Topical TOR-582 Treatment of Epistaxis in HHT

Part of paid clinical trials in New York, New York.

Sponsor
Columbia University
Study ID
NCT07667413
Phase
PHASE1
Status
Not Yet Recruiting

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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

First listed
Jun 25, 2026
Start date
Oct 31, 2026
Status verified
Aug 2026
Primary completion
Jul 31, 2027
Completion
Dec 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

Locations (1)

FacilityCityStateZIPSite coordinators
Columbia University Irving Medical CenterNew YorkNew York10032
Jonathan B Overdevest, MD, PhD
[email protected]
(212) 305-6130
Jonathan B Overdevest, MD, PhD (PRINCIPAL_INVESTIGATOR)

Find similar trials in New York, NY

By research site
Columbia University Irving Medical Center· New York, NY

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