Clinical trial • Phase III • Ophthalmology|Endocrinology
VRDN-003 for Thyroid eye disease
Phase III trial of VRDN-003 for Thyroid eye disease.
Overview
- Trial Therapeutic Area
- Ophthalmology|Endocrinology
- 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 (ted sc placebo) administered as matching subcutaneous injections; active treatment vrdn-003 administered as subcutaneous injections given every 4 weeks or every 8 weeks (as described in protocol).-controlled Phase III trial across 19 sites in Poland, Germany, Spain and others.
- Randomised
- Yes
- Comparator
- Placebo (Ted sc placebo) administered as matching subcutaneous injections; active treatment VRDN-003 administered as subcutaneous injections given every 4 weeks or every 8 weeks (as described in protocol).
- Target Sample Size
- 113
- Trial Duration For Participant
- 168
Eligibility
Recruits 113 Vulnerable population selected. Study enrols adults (≥18 years); no minors/assent procedures are indicated. Pregnancy and breastfeeding are specifically addressed (pregnant or breastfeeding participants are excluded) and there are dedicated country-specific ICFs for pregnant participants and partner information (e.g. documents titled 'Country ICF Other Pregnant Participant' and 'SIS and ICF Pregnant Partner'). Informed consent is obtained from adult participants via country-specific ICFs (documents available in Polish, German, Spanish, Hungarian, Dutch and English as listed)..
- Pregnancy Exclusion
- Female participants who are pregnant or breastfeeding
- Vulnerable Population
- Vulnerable population selected. Study enrols adults (≥18 years); no minors/assent procedures are indicated. Pregnancy and breastfeeding are specifically addressed (pregnant or breastfeeding participants are excluded) and there are dedicated country-specific ICFs for pregnant participants and partner information (e.g. documents titled 'Country ICF Other Pregnant Participant' and 'SIS and ICF Pregnant Partner'). Informed consent is obtained from adult participants via country-specific ICFs (documents available in Polish, German, Spanish, Hungarian, Dutch and English as listed).
Inclusion criteria
- {"criterion_text":"- Adult males or females, ≥18 to ≤75 years of age who have a clinical diagnosis of TED with any value on the CAS scale (0-7)\n- Must have moderate to severe chronic TED with eye bulging value in the study eye before the first dose as defined in the protocol\n- Have experienced eye-related signs or symptoms that began more than 15 months before the first study visit\n- Must agree to use highly effective contraception as specified in the protocol\n- 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\n- Received corticosteroids (drugs that reduce inflammation in the body) for any condition, including TED, within 2 weeks prior to first dose.\n- 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\n- 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\n- Have had abnormal hearing test before first dose. History of ear conditions considered significant by study doctor\n- Have inflammatory bowel disease (conditions that involve inflammation of the gastrointestinal [GI] tract)\n- 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 no worsening in Clinical Activity Score or CAS (that is an estimate of disease severity/symptoms) at Week 24 versus measurements before the first dose.","definition_or_measurement_approach":"Overall Responder Rate at Week 24; defined as participants achieving a ≥2 mm reduction in eye bulging AND no worsening in Clinical Activity Score (CAS) at Week 24 versus pre-dose measurements."}
Secondary endpoints
- {"endpoint_text":"- The secondary endpoints will assess changes in eye bulging 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":"Assess change in eye bulging (proptosis) versus pre-dose measurements; percent achieving required reduction in eye bulging, disease severity/symptoms, and reduction/resolution of diplopia at Week 24."}
- {"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":"Safety events (adverse events/side effects) monitored and evaluated throughout study duration; assessment of effects of VRDN-003 on participants and vice-versa."}
Recruitment
- Planned Sample Size
- 113
- Recruitment Window Months
- 22
- Consent Approach
- Informed consent obtained from adult participants via country-specific subject information and informed consent forms (L1 Country ICF Main Adult and related ICFs). There are dedicated ICFs and information for pregnant participants and partner information where relevant. ICF documents are available in multiple languages corresponding to participating countries (Polish, German, Spanish, Hungarian, Dutch and English). No assent/parental consent is indicated (minors excluded).
Methods
- Country-specific recruitment materials and procedures documented: posters, flyers, brochures, flipbooks and recruitment procedure descriptions exist for Poland (K1_POL etc.), Germany (K1_DEU, K2_DEU), Spain (K1_ESP, K2_ESP), Hungary (K2_HUN, K1_HUN) and Netherlands (K1_NLD, K2_NLD). Materials are provided in country/language-specific versions (e.g. Polish, German, Spanish, Hungarian, Dutch and English) as listed in the CTIS documents.
- Site-based/clinic recruitment indicated by trial sites (hospital/clinic sites listed per country); recruitment procedure descriptions associated with country Part II submissions.
Geography
- Total Number Of Sites
- 19
- Total Number Of Participants
- 91
Poland
- Earliest CTIS Part Ii Submission Date
- 03-02-2025
- Latest Decision Or Authorization Date
- 31-03-2026
- Processing Time Days
- 422
- Number Of Sites
- 5
- Number Of Participants
- 34
Sites
- Site Name
- Eskulap Pabianice Sp. z o.o.
- Department Name
- 2214:Eskulap Pabianice Sp. z o.o.
- Contact Person Name
- Agnieszka Waliszek-Iwanicka
- Contact Person Email
- a.waliszek@eskulapbk.pl
- Site Name
- Santa Sp. z o.o.
- Department Name
- 2208:Santa Familia PTG Łódź
- Contact Person Name
- Michal Nowak
- Contact Person Email
- michal.nowak@ptg-network.com
- Site Name
- Polimedica Centrum Badan Profilaktyki I Leczenia Sp. z o.o.
- Department Name
- 2216:Polimedica PTG
- Contact Person Name
- Magdalena Guminska
- Contact Person Email
- magdalena.guminska@ptg-network.com
- Site Name
- Warszawski Szpital Okulistyczny Sp. z o.o.
- Department Name
- 2212:Warszawski Szpital Okulistyczny
- Contact Person Name
- Agnieszka Nowosielska
- Contact Person Email
- nowosielska.aga@gmail.com
- Site Name
- Pro Life Medica Sp. z o.o.
- Department Name
- 2215:ETG Lublin
- Contact Person Name
- Iwona Chmiel - Perzyńska
- Contact Person Email
- i.chmielperzynska@etg-network.com
Germany
- Earliest CTIS Part Ii Submission Date
- 30-01-2025
- Latest Decision Or Authorization Date
- 31-03-2026
- Processing Time Days
- 425
- Number Of Sites
- 3
- Number Of Participants
- 9
Sites
- Site Name
- Charite Universitaetsmedizin Berlin KöR
- Department Name
- 1603: Klinik für Augenheilkunde
- Contact Person Name
- Eckart Bertelmann
- Contact Person Email
- Eckart.bertelmann@charite.de
- Site Name
- Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz KöR
- Department Name
- 1605: Endokrinologie & Stoffwechsele
- Contact Person Name
- George Kahaly
- Contact Person Email
- george.kahaly@unimedizin-mainz.de
- Site Name
- Universitaetsmedizin Goettingen
- Department Name
- 1602: Klinik fur Augenheilkunde
- Contact Person Name
- Maren Horn
- Contact Person Email
- maren.horn@med.uni-goettingen.de
Spain
- Earliest CTIS Part Ii Submission Date
- 23-01-2025
- Latest Decision Or Authorization Date
- 01-04-2026
- Processing Time Days
- 433
- Number Of Sites
- 6
- Number Of Participants
- 34
Sites
- Site Name
- Hospital Universitario Miguel Servet
- Department Name
- #2105:Oftalmología
- Contact Person Name
- Laura Maria Gil Arribas
- Contact Person Email
- lauragilarribas@gmail.com
- Site Name
- Hospital Universitario Virgen De La Macarena
- Department Name
- #2101: Oftalmología
- Contact Person Name
- Antonio Manuel Garrido Hermosilla
- Contact Person Email
- ensayosoftalmohuvm2@gmail.com
- Site Name
- Clinica De Oftalmologia De Cordoba S.L.
- Department Name
- #2106: Oftalmología
- Contact Person Name
- Javier Vicente Andreu
- Contact Person Email
- jvicente@hospitalarruzafa.com
- Site Name
- Complexo Hospitalario Universitario De Santiago
- Department Name
- #2104: Oftalmología
- Contact Person Name
- José Manuel Abalo Lojo
- Contact Person Email
- Jose.Manuel.Abalo.Lojo@sergas.es
- Site Name
- Hospital Clinico San Carlos
- Department Name
- #2107: Oftalmología
- 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
- Contact Person Name
- Javier Vicente Andreu
- Contact Person Email
- jvicente@hospitalarruzafa.com
Hungary
- Earliest CTIS Part Ii Submission Date
- 28-10-2024
- Latest Decision Or Authorization Date
- 30-03-2026
- Processing Time Days
- 518
- Number Of Sites
- 3
- Number Of Participants
- 7
Sites
- Site Name
- Nozologen Kft.
- Department Name
- 1901:Szemészet
- Contact Person Name
- Balázs Varsányi
- Contact Person Email
- varsanyi.balazs@ganglion.hu
- Site Name
- University Of Pecs
- Department Name
- 1903:Szemészeti Klinika
- Contact Person Name
- Adrienne Csutak
- Contact Person Email
- csutak.adrienne@pte.hu
- Site Name
- Budapest Retina Associates Kft.
- Department Name
- 1902:Szemészet
- Contact Person Name
- András Seres
- Contact Person Email
- seres@budapestretina.hu
Netherlands
- Earliest CTIS Part Ii Submission Date
- 21-01-2025
- Latest Decision Or Authorization Date
- 26-03-2026
- Processing Time Days
- 429
- Number Of Sites
- 2
- Number Of Participants
- 7
Sites
- Site Name
- Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
- Department Name
- 2802:Internal Medicine
- Contact Person Name
- Marco Medici
- Contact Person Email
- m.medici@erasmusmc.nl
- Site Name
- Amsterdam UMC Stichting
- Department Name
- 2801:Ophtalmology
- Contact Person Name
- Peerooz Saeed
- Contact Person Email
- p.saeed@amsterdamumc.nl
Sponsor
Primary sponsor
- Full Name
- Viridian Therapeutics Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Contract research organisations
- Name
- Suvoda LLC
- Responsibilities
- codes: 3
- Name
- Parexel International (IRL) Limited
- Responsibilities
- codes: 1,10,11,12,13,2,5,6,8
- Name
- Medidata Solutions Inc.
- Responsibilities
- codes: 7
- Name
- Bioclinica Inc.
- Responsibilities
- codes: 15 (Imaging)
- Name
- Scout Clinical
- Responsibilities
- codes: 15 (Patient Reimbursement; Investigator Meeting)
- Name
- Professional Case Management Clinical Trials LLC
- Responsibilities
- codes: 15 (Home Health Care)
Third parties
- {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"codes: 3","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":"Xerimis Inc.","duties_or_roles":"codes: 14,4","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Kcas LLC","duties_or_roles":"codes: 4","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Acm Global Central Laboratory Limited","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":"Mayo Collaborative Services LLC","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":"Bioclinica Inc.","duties_or_roles":"codes: 15 (Imaging)","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"codes: 15 (Patient Reimbursement; Investigator Meeting)","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"South Africa","full_name":"Hearx SA (Pty) Ltd.","duties_or_roles":"codes: 15 (Audiometry assessments)","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Professional Case Management Clinical Trials LLC","duties_or_roles":"codes: 15 (Home Health Care)","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Acm Medical Laboratory Inc.","duties_or_roles":"codes: 4","organisation_type":"Laboratory/Research/Testing facility"}
- {"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"}
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
- Maximum Dose
- 600 mg (max daily); 4200 mg (max total)
- Investigational Product Name
- Ted sc placebo
- Modality
- Other
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