Study to Assess K1-70 in Participants with Active Thyroid Eye Disease

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Av7 Limited

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Eye Diseases [C11], Diseases [C] - Immune System Diseases [C20]

Trial duration

28 May 2026 → ongoing

Decision date (initial)

2026-02-16

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

AV7 Limited

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

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

To compare the exophthalmos improvement rate achieved with K1-70 once every 6 weeks (q6W) for 24 weeks with that achieved with placebo in subjects with Graves’ disease and active thyroid eye disease.

Secondary objectives 1

1. To evaluate the efficacy, safety, tolerability, thyroid function, pharmacokinetics (PK), immunogenicity, and the following assessments after K1-70 q6W for 24 weeks in subjects with Graves’ disease and active thyroid eye disease: a) Orbital inflammation b) Graves’ ophthalmopathy-specific quality of life (GO-QOL) c) Clinical activity score (CAS) d) Exophthalmos e) Diplopia f) Retrobulbar volume

Conditions and MedDRA coding

Graves’ disease and active thyroid eye disease

Study design 2 periods

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 8

1. Subjects must satisfy all the following criteria to be enrolled in the study 1. Is a male or female subject aged 18 to 75 years of age inclusive, at the time of signing and dating the informed consent form (ICF).
2. 2. Must sign and date the ICF indicating that they understand the purpose of and procedures required for the study and are willing to participate in the study.
3. 3. Have clinically confirmed Graves’ disease with active thyroid eye disease at screening and baseline, with the study eye satisfying criterion a) or b). Criterion a) will be used if magnetic resonance imaging (MRI) is available and criterion b) will be used if not. a) Have inflammatory hypertrophy of the extraocular muscles as shown by MRI according to the criteria of the 2021 European Group on Graves' orbitopathy (EUGOGO) clinical practice guidelines for the medical management of Graves' orbitopathy. b) The Clinical activity score (CAS) is at least 3.
4. 4. Has exophthalmos of ≥18 mm in the study eye, or if <18mm in the study eye there is at least 3 mm greater proptosis in the study eye compared to the fellow eye.
5. 5. Have any symptoms of eyelid retraction, soft-tissue findings, or diplopia as defined in the criteria for moderate to severe eye disease in the 2021 EUGOGO clinical practice guidelines for the medical management of Graves' orbitopathy.
6. 6. Has self-reported symptoms of active thyroid eye disease within the 12 months prior to Screening.
7. 7. Has Graves' disease whose underlying condition is controlled and has normal thyroid function at the time of screening or has mild hypothyroidism or hyperthyroidism.Every effort should be made to maintain thyroid function throughout the study”.
8. 8. Is a female of childbearing potential or male with a female partner of childbearing potential who consents to use acceptable contraceptive methods as defined in the protocol. Additional inclusion criteria are outlined in the study protocol.

Exclusion criteria 8

1. Individuals who meet any of the following criteria will be excluded from the study : 1. Has dysthyroid optic neuropathy and/or very severe eye disease (with the risk of blindness) that features ulceration, perforation or necrosis of the cornea and requires urgent therapeutic intervention.
2. 2. Whose CAS decreased by 2 or more points in the study eye from screening to baseline
3. 3. Whose exophthalmos decreased by 2 mm or more in the study eye from screening to baseline.
4. 4. Has a history of orbital radiotherapy or surgery for thyroid eye disease.
5. 5. Has received a systemic corticosteroid (IV or orally), intraorbital corticosteroid injection, or ophthalmic corticosteroid drug for treating thyroid eye disease or another medical condition within the 3 weeks prior to providing informed consent. None of these corticosteroid treatments may be started during the study period. This excludes corticosteroids that are locally administered to an area not near the orbits (e.g., topical corticosteroids, intraarticular corticosteroid injections, and inhaled corticosteroids) and corticosteroids for treating infusion-related reactions
6. 6. Has had any previous systemic treatment with rituximab (Rituxan®) within the 12 months prior to the start of study drug administration, tocilizumab (Actemra®) within the 6 months prior to the start of study drug administration, or any systemic non-steroid immunosuppressive agent within the 3 months prior to the start of study drug administration.
7. 7. Have received treatment around the eyes with botulinum toxin within the 3 months prior to the start of study drug administration.
8. 8.Has a pre-existing ophthalmic disease that, in the opinion of the investigator, would preclude study participation or complicate interpretation of the study results Additional exclusion criteria are outlined in the study protocol.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Exophthalmos improvement rate at Week 24* *The proportion (%) of subjects who achieve at least a 2-mm decrease in exophthalmos of the study eye from baseline and have no worsening in exophthalmos (defined as an increase of at least 2 mm) in the fellow eye.

Secondary endpoints 2

1. Change from baseline in the ratio of signal intensity in the extraocular muscles shown by MRI. Change from baseline in the GO-QOL score Change from baseline in the CAS. Change from baseline in exophthalmos measurement . Change from baseline in diplopia grade (Bahn-Gorman scale) (diplopia improvement rate) Change from baseline in retrobulbar volume shown by MRI
2. Safety, determined by: a) AEs b) Vital sign measurements c) 12-lead ECG measurements d) Laboratory test results e) Physical examination results f) Eye examination results Changes in levels of TSH, thyroid hormones (FT4 and FT3), TSH receptor autoantibodies and stimulating TSH receptor autoantibodies Change over time in blood concentrations of K1-70 Change over time in blood concentrations of Anti-drug antibodies

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Av7 Limited

Sponsor organisation

Av7 Limited

Address

7 Robin Lane, Bentham Bentham

City

Lancaster

Postcode

LA2 7AB

Country

United Kingdom

Scientific contact point

Organisation

Av7 Limited

Contact name

Dr Shu Chen

Public contact point

Organisation

Av7 Limited

Contact name

Dr Shu Chen

Third parties 6

Locations

2 EU/EEA countries · 9 investigational sites

By country

Investigational sites

Country notifications

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

Results and documents

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

Documents 24 files

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

Application history

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