Phase 3 Study to Assess Efficacy and Safety of Batoclimab as Induction and Maintenance Therapy in Adult Participants with Generalized Myasthenia Gravis (gMG)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Immunovant Sciences GmbH

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

3 Feb 2023 → 21 Apr 2026

Decision date (initial)

2024-07-10

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

External identifiers

EU CT number

2024-512258-91-00

EudraCT number

2021-000249-42

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacodynamic, Pharmacokinetic, Safety, Pharmacogenetic, Therapy, Dose response, Efficacy

To evaluate the efficacy of batoclimab 680 mg SC QW or 340 mg SC QW compared to placebo as Induction Therapy in AChRAb+ participants as assessed by change in the Myasthenia Gravis Activities of Daily Living (MG-ADL) score.

Secondary objectives 1

1. To evaluate the efficacy of batoclimab 680 mg SC QW or 340 mg SC QW compared to placebo as Induction Therapy in AChRAb+ participants as assessed by change in Quantitative Myasthenia Gravis (QMG) score.

Conditions and MedDRA coding

Generalized Myasthenia Gravis (gMG)

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

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 6

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, etc.) 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.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Change from Period 1 baseline in MG-ADL score to Week 12 for AChRAb+ participants (Timeframe: Period 1 baseline to Week 12).

Secondary endpoints 5

1. Change from Period 1 baseline in QMG score to Week 12 for AChRAb+ participants (Timeframe: Period 1 baseline to Week 12).
2. Change from Period 2 baseline in MG-ADL score to Week 24 for AChRAb+ randomized withdrawal participants (Timeframe: Week 12 to Week 24).
3. Proportion of AChRAb+ participants with ≥ 3-point improvement (decrease in score) in QMG from Period 1 baseline to Week 12 (Timeframe: Period 1 baseline to Week 12).
4. Proportion of AChRAb+ participants achieving MG-ADL score of 0 or 1 by Week 12 (Timeframe: Period 1 baseline to Week 12).
5. Change from Period 1 baseline in MG-ADL score to Week 12 for AChRAb- participants (Timeframe: Period 1 baseline to Week 12).

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Immunovant Sciences GmbH

Sponsor organisation

Immunovant Sciences GmbH

Address

Viaduktstrasse 8

City

Basel Town

Postcode

4051

Country

Switzerland

Scientific contact point

Organisation

Immunovant Sciences GmbH

Contact name

Immunovant Clinical Trials

Public contact point

Organisation

Immunovant Sciences GmbH

Contact name

Immunovant Clinical Trials

Third parties 13

Locations

6 EU/EEA countries · 31 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Oversight and notifications

Regulatory notifications under CTR Articles 38, 52, 53, 54 and 77

Urgent safety measures 1 · Art. 54 CTR

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 83 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications