TER-1754 for Hereditary Hemorrhagic Telangiectasia

Overseen By Terremoto Bioscience

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Terremoto Biosciences Inc.

Disqualifiers: Diabetes, Significant bleeding, Anemia, others

Compensation: Varies

Number of Visits: Unknown

90 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test the safety and effectiveness of a new drug, TER-1754, for people with Hereditary Hemorrhagic Telangiectasia (HHT), a condition that causes abnormal blood vessels. Researchers seek to understand how this medication, which blocks a specific protein (AKT1) believed to play a role in the disease, affects patients. The study includes several parts, such as testing different doses and comparing the drug to a placebo (a harmless pill with no active medication). People with HHT who have been dealing with anemia (low red blood cells) requiring treatment might be suitable for this trial. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new medication.

Is there any evidence suggesting that TER-1754 is likely to be safe for humans?

Research has shown that TER-1754 is being tested for safety and tolerance in people with Hereditary Hemorrhagic Telangiectasia (HHT). This marks the first human trial, with a primary focus on safety. Detailed information on side effects from earlier studies is unavailable because testing remains in the early stages.

As a Phase 1 trial, the main goal is to assess how well participants tolerate the treatment. The trial tests different doses to identify the safest one. Although no existing data on TER-1754's tolerance is available, Phase 1 trials are carefully designed to monitor safety closely. Participants are observed for side effects, and doses are adjusted to maintain a safe balance.

For those considering participation, the staff will closely monitor health and make necessary adjustments to ensure safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike other treatments for Hereditary Hemorrhagic Telangiectasia (HHT), such as anti-angiogenic drugs and hormonal therapies, TER-1754 might bring something new to the table. This experimental treatment is administered orally, which could offer a more convenient option compared to injections or infusions. Researchers are particularly interested in TER-1754 because it could target the underlying mechanisms of HHT in a novel way, potentially leading to improved outcomes. If successful, TER-1754 might provide a more effective and patient-friendly approach to managing this condition.

What evidence suggests that TER-1754 might be an effective treatment for HHT?

Research shows that TER-1754 is a new medicine targeting a protein involved in cell growth and blood vessel formation. This is crucial for treating Hereditary Hemorrhagic Telangiectasia (HHT), a condition where unusual blood vessels cause bleeding. Early findings suggest that inhibiting this protein could help reduce bleeding. Although extensive data on TER-1754's effectiveness is not yet available, similar treatments have shown promise in reducing bleeding in HHT patients. This raises hope that TER-1754 might also be effective. Participants in this trial will receive either a placebo or one of the two doses of TER-1754 determined in the dose escalation phase. ^{1 2 4 5 6}

Are You a Good Fit for This Trial?

This trial is for patients with Hereditary Hemorrhagic Telangiectasia (HHT) who have an ECOG score of 2 or less, indicating they can walk and care for themselves. They must have had anemia treatments recently, be able to consent to the study, and meet specific criteria for liver, kidney, and bone marrow function.

Inclusion Criteria

My kidney function is normal.

I can sign and date a consent form for study procedures.

My sleepiness score is 4 or higher.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Phase 1a (Dose Escalation)

Evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of TER-1754 and determine the maximum tolerated or administered dose

4 weeks

Phase 1b (Proof of Concept)

Evaluate clinical activity and further characterize the safety profile of TER-1754, including a blinded treatment segment

24 weeks

Open-label Extension

Participants may opt into continuation of treatment long-term in an open-label extension segment

Long-term

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• TER-1754

Trial Overview

The trial is testing TER-1754, a new potential drug that inhibits a protein called AKT1. It's in early stages (Phase 1a/1b), focusing on how safe it is and how the body responds to it compared to a placebo in people with HHT.

How Is the Trial Designed?

Group I: Phase 1b (Proof of Concept) - Phase 1b TER-1754 lower dose to begin post determination in Phase 1a Experimental Treatment 1 Intervention

Patient will receive one of the two doses determined post Phase 1a

Group II: Phase 1b (Proof of Concept) - Phase 1b TER-1754 higher dose to begin post determination in Phase 1a. Experimental Treatment 1 Intervention

Patient will receive one of the two doses determined post Phase 1a

Group III: Phase 1a (Dose Escalation) TER-1754 dose escalation Experimental Treatment 1 Intervention

TER-1754 Oral tablets

Group IV: Phase 1b (Proof of Concept) Placebo Group 1 Intervention

Placebo oral tablets

Who Is Running the Clinical Trial?

Terremoto Biosciences Inc.

Lead Sponsor

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT07255846

A Trial of TER-1754 in Patients With Hereditary ...

This is a Phase 1a/1b, multicenter study evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and clinical activity of TER- ...

2.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC12627177/

Executive summary of the 15th HHT international scientific ...

Dupuis-Girod (O5) reported the long-term outcome of 210 patients with HHT who were treated with bevacizumab from 2009 to 2023 with a median ...

3.

massgeneralbrigham.org

massgeneralbrigham.org/en/about/newsroom/press-releases/engasertib-promising-trial-results

Drug Developed for Inherited Bleeding Disorder Shows ...

A study led by Mass General Brigham researchers found that engasertib lessened bleeding events for patients with HHT, the second most common ...

4.

centerwatch.com

centerwatch.com/clinical-trials/listings/NCT07255846/a-trial-of-ter-1754-in-patients-with-hereditary-hemorrhagic-telangiectasia

A Trial of TER-1754 in Patients With Hereditary ...

This is a Phase 1a/1b, multicenter study evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and clinical activity of TER- ...

5.

curehht.org

curehht.org/cure-hht-leads-global-breakthrough-in-bleeding-standards-paving-the-way-for-future-hht-therapies/

Cure HHT Leads Global Breakthrough in Bleeding ...

These AVMs can cause life-threatening issues including stroke, brain abscess, heart failure, and internal hemorrhage. Despite its prevalence and ...

6.

adisinsight.springer.com

adisinsight.springer.com/trials/700388421

A Phase 1 Trial of TER-1754 in Patients With Hereditary ...

This is a Phase 1a/1b, multicenter study evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and clinical activity of ...