Hereditary Hemorrhagic Telangiectasia (HHT)PATH-HHT ATLASNCT07018401
Pomalidomide for the Treatment of Bleeding in Hereditary Hemorrhagic Telangiectasia Longitudinal Assessment Study
An observational study of Hereditary Hemorrhagic Telangiectasia (HHT), run by Massachusetts General Hospital at 11 sites in United States.
Trial summary
This is a multicenter U.S. longitudinal study evaluating patients with hereditary hemorrhagic telangiectasia who participated in the PATH-HHT clinical trial of pomalidomide for the treatment of HHT. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hereditary Hemorrhagic Telangiectasia (HHT)
- What is being tested?
- Pomalidomide
- Type of treatment
- Pill or oral medicine
- How many people
- 62 participants
- Where
- 11 sitesUnited States
- Study dates
- 1 May 2024to 1 Jun 2026
- Phase
- Observational
- Are there results?
- None posted in the registry
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Individuals with hereditary hemorrhagic telangiectasia and moderate-to-severe bleeding who previously enrolled in the PATH-HHT trial
Who may be able to join
- A clinical diagnosis of HHT as defined by the Curacao criteria
- Age > 18 years
- Platelet count ≥ 100 x 109/L prior to pomalidomide initiation
- WBC ≥ 2.5 x 109/L prior to pomalidomide initiation
- INR ≤ 1.4 and normal ± 2 sec activated partial thromboplastin time (aPTT) by local laboratory criteria (except for patients on a stable dose of warfarin or direct oral anticoagulants)
- Epistaxis severity score ≥ 3 measured over the preceding month
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- A requirement for anemia, as determined by local laboratory normal ranges, and/or parenteral infusion of at least 250 mg of iron or transfusion of 1 unit of blood over the 24 weeks preceding the screening visit
- All study participants must agree to be registered into the FDA mandated POMALYST REMS program, and be willing and able to comply with the requirements of the POMALYST REMS program
- Females of childbearing potential (FCBP)† must adhere to the pregnancy testing schedule mandated by the POMALYST REMS program
- Prior enrollment on PATH-HHT study (NCT03910244)
- A female of childbearing potential is a sexually mature woman who: 1) has not undergone a hysterectomy or bilateral oophorectomy; or 2) has not been naturally postmenopausal for at least 24 consecutive months (i.e., has had menses at any time in the preceding 24 consecutive months).
Who cannot join
- Women currently breast feeding or pregnant
- Renal insufficiency, serum creatinine > 2.0 mg/dl
- Hepatic insufficiency, bilirubin > 2.0 (or >4.0 in the setting of a prior clinical or genetic diagnosis of Gilbert's syndrome) or transaminases > 3.0x normal
- Prior treatment with thalidomide or other non-pomalidomide immunomodulatory imide drugs (IMiDs) within previous 6 months
- Prior treatment with bevacizumab (systemic or nasal) within previous 6 weeks
- Prior treatment with pazopanib within previous 6 weeks
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- The use of octreotide or estrogens within the previous month
- History of prior unprovoked thromboembolism confirmed by venous ultrasound or other imaging modalities
- Known peripheral neuropathy, confirmed by neurologic consultation
- Known underlying hypoproliferative anemia (i.e. myelodysplasia, aplastic anemia)
- Currently enrolled in other drug trials
- Known hypersensitivity to thalidomide or lenalidomide
- The development of erythema nodosum if characterized by a desquamating rash while taking thalidomide or similar drugs
- Known SMAD-4 mutation, unless there has been a colonoscopy with normal (negative) results, or in which the patient has had no more than 5 small (in the opinion of the gastroenterologist) colonic polyps completely removed within the preceding 18 months
- Anything that in the investigator's opinion is likely to interfere with completion of the study
Trial locations and contacts
11 sites in 1 country.
11 sites in 1 country
United States11 sites
Johns Hopkins University
Baltimore, Maryland, 21218
Massachusetts General Hospital
Boston, Massachusetts, 02114
University of North Carolina, Chapel HIll
Chapel Hill, North Carolina, 27599
Cleveland Clinic
Cleveland, Ohio, 44195
University of Florida
Gainesville, Florida, 32610
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Mayo Clinic
Rochester, Minnesota, 55905
University of Utah
Salt Lake City, Utah, 84112
University of California-San Diego
San Diego, California, 92093
University of California-San Francisco
San Francisco, California, 94143
About this study
Official title: Pomalidomide for the Treatment of Bleeding in Hereditary Hemorrhagic Telangiectasia After Trial Longitudinal Assessment Study (PATH-HHT ATLAS)
This is a multicenter U.S. longitudinal study evaluating patients with hereditary hemorrhagic telangiectasia who participated in the PATH-HHT clinical trial of pomalidomide for the treatment of HHT. This study is a longitudinal assessment of safety and effectiveness of pomalidomide in HHT in clinical trial participants following completion of the double-blind, placebo-controlled study.
Read the detailed description
Hereditary Hemorrhagic Telangiectasia (HHT) is an autosomal dominant condition characterized by disordered angiogenesis that affects 1 in 5,000 people. It results in numerous clinical complications including severe recurrent epistaxis, gastrointestinal bleeding, chronic iron deficiency anemia (and possible transfusion dependence), high-output cardiac failure, and many others. In recognition that elevated levels of vascular endothelial growth factor (VEGF) are elevated in HHT, anti-angiogenic drugs are now being used to treat HHT off-label to manage HHT-associated bleeding. A primary agent used for this purpose is pomalidomide, an oral immunomodulatory drug with antiangiogenic properties. Pomalidomide was demonstrated to be efficacious over a 6-month treatment period in the multicenter U.S. randomized controlled PATH-HHT Study. The present study is the successor to PATH-HHT, the PATH-HHT ATLAS (After Trial Longitudinal Assessment Study). This study will evaluate the long-term impact of pomalidomide on epistaxis (as measured by the validated ESS, epistaxis severity score), gastrointestinal bleeding, and iron deficiency anemia (as assessed by hemoglobin measurements, red blood cell transfusions, and intravenous iron infusions).
- Being tested
- Pomalidomide
- Keywords
- hereditary hemorrhagic telangiectasia, HHT, Osler-Weber-Rendu, pomalidomide, PATH-HHT, PATH-HHT ATLAS
- 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.
PomalidomideDrug
Also called Pomalyst
Oral pomalidomide up to 4 mg daily
What is being measured
The primary outcome is the main question the study is designed to answer.
Epistaxis Severity Score
Validated bleeding scale in HHT scored between 0-10, higher scores indicate worse bleeding
Measured: 12 months
Secondary outcomes (5)
Serum hemoglobin (g/dL)
Measured: 12 months
Hematologic Support Score
Composite hematologic endpoint, higher scores indicate more hematologic support requirements
Measured: 12 months
Intravenous iron infusion (mg elemental iron)
Measured: 12 months
Red cell transfusion (units of packed red blood cells)
Measured: 12 months
Incidence of treatment-emergent adverse events (safety)
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jun 2025
- Study start1 May 2024
- Primary completion8 Apr 2025
- Study completion1 Jun 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hereditary Hemorrhagic Telangiectasia (HHT)Studies of the same condition, open and under way.
- More trials run by Massachusetts General HospitalThe sponsor's other registered studies.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 62 participants (actual)
Sponsor and investigators
- Lead sponsor
- Massachusetts General Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- The Cleveland Clinic, University of North Carolina, Chapel Hill, University of California, San Francisco, Mayo Clinic, University of Utah, University of California, San Diego, University of Florida, Medical College of Wisconsin, Johns Hopkins University, University of Pennsylvania
- Responsible party
- Principal investigator, Hanny Al-Samkari, MD, Massachusetts General Hospital
- Investigators
- Ellen Zhang, MD, Study director, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07018401
- Sponsor's protocol ID
- 2022P000149
- Acronym
- PATH-HHT ATLAS
- Status last verified
- Jun 2025
- First submitted
- 28 May 2025
- Last updated
- 10 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Zhang E, Kasthuri RS, Parambil J, Prasad V, Iyer VN, Whitehead KJ, Hodges PG, Pishko AM, Conrad MB, Phelan D, Rodriguez-Lopez J, McCrae KR, Al-Samkari H. Pomalidomide for hereditary hemorrhagic telangiectasia: after trial longitudinal assessment study (PATH-HHT ATLAS). Blood Adv. 2026 Mar 10;10(5):1799-1808. doi: 10.1182/bloodadvances.2025018382.
PubMed 41512167
Study at a glance
From the registry record, last updated by the sponsor on 10 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pomalidomide for the Treatment of Bleeding in Hereditary Hemorrhagic Telangiectasia After Trial Longitudinal Assessment Study (PATH-HHT ATLAS)
- ClinicalTrials.gov ID
- NCT07018401
- Acronym / sponsor ID
- PATH-HHT ATLAS / 2022P000149
- Keywords
- hereditary hemorrhagic telangiectasia, HHT, Osler-Weber-Rendu, pomalidomide, PATH-HHT, PATH-HHT ATLAS
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 62 participants (actual)
- Lead sponsor
- Massachusetts General HospitalUniversity, hospital or other
- Collaborators
- The Cleveland Clinic, University of North Carolina, Chapel Hill, University of California, San Francisco, Mayo Clinic, University of Utah, University of California, San Diego, University of Florida, Medical College of Wisconsin, Johns Hopkins University, University of Pennsylvania
- Study start
- 1 May 2024 (actual)
- Primary completion
- 8 Apr 2025 (actual)
- Study completion
- 1 Jun 2026 (actual)
- First posted
- 12 Jun 2025 (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.
This is a multicenter U.S. longitudinal study evaluating patients with hereditary hemorrhagic telangiectasia who participated in the PATH-HHT clinical trial of pomalidomide for the treatment of HHT. This study is a longitudinal assessment of safety and effectiveness of pomalidomide in HHT in clinical trial participants following completion of the double-blind, placebo-controlled study.
Detailed description
Hereditary Hemorrhagic Telangiectasia (HHT) is an autosomal dominant condition characterized by disordered angiogenesis that affects 1 in 5,000 people. It results in numerous clinical complications including severe recurrent epistaxis, gastrointestinal bleeding, chronic iron deficiency anemia (and possible transfusion dependence), high-output cardiac failure, and many others. In recognition that elevated levels of vascular endothelial growth factor (VEGF) are elevated in HHT, anti-angiogenic drugs are now being used to treat HHT off-label to manage HHT-associated bleeding. A primary agent used for this purpose is pomalidomide, an oral immunomodulatory drug with antiangiogenic properties. Pomalidomide was demonstrated to be efficacious over a 6-month treatment period in the multicenter U.S. randomized controlled PATH-HHT Study. The present study is the successor to PATH-HHT, the PATH-HHT ATLAS (After Trial Longitudinal Assessment Study). This study will evaluate the long-term impact of pomalidomide on epistaxis (as measured by the validated ESS, epistaxis severity score), gastrointestinal bleeding, and iron deficiency anemia (as assessed by hemoglobin measurements, red blood cell transfusions, and intravenous iron infusions).
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Individuals with hereditary hemorrhagic telangiectasia and moderate-to-severe bleeding who previously enrolled in the PATH-HHT trial
Key inclusion criteria (11 listed)
- A clinical diagnosis of HHT as defined by the Curacao criteria
- Age > 18 years
- Platelet count ≥ 100 x 109/L prior to pomalidomide initiation
- WBC ≥ 2.5 x 109/L prior to pomalidomide initiation
- INR ≤ 1.4 and normal ± 2 sec activated partial thromboplastin time (aPTT) by local laboratory criteria (except for patients on a stable dose of warfarin or direct oral anticoagulants)
Key exclusion criteria (15 listed)
- Women currently breast feeding or pregnant
- Renal insufficiency, serum creatinine > 2.0 mg/dl
- Hepatic insufficiency, bilirubin > 2.0 (or >4.0 in the setting of a prior clinical or genetic diagnosis of Gilbert's syndrome) or transaminases > 3.0x normal
- Prior treatment with thalidomide or other non-pomalidomide immunomodulatory imide drugs (IMiDs) within previous 6 months
- Prior treatment with bevacizumab (systemic or nasal) within previous 6 weeks
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 |
|---|---|---|
| PomalidomideAlso: PomalystOral pomalidomide up to 4 mg daily | Drug | – |
Primary outcome
Epistaxis Severity Score
Time frame: 12 months
Validated bleeding scale in HHT scored between 0-10, higher scores indicate worse bleeding
5 secondary outcomes
Serum hemoglobin (g/dL)
Time frame: 12 months
Hematologic Support Score
Time frame: 12 months
Intravenous iron infusion (mg elemental iron)
Time frame: 12 months
Red cell transfusion (units of packed red blood cells)
Time frame: 12 months
Incidence of treatment-emergent adverse events (safety)
Time frame: 12 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jul 2026.
- Derived from this study: PubMed 41512167Zhang E, Kasthuri RS, Parambil J, Prasad V, Iyer VN, Whitehead KJ, Hodges PG, Pishko AM, Conrad MB, Phelan D, Rodriguez-Lopez J, McCrae KR, Al-Samkari H. Pomalidomide for hereditary hemorrhagic telangiectasia: after trial longitudinal assessment study (PATH-HHT ATLAS). Blood Adv. 2026 Mar 10;10(5):1799-1808. doi: 10.1182/bloodadvances.2025018382.
- Individual participant dataSharing planned: Undecided
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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. This study is not recruiting at the moment.
Ellen Zhang, MD study director
Stanford University
11 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hereditary Hemorrhagic Telangiectasia (HHT), run by Massachusetts General Hospital at 11 sites in United States.
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 11 inclusion and 15 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: finished, as of the sponsor's last update on 10 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Epistaxis Severity Score (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT07018401, which also lists 1 linked publication.
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 10 Jul 2026.