Subcutaneous VGA039 to prevent bleeds in people with von Willebrand disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a Phase 3, multicenter, open-label, single-sequence crossover study that gives each participant an observational period followed by active treatment with subcutaneous VGA039. After at least 24 weeks of observational data collection, participants will receive approximately 49 weeks of VGA039 prophylaxis. Patients will keep diaries recording the number, duration, location, and type of bleeds and any treatments used, while investigators monitor adverse events and laboratory findings. The study enrolls people with all types of VWD who have a high historical bleeding rate and are considered candidates for routine prophylaxis.

Why it matters

Who can join: the full rules

Key Inclusion Criteria

* 12 to 75 years of age, inclusive
* No clinically significant laboratory, ECG, or vital signs results
* Documented diagnosis consistent with VWD of any type
* Historical annualized bleeding rate (ABR; excluding menstrual bleeds and bleeds under the skin) of both untreated and treated bleeds ≥12 per year
* Patients with VWD who are judged by the investigator to be suitable candidates for routine prophylaxis to reduce the frequency of bleeding episodes
* Hemoglobin level ≥ 8 g/dL and platelet count ≥ 100 x 109/L at Screening

Key Exclusion Criteria

* Use of routine prophylaxis of VWF-containing concentrates defined as at least 1 VWF-containing concentrate infusion to prevent or reduce bleeding per week during the previous 6 months prior to screening
* Planning to initiate routine prophylaxis with VWF-containing concentrates or any other hemostatic treatment during the study
* Patients with pro-thrombotic disorders or abnormal findings on laboratory thrombophilia evaluation performed at screening or previously documented
* 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
* Baseline FVIII activity \> lower limit of normal (LLN)

Where it's running

• Arkansas Children's Hospital
  Little Rock, Arkansas, United States Recruiting
• Luskin Orthopaedic Institute For Children
  Los Angeles, California, United States Recruiting
• Stanford University
  Palo Alto, California, United States Recruiting
• UC Davis Medical Center
  Sacramento, California, United States Recruiting
• University of California San Francisco
  San Francisco, California, United States Recruiting
• Yale University School of Medicine
  New Haven, Connecticut, United States Recruiting

Study contacts

• Study coordinator: Clinical Trials- Vega Therapeutics
• Email: trialtransparency@incyte.com
• Phone: 650-466-8041

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Endothelial colony-forming cell behavior in people with von Willebrand disease and acquired von Willebrand syndrome

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• Assessing HMB-002 for treating Von Willebrand Disease

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.