Hereditary Hemorrhagic Telangiectasia (HHT)Phase 1NCT07255846
A Trial of TER-1754 in Patients With Hereditary Hemorrhagic Telangiectasia
A Phase 1 study testing TER-1754 in people with Hereditary Hemorrhagic Telangiectasia (HHT), run by Terremoto Biosciences Inc. at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hereditary Hemorrhagic Telangiectasia (HHT)
- What is being tested?
- TER-1754
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 15 Dec 2025to Mar 2028
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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 locations and contacts
1 site in 1 country.
United States1 site
Innovative Hematology, Inc.
Indianapolis, Indiana, 46260
About this study
Official title: 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.
Read the detailed 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.
- Being tested
- TER-1754, Placebo
- Keywords
- Hereditary hemorrhagic telangiectasia, ENG, ACVRL1/ALK-1 alterations, Osler-Weber-Rendu, HHT, Anemia, Nosebleed, Epistaxis, AVM, Arteriovenous Malformation
- Medical subject headings
- Telangiectasia, Hereditary Hemorrhagic, Anemia, Epistaxis, Arteriovenous Malformations
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TER-1754Drug
QD or BID, orally in 28-day cycles
PlaceboDrug
Number of tablets will be confirmed post Phase 1a
TER-1754Drug
Phase 1b dose to be determined post Phase 1a
| Study group | Role | What they receive |
|---|---|---|
| Phase 1a (Dose Escalation) TER-1754 dose escalation | Experimental | TER-1754 Oral tablets Receives: Drug: TER-1754 |
| Phase 1b (Proof of Concept) | Placebo comparator | Placebo oral tablets Receives: Drug: Placebo |
| Phase 1b (Proof of Concept) - Phase 1b TER-1754 lower dose to begin post determination in Phase 1a | Experimental | Patient will receive one of the two doses determined post Phase 1a Receives: Drug: TER-1754 |
| Phase 1b (Proof of Concept) - Phase 1b TER-1754 higher dose to begin post determination in Phase 1a. | Experimental | Patient will receive one of the two doses determined post Phase 1a Receives: Drug: TER-1754 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Phase 1a: Safety and Tolerability of TER-1754
Incidence of adverse events characterized by type, seriousness, relationship to study treatment, and severity according to the National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE) v5.0
Measured: up to 48 weeks
Phase 1a: Maximum Tolerated Dose (MTD) and/or Maximum Administrated Dose (MAD) of TER-1754
Incidence dose-limiting toxicities (DLTs) characterized by type, seriousness, and severity according to NCI CTCAE v5.0 (dose escalation only).
Measured: 28 days
Phase 1b - Evaluate the Change from baseline in epistaxis and symptom-related clinical activity scores at Week 24
Assess change from baseline in epistaxis duration
Measured: 48 weeks
Secondary outcomes (11)
Phase 1a and 1b: Evaluate TER-1754 Peak Plasma Concentration (Cmax)
Cmax of TER-1754 following single and multiple dosing.
Measured: up to 48 weeks
Phase 1a and 1b: Evaluate change from baseline in Daily Epistaxis (EQ) Questionnaire
Change from baseline in EQ Questionnaire based on patient self-reported epistaxis frequency, severity and duration, where:
Epistaxis frequency: number of epistaxis events per week Epistaxis duration: total minutes of bleeding Epistaxis intensity - From mild to severe: Spotting, dripping, dripping quickly, steady stream, pouring, and gushing.
Measured: up to 48 weeks
Phase 1a and 1b: Evaluate HHT-specfic quality of life
Change from baseline in HHT-specific QoL (HHT-QoL), where The total score ranges from 0 (no limitations) to 16 (severe limitations).
Measured: Up to 48 weeks
Phase 1a and 1b: Evaluate TER-1754 Time to Peak Concentration (Tmax)
Time at which maximum observed plasma concentration occurs.
Measured: Up to 48 weeks
Phase 1a and 1b: Evaluate TER-1754 Area Under the Concentration-Time Curve (AUC0-last)
Area under the plasma concentration-time curve from time zero to last measurable concentration (AUC0-last)
Measured: Up to 48 weeks
Phase 1a and 1b: Evaluate TER-1754 AUC0-24
Area under the plasma concentration-time curve from time zero to 24 hours (AUC0-24)
Measured: Up to 48 weeks
Phase 1a and 1b: Evaluate TER-1754 AUC∞
Area under the plasma concentration versus time curve extrapolated to infinity (AUC∞) of TER-1754 following single and multiple dosing.
Measured: Up to 48 weeks
Phase 1a and 1b: Evaluate TER-1754 Terminal Half-Life (t½)
Terminal elimination half-life (t½) of TER-1754 calculated from the terminal slope of the plasma concentration versus time profile.
Measured: Up to 48 weeks
Phase 1a and 1b: Evaluate TER-1754 Mean Residence Time (MRT)
Mean residence time (MRT) of TER-1754.
Measured: Up to 48 weeks
Phase 1a and 1b: Evaluate TER-1754 Accumulation Ratio at Steady State
Accumulation ratio of TER-1754 at steady state, calculated as the ratio of exposure at steady state to exposure after the first dose.
Measured: Up to 48 weeks
Phase 1a and 1b: Change from baseline in Nasal Obstruction Symptom Evaluation Score for HHT (NOSE HHT)
Change from baseline in the NOSE HHT score, a validated scale measuring nasal obstruction in HHT. The score ranges from 0 to 4, with higher scores indicating more severe symptoms.
Measured: up to 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Dec 2025
- Study start15 Dec 2025
- Primary completionMar 2028 (estimated)
- Study completionMar 2028 (estimated)
Why it stopped: This decision is based solely on internal company prioritization and resource allocation considerations. No patients have been enrolled or dosed in this study and no clinical data have been generated.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary Hemorrhagic Telangiectasia (HHT)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- 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
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Terremoto Biosciences Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07255846
- Sponsor's protocol ID
- TER-1754-C01
- Status last verified
- Apr 2026
- First submitted
- 14 Nov 2025
- Last updated
- 13 Apr 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Trial of TER-1754 in Patients With Hereditary Hemorrhagic Telangiectasia
- ClinicalTrials.gov ID
- NCT07255846
- Acronym / sponsor ID
- TER-1754-C01
- Keywords
- Hereditary hemorrhagic telangiectasia, ENG, ACVRL1/ALK-1 alterations, Osler-Weber-Rendu, HHT, Anemia, Nosebleed, Epistaxis, AVM
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: This decision is based solely on internal company prioritization and resource allocation considerations. No patients have been enrolled or dosed in this study and no clinical data have been generated.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Terremoto Biosciences Inc.Industry (pharma, biotech, devices)
- Study start
- 15 Dec 2025 (actual)
- Primary completion
- Mar 2028 (estimated)
- Study completion
- Mar 2028 (estimated)
- First posted
- 1 Dec 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
- Model description
- 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
| Arm | Type | Receives |
|---|---|---|
| Phase 1a (Dose Escalation) TER-1754 dose escalation | Experimental | Drug: TER-1754TER-1754 Oral tablets |
| Phase 1b (Proof of Concept) | Placebo comparator | Drug: PlaceboPlacebo oral tablets |
| Phase 1b (Proof of Concept) - Phase 1b TER-1754 lower dose to begin post determination in Phase 1a | Experimental | Drug: TER-1754Patient will receive one of the two doses determined post Phase 1a |
| Phase 1b (Proof of Concept) - Phase 1b TER-1754 higher dose to begin post determination in Phase 1a. | Experimental | Drug: TER-1754Patient will receive one of the two doses determined post Phase 1a |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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.
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TER-1754QD or BID, orally in 28-day cycles | Drug | Phase 1a (Dose Escalation) TER-1754 dose escalation |
| PlaceboNumber of tablets will be confirmed post Phase 1a | Drug | Phase 1b (Proof of Concept) |
| TER-1754Phase 1b dose to be determined post Phase 1a | Drug | Phase 1b (Proof of Concept) - Phase 1b TER-1754 higher dose to begin post determination in Phase 1a., Phase 1b (Proof of Concept) - Phase 1b TER-1754 lower dose to begin post determination in Phase 1a |
Primary outcome
Phase 1a: Safety and Tolerability of TER-1754
Time frame: up to 48 weeks
Incidence of adverse events characterized by type, seriousness, relationship to study treatment, and severity according to the National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE) v5.0
Phase 1a: Maximum Tolerated Dose (MTD) and/or Maximum Administrated Dose (MAD) of TER-1754
Time frame: 28 days
Incidence dose-limiting toxicities (DLTs) characterized by type, seriousness, and severity according to NCI CTCAE v5.0 (dose escalation only).
Phase 1b - Evaluate the Change from baseline in epistaxis and symptom-related clinical activity scores at Week 24
Time frame: 48 weeks
Assess change from baseline in epistaxis duration
11 secondary outcomes
Phase 1a and 1b: Evaluate TER-1754 Peak Plasma Concentration (Cmax)
Time frame: up to 48 weeks
Phase 1a and 1b: Evaluate change from baseline in Daily Epistaxis (EQ) Questionnaire
Time frame: up to 48 weeks
Phase 1a and 1b: Evaluate HHT-specfic quality of life
Time frame: Up to 48 weeks
Phase 1a and 1b: Evaluate TER-1754 Time to Peak Concentration (Tmax)
Time frame: Up to 48 weeks
Phase 1a and 1b: Evaluate TER-1754 Area Under the Concentration-Time Curve (AUC0-last)
Time frame: Up to 48 weeks
Phase 1a and 1b: Evaluate TER-1754 AUC0-24
Time frame: Up to 48 weeks
Phase 1a and 1b: Evaluate TER-1754 AUC∞
Time frame: Up to 48 weeks
Phase 1a and 1b: Evaluate TER-1754 Terminal Half-Life (t½)
Time frame: Up to 48 weeks
Phase 1a and 1b: Evaluate TER-1754 Mean Residence Time (MRT)
Time frame: Up to 48 weeks
Phase 1a and 1b: Evaluate TER-1754 Accumulation Ratio at Steady State
Time frame: Up to 48 weeks
Phase 1a and 1b: Change from baseline in Nasal Obstruction Symptom Evaluation Score for HHT (NOSE HHT)
Time frame: up to 48 weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Apr 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing TER-1754 in people with Hereditary Hemorrhagic Telangiectasia (HHT), run by Terremoto Biosciences Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 13 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Phase 1a: Safety and Tolerability of TER-1754 (time frame: up to 48 weeks). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Apr 2026.