-8765, Japan

Location

Site Number - 4015

Yokohama, 236-0004, Japan

Location

Site Number - 2601

Mexico City, 06700, Mexico

Location

Site Number - 3007

Gdansk, 80-214, Poland

Location

Site Number -3001

Katowice, 40-123, Poland

Location

Site Number -3004

Krakow, 31-324, Poland

Location

Site Number - 3003

Krakow, 31-503, Poland

Location

Site Number -3002

Krakow, 31-505, Poland

Location

Site Number - 3008

Lublin, 20-701, Poland

Location

Site Number -3006

Poznan, 61-731, Poland

Location

Site Number - 3005

Warsaw, 01-684, Poland

Location

Site Number -7502

Constanța, 900628, Romania

Location

Site Number -7501

Târgu Mureş, 540136, Romania

Location

Site Number -7503

Timișoara, 300723, Romania

Location

Site Number - 9001

Belgrade, 11000, Serbia

Location

Site Number - 9002

Niš, 1800, Serbia

Location

Site Number - 4505

Daegu, 41944, South Korea

Location

Site Number - 4501

Seoul, 06351, South Korea

Location

Site Number -3502

Barcelona, 08195, Spain

Location

Site Number -3505

Barcelona, 8041, Spain

Location

Site Number -3501

Madrid, 028034, Spain

Location

Site Number - 3503

Madrid, 28046, Spain

Location

Site Number - 7002

Sheffield, S102JF, United Kingdom

Location

Related Publications (1)

• Benatar M, Wiendl H, Nowak R, Zheng Y, Macias W. Batoclimab as induction and maintenance therapy in patients with myasthenia gravis: rationale and study design of a phase 3 clinical trial. BMJ Neurol Open. 2024 Jan 10;6(1):e000536. doi: 10.1136/bmjno-2023-000536. eCollection 2024.

  PMID: 38268752 DERIVED

MeSH Terms

Conditions

Myasthenia Gravis

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Sponsor Staff is also masked, so the study is Quadruple-blind.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 26, 2022
• First Posted: June 3, 2022
• Study Start: June 27, 2022
• Primary Completion: January 10, 2025
• Study Completion (Estimated): February 1, 2027
• Last Updated: March 25, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

DE (5); BR (2); KR (2); IT (6); GB (1); US (30); SE (2); RO (3); ES (4); PL (8); GE (5); CA (3); HU (2); AR (3); ME (1); JP (16)