A Trial of TER-1754 in Patients With Hereditary Hemorrhagic Telangiectasia

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1

Total Enrollment

90 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-12-15

Study Completion Date

2028-03-31

Brief Summary

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This is a Phase 1a/1b, multicenter study evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and clinical activity of TER-1754 (a novel AKT1 inhibitor) in patients with HHT.

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Detailed Description

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This is a first-in-human, Phase 1, multicenter trial that includes two parts:

* Phase 1a (dose escalation) will evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity of TER-1754 in patients with HHT and determine the maximum tolerated or administered dose.
* Phase 1b (proof of concept) will evaluate clinical activity and further characterize the safety profile of TER-1754 in patients with HHT. The Phase 1b treatment period is separated into a blinded treatment segment followed by an open-label extension (OLE) segment.

Conditions

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Hereditary Hemorrhagic Telangiectasia (HHT)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Phase 1a is an open-label dose-escalation trial. Phase 1b is a 3-arm randomized placebo-controlled design. The Phase 1b treatment period is separated into a blinded treatment segment followed by an open-label extension (OLE) segment

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Phase 1a is open label, dose escalation. The Phase 1b treatment period is separated into a blinded treatment segment followed by an open-label extension (OLE) segment

Study Groups

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Phase 1a (Dose Escalation) TER-1754 dose escalation

TER-1754 Oral tablets

Group Type EXPERIMENTAL

TER-1754

Intervention Type DRUG

QD or BID, orally in 28-day cycles

Phase 1b (Proof of Concept)

Placebo oral tablets

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Number of tablets will be confirmed post Phase 1a

Phase 1b (Proof of Concept) - Phase 1b TER-1754 lower dose to begin post determination in Phase 1a

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

Group Type EXPERIMENTAL

TER-1754

Intervention Type DRUG

Phase 1b dose to be determined post Phase 1a

Phase 1b (Proof of Concept) - Phase 1b TER-1754 higher dose to begin post determination in Phase 1a.

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

Group Type EXPERIMENTAL

TER-1754

Intervention Type DRUG

Phase 1b dose to be determined post Phase 1a

Interventions

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TER-1754

QD or BID, orally in 28-day cycles

Intervention Type DRUG

Placebo

Number of tablets will be confirmed post Phase 1a

Intervention Type DRUG

TER-1754

Phase 1b dose to be determined post Phase 1a

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Able to provide a signed and dated written informed consent prior to any study-specific procedures, sampling, or data collection.
* A clinical diagnosis of HHT as defined by the Curaçao criteria
* Baseline (1-month) ESS ≥ 4
* ECOG ≤ 2
* Anemia or parental iron infusion of at least 500 mg or transfusion of at least 2 units of RBCs within the preceding 24 weeks.
* Adequate bone marrow function
* Adequate renal function
* Adequate hepatic function

Exclusion Criteria

* Prior nonresponse or loss of response to an agent that inhibits AKT1 and/or AKT2 as the primary mechanism of action.
* Diagnosis of DM requiring insulin treatment
* Known significant bleeding sources other than nasal, GI, or menstrual/ uterine
* Known underlying hypoproliferative anemia or clinically significant hemolytic anemia

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No