Overview
Sponsor-declared trial summary
Phase
Therapeutic confirmatory (Phase III)
Status
Ended
Participants planned
290
Countries
6
Sites
28
Thyroid eye disease
To determine the safety, tolerability, and efficacy of VRDN- 001 when given as 5 infusions of 10 mg/kg compared to placebo in participants with chronic TED.
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
- 12 Apr 2024 → 25 Jul 2025
- Decision date (initial)
- 2024-04-02
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- Yes
- Funding sources
- Viridian Therapeutics, Inc.
External identifiers
- EU CT number
- 2023-507217-10-00
- WHO UTN
- U1111-1299-4825
- ClinicalTrials.gov
- NCT06021054
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Others, Safety, Therapy, Efficacy
To determine the safety, tolerability, and efficacy of VRDN- 001 when given as 5 infusions of 10 mg/kg compared to placebo in participants with chronic TED.
Conditions and MedDRA coding
Thyroid eye disease
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 23.1 | LLT | 10084358 | Thyroid eye disease | 100000004848 |
| 20.1 | PT | 10060742 | Endocrine ophthalmopathy | 100000004853 |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 5
- 1. Males and/or females of age ≥18 to ≤75 years of age and older who have a clinical diagnosis of TED with any degree of disease severity and symptoms as measured by the CAS scale (0-7).
- 2. Participants must have moderate to severe chronic TED with proptosis (eye bulging) of at least 3 mm and >17 mm measurement at pre-dose baseline (Day -1 or Day 1) in the study eye.
- 3. Have experienced eye-related symptoms or signs that began more than 15 months before study screening.
- 4. Must agree to use highly effective contraception as specified in the protocol.
- 5. Female TED participants must have a negative serum pregnancy test at Screening.
Exclusion criteria 8
- 1. Participants who have previously received treatment with other anti-IGF-1R therapy
- 2. Participants who have taken corticosteroids (except topically) or selenium (except multivitamins) for any condition, including TED, within 2 weeks before starting study medication. Also exclusionary are injections of corticosteroids into the eye within 3 months prior to the first dose of study medication or having received greater than 3 injections of corticosteroids into the eye at any time.
- 3. Participants who have received immunosuppressive agents, radioactive iodine, any other therapy for TED, or an investigational agent for any condition within 8 weeks prior to the first dose of study medication.
- 4. Participants who have a decrease in disease severity and symptoms as measured by CAS scale of at least 2 points in the study eye between screening and one day before treatment, for participants who had a CAS greater than 2 at screening.
- 5. Participants who have a decrease of at least 2 mm in proptosis (eye bulging) in the study eye from screening to one day before treatment.
- 6. Participants who require immediate surgical intervention and/or have other eye conditions as evaluated by the study doctor that are not responding to treatment.
- 7. Female participants who are pregnant or breastfeeding.
- 8. Have abnormal baseline hearing assessment
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 2
- Overall Response Rate comprised of Proptosis Responder Rate in the most proptotic eye at 3 weeks post the fifth IV infusion and Clinical Activity Responder Rate in the most proptotic eye at 3 weeks post the fifth IV infusion.
- Safety Endpoint: Adverse Events (AEs) and Serious Adverse Events (SAEs) will be monitored and recorded throughout the duration of the study. All clinically significant changes in other safety measurements will be recorded as AEs.
Secondary endpoints 3
- Change from baseline in proptosis for the most proptotic eye at Week 15.
- Proptosis change from baseline, general and measured by exophtalmometer and responder rate at Week 15.
- Diplopia Responder and Resolution Rate (i.e., reduction in Diplopia Score to 0 from baseline for participants with baseline Diplopia Score >0) at Week 15.
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
VRDN-001 (Insulin-like growth factor-1 receptor [IGF-1R] inhibitor)
PRD10829291 · Product
- Active substance
- VRDN-001
- Pharmaceutical form
- CONCENTRATE FOR SOLUTION FOR INFUSION
- Route of administration
- INTRAVENIOUS INFUSION
- Max daily dose
- 10 mg/kg milligram(s)/kilogram
- Max total dose
- 50 mg/Kg milligram(s)/kilogram
- Max treatment duration
- 12 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- VIRIDIAN THERAPEUTICS, INC.
- Paediatric formulation
- No
- Orphan designation
- No
Placebo 1
SUB12581MIG · Substance
- Active substance
- Sodium Chloride
- Pharmaceutical form
- SOLUTION FOR INFUSION
- Route of administration
- INTRAVENOUS INFUSION
- Max daily dose
- 250 ml millilitre(s)
- Max total dose
- 1250 ml millilitre(s)
- Max treatment duration
- 12 Week(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- Yes
- Modification description
- At clinical trial sites: if starting with a 500 ml or 1000 ml bag, normal saline must be removed to achieve 250 ml of volume. After dosing preparation, masked label will be applied on the infusion bag.
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 12
| Organisation | City, country | Duties |
|---|---|---|
| Medidata Solutions Inc. ORG-100016256
|
New York, United States | E-data capture |
| International Drug Development Institute ORG-100028563
|
Ottignies-Louvain-La-Neuve, Belgium | Code 10 |
| Leapcure Inc. ORL-000003865
|
Redwood City, CA, United States | Other |
| JMAC Partners LLC ORL-000003763
|
Vero Beach, United States | Other |
| Acm Global Central Laboratory Limited ORG-100042459
|
York, United Kingdom | Laboratory analysis |
| Bioclinica Inc. ORG-100033079
|
Princeton, United States | Other |
| HearX SA (PTY) LTD ORL-000003765
|
Pretoria, South Africa | Other |
| Greenphire LLC ORG-100041621
|
King Of Prussia, United States | Other |
| ScienceMedia, Inc. ORL-000003766
|
San Diego, CA, United States | Other |
| CluePoints INC ORL-000002186
|
King of Prussia, United States | Other |
| Parexel International (IRL) Limited ORG-100022780
|
Dublin 2, Ireland | On site monitoring, Code 11, Code 12, Code 13, Code 2, Code 5, Data management, Code 8 |
| Suvoda LLC ORG-100043523
|
Conshohocken, United States | Interactive response technologies (IRT) |
Locations
6 EU/EEA countries · 28 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| France | Ended | 43 | 4 |
| Germany | Ended | 30 | 4 |
| Hungary | Ended | 30 | 3 |
| Italy | Ended | 17 | 4 |
| Poland | Ended | 20 | 5 |
| Spain | Ended | 30 | 8 |
| Rest of world
Canada, Israel, Australia, Turkey, United States, United Kingdom
|
— | 120 | — |
Investigational sites
Centre Hospitalier Universitaire De Nantes
Endocrinologie, Diabétologie et Nutrition, Boulevard Du Professeur Jacques Monod, 44800, Saint Herblain
Centre Hospitalier Universitaire De Nice
Ophthalmologie, 30 Voie Romaine, 06000, Nice
Centre Hospitalier Universitaire D'Angers
Endocrinologie, Diabétologie et Nutrition, 4 Rue Larrey, 49100, Angers
Centre Hospitalier Universitaire De Bordeaux
Endocrinologie Et Diabétologie, 1 Rue Jean Burguet, 33000, Bordeaux
Universitaetsmedizin Goettingen
Abteilung Augenheilkunde, Robert-Koch-Strasse 40, Weende, Goettingen
Charite Universitaetsmedizin Berlin KöR
Klinik für Augenheilkunde, Augustenburger Platz 1, Wedding, Berlin
Medical Center - University Of Freiburg
Augenklinik, Killianstrasse 5, Stuehlinger, Freiburg Im Breisgau
Universitaetsklinikum Essen AöR
Zentrum für Augenheilkunde, Hufelandstrasse 55, Holsterhausen, Essen
Nozologen Kft.
Szemészet, Varady Antal Utca 10 Fszt. 5, 7621, Pecs
Budapest Retina Associates Kft.
Szemészet, Vaci Ut 76, Kerulet, Budapest XIII
University Of Pecs
Szemészeti Klinika, Rakoczi Ut 2, 7623, Pecs
University Hospital Consorziale Policlinico
UO Oftalmologia Universitaria,, Piazzale Giulio Cesare 11, 70124, Bari
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Endocrinology Unit, Via Francesco Sforza 35, 20122, Milan
Azienda Ospedaliero Universitaria Pisana
SD Medicina interna ad indirizzo immuno-endocrino, Via Roma 67, 56126, Pisa
Istituto Auxologico Italiano IRCCS
U.O. di Endocrinologia, Andrologia e Malattie Metaboliche, Piazzale Brescia 20, 20149, Milan
Centrum Medyczne Pulawska Sp. z o.o.
Ophthalmology, Ul. Pulawska 49, 05-500, Piaseczno
Uniwersytecki Szpital Kliniczny W Bialymstoku
Klinika Okulistyki, Centrum Badan Klinicznych, Ul. Marii Curie-Sklodowskiej 24a, 15-276, Bialystok
Optimum Profesorskie Centrum Okulistyki Sp. z o.o.
Ophthalmology, Ul. Cienista 30, 80-809, Gdansk
Centrum Medyczne Piasta 47 Sp. z o.o.
Ophthalmology, Ul. Piasta 47c, 58-304, Walbrzych
Oculomedica Sp. z o.o.
Ophthalmology, Gorzyskowo 14, 85-157, Bydgoszcz
University Hospital Virgen Del Rocio S.L.
Oftalmología, Avenida De Manuel Siurot S/n, 41013, Sevilla
Complexo Hospitalario Universitario De Santiago
Oftalmología, Calle Choupana Da S/n, 15706, Santiago De Compostela
Clinica De Oftalmologia De Cordoba S.L.
Instituto de Oftalmología La Arruzafa, Avenida De La Arruzafa 9, 14012, Cordoba
Centro De Oftalmologia Barraquer S.A.
Oftalmología, Calle Muntaner 314, 08021, Barcelona
Hospital Universitario Ramon Y Cajal
Oftalmología, Carretera Del Colmenar Viejo Km 9 100, Por El Pardo, Madrid
Hospital Universitario Y Politecnico La Fe
Oftalmología, Avenida De Fernando Abril Martorell 106, 46026, Valencia
Hospital Unviersitario Miguel Servet
Oftalmología, Paseo De Isabel La Catolica 1-3, 50009, Zaragoza
Hospital Universitario Virgen De La Macarena
Oftalmología, Avenida Del Doctor Fedriani 3, 41009, Sevilla
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| France | 2024-04-18 | 2025-07-10 | 2024-04-22 | 2024-07-05 | |
| Germany | 2024-04-17 | 2025-07-18 | 2024-04-22 | 2024-07-05 | |
| Hungary | 2024-04-26 | 2025-07-02 | 2024-04-29 | 2024-07-05 | |
| Italy | 2024-05-07 | 2024-07-06 | 2024-06-13 | 2024-07-05 | |
| Poland | 2024-04-17 | 2025-07-10 | 2024-05-28 | 2024-07-05 | |
| Spain | 2024-04-12 | 2025-07-02 | 2024-04-24 | 2024-07-05 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 77 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol Amendment Main English VRDN-001-301 Public | 5.0 |
| Protocol (for publication) | Subject Questionnaire EQ-5D-5L Italian VRDN-001-301 Public | 1.1 |
| Protocol (for publication) | Subject Questionnaire EQ-5D-5L English VRDN-001-301 Public | 1.1 |
| Protocol (for publication) | Subject Questionnaire EQ-5D-5L French VRDN-001-301 Public | 1.2 |
| Protocol (for publication) | Subject Questionnaire EQ-5D-5L German VRDN-001-301 Public | 1.0 |
| Protocol (for publication) | Subject Questionnaire EQ-5D-5L Hungarian VRDN-001-301 Public | 1.3 |
| Protocol (for publication) | Subject Questionnaire EQ-5D-5L Polish VRDN-001-301 Public | 1.0 |
| Protocol (for publication) | Subject Questionnaire EQ-5D-5L Spanish VRDN-001-301 Public | 1.0 |
| Protocol (for publication) | Subject Questionnaire GO-QoL English VRDN-001-301 Public | 1.0 |
| Protocol (for publication) | Subject Questionnaire GO-QoL French VRDN-001-301 Public | 1.0 |
| Protocol (for publication) | Subject Questionnaire GO-QoL German VRDN-001-301 Public | 1.0 |
| Protocol (for publication) | Subject Questionnaire GO-QoL Hungarian VRDN-001-301 Public | 1.0 |
| Protocol (for publication) | Subject Questionnaire GO-QoL Italian VRDN-001-301 Public | 1.0 |
| Protocol (for publication) | Subject Questionnaire GO-QoL Polish VRDN-001-301 Public | 1.0 |
| Protocol (for publication) | Subject Questionnaire GO-QoL Spanish VRDN-001-301 Public | 1.0 |
| Recruitment arrangements (for publication) | DEU Recruitment Brochure German VRDN-001-301 Public | 1.0 |
| Recruitment arrangements (for publication) | DEU Recruitment Other Physician Letter German VRDN-001-301 Public | 1.0 |
| Recruitment arrangements (for publication) | DEU Recruitment Poster German VRDN-001-301 Public | 1.0 |
| Recruitment arrangements (for publication) | DEU Recruitment Procedure Description English VRDN-001-301 Public | 1.0 |
| Recruitment arrangements (for publication) | ESP Recruitment Brochure Spanish VRDN-001-301 Public | 1.0 |
| Recruitment arrangements (for publication) | ESP Recruitment Poster Spanish VRDN-001-301 Public | 1.0 |
| Recruitment arrangements (for publication) | ESP Recruitment Procedure Description English VRDN-001-301 Public | 1.0 |
| Recruitment arrangements (for publication) | FRA Recruitment Brochure French VRDN-001-301 Public | 1.0 |
| Recruitment arrangements (for publication) | FRA Recruitment Dear Colleague Letter French VRDN-001-301 Public | 1.0 |
| Recruitment arrangements (for publication) | FRA Recruitment Poster French VRDN-001-301 Public | 1.0 |
| Recruitment arrangements (for publication) | FRA Recruitment Procedure Description French VRDN-001-301 Public | 1.1 |
| Recruitment arrangements (for publication) | HUN Recruitment Brochure Hungarian VRDN-001-301 Public | 1.0 |
| Recruitment arrangements (for publication) | HUN Recruitment Dear Colleague Letter Hungarian VRDN-001-301 Public | 1.0 |
| Recruitment arrangements (for publication) | HUN Recruitment Poster Hungarian VRDN-001-301 Public | 1.0 |
| Recruitment arrangements (for publication) | ITA Recruitment Brochure Italian VRDN-001-301 Public | 1.0 |
| Recruitment arrangements (for publication) | ITA Recruitment Other Italian VRDN-001-301 Public | 1.0 |
| Recruitment arrangements (for publication) | ITA Recruitment Poster Italian VRDN-001-301 Public | 1.0 |
| Recruitment arrangements (for publication) | ITA Recruitment Social Media AdvocacyFlyers Italian VRDN-001-301 Public | 1.0 |
| Recruitment arrangements (for publication) | ITA Recruitment Social Media CampaignCopy Italian VRDN-001-301 Public | 1.0 |
| Recruitment arrangements (for publication) | ITA Recruitment Social Media CampaignImagery Italian VRDN-001-301 Public | 1.0 |
| Recruitment arrangements (for publication) | ITA Recruitment Social Media PatientJourney Italian VRDN-001-301 Public | 1.0 |
| Recruitment arrangements (for publication) | ITA Recruitment Social Media PatientReengagement Italian VRDN-001-301 Public | 1.0 |
| Recruitment arrangements (for publication) | ITA Recruitment Social Media Pre-screenerQuestionnaire Italian VRDN-001-301 Public | 1.0 |
| Recruitment arrangements (for publication) | ITA Recruitment Social Media PrivacyPolicy Italian VRDN-001-301 Public | 1.0 |
| Recruitment arrangements (for publication) | POL Recruitment Brochure Polish VRDN-001-301 Public | 1.0 |
| Recruitment arrangements (for publication) | POL Recruitment Poster Polish VRDN-001-301 Public | 1.0 |
| Recruitment arrangements (for publication) | POL Recruitment Procedure Description Polish VRDN-001-301 Public | 1.0 |
| Subject information and informed consent form (for publication) | DEU Country ICF Main German VRDN-001-301 Public | 2.1 |
| Subject information and informed consent form (for publication) | DEU Country ICF Other Pregnant Participant German VRDN-001-301 Public | 1.1 |
| Subject information and informed consent form (for publication) | DEU Country ICF Other Pregnant Partner German VRDN-001-301 Public | 1.1 |
| Subject information and informed consent form (for publication) | DEU Country ICF Procedure English VRDN-001-301 Public | 1.0 |
| Subject information and informed consent form (for publication) | FRA Country ICF Main French VRDN-001-301 Public | 3.0 |
| Subject information and informed consent form (for publication) | FRA Country ICF Other Newborn FU French VRDN-001-301 Public | 1.2 |
| Subject information and informed consent form (for publication) | FRA Country ICF Other Pregnant Participant French VRDN-001-301 Public | 2.2 |
| Subject information and informed consent form (for publication) | FRA Country ICF Other Pregnant Partner French VRDN-001-301 Public | 2.2 |
| Subject information and informed consent form (for publication) | HUN Country ICF Main Hungarian VRDN-001-301 Public | 2.0 |
| Subject information and informed consent form (for publication) | HUN Country ICF Other Hungarian VRDN-001-301 Public | 1.1 |
| Subject information and informed consent form (for publication) | HUN Country ICF Pregnant Partner Information Other Hungarian VRDN-001-301 Public | 1.1 |
| Subject information and informed consent form (for publication) | HUN Subject Participation Card English VRDN-001-301 Public | 1.0 |
| Subject information and informed consent form (for publication) | HUN Subject Participation Card Hungarian VRDN-001-301 Public | 2.0 |
| Subject information and informed consent form (for publication) | ITA Country ICF Data Protection Italian VRDN-001-301 Public | 1.0 |
| Subject information and informed consent form (for publication) | ITA Country ICF Main Adult Italian VRDN-001-301 Public | 2.0 |
| Subject information and informed consent form (for publication) | ITA Country ICF Other Italian VRDN-001-301 Public | 1.0 |
| Subject information and informed consent form (for publication) | ITA Country ICF Other Pregnant participant Italian VRDN-001-301 Public | 1.0 |
| Subject information and informed consent form (for publication) | ITA Country ICF Other Pregnant partner Italian VRDN-001-301 Public | 1.0 |
| Subject information and informed consent form (for publication) | ITA Country ICF Procedure English VRDN-001-301 Public | 1.0 |
| Subject information and informed consent form (for publication) | ITA Subject Materials Other GP letter Italian VRDN-001-301 Public | 2.0 |
| Subject information and informed consent form (for publication) | L1_ESP Country ICF Main Spanish VRDN-001-301 Public | 2.1 |
| Subject information and informed consent form (for publication) | L1_ESP Country ICF Other Pregnant Partner Spanish VRDN-001-301 Public | 1.1 |
| Subject information and informed consent form (for publication) | L1_ESP Country ICF Other Pregnant participant Spanish VRDN-001-301 Public | 1.1 |
| Subject information and informed consent form (for publication) | POL Country ICF Main Adult Polish VRDN-001-301 Public | 2.0 |
| Subject information and informed consent form (for publication) | POL Country ICF Other Adult Pregnant Participant Polish VRDN-001-301 Public | 1.1 |
| Subject information and informed consent form (for publication) | POL Country ICF Other Adult Pregnant Partner Polish VRDN-001-301 Public | 1.1 |
| Subject information and informed consent form (for publication) | POL Country ICF Other Adult Reimbursement through Greenphire Polish VRDN-001-301 Public | 1.0 |
| Subject information and informed consent form (for publication) | POL Country ICF Procedure Polish VRDN-001-301 Public | 1.0 |
| Synopsis of the protocol (for publication) | D1_Lay Protocol Synopsis Main English VRDN-001-301 Public | 3.0 |
| Synopsis of the protocol (for publication) | D1_Lay Protocol Synopsis Main French VRDN-001-301 Public | 3.0 |
| Synopsis of the protocol (for publication) | D1_Lay Protocol Synopsis Main German VRDN-001-301 Public | 3.0 |
| Synopsis of the protocol (for publication) | D1_Lay Protocol Synopsis Main Hungarian VRDN-001-301 Public | 3.0 |
| Synopsis of the protocol (for publication) | D1_Lay Protocol Synopsis Main Italian VRDN-001-301 Public | 3.0 |
| Synopsis of the protocol (for publication) | D1_Lay Protocol Synopsis Main Polish VRDN-001-301 Public | 3.0 |
| Synopsis of the protocol (for publication) | D1_Lay Protocol Synopsis Main Spanish VRDN-001-301 Public | 3.0 |
Application history
6 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2023-11-28 | Spain | Acceptable 2024-04-01
|
2024-04-02 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2024-06-28 | Spain | Acceptable 2024-08-12
|
2024-08-13 |
| 3 | SUBSTANTIAL MODIFICATION | SM-2 | 2024-10-11 | Acceptable | 2024-11-15 | |
| 4 | SUBSTANTIAL MODIFICATION | SM-3 | 2024-12-05 | Spain | Acceptable 2025-03-10
|
2025-03-10 |
| 5 | SUBSTANTIAL MODIFICATION | SM-4 | 2025-04-11 | Spain | Acceptable | 2025-05-19 |
| 6 | SUBSTANTIAL MODIFICATION | SM-5 | 2025-06-27 | Spain | Acceptable 2025-08-29
|
2025-09-01 |