Phase 3 Study to Assess Batoclimab in Participants with Active Thyroid Eye Disease.

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] - Eye Diseases [C11]

Trial duration

30 Nov 2023 → 18 Nov 2025

Decision date (initial)

2024-07-19

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

External identifiers

EU CT number

2024-512648-45-00

EudraCT number

2022-002787-68

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Others, Therapy, Pharmacokinetic, Efficacy, Pharmacodynamic, Safety

To evaluate the efficacy of batoclimab 680 mg subcutaneously (SC) once a week (QW) for 12 weeks followed by 340 mg SC QW for 12 weeks versus placebo on proptosis responder rate at Week 24.

Secondary objectives 7

1. To evaluate the efficacy of batoclimab compared to placebo as assessed by proptosis and Clinical Activity Score (CAS)
2. To evaluate the efficacy of batoclimab compared to placebo as assessed by CAS.
3. To evaluate the efficacy of batoclimab compared to placebo assessed by change in binding anti-TSHR antibodies (Abs).
4. To evaluate the efficacy of batoclimab compared to placebo as assessed by Gorman score for diplopia.
5. To evaluate the efficacy of batoclimab compared to placebo as assessed by proptosis.
6. To evaluate the efficacy of batoclimab compared to placebo as assessed by Graves’ ophthalmology-specific quality of life (GO-QOL).
7. To evaluate the efficacy of batoclimab compared to placebo as assessed by motility.

Conditions and MedDRA coding

Thyroid Eye Disease (TED)

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

EMA paediatric investigation plan (PIP)

EMEA-003162-PIP02-22

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

1. Are ≥18 years of age at screening.
2. Have a clinical diagnosis of TED associated with active, moderate to severe TED with a CAS ≥4 in either eye, and clinical evidence of worsened proptosis with: - Proptosis ≥ 18 mm and/or - Proptosis ≥ 3 mm increase from participant's baseline (prior to diagnosis of TED), as estimated by the Investigator/assessor
3. Have moderate to severe active TED, as defined by European Group on Graves' Orbitopathy (EUGOGO) guidelines.
4. Have onset of active TED within 12 months prior to screening.
5. Have documented evidence of detectable anti-TSHR-Ab at screening.
6. Are not expected to require immediate surgical intervention and are not planning corrective surgery/irradiation or medical therapy for TED during the course of the study.
7. Are euthyroid with the baseline disease under control or have mild hypo- or hyperthyroidism.

Exclusion criteria 6

1. Have decreased best corrected visual acuity due to optic neuropathy.
2. Have at least a 2-point decrease in CAS or ≥2 mm decrease in proptosis between screening and Baseline assessments in either eye.
3. Have used any steroid (intravenous or oral) for the treatment of TED or other conditions within 4 weeks prior to screening.
4. Have used any steroid (Intravenous or oral) with a cumulative dose equivalent to ≥ 1 g of methylprednisolone for the treatment of TED.
5. Have known autoimmune disease other than TED, that, in the opinion of the Investigator, would interfere with the course and conduct of the study.
6. Had previous orbital irradiation or surgery for TED.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Proportion of proptosis responders (proptosis responder rate) at Week 24 where proptosis responder is defined as the participant with a ≥ 2 mm reduction in the study eye without deterioration (≥ 2 mm increase) in the fellow eye.

Secondary endpoints 8

1. Proportion of participants with proptosis ≥ 2 mm reduction and CAS of ≤ 3 at Week 24 from baseline in the study eye
2. Proportion of participants with CAS of 0 or 1 at Week 24 in the study eye
3. Mean change from baseline to Week 24 in CAS in the study eye
4. Proportion of participants with positive binding anti-TSHR Ab at baseline who achieve seroconversion at Week 24
5. Proportion of participants with decrease of at least 1 grade from baseline in Gorman score for diplopia at Week 24 in participants who have diplopia at baseline.
6. Mean change from baseline to Week 24 in proptosis (mm) in the study eye
7. Proportion of participants with ≥ 6-point increase from baseline in total GO-QOL score at Week 24 in participants who have the ability to increase by ≥ 6-points from baseline
8. Proportion of participants with ≥ 8-degree increase from baseline in motility (in at least 1 of 4 directions) at Week 24 in the study eye in participants who have the ability to increase by ≥ 8 degrees from baseline.

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 9

Locations

3 EU/EEA countries · 23 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 53 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