Topical TOR-582 Treatment of Epistaxis in HHT

Conditions

• Epistaxis
• Hereditary Hemorrhagic Telangiectasia (HHT)

Eligibility Criteria

Sex

ALL

Age

18 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• Topical sirolimus ointment — DRUG
  The active formulation of TOR-582 incorporates sirolimus at concentrations of 5 mg/mL (0.5%), 10 mg/mL (1.0%), and 20 mg/mL (2.0%) along with other active ingredients in an oil-based carrier ointment. Ointment will be applied intranasally twice daily in 0.2mL applications using TC5-R - Topi-CLICK Micro® 5 mL devices, which dispense metered 0.05 mL doses. Dose finding will begin at a dose of 10 mg/mL (1.0%). Doses will escalate sequentially across planned dose levels (Level 1: 5 mg/mL, Level 2: 10 mg/mL, Level 3: 20 mg/mL) as guided by the BOIN-AT design until the Maximum Tolerated Dose is identified.

Study Details

People with hereditary hemorrhagic telangiectasia (HHT) often experience frequent and severe nosebleeds that can disrupt daily life and lead to anemia, medical procedures, and reduced quality of life. This study is testing a new nasal ointment called TOR-582, which contains sirolimus, to determine whether it can be used safely when applied inside the nose. Adults with HHT and frequent nosebleeds will be invited to participate. Participants will first complete one week of observation without treatment, followed by up to 12 weeks of applying the study ointment inside each nostril twice daily. Different participants will receive different strengths of the ointment so researchers can identify the safest dose. During the study, participants will attend study visits, complete questionnaires about their nosebleeds and quality of life, keep a daily nosebleed diary, undergo nasal examinations, and have blood tests to monitor safety and medication levels. The information gained from this study will help determine whether this topical treatment can be safely studied further and will support the development of a new, less invasive option for managing nosebleeds in people with HHT.

Key Dates

Start date

Jul 31, 2026

Status verified

Jun 2026

Primary completion

Dec 31, 2026

Completion

May 31, 2027

Study Design

Enrollment

27 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Arms

• Experimental: Dose Level 1 - 5 mg/mL (0.5%)
• Experimental: Dose Level 2 - 10 mg/mL (1.0%)
• Experimental: Dose Level 3 - 20 mg/mL (2.0%)

Primary Outcome Measure

Incidence of dose-limiting toxicities (DLTs) [ Time Frame: From enrollment through the first 4 weeks of treatment for each cohort (the DLT evaluation window) ]

Central Contacts

• Jonathan B Overdevest, MD, PhD
  212-305-6130
  [email protected]

Locations (1)

Find similar trials in New York, NY

Related Studies

• Comprehensive HHT Outcomes Registry of the United States (CHORUS)
  Recruiting · Cure HHT · Birmingham, Alabama