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

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

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.

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.

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