Phase 1b Study of TX002100 in Participants With Hematologic Support-Dependent Hereditary Hemorrhagic Telangiectasia (HHT)

NCT07863609 | Not Yet Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: TX002100-002
• Study Type: interventional
• Target: 20
• Locations: 0 countries
• Sites: N/A

Brief 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.

Conditions

Hereditary Hemorrhagic Telangiectasia (HHT)

Outcome Measures

Primary Outcomes (2)

• Incidence of adverse events (AEs), including serious adverse events (SAEs)

  Screening up to Day 225

• Incidence of clinically significant changes from baseline in safety laboratory assessments and other safety assessments

  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

  Baseline up to 6 months

• To assess hematologic impact score

  Measured at 6 months

• To assess hemoglobin change from baseline

  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

  Baselineup to 6 months

Study Arms (1)

TX002100

EXPERIMENTAL

Participants will receive subcutaneous (SC) injection of TX002100 administered weekly.

Biological: TX002100

Interventions

TX002100 BIOLOGICAL

Subcutaneous injection

TX002100

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Tectonic Operating Company, Inc.: lead

MeSH Terms

Conditions

Telangiectasia, Hereditary Hemorrhagic

Condition Hierarchy (Ancestors)

Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Telangiectasis; Hemorrhagic Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Vascular Malformations; Cardiovascular Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Clinical Trials

CONTACT

+1 339 337 4053; ClinicalTrials@tectonictx.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 2, 2026
• First Posted: October 7, 2026
• Study Start (Estimated): December 31, 2026
• Primary Completion (Estimated): April 30, 2028
• Study Completion (Estimated): July 30, 2028
• Last Updated: October 15, 2026

Record last verified: 2026-10