A Safety and Tolerability Study of VRDN-001 in Persons with 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], Diseases [C] - Immune System Diseases [C20]

Trial duration

5 Nov 2024 → 20 Jan 2026

Decision date (initial)

2024-10-31

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Viridian Therapeutics, Inc.

External identifiers

EU CT number

2023-507563-19-00

WHO UTN

U1111-1299-4825

ClinicalTrials.gov

NCT06384547

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Others, Safety, Dose response

To confirm the safety and tolerability of VRDN-001 when given as 5 intravenous (IV) infusions of 10 mg/kg every three weeks in participants with TED of any duration.

Conditions and MedDRA coding

Thyroid eye disease

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Be an adult male or female participant, >18 to ≤75 years of age
2. Have a clinical diagnosis of TED with or without proptosis and with any CAS (0 – 7) and in the opinion of the investigator may benefit from treatment
3. If female, have a negative serum pregnancy test at screening and further negative urine tests immediately before each dose of study medication and following the last dose of study medication
4. Be surgically sterile males for at least 6 weeks prior to the first dose of VRDN-001, or agree to use an acceptable method of contraception such as a condom and a second highly effective method of contraception

Exclusion criteria 10

1. Have received prior treatment with another anti-IGF-1R therapy
2. Have used systemic corticosteroids for any condition, including TED, or selenium within 2 weeks prior to the first dose of study medication (topical steroids including eye drops or multivitamins that contain selenium are permitted). Also exclusionary is periocular (including intraorbital) or intraocular administration of corticosteroids within 3 months prior to the first dose of study medication or having received greater than 3 periocular or intraocular corticosteroid injections at any time
3. Have received other immunosuppressive agents, including rituximab, tocilizumab, secukimumab, satralizumab or anti-FcRn’s for any condition (including TED) within 8 weeks prior to the first dose of study medication or have received intraorbital administration of other such immunosuppressive agents at any time
4. Have received any other therapy for TED within 8 weeks prior to the first dose of study medication (artificial tears are permitted)
5. Have received an investigational agent for any condition within 8 weeks prior to the first dose of study medication
6. Have had previous orbital irradiation or decompression surgery involving excision of fat for TED to the study eye’s orbit
7. Have a pre-existing ophthalmic condition in the study eye which in the opinion of the Investigator, would confound interpretation of the study results
8. Have abnormal baseline audiometry Pure Tone Average (PTA) assessment or history of significant (as determined by the Investigator) ear pathology, relevant ear surgery or hearing loss
9. Have inflammatory bowel disease
10. Be a pregnant or lactating woman

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Treatment Emergent Adverse Event (TEAE) incidence rate through Week 15

Secondary endpoints 2

1. Change from baseline in proptosis in the study eye as measured by exophthalmometer at Week 15
2. Treatment Emergent Adverse Event (TEAE) incidence rate through Week 52

Investigational products

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

Test 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 S.à.r.l.

Contact name

Chief Development Officer

Public contact point

Organisation

Viridian Therapeutics S.à.r.l.

Contact name

Chief Development Officer

Third parties 14

Locations

5 EU/EEA countries · 19 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 45 files

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

Application history

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