VAD044 for Hereditary Hemorrhagic Telangiectasia

Not currently recruiting at 9 trial locations

Overseen By Damien Picard

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Vaderis Therapeutics AG

Must not be taking: Anti-angiogenics, Anticoagulants

Disqualifiers: Diabetes, Active infections, others

Compensation: Varies

Number of Visits: Unknown

Duration: 12 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests VAD044 for individuals with Hereditary Hemorrhagic Telangiectasia (HHT), a condition that causes frequent nosebleeds and anemia (low red blood cell count). The first part aims to determine if VAD044, an experimental treatment, is safe and effective in controlling symptoms. The second part will assess its safety and effectiveness over a longer period. Suitable candidates have HHT, experience several nosebleeds weekly, and have anemia. As a Phase 1, Phase 2 trial, this research focuses on understanding how VAD044 works in people and measuring its effectiveness in an initial, smaller group.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot participate if you have used drugs with anti-angiogenic properties in the past 8 weeks or if you require therapeutic anticoagulation.

Is there any evidence suggesting that VAD044 is likely to be safe for humans?

Research has shown that VAD044, a new treatment for Hereditary Hemorrhagic Telangiectasia (HHT), was tested for safety in earlier studies. These studies focused on two doses: 30 mg and 40 mg. The findings indicated that patients generally tolerated VAD044 well. Some participants experienced side effects, but these were mostly mild, and no serious safety issues were reported. This suggests that VAD044 might be safe for humans, but discussing any concerns with a doctor before joining a trial is always advisable. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for Hereditary Hemorrhagic Telangiectasia, which typically focus on managing symptoms, VAD044 offers a novel approach by targeting the underlying mechanisms of blood vessel formation. Researchers are excited about this treatment because it has the potential to more effectively reduce the frequency and severity of bleeding episodes. VAD044 is being explored in both 30 mg and 40 mg doses, providing flexibility in dosing that could optimize its effectiveness. This new drug could represent a significant advancement in treating a condition that currently has limited therapeutic options.

What evidence suggests that VAD044 might be an effective treatment for Hereditary Hemorrhagic Telangiectasia?

Research shows that VAD044 is undergoing testing for its effectiveness in treating hereditary hemorrhagic telangiectasia (HHT), a condition that causes abnormal blood vessel formation. Studies have found that VAD044 affects a protein involved in controlling cell growth and survival, potentially improving HHT symptoms. Early results suggest that VAD044 is safe, with side effects similar to those of a placebo, and any drug-related effects were mostly mild and resolved independently. The data so far are promising, but further research is needed to confirm its efficacy. ^{1 2 3 4 6}

Who Is on the Research Team?

Damien Picard

Principal Investigator

Vaderis Therapeutics AG

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

I experience nosebleeds several times a week.

I have anemia.

You have been diagnosed with HHT using the Curaçao criteria.

Exclusion Criteria

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

Abnormal test results from blood or other lab tests.

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

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

8 weeks

Treatment

Participants are randomized to receive either 30mg VAD044, 40mg VAD044, or placebo once daily

12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants may opt into continuation of treatment with VAD044 for long-term safety and efficacy assessment

12 months

What Are the Treatments Tested in This Trial?

Interventions

• VAD044

Trial Overview 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.

How Is the Trial Designed?

Group I: 40 mg Experimental Treatment 2 Interventions

40 mg VAD044

Group II: 30 mg Experimental Treatment 2 Interventions

30 mg VAD044

Group III: Placebo Placebo Group 1 Intervention

Placebo

Who Is Running the Clinical Trial?

Vaderis Therapeutics AG

Lead Sponsor

Trials

1

Recruited

80+

Citations

1. ashpublications.org ashpublications.org/blood/article/144/Supplement%201/553/531132/A-Randomized-Placebo-Controlled-Multicenter-Proof

A Randomized, Placebo-Controlled, Multicenter Proof-of ... ... Efficacy of the Novel Allosteric AKT Inhibitor, VAD044, in Adults with Hereditary Hemorrhagic Telangiectasia (HHT) Available. Hanny Al ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT05406362

Assess Safety and Efficacy of VAD044 in HHT Patients A randomised, placebo controlled, double blind, multicentre proof of concept study to assess the safety and efficacy of two doses of VAD044 in patients with ...

3. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0006497124033007

A Randomized, Placebo-Controlled, Multicenter Proof-of- ... We report primary results from a POC study (NCT05406362) of VAD044 in adults with HHT. Methods: This randomized, placebo-controlled study was conducted in the ...

4. jadpro.com jadpro.com/videos/meeting-highlights/2024-ash-annual-meeting-exposition/assessing-the-safety-and-efficacy-of-the-novel-allosteric-akt-inhibitor-vad044-in-hereditary-hemorrhagic-telangiectasia/

Assessing the Safety and Efficacy of the Novel Allosteric ... Results showed that safety and tolerability of VAD044 was similar to placebo except for drug class effects, which were mostly mild and resolved ...

5. pharmacytimes.com pharmacytimes.com/view/fda-grants-fast-track-designation-to-vad044-for-treatment-of-adults-with-hht

FDA Grants Fast Track Designation to VAD044 for ... Clinical trial analysis results studying VAD-44 in patients with hereditary hemorrhagic telangiectasia (HTT) will be presented at the 2024 ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10543799/

Executive summary of the 14th HHT international scientific ... Hereditary Hemorrhagic Telangiectasia (HHT) is an autosomal dominant vascular disorder characterized by small, dilated clustered vessels ...