A dose ranging study to investigate efficacy and safety of subcutaneous TOUR006 compared with placebo in participants 18 to 80 years of age inclusive with thyroid eye disease.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Tourmaline Bio Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Trial duration

2 Apr 2025 → ongoing

Decision date (initial)

2025-03-12

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Tourmaline Bio, Inc.

External identifiers

EU CT number

2024-514801-69-00

WHO UTN

U1111-1308-4718

ClinicalTrials.gov

NCT06088979

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Pharmacokinetic, Pharmacodynamic, Efficacy, Safety

To evaluate the efficacy of TOUR006 in reducing proptosis

Secondary objectives 4

1. 1. To explore the efficacy of different dose levels of TOUR006
2. 2. To characterize the efficacy of TOUR006 in outcome measure
3. 3. To evaluate the long-term efficacy outcomes of TOUR006
4. 4. To characterize the safety of TOUR006

Conditions and MedDRA coding

Thyroid Eye Disease

Regulatory references

Scientific advice from competent authorities

Food And Drug Administration

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. 1. Clinical diagnosis of Graves' disease associated with moderate to severe active TED
2. 2. Onset of active TED symptoms within approximately 15 months
3. 3. Proptosis (exophthalmos) ≥3 mm above the normal range per investigator judgment (based upon race and gender) for the study eye.
4. 4. CAS ≥4 (on the 7-item scale) for the study eye.
5. 5. Presence of TSI >130% of the normal reference standard or >0.55 IU/L (depending on assay method) and laboratory reference ranges

Exclusion criteria 7

1. 1. Anticipated need for intervention due to sight-threatening complications or other significant and acute deterioration in vision
2. 2. Any previous treatment with teprotumumab or other agent that inhibits the IGF-1 receptor
3. 3. History of systemic (eg, oral or IV) steroid use with a cumulative dose equivalent to >1 g of methylprednisolone for the treatment of TED. Previous oral steroid use with a cumulative dose of ≤1 g methylprednisolone (or equivalent dosage for other systemic corticosteroid) for the treatment of TED, however, is allowed if the corticosteroid was discontinued at least 6 weeks before baseline (Day 1) and completely tapered by Baseline (if applicable)
4. 4. Systemic (oral or IV) corticosteroid use for conditions other than TED within 6 weeks of baseline (Day 1) or not completely tapered by baseline (if applicable)
5. 5. Any major illness/condition or evidence of an unstable clinical condition that, in the investigator's judgment, will substantially increase the risk to the participant, or confound the interpretation of safety assessments, if they were to participate in the study
6. 6. Any other condition that, in the opinion of the investigator, would impair the ability of the participant to comply with the study procedures or impair the ability to interpret data from the participant's participation in the study
7. 7. Pregnant or lactating

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Percentage of participants achieving proptosis response (defined as a ≥2 mm reduction from baseline in the study eye without deterioration [≥2 mm increase] of proptosis in the fellow eye and without need for rescue therapy/intervention) in the TOUR006 treatment arms compared with the placebo arm at Week 20.

Secondary endpoints 9

1. 1. Percentage of participants achieving a proptosis response in the TOUR006 treatment arms at Week 8, Week 16, Week 24, Week 32, Week 40, Week 44, Week 48, Week 56, Week 64, and Week 72.
2. 2. Mean change from baseline in proptosis in the study eye at Week 8, Week 16, Week 20, Week 24, Week 32, Week 40, Week 44, Week 48, Week 56, Week 64, and Week 72.
3. 3. Percentage of participants attaining a complete or near-complete CAS response (defined as CAS ≤1 in the study eye, without ≥2 point increase in CAS from baseline in the fellow eye, and without need for rescue therapy/intervention) at Week 8, Week 16, Week 20, Week 24, Week 32, Week 40, Week 44, Week 48, Week 56, Week 64, and Week 72.
4. 4. Mean change from baseline in CAS in the study eye at Week 8, Week 16, Week 20, Week 24, Week 32, Week 40, Week 44, Week 48, Week 56, Week 64, and Week 72.
5. 5. Percentage of participants attaining ≥1 grade decrease in diplopia at Week 8, Week 16, Week 20, Week 24, Week 32, Week 40, Week 44, Week 48, Week 56, Week 64, and Week 72.
6. 6. Percentage of participants attaining resolution in diplopia at Week 8, Week 16, Week 20, Week 24, Week 32, Week 40, Week 44, Week 48, Week 56, Week 64, and Week 72.
7. 7. Percentage of participants with inconstant diplopia at baseline attaining resolution of inconstant diplopia at Week 8, Week 16, Week 20, Week 24, Week 32, Week 40, Week 44, Week 48, Week 56, Week 64, and Week 72.
8. 8. Percentage of participants with constant diplopia at baseline attaining resolution of constant diplopia at Week 8, Week 16, Week 20, Week 24, Week 32, Week 40, Week 44, Week 48, Week 56, Week 64, and Week 72.
9. 9. Incidence of TEAEs by severity and SAEs through Week 72.

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Tourmaline Bio Inc.

Sponsor organisation

Tourmaline Bio Inc.

Address

27 West 24th Street Ste 702

City

New York

Postcode

10010-3265

Country

United States

Scientific contact point

Organisation

Tourmaline Bio Inc.

Contact name

VP and Therapeutic Area Head, Ophthalmology

Public contact point

Organisation

Tourmaline Bio Inc.

Contact name

Senior Clinical Trial Manager

Third parties 13

Locations

6 EU/EEA countries · 15 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

Unexpected events 1 · Art. 53 CTR

Note: SUSARs are reported via EudraVigilance, not CTIS — events shown here are CTIS-public notifications only.

Results and documents

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

Documents 109 files

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

Application history

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