Clinical trial NCT05776069: Study of VGA039 in Healthy Volunteers and Patients with Von Willebrand Disease (VIVID)
Official title: A Multi-Modular Trial to Evaluate VGA039 in Healthy Volunteers and Patients with Von Willebrand Disease and Other Bleeding Disorders (VIVID)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: Von Willebrand Diseases
Keywords: Von Willebrand Disorder
Lead sponsor: Vega Therapeutics, Inc
Sponsor class: INDUSTRY
First posted: 2023-03-20
Enrollment: 116
Sex: ALL
Minimum age (years): 12
Maximum age (years): 60
Standard ages: CHILD
ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief summary: The VIVID study is structured in a master protocol format comprised of multiple parts that evaluate intravenous (IV) and subcutaneous (SC) VGA039 in healthy volunteers and subjects with von Willebrand Disease (VWD) and other bleeding disorders.

Detailed description: This first-in-human study consists of 5 parts based on the subject population. Part 1 is a randomized, double-blind, placebo-controlled, single-ascending dose (SAD) evaluation of IV or SC VGA039 or placebo in up to 8 cohorts in healthy volunteers. Part 2 is an open-label, SAD of SC or IV VGA039 in up to 8 cohorts in subjects diagnosed with VWD. All participants will be enrolled, treated, and followed up for 15 weeks (IV SAD) or 8 weeks(SC SAD). Part 3 is an open-label, Phase 1b study of SC multiple doses (MD) of VGA039 in up to 4 cohorts. Part 4 is an open-label, Phase 2 study of SC single, surgical prophylaxis (SP) doses of VGA039 administered prior to a minor surgical procedure in subjects diagnosed with VWD in up to 2 cohorts. Part 5 is an open-label extension (OLE) study of SC MD of VGA039 in eligible subjects diagnosed with VWD who have previously participated in a VGA039 interventional trial.

Eligibility criteria: Key Inclusion Criteria (All Subjects)

* Subjects, 18 to 60 years of age, inclusive for Parts 1 and 2
* Subjects, 12 to 60 years of age, inclusive for Parts 3 and 5
* No clinically significant laboratory, ECG, or vital signs results.

Additional Key Inclusion Criteria (for Subjects in Part 1 Only) • Body mass index of 18-32 kg/m2

Additional Key Inclusion Criteria (for Subjects in Part 2 Only)

* Subjects with VWD who are symptomatic, defined as having a history of bleeding or bruising.
* Hemoglobin level ≥ 8 g/dL and platelet count ≥ 150 × 109/L at Screening.

Exclusion Key Criteria (All Subjects)

* Use of hormonal contraceptives within 56 days prior to administration of the study drug.
* Subjects with detection of FV Leiden or Prothrombin G20210A mutation, protein C or S deficiency, antithrombin deficiency, or antiphospholipid antibody syndrome at Screening.
* Subjects with other known pro-thrombotic disorders or abnormal findings in any prior laboratory thrombophilia evaluation.
* History of arterial or venous thrombosis, including superficial thrombophlebitis, or embolism.
* Evidence of renal, hepatic, central nervous system, respiratory, cardiovascular disease, cerebrovascular disease, peripheral vascular disease, or metabolic dysfunction.

Additional Key Exclusion Criterion (Subjects in Part 1 Only)

• Baseline FVIII activity \> 150 IU/dL.

Additional Key Exclusion Criteria (Subjects in Parts 2, 3, 4 and 5 Only)

* Baseline FVIII activity \> 50 IU/dL.
* Any acute, clinically significant bleeding event requiring surgical or procedural intervention within 7 days prior to receiving study drug.

Primary outcomes:
measure: Incidence of Treatment-Emergent Adverse Events [Safety and tolerability]
timeFrame: From start of study drug administration until 15 or 8 weeks after IV or SC study drug administration, respectively
Secondary outcomes:
measure: Plasma Concentrations of single IV and SC doses of VGA039
timeFrame: From baseline until 15 or 8 weeks after IV or SC study drug administration, respectively
measure: Pharmacodynamics of single IV and SC doses of VGA039
timeFrame: From baseline until 15 or 8 weeks after IV or SC study drug administration, respectively
measure: Incidence of Anti-drug antibodies to VGA039
timeFrame: From baseline until 15 or 8 weeks after IV or SC study drug administration, respectively
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Orthopedic Institute for Children (UCLA)
state: California
city: Sacramento
country: United States
facility: UC Davis Medical Center
state: California
city: Aurora
country: United States
facility: University of Colorado School of Medicine
state: Colorado
city: Atlanta
country: United States
facility: Hemophilia of Georgia Center for Bleeding & Clotting Disorders of Emory
state: Georgia
city: Pittsburgh
country: United States
facility: Hemophilia Center of Western PA
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Dallas
country: United States
facility: University of Texas Southwestern
state: Texas
city: Seattle
country: United States
facility: Washington Center for Bleeding Disorders
state: Washington
city: Milwaukee
country: United States
facility: Versiti Comprehensive Center for Bleeding Disorders
state: Wisconsin
city: Herston
country: Australia
facility: Royal Brisbane & Women's Hospital, Queensland Haemophilia Centre
state: Queenland
city: Vienna
country: Austria
facility: Medical University of Vienna
city: Campinas
country: Brazil
facility: Hemocentro Unicamp
state: Sao Paulo
city: Rio De Janeiro
country: Brazil
facility: Centro de Hemoterapia e Hematologia do Rio de Janeiro HEMORIO
city: São Paulo
country: Brazil
facility: Hospital das Clinicas - USP Endereco
city: Hamilton
country: Canada
facility: Hamilton Health Sciences Corporation
state: Ontario
city: Kingston
country: Canada
facility: Queens University
state: Ontario
city: Toronto
country: Canada
facility: St. Michaels Hospital
state: Ontario
city: Sion
country: India
facility: K J Somaiya Super Speciality Hospital & Research Centre
state: Mumbai
city: Johannesburg
country: South Africa
facility: Charlotte Maxeke Johannesburg Academic Hospital
city: Birmingham
country: United Kingdom
facility: Queen Elizabeth Hospital Birmingham
state: Edgbaston
city: Southampton
country: United Kingdom
facility: University Hospital Southampton NHS Foundation Trust
state: Hampshire
city: Whitechapel
country: United Kingdom
facility: Royal London Hospital, Clinical Haematology Research
state: London
city: London
country: United Kingdom
facility: Royal Free Hospital
city: London
country: United Kingdom
facility: Imperial College Healthcare NHS Trust- Queen Charlotte's & Chelsea Hospital
Study officials:
References: