Clinical trial NCT06179875: An Open-label Study for Non-responders of VRDN-001-101 and VRDN-001-301 (OLE)
Official title: An Open-label Study for Participants Who Are Non-responders at the End of Treatment Assessment on the VRDN-001-101 and VRDN-001-301 Pivotal Studies
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Thyroid Eye Disease
Keywords: Graves
Thyroid-Associated Ophthalmopathy
Thyroid Eye Disease
Dysthyroid Ophthalmopathy
Graves Eye Disease
Graves Orbitopathy
Myopathic Ophthalmopathy
Congestive Ophthalmopathy
Edematous Ophthalmopathy
Infiltrative Ophthalmopathy
Lead sponsor: Viridian Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2023-12-22
Enrollment: 143
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Single treatment assignment
Primary purpose: TREATMENT

Brief summary: An open-label study for participants who received VRDN-001 or placebo and were non-responders at end of the treatment period assessment (i.e., 15 weeks) in the VRDN-001-101 (THRIVE) and VRDN-001-301 (THRIVE-2) pivotal studies


Eligibility criteria: Key Inclusion Criteria:

1. Be able to understand the study procedures and the risks involved and be willing to provide written informed consent before the first study-related activity
2. Have completed at least 5 IV infusions and assessments required to determine proptosis responder status 3 weeks post the fifth IV infusion (i.e., Week 15) as defined in either the VRDN-001-101 or VRDN-001-301 pivotal studies
3. Been a participant in either the VRDN-001-101 or VRDN-001-301 studies and found to be a non-responder as defined within the VRDN-001-101 or VRDN-001-301 study
4. Not require immediate surgical ophthalmological or orbital surgery in the study eye for any reason
5. Must agree to use highly effective contraception as specified in the protocol

   ⦁ Female TED participants must have a negative urine pregnancy test at screening
6. Be willing and able to comply with all the requirements of the protocol for the entire duration of the study

Key Exclusion Criteria:

Participants must not:

1. Have received prior treatment with another anti-IGF-1R agent
2. Have received systemic corticosteroids for any condition, including TED, or selenium within 2 weeks prior to first dose
3. Have received other immunosuppressive drugs or another investigational agent for any condition, including TED (other than VRDN-001 or placebo associated with the VRDN-001-101 or VRDN-001-301 pivotal studies), or any other therapy for TED, within 8 weeks prior to first dose
4. Have received radioactive iodine (RAI) treatment within 8 weeks prior to first dose
5. Have had previous orbital irradiation or decompression surgery involving excision of fat for TED to the study eye's orbit
6. Must not have abnormal hearing test before first dose. Must also not have a history of ear conditions considered significant by study doctor

Primary outcomes:
measure: Proptosis Responder Rate in the most proptotic eye
timeFrame: Week 15
Secondary outcomes:
measure: Change from Baseline in proptosis in the most proptotic eye
timeFrame: Week 15
measure: Clinical Activity Responder Rate in the most proptotic eye
timeFrame: Week 15
measure: Overall Responder Rate in the most proptotic eye
timeFrame: Week 15
measure: Diplopia Responder Rate
timeFrame: Week 15
measure: Diplopia Resolution Rate
timeFrame: Week 15
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Advancing Research International, LLC
state: California
city: Los Angeles
country: United States
facility: USC Eye Institute
state: California
city: Newport Beach
country: United States
facility: Advancing Research International, LLC
state: California
city: Palo Alto
country: United States
facility: Stanford Byers Eye Institute
state: California
city: San Diego
country: United States
facility: Cockerham Eye Consultants, PC
state: California
city: Miami
country: United States
facility: University of Miami Miller School of Medicine, Bascom Palmer Eye Institute
state: Florida
city: Sarasota
country: United States
facility: Sarasota Retina Institute
state: Florida
city: Oak Lawn
country: United States
facility: Vision Medical Research
state: Illinois
city: East Weymouth
country: United States
facility: Ophthalmic Consultants of Boston
state: Massachusetts
city: Livonia
country: United States
facility: Kahana Oculoplastic & Orbital Surgery
state: Michigan
city: Las Vegas
country: United States
facility: Ophthalmic Plastic, Reconstructive, Orbital and Cosmetic Surgery
state: Nevada
city: Newark
country: United States
facility: Rutgers New Jersey Medical School
state: New Jersey
city: Somerset
country: United States
facility: The Center for Eye and Facial Plastic Surgery
state: New Jersey
city: Philadelphia
country: United States
facility: Hospital of the University of Pennsylvania Perleman Center
state: Pennsylvania
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
city: Houston
country: United States
facility: Neuro-Eye Clinical Trials
state: Texas
city: Burlington
country: United States
facility: University of Vermont Medical Center
state: Vermont
city: Seattle
country: United States
facility: University of Washington Medical Center
state: Washington
city: Montpellier Cedex 5
country: France
facility: Centre Hospitalier Universitaire de Montperllier Hopital Lapeyronie
state: Hérault
city: Saint-Herblain
country: France
facility: Centre Hospitalier Universitaire De Nantes G.R. Laenne
state: Loire-Atlantique
city: Angers Cedex
country: France
facility: CHU Angers
city: Göttingen
country: Germany
facility: Universitatmedizin Gottingen
state: Niedersachsen
city: Berlin
country: Germany
facility: Charite - Universitatsmedizin Berlin KoR, Campus Virchow Klinikum, Klinik tor Augenheilkunde
city: Dresden
country: Germany
facility: Universitatsklinikum Carl Gustav Carus
city: Essen
country: Germany
facility: Universitatsklinikum Essen AoR - Klinik fur Augenheilkunde
city: Freiburg
country: Germany
facility: University Medical Center Freiburg
city: Amsterdam
country: Netherlands
facility: Amsterdam UMC
city: Walbrzych
country: Poland
facility: Centrum Medyczne Piasta
state: Piasta
city: Bialystok
country: Poland
facility: Uniwersytecki Szpital Kliniczny w Bialymstoku
city: Gdansk
country: Poland
facility: Optimum Profesorskie Centrum Okulistyki
city: Piaseczno
country: Poland
facility: Centrum Medyczne Pulawska
city: Córdoba
country: Spain
facility: Hospital Arruzafa. Servicio de Oftalmologia
city: Madrid
country: Spain
facility: Hospital Universitario Ramon y Cajal
city: Madrid
country: Spain
facility: Hospital Clinico San Carlos
city: Sevilla
country: Spain
facility: Hospital Universitario Virgen de la Macarena
city: Zaragoza
country: Spain
facility: Hospital Universitario Miguel Servel
city: Ankara
country: Turkey
facility: Hacettepe Universitesi Tip Fakultesi
city: Ankara
country: Turkey
facility: Gazi University Medical Faculty Hospital
city: Antalya
country: Turkey
facility: Akdeniz University Medical Faculty Hospital
city: Istanbul
country: Turkey
facility: Marmara University Faculty of Medicine
city: Norwich
country: United Kingdom
facility: Norfolk and Norwich University ospital NHS Foundation Trust
state: London
city: Bristol
country: United Kingdom
facility: University Hospital Bristol and Weston NHS Foundation Trust- Bristol Eye Hospital
city: London
country: United Kingdom
facility: Imperial College Healthcare NHS Trust Western Eye Hospital
city: London
country: United Kingdom
facility: Guy's and St. Thomas Trust
city: Newcastle
country: United Kingdom
facility: Newcastle Eye Centre
Study officials:
References: