Hereditary Hemorrhagic Telangiectasia (HHT)Phase 1NCT07863609
Phase 1b Study of TX002100 in Participants With Hematologic Support-Dependent Hereditary Hemorrhagic Telangiectasia (HHT)
A Phase 1 study testing TX002100 in people with Hereditary Hemorrhagic Telangiectasia (HHT), run by Tectonic Operating Company, Inc..
Trial summary
The primary purpose of this study is to assess the safety and tolerability of TX002100 in participants with hereditary hemorrhagic telangiectasia (HHT). The study will also look at whether TX002100 reduces the need for blood or iron treatments, and whether it improves blood counts, such as hemoglobin levels. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Hereditary Hemorrhagic Telangiectasia (HHT)
- What is being tested?
- TX002100
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- No sites listed yet
- Study dates
- 31 Dec 2026to 30 Jul 2028
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adults with HHT who need regular intravenous iron or blood transfusions.
- Females of childbearing potential and male participants must agree to comply with the contraception requirements.
- Able to understand and is willing and able to comply with the study requirements and study procedures and agrees to participate in the study by giving written informed consent.
Who cannot join
- History of anemia of any cause other than HHT-related bleeding, or any other clinically significant inherited bleeding disorder aside from HHT.
- Has undergone or is anticipated to undergo liver transplantation.
- Medical conditions or comorbidities that, in the opinion of the investigator, would interfere with study compliance or data interpretation; or, in the opinion of the Investigator, taking part in the study would jeopardize the safety of the patient.
- History of hemorrhage from untreated pulmonary arteriovenous malformations (AVMs) manifesting as hemoptysis or hemothorax within 12 weeks before screening.
- Receipt of any vaccination within 2 weeks prior to first dose.
- Recent major surgery (within 4 weeks before the screening visit) or planned major surgery during the study.
- GI bleeding due to non-HHT disease (e.g., GI ulcers, diverticulosis), in the opinion of the investigator, within 6 months before screening.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: An Open-label, Phase 1b Study to Assess the Safety, Tolerability, and Efficacy of TX002100 in Participants With Hematologic Support-Dependent Hereditary Hemorrhagic Telangiectasia (HHT)
The primary purpose of this study is to assess the safety and tolerability of TX002100 in participants with hereditary hemorrhagic telangiectasia (HHT). The study will also look at whether TX002100 reduces the need for blood or iron treatments, and whether it improves blood counts, such as hemoglobin levels.
- Being tested
- TX002100
- Medical subject headings
- Telangiectasia, Hereditary Hemorrhagic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TX002100Biological
Subcutaneous injection
| Study group | Role | What they receive |
|---|---|---|
| TX002100 | Experimental | Participants will receive subcutaneous (SC) injection of TX002100 administered weekly. Receives: Biological: TX002100 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse events (AEs), including serious adverse events (SAEs)
Measured: Screening up to Day 225
Incidence of clinically significant changes from baseline in safety laboratory assessments and other safety assessments
Measured: Baseline up to Day 225
Secondary outcomes (4)
To assess hematologic support score change from baseline calculated over the 12 weeks preceding the time point
Measured: Baseline up to 6 months
To assess hematologic impact score
Measured: Measured at 6 months
To assess hemoglobin change from baseline
Measured: Baseline up to 6 months
Change from baseline in the following calculated over the 12 weeks preceding the time point: - Total parenteral iron (mg) infused - Number of pRBCs transfused
Measured: Baselineup to 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Oct 2026
- Study start31 Dec 2026 (estimated)
- Primary completion30 Apr 2028 (estimated)
- Study completion30 Jul 2028 (estimated)
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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Tectonic Operating Company, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07863609
- Sponsor's protocol ID
- TX002100-002
- Status last verified
- Oct 2026
- First submitted
- 2 Oct 2026
- Last updated
- 15 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Phase 1b Study to Assess the Safety, Tolerability, and Efficacy of TX002100 in Participants With Hematologic Support-Dependent Hereditary Hemorrhagic Telangiectasia (HHT)
- ClinicalTrials.gov ID
- NCT07863609
- Acronym / sponsor ID
- TX002100-002
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Tectonic Operating Company, Inc.Industry (pharma, biotech, devices)
- Study start
- 31 Dec 2026 (estimated)
- Primary completion
- 30 Apr 2028 (estimated)
- Study completion
- 30 Jul 2028 (estimated)
- First posted
- 7 Oct 2026 (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.
The primary purpose of this study is to assess the safety and tolerability of TX002100 in participants with hereditary hemorrhagic telangiectasia (HHT). The study will also look at whether TX002100 reduces the need for blood or iron treatments, and whether it improves blood counts, such as hemoglobin levels.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| TX002100 | Experimental | Biological: TX002100Participants will receive subcutaneous (SC) injection of TX002100 administered weekly. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Adults with HHT who need regular intravenous iron or blood transfusions.
- Females of childbearing potential and male participants must agree to comply with the contraception requirements.
- Able to understand and is willing and able to comply with the study requirements and study procedures and agrees to participate in the study by giving written informed consent.
Key exclusion criteria (7 listed)
- History of anemia of any cause other than HHT-related bleeding, or any other clinically significant inherited bleeding disorder aside from HHT.
- Has undergone or is anticipated to undergo liver transplantation.
- Medical conditions or comorbidities that, in the opinion of the investigator, would interfere with study compliance or data interpretation; or, in the opinion of the Investigator, taking part in the study would jeopardize the safety of the patient.
- History of hemorrhage from untreated pulmonary arteriovenous malformations (AVMs) manifesting as hemoptysis or hemothorax within 12 weeks before screening.
- Receipt of any vaccination within 2 weeks prior to first dose.
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 |
|---|---|---|
| TX002100Subcutaneous injection | Biological | TX002100 |
Primary outcome
Incidence of adverse events (AEs), including serious adverse events (SAEs)
Time frame: Screening up to Day 225
Incidence of clinically significant changes from baseline in safety laboratory assessments and other safety assessments
Time frame: Baseline up to Day 225
4 secondary outcomes
To assess hematologic support score change from baseline calculated over the 12 weeks preceding the time point
Time frame: Baseline up to 6 months
To assess hematologic impact score
Time frame: Measured at 6 months
To assess hemoglobin change from baseline
Time frame: Baseline up to 6 months
Change from baseline in the following calculated over the 12 weeks preceding the time point: - Total parenteral iron (mg) infused - Number of pRBCs transfused
Time frame: Baselineup to 6 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Oct 2026.
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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.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing TX002100 in people with Hereditary Hemorrhagic Telangiectasia (HHT), run by Tectonic Operating Company, Inc..
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 3 inclusion and 7 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: opening soon, as of the sponsor's last update on 15 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Clinical Trials (+1 339 337 4053, ClinicalTrials@tectonictx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of adverse events (AEs), including serious adverse events (SAEs) (time frame: Screening up to Day 225). The study lists 2 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 15 Oct 2026.