NCT04404881

Bevacizumab In Hereditary Hemorrhagic Telangiectasia

A Phase 2 Study of Bevacizumab for Chronic Bleeding and Iron Deficiency Anemia in Hereditary Hemorrhagic Telangiectasia (Trial Using Systemic Bevacizumab to Treat HHT, TrUST-HHT)

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 33 (actual)
• Sponsor: Hanny Al-Samkari, MD · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This research study is studying to see whether bevacizumab may treat chronic bleeding and iron deficiency anemia in Hereditary Hemorrhagic Telangiectasia (HHT). Hereditary Hemorrhagic Telangiectasia (HHT) is a disorder that causes abnormal blood vessel formation. In HHT, there is a mutation in the TGF-β pathway, which results in an increase of vascular endothelial growth factor (VEGF) levels. An increase in VEGF levels can result in poorly formed blood vessels that have a higher rate of bleeding than normal blood vessels. Bevacizumab is designed to block VEGF activity. It is believed that targeting increased VEGF levels may be able to treat HHT. This research study involves the following study drug: \- Bevacizumab

Detailed description

In this research study, the investigators are studying the study drug, bevacizumab. Researchers would like to see whether bevacizumab effectively treats Hereditary Hemorrhagic Telangiectasia (HHT) and what side effects occur. \- The research study procedures include: screening for eligibility, pretreatment period, study treatment, end-of-study visit, and follow-up visit. * Participants will be observed for 12 weeks and then receive study treatment for 24 weeks and will be followed for 30 days after ending study treatment. * This research study involves the following study drug: Bevacizumab * It is expected that about 33 people will take part in this research study. This research study is a Phase II clinical trial. Phase II clinical trials test the safety and effectiveness of an investigational drug to learn whether the drug works in treating Hereditary Hemorrhagic Telangiectasia (HHT). "Investigational" means that the drug is being studied. The U.S. Food and Drug Administration (FDA) has not approved bevacizumab for Hereditary Hemorrhagic Telangiectasia (HHT), but it has been approved for other uses.

Conditions

• Hereditary Hemorrhagic Telangiectasia

Interventions

Type	Name	Description
DRUG	Bevacizumab (Avastin®)	* Induction Period (first 3 months of bevacizumab treatment): \-- Bevacizumab: once every 2 weeks via intravenous infusion for up to 12 weeks. * Maintenance Period (second 3 months of bevacizumab treatment): * Bevacizumab: once every 4 weeks via intravenous infusion for up to 12 weeks.

Timeline

• Start date: 2020-11-23
• Primary completion: 2026-04-01
• Completion: 2027-02-01
• First posted: 2020-05-28
• Last updated: 2026-03-27

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study