What side effects are associated with this type of intervention?

Common treatments for Osler-Weber-Rendu Syndrome (HHT) include anti-angiogenic agents like bevacizumab, immunosuppressants such as sirolimus, and other supportive therapies. The known side effects of these treatments can vary. Bevacizumab may cause hypertension, proteinuria, and increased risk of bleeding. Sirolimus is generally well-tolerated but can lead to side effects such as mouth ulcers, infections, and hyperlipidemia. Supportive therapies, including iron supplementation and blood transfusions, are generally safe but can have minor side effects like gastrointestinal discomfort and allergic reactions.

What prior FDA approvals exist?

The provided context does not mention any specific FDA approvals or related drugs for the treatment of Osler-Weber-Rendu Syndrome (HHT). The context includes information about various treatments and studies for other conditions, but none directly related to HHT or similar to the VAD044 trial. Therefore, it is not possible to provide a detailed summary of FDA-approved treatments for HHT based on the given information.

What other types of research exist?

Promising novel alternatives for treating Osler-Weber-Rendu Syndrome (HHT) in 2024 include therapies targeting the underlying vascular abnormalities. These may involve anti-angiogenic agents, such as bevacizumab, which has shown efficacy in reducing epistaxis and gastrointestinal bleeding. Additionally, thalidomide and its analogs, which inhibit angiogenesis, are being explored for their potential benefits in HHT. Gene therapy approaches targeting the defective genes (ENG, ACVRL1) responsible for HHT are also under investigation and hold promise for future treatment options.

← Back to Search

Unknown

VAD044 for Hereditary Hemorrhagic Telangiectasia

Phase 1

Recruiting

Research Sponsored by Vaderis Therapeutics AG

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Several epistaxis per week

Anemia

Must not have

Active uncontrolled infection or known to be serologically positive for HIV, Hep B, Hep C infection

Type 1 diabetes or uncontrolled type II diabetes (insulin or non-insulin dependent)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 weeks

Summary

This trial is testing a new medication called VAD044 to see if it can help adults with Hereditary Hemorrhagic Telangiectasia (HHT). The study will compare two different doses of VAD044 to check its safety and effectiveness. Participants will be assigned to one of the groups, and neither they nor the researchers will know which treatment they are receiving.

Who is the study for?

This trial is for adults with Hereditary Hemorrhagic Telangiectasia (HHT) who experience several nosebleeds per week and have anemia. Participants must be vaccinated against or have antibodies for COVID-19. Those on blood thinners, recent anti-angiogenic drugs, with certain diabetes conditions, active infections including COVID-19, or recent nasal procedures cannot join.

What is being tested?

The study tests the safety and effectiveness of two doses of a new medication called VAD044 in HHT patients. It's a Phase 1b trial where participants are randomly assigned to receive either VAD044 or a placebo without knowing which one they get.

What are the potential side effects?

As this is an early-stage trial for VAD044, specific side effects are being studied but may include typical drug reactions such as headaches, nausea, allergic reactions or other unforeseen issues related to the medication.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I experience nosebleeds several times a week.

Select...

I have anemia.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I do not have an active infection and am not HIV or hepatitis B/C positive.

Select...

I have type 1 diabetes or my type 2 diabetes is not under control.

Select...

I haven't taken any anti-angiogenic drugs in the last 8 weeks.

Select...

I currently have an active COVID-19 infection.

Select...

I need medication to prevent blood clots.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~12 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Safety and Tolerability

Secondary study objectives

Change in Epistaxis Frequency

Change in the Nasal Outcome for Epistaxis in Hereditary Hemorrhagic Telangiectasia score

Pharmacokinetics (PK) of VAD044

+1 more

Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: 40 mgExperimental Treatment1 Intervention

40 mg VAD044

Group II: 30 mgExperimental Treatment1 Intervention

30 mg VAD044

Group III: PlaceboPlacebo Group1 Intervention

Placebo

0 / 7Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Osler-Weber-Rendu Syndrome treatments often include anti-angiogenic agents to inhibit new blood vessel formation and reduce bleeding, antifibrinolytic agents to stabilize blood clots, and supportive treatments like iron supplements and blood transfusions to manage anemia. These treatments are crucial for managing symptoms and preventing complications in HHT patients. The trial VAD044 is likely exploring a new treatment targeting these pathways to enhance safety and efficacy.

Kasabach-Merritt syndrome.[Prostacyclin in the treatment of hemolytic-uremic syndrome: apropos of a case].[Our experience with sirolimus for the treatment of complicated vascular anomalies].