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New treatment option for Hereditary Hemorrhagic Telangiectasia (HHT)
Official title Topical TOR-582 Treatment of Epistaxis in HHT
ClinicalTrials.gov ID: NCT07667413
What this study is testing
What is Topical sirolimus ointment?
Topical sirolimus ointment is an investigational medicine, given as an once-daily treatment applied to the skin, being studied as a potential treatment for hereditary hemorrhagic telangiectasia (hht).
Also referred to as TOR-582.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Age 18 years or older at the time of consent.
- Confirmed diagnosis of HHT, defined as meeting at least three of the four Curaçao criteria or preferably by genetic testing.
- Moderate nasal epistaxis represented by an Epistaxis Severity Score (ESS) between 3 and 8 at screening, average NOSE-HHT score of 1.01-2, with a...
- Stable nasal hygiene regimen and epistaxis-related medical management for at least 3 months prior to enrollment.
- Stable epistaxis pattern for at least 3 months prior to enrollment
You likely can't join if
- Any medical contraindication to systemic sirolimus use.
- Prior use of any mTOR inhibitor within the past 3 months.
- Endoscopic evaluation of nasal cavity (HES-based)
- Site: No numerical site exclusion
- Pattern: AVM-type vascular pattern (HES pattern score = 2).
- Crusting: Moderate to severe nasal crusting (HES crusting score ≥ 2).
See the full eligibility criteria
- Age 18 years or older at the time of consent.
- Confirmed diagnosis of HHT, defined as meeting at least three of the four Curaçao criteria or preferably by genetic testing.
- Moderate nasal epistaxis represented by an Epistaxis Severity Score (ESS) between 3 and 8 at screening, average NOSE-HHT score of 1.01-2, with a self-reported history of at least four spontaneous nosebleeds per week and...
- Stable nasal hygiene regimen and epistaxis-related medical management for at least 3 months prior to enrollment.
- Stable epistaxis pattern for at least 3 months prior to enrollment
- Adequate bone marrow function defined as:
- Platelet count ≥ 100 × 10⁹/L (≥ 100,000/mm3)
- WBC count ≥ 2.5 × 10⁹/L at screening (≥ 2,500/mm3)
- Hgb ≥ 6g/dL with no transfusion in the prior 2 months
- INR ≤ 1.4 and activated partial thromboplastin time (aPTT) within institutional normal limits.
- Willingness to avoid initiation of other investigational or targeted therapeutic agents for epistaxis (including antiangiogenic or mTOR-modulating therapies) from the time of enrollment through study completion.
- Female participants of childbearing potential must have a negative pregnancy test at screening and agree to use effective contraception during the study and for 28 days following the final dose.
- Ability to comply with study procedures and follow-up visits, and capacity to provide written informed consent.
- Any medical contraindication to systemic sirolimus use.
- Prior use of any mTOR inhibitor within the past 3 months.
- Endoscopic evaluation of nasal cavity (HES-based)
- Site: No numerical site exclusion
- Pattern: AVM-type vascular pattern (HES pattern score = 2).
- Crusting: Moderate to severe nasal crusting (HES crusting score ≥ 2).
- Location: Telangiectasias isolated to the middle or posterior turbinates (HES location score ≥ 2).
- Perforation: Presence of a nasal septal perforation
- Surgical cautery or sclerotherapy within the past 3 months prior to enrollment
- Vascular embolization of nasal vasculature within the past 3 months prior to enrollment
- Clinically significant peripheral vascular disease or circulatory compromise.
- Current use of strong CYP3A4 modulators, including inhibitors (e.g., ketoconazole, clarithromycin) or inducers (e.g., rifampin, phenytoin, carbamazepine, St. John's wort).
- Use of anti-angiogenic therapies within 30 days prior to screening (e.g., bevacizumab, pazopanib, thalidomide, lenalidomide).
- Use of illicit substances within the past 30 days, excluding marijuana.
- Use of anticoagulant, antiplatelet, or fibrinolytic medications within the past 30 days, except for low-dose aspirin (81 mg or less).
- Use of octreotide or systemic estrogen therapy within the past 30 days.
- Clinical laboratory evaluation:
- Renal dysfunction, defined as a serum creatinine level greater than 2.0 mg/dL.
- Hepatic impairment, indicated by total bilirubin above 2.0 mg/dL (or above 4.0 mg/dL in patients with a known diagnosis of Gilbert's syndrome) or liver transaminases exceeding three times the upper limit of normal.
- Known SMAD4 mutation with a history of significant gastrointestinal polyposis, unless colonoscopy within the past 18 months demonstrated either no polyps or ≤5 polyps judged to be clinically insignificant by a...
- History of unprovoked venous thromboembolism, confirmed by imaging.
- Diagnosis of peripheral neuropathy as confirmed by neurologic evaluation.
- Documented hyperproliferative anemia (myelodysplastic syndrome, aplastic anemia, etc.). a. Current pregnancy or planned pregnancy within the next 6 months, or active breastfeeding.
- Concurrent participation in another treatment research study.
- Any condition, circumstance, language, or literacy limitation, that in the judgment of the investigator, would interfere with study participation or completion.
The study team makes the final eligibility decision.
Where it's taking place
- New York, New York, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include New York, New York, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.