An Efficacy, Safety, and Tolerability Study of VRDN-003 in Participants with Active Thyroid Eye Disease (TED)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Viridian Therapeutics Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Eye Diseases [C11]

Trial duration

25 Feb 2025 → ongoing

Decision date (initial)

2025-02-11

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Viridian Therapeutics Inc.

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

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

To determine if VRDN-003 is efficacious (meaning if VRDN-003 affects the signs and symptoms of TED), safe and tolerable (meaning if the body reacts acceptably to VRDN-003) when administered as a series of subcutaneous/SC (injection into layer of fat between skin and muscle) injections given every 4 weeks or every 8 weeks compared to placebo (a substance with no active drug ingredients) in participants with active TED

Conditions and MedDRA coding

Thyroid eye disease

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. Adult males or females, ≥18 to ≤75 years of age who have a clinical diagnosis of TED with a value ≥ 3 on the CAS scale
2. Must have moderate to severe active TED with eye bulging value in the study eye before the first dose as defined in the protocol and at least one additional sign/symptom as described in the protocol
3. Have experienced eye-related signs or symptoms that began within 15 months before the first study visit
4. Must agree to use highly effective contraception as specified in the protocol
5. Female participants must have a negative pregnancy test at first study visit

Exclusion criteria 7

1. Received prior treatment with another anti-IGF-1R therapy
2. Received corticosteroids (drugs that reduce inflammation in the body) for any condition, including TED, within 2 weeks prior to first dose
3. Received other immunosuppressive drugs (drugs that dampen the body’s immune response) or any other therapy for TED within 12 weeks prior to first dose or another investigational agent for any condition, including TED, within 8 weeks or longer duration (depending on the type of investigational agent) prior to first dose
4. Have had previous orbital irradiation or decompression surgery for TED or a pre-existing eye condition which in the study doctor’s opinion, would interfere with interpretation of study results
5. Have abnormal hearing test before first dose. History of ear conditions considered significant by study doctor
6. Have inflammatory bowel disease (conditions that involve inflammation of the gastrointestinal [GI] tract)
7. Female participants who are pregnant or breastfeeding

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The primary study endpoint will assess the percentage of participants who have a response called Overall Responder Rate at Week 24. An Overall Responder is a participant who achieves a ≥ 2 mm reduction in eye bulging AND shows a reduction in Clinical Activity Score or CAS (that is an estimate of disease severity/symptoms) ≥ 2 points at Week 24 versus measurements before the first dose.

Secondary endpoints 2

1. The secondary endpoints will assess changes in eye bulging, disease severity and symptoms versus measurements before the first dose, and percent of participants who achieve the required reduction in eye bulging, disease severity and symptoms, and reduction and resolution of double vision at Week 24.
2. Safety Endpoint: the study will also evaluate safety events (side effects), the effects of VRDN-003 on the body and vice-versa throughout the study.

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Viridian Therapeutics Inc.

Sponsor organisation

Viridian Therapeutics Inc.

Address

221 Crescent Street Suite 103a

City

Waltham

Postcode

02453-3425

Country

United States

Scientific contact point

Organisation

Viridian Therapeutics Inc.

Contact name

Chief Medical Officer

Public contact point

Organisation

Viridian Therapeutics Inc.

Contact name

Chief Medical Officer

Third parties 14

Locations

5 EU/EEA countries · 17 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 64 files

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

Application history

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