Clinical trial • Phase III • Ophthalmology
VRDN-003 for Thyroid eye disease
Phase III trial of VRDN-003 for Thyroid eye disease.
Overview
- Trial Therapeutic Area
- Ophthalmology
- Trial Disease
- Thyroid eye disease
- Trial Stage
- Phase III
- Drug Modality
- Peptide/protein/enzyme
Key dates
- Initial CTIS Submission Date
- 08-10-2024
- First CTIS Authorization Date
- 11-02-2025
Trial design
Randomised, placebo arm: ted sc placebo (placebo comparator). dosing/schedule: placebo administered as matched subcutaneous injections to vrdn-003 (vrdn-003 given subcutaneously every 4 weeks or every 8 weeks; placebo schedule matched accordingly).-controlled Phase III trial in Germany, Hungary, Spain and others.
- Randomised
- Yes
- Comparator
- Placebo arm: TED SC Placebo (placebo comparator). Dosing/schedule: placebo administered as matched subcutaneous injections to VRDN-003 (VRDN-003 given subcutaneously every 4 weeks or every 8 weeks; placebo schedule matched accordingly).
- Target Sample Size
- 88
- Trial Duration For Participant
- 168
Eligibility
Recruits 88 Vulnerable population selected (isVulnerablePopulationSelected = true). Country-specific informed consent forms and additional ICFs for pregnant participants/partners are provided (multiple L1 documents listed). Only adult participants (≥18) are eligible and provide informed consent; dedicated documents are available for pregnant participants and pregnant partners in several countries. No assent procedures for minors are specified (minors excluded)..
- Pregnancy Exclusion
- Female participants who are pregnant or breastfeeding
- Vulnerable Population
- Vulnerable population selected (isVulnerablePopulationSelected = true). Country-specific informed consent forms and additional ICFs for pregnant participants/partners are provided (multiple L1 documents listed). Only adult participants (≥18) are eligible and provide informed consent; dedicated documents are available for pregnant participants and pregnant partners in several countries. No assent procedures for minors are specified (minors excluded).
Inclusion criteria
- {"criterion_text":"- 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"}
- {"criterion_text":"- 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"}
- {"criterion_text":"- Have experienced eye-related signs or symptoms that began within 15 months before the first study visit"}
- {"criterion_text":"- Must agree to use highly effective contraception as specified in the protocol"}
- {"criterion_text":"- Female participants must have a negative pregnancy test at first study visit"}
Exclusion criteria
- {"criterion_text":"- Received prior treatment with another anti-IGF-1R therapy"}
- {"criterion_text":"- Received corticosteroids (drugs that reduce inflammation in the body) for any condition, including TED, within 2 weeks prior to first dose"}
- {"criterion_text":"- 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"}
- {"criterion_text":"- 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"}
- {"criterion_text":"- Have abnormal hearing test before first dose. History of ear conditions considered significant by study doctor"}
- {"criterion_text":"- Have inflammatory bowel disease (conditions that involve inflammation of the gastrointestinal [GI] tract)"}
- {"criterion_text":"- Female participants who are pregnant or breastfeeding"}
Endpoints
Primary endpoints
- {"endpoint_text":"- 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.","definition_or_measurement_approach":"Overall Responder Rate at Week 24 defined as percentage of participants achieving both: ≥2 mm reduction in eye bulging (proptosis) AND ≥2-point reduction in Clinical Activity Score (CAS) at Week 24 compared to pre-dose measurements."}
Secondary endpoints
- {"endpoint_text":"- 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.","definition_or_measurement_approach":"Assessment of change from baseline in eye bulging (proptosis), disease severity measures and symptoms, percent achieving prespecified reductions, and reduction/resolution of diplopia at Week 24 versus pre-dose measurements."}
- {"endpoint_text":"- 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.","definition_or_measurement_approach":"Collection and assessment of safety events/adverse events, laboratory and clinical safety assessments and overall safety/tolerability of VRDN-003 during the study."}
Recruitment
- Planned Sample Size
- 88
- Recruitment Window Months
- 23
- Consent Approach
- Informed consent obtained from adult participants (≥18 years) via country-specific Subject Information and Informed Consent Forms (L1 documents). Multiple language versions available (English, German, Dutch, Spanish, Hungarian, Polish as indicated by L1 documents). There are specific ICFs/forms for pregnant participants and for pregnant partners where applicable. No assent for minors is described (minors excluded).
Methods
- Country-specific recruitment arrangements documents (K1) and recruitment materials (K2) are provided: posters, brochures, flyers, flipbooks (documents listed per country).
- Recruitment materials are available in country languages and English (examples: Polish recruitment poster/brochure/flipbook for Poland; German and English recruitment materials for Germany; Hungarian materials for Hungary; Spanish materials for Spain; Dutch materials for Netherlands).
- Site-based recruitment through participating hospitals/clinics (listed trial sites per country).
Geography
- Total Number Of Sites
- 18
- Total Number Of Participants
- 54
Germany
- Earliest CTIS Part Ii Submission Date
- 24-01-2025
- Latest Decision Or Authorization Date
- 11-02-2025
- Processing Time Days
- 18
- Number Of Sites
- 3
- Number Of Participants
- 12
Sites
- Site Name
- Universitaetsklinikum Carl Gustav Carus Dresden an der Technischen Universitaet Dresden AöR
- Department Name
- 1606:Klinik und Poliklinik für Augenheilkunde
- Principal Investigator Name
- Dirk Sandner
- Principal Investigator Email
- dirk.sandner@ukdd.de
- Contact Person Name
- Dirk Sandner
- Contact Person Email
- dirk.sandner@ukdd.de
- Site Name
- Charite Universitaetsmedizin Berlin KöR
- Department Name
- 1603:Klinik für Augenheilkunde
- Principal Investigator Name
- Eckart Bertelmann
- Principal Investigator Email
- Eckart.bertelmann@charite.de
- Contact Person Name
- Eckart Bertelmann
- Contact Person Email
- Eckart.bertelmann@charite.de
- Site Name
- Universitaetsmedizin Goettingen
- Department Name
- 1602:Klinik für Augenheilkunde
- Principal Investigator Name
- Maren Horn
- Principal Investigator Email
- maren.horn@med.uni-goettingen.de
- Contact Person Name
- Maren Horn
- Contact Person Email
- maren.horn@med.uni-goettingen.de
Hungary
- Earliest CTIS Part Ii Submission Date
- 23-01-2025
- Latest Decision Or Authorization Date
- 13-02-2025
- Processing Time Days
- 21
- Number Of Sites
- 3
- Number Of Participants
- 4
Sites
- Site Name
- Budapest Retina Associates Kft.
- Department Name
- 1902:Szemészet
- Principal Investigator Name
- András Seres
- Principal Investigator Email
- seres@budapestretina.hu
- Contact Person Name
- András Seres
- Contact Person Email
- seres@budapestretina.hu
- Site Name
- University Of Pecs
- Department Name
- 1903:Szemészeti Klinika
- Principal Investigator Name
- Adrienne Csutak
- Principal Investigator Email
- csutak.adrienne@pte.hu
- Contact Person Name
- Adrienne Csutak
- Contact Person Email
- csutak.adrienne@pte.hu
- Site Name
- Vas Varmegyei Markusovszky Egyetemi Oktatokorhaz
- Department Name
- 1904:Szemészeti Osztály
- Principal Investigator Name
- György Bátor
- Principal Investigator Email
- bator.gyorgy@markusovszky.hu
- Contact Person Name
- György Bátor
- Contact Person Email
- bator.gyorgy@markusovszky.hu
Spain
- Earliest CTIS Part Ii Submission Date
- 23-01-2025
- Latest Decision Or Authorization Date
- 12-02-2025
- Processing Time Days
- 20
- Number Of Sites
- 7
- Number Of Participants
- 21
Sites
- Site Name
- Complexo Hospitalario Universitario De Santiago
- Department Name
- 2104:Oftalmología
- Principal Investigator Name
- José Manuel Abalo Lojo
- Principal Investigator Email
- Jose.Manuel.Abalo.Lojo@sergas.es
- Contact Person Name
- José Manuel Abalo Lojo
- Contact Person Email
- Jose.Manuel.Abalo.Lojo@sergas.es
- Site Name
- Hospital Universitario Ramon Y Cajal
- Department Name
- 2103: Oftalmología
- Principal Investigator Name
- Marco Sales Sanz
- Principal Investigator Email
- marco.sales@salud.madrid.org
- Contact Person Name
- Marco Sales Sanz
- Contact Person Email
- marco.sales@salud.madrid.org
- Site Name
- Hospital Universitario Virgen De La Macarena
- Department Name
- 2101: Oftalmología
- Principal Investigator Name
- Antonio Garrido Hermosilla
- Principal Investigator Email
- ensayosoftalmohuvm2@gmail.com
- Contact Person Name
- Antonio Garrido Hermosilla
- Contact Person Email
- ensayosoftalmohuvm2@gmail.com
- Site Name
- Hospital Universitario Miguel Servet
- Department Name
- 2105:Oftalmología
- Principal Investigator Name
- Laura Maria Gil Arribas
- Principal Investigator Email
- lauragilarribas@gmail.com
- Contact Person Name
- Laura Maria Gil Arribas
- Contact Person Email
- lauragilarribas@gmail.com
- Site Name
- Hospital Clinico San Carlos
- Department Name
- 2107:Oftalmología
- Principal Investigator Name
- Ángel Romo López
- Principal Investigator Email
- angel.romo@salud.madrid.org
- Contact Person Name
- Ángel Romo López
- Contact Person Email
- angel.romo@salud.madrid.org
- Site Name
- Clinica De Oftalmologia De Cordoba S.L.
- Department Name
- 2106:Oftalmología
- Principal Investigator Name
- Javier Vicente Andreu
- Principal Investigator Email
- jvicente@hospitalarruzafa.com
- Contact Person Name
- Javier Vicente Andreu
- Contact Person Email
- jvicente@hospitalarruzafa.com
- Site Name
- Hospital Universitario Virgen De La Macarena (additional entry)
- Department Name
- 2101: Oftalmología
- Principal Investigator Name
- Antonio Garrido Hermosilla
- Principal Investigator Email
- ensayosoftalmohuvm2@gmail.com
- Contact Person Name
- Antonio Garrido Hermosilla
- Contact Person Email
- ensayosoftalmohuvm2@gmail.com
Netherlands
- Earliest CTIS Part Ii Submission Date
- 21-01-2025
- Latest Decision Or Authorization Date
- 12-02-2025
- Processing Time Days
- 22
- Number Of Sites
- 1
- Number Of Participants
- 7
Sites
- Site Name
- Amsterdam UMC Stichting
- Department Name
- 2801:Ophtalmology
- Principal Investigator Name
- Peerooz Saeed
- Principal Investigator Email
- p.saeed@amsterdamumc.nl
- Contact Person Name
- Peerooz Saeed
- Contact Person Email
- p.saeed@amsterdamumc.nl
Poland
- Earliest CTIS Part Ii Submission Date
- 04-02-2025
- Latest Decision Or Authorization Date
- 17-02-2025
- Processing Time Days
- 13
- Number Of Sites
- 4
- Number Of Participants
- 10
Sites
- Site Name
- Warszawski Szpital Okulistyczny Sp. z o.o.
- Department Name
- 2212: Warszawski Szpital Okulistyczny
- Principal Investigator Name
- Agnieszka Nowosielska
- Principal Investigator Email
- nowosielska.aga@gmail.com
- Contact Person Name
- Agnieszka Nowosielska
- Contact Person Email
- nowosielska.aga@gmail.com
- Site Name
- Santa Sp. z o.o.
- Department Name
- 2208: Santa Familia PTG Łódź
- Principal Investigator Name
- Michal Nowak
- Principal Investigator Email
- michal.nowak@ptg-network.com
- Contact Person Name
- Michal Nowak
- Contact Person Email
- michal.nowak@ptg-network.com
- Site Name
- Centrum Medyczne Pulawska Sp. z o.o.
- Department Name
- 2203: Centrum Medyczne Puławska Sp. z o.o.
- Principal Investigator Name
- Malgorzata Zaras
- Principal Investigator Email
- malgorzata.zaras@cmp.med.pl
- Contact Person Name
- Malgorzata Zaras
- Contact Person Email
- malgorzata.zaras@cmp.med.pl
- Site Name
- Eskulap Pabianice Sp. z o.o.
- Department Name
- 2214: Eskulap Pabianice Sp. z o.o.
- Principal Investigator Name
- Barbara Gluck
- Principal Investigator Email
- b.gluck@eskulapbk.pl
- Contact Person Name
- Barbara Gluck
- Contact Person Email
- b.gluck@eskulapbk.pl
Sponsor
Primary sponsor
- Full Name
- Viridian Therapeutics Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Contract research organisations
- Name
- Parexel International (IRL) Limited
- Responsibilities
- codes: [1,10,11,12,13,2,5,6,8]
- Name
- Suvoda LLC
- Responsibilities
- codes: [3]
- Name
- Bioclinica Inc.
- Responsibilities
- value: Imaging
- Name
- Kcas LLC
- Responsibilities
- codes: [4]
- Name
- Medidata Solutions Inc.
- Responsibilities
- codes: [7]
Third parties
- {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"codes: [1,10,11,12,13,2,5,6,8]","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Xerimis Inc.","duties_or_roles":"codes: [14,4]","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Acm Medical Laboratory Inc.","duties_or_roles":"codes: [4]","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United Kingdom","full_name":"The Doctors Laboratory Limited","duties_or_roles":"codes: [4]","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"codes: [7]","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Arup Laboratories Inc.","duties_or_roles":"codes: [4]","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Kcas LLC","duties_or_roles":"codes: [4]","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"codes: [3]","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Mayo Collaborative Services LLC","duties_or_roles":"codes: [4]","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Professional Case Management Clinical Trials LLC","duties_or_roles":"value: Home Health Care","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Bioclinica Inc.","duties_or_roles":"value: Imaging","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United Kingdom","full_name":"Acm Global Central Laboratory Limited","duties_or_roles":"codes: [4]","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"South Africa","full_name":"Hearx SA (Pty) Ltd.","duties_or_roles":"value: Audiometry assessments","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"value: Patient Reimbursement; Investigator Meeting","organisation_type":"Hospital/Clinic/Other health care facility"}
Investigational products
- Investigational Product Name
- VRDN (Insulin-like growth factor-1 receptor [IGF-1R] inhibitor with half-life extension)
- Active Substance
- VRDN-003
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- SUBCUTANEOUS USE
- Route
- Subcutaneous
- Frequency
- Every 4 weeks or every 8 weeks (per protocol dosing schedules)
- Maximum Dose
- max daily 600 mg; max total 4200 mg (as listed in product entry)
- Investigational Product Name
- TED SC Placebo
- Modality
- Other
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