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

Terremoto Biosciences Inc.

Status and phase: Enrolling; Phase 1

Conditions: Hereditary Hemorrhagic Telangiectasia (HHT)

Treatments: Drug: TER-1754; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT07255846

TER-1754-C01

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 90 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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90 participants in 4 patient groups, including a placebo group

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

Experimental group

Description:

TER-1754 Oral tablets

Treatment:

Drug: TER-1754

Drug: TER-1754

Phase 1b (Proof of Concept)

Placebo Comparator group

Description:

Placebo oral tablets

Treatment:

Drug: Placebo

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

Experimental group

Description:

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

Treatment:

Drug: TER-1754

Drug: TER-1754

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

Experimental group

Description:

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

Treatment:

Drug: TER-1754

Drug: TER-1754