Pomalidomide for the Treatment of Bleeding in Hereditary Hemorrhagic Telangiectasia Longitudinal Assessment Study (PATH-HHT ATLAS)

September 22, 2025 updated by: Hanny Al-Samkari, MD, Massachusetts General Hospital

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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 Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
62

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • San Diego, California, United States, 92093
        • University of California-San Diego
      • San Francisco, California, United States, 94143
        • University of California-San Francisco
    • Florida
      • Gainesville, Florida, United States, 32610
        • University of Florida
    • Maryland
      • Baltimore, Maryland, United States, 21218
        • Johns Hopkins University
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Massachusetts General Hospital
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Mayo Clinic
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27599
        • University of North Carolina, Chapel Hill
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Cleveland Clinic
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • University of Pennsylvania
    • Utah
      • Salt Lake City, Utah, United States, 84112
        • University of Utah
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Medical College of Wisconsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Individuals with hereditary hemorrhagic telangiectasia and moderate-to-severe bleeding who previously enrolled in the PATH-HHT trial

Description

Inclusion Criteria:

  1. A clinical diagnosis of HHT as defined by the Curacao criteria
  2. Age > 18 years
  3. Platelet count ≥ 100 x 109/L prior to pomalidomide initiation
  4. WBC ≥ 2.5 x 109/L prior to pomalidomide initiation
  5. 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)
  6. Epistaxis severity score ≥ 3 measured over the preceding month
  7. 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
  8. 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
  9. Females of childbearing potential (FCBP)† must adhere to the pregnancy testing schedule mandated by the POMALYST REMS program

  10. 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).

Exclusion Criteria:

  1. Women currently breast feeding or pregnant
  2. Renal insufficiency, serum creatinine > 2.0 mg/dl
  3. 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
  4. Prior treatment with thalidomide or other non-pomalidomide immunomodulatory imide drugs (IMiDs) within previous 6 months
  5. Prior treatment with bevacizumab (systemic or nasal) within previous 6 weeks
  6. Prior treatment with pazopanib within previous 6 weeks
  7. The use of octreotide or estrogens within the previous month
  8. History of prior unprovoked thromboembolism confirmed by venous ultrasound or other imaging modalities
  9. Known peripheral neuropathy, confirmed by neurologic consultation
  10. Known underlying hypoproliferative anemia (i.e. myelodysplasia, aplastic anemia)
  11. Currently enrolled in other drug trials
  12. Known hypersensitivity to thalidomide or lenalidomide
  13. The development of erythema nodosum if characterized by a desquamating rash while taking thalidomide or similar drugs
  14. 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
  15. Anything that in the investigator's opinion is likely to interfere with completion of the study

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Epistaxis Severity Score
Time Frame: 12 months
Validated bleeding scale in HHT scored between 0-10, higher scores indicate worse bleeding
12 months

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Serum hemoglobin (g/dL)
Time Frame: 12 months
12 months
Hematologic Support Score
Time Frame: 12 months
Composite hematologic endpoint, higher scores indicate more hematologic support requirements
12 months
Intravenous iron infusion (mg elemental iron)
Time Frame: 12 months
12 months
Red cell transfusion (units of packed red blood cells)
Time Frame: 12 months
12 months
Incidence of treatment-emergent adverse events (safety)
Time Frame: 12 months
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Massachusetts General Hospital

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Johns Hopkins University
Mayo Clinic
University of Pennsylvania
University of California, San Diego
The Cleveland Clinic
University of Florida
Medical College of Wisconsin
University of California, San Francisco
University of North Carolina, Chapel Hill
University of Utah

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Ellen Zhang, MD, Stanford University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
May 1, 2024

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
April 8, 2025

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
June 1, 2026

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
May 28, 2025

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 4, 2025

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
June 12, 2025

Study Record Updates

Last Update Posted (Estimated)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
September 23, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 22, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
June 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • InHIBIT-002

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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