Telangiectasia, Hereditary HemorrhagicPhase 2PATH-HHTNCT03910244
Pomalidomide for the Treatment of Bleeding in HHT
A Phase 2 study testing Pomalidomide Oral Product in people with Telangiectasia, Hereditary Hemorrhagic, run by The Cleveland Clinic at 14 sites in United States.
Trial summary
This is a Phase II placebo-controlled double-blind study of pomalidomide in patients with hereditary hemorrhagic telangiectasia (HHT) with moderate to severe epistaxis who have anemia and/or require parenteral iron infusions or blood transfusions. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Telangiectasia, Hereditary Hemorrhagic
- What is being tested?
- Pomalidomide Oral Product
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 145 participants
- Where
- 14 sitesUnited States
- Study dates
- 17 Oct 2019to 8 Sep 2023
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- A clinical diagnosis of HHT as defined by the Curacao criteria
- Age ≥ 18 years
- Platelet count ≥ 100,000/µl
- White Blood Count (WBC) ≥ 2,500/µl
- International Normalized Ratio (INR) ≤ 1.4 and normal ± 2 sec activated partial thromboplastin time (aPTT or partial thromboplastin time (PTT) per local laboratory designation) 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 three months, measured at the screening visit
Show all 10Show fewer
- A requirement for anemia, as determined by local laboratory hemoglobin assessment and 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 Risk Evaluation and Mitigation Strategy (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 scheduled pregnancy testing as required in the POMALYST REMS program. FCBP must have a negative pregnancy test with a sensitivity of at least 50 milli-international units per milliliter (mIU/mL) within 10 - 14 days prior to and again within 24 hours prior to prescribing pomalidomide and must either commit to continued abstinence from heterosexual intercourse or use two (2) acceptable methods of birth control, one highly effective method and one additional effective method at the same time, at least 28 days before she starts taking pomalidomide, during therapy and for at least 4 weeks following discontinuation of therapy. Men must agree to use a latex condom during sexual contact with a FCBP even if they have had a vasectomy.
- Ability to understand and sign informed consent
Who cannot join
- Women currently breast feeding
- 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 Immunomodulatory imide drugs within previous 6 months
- Prior treatment with bevacizumab (systemic or nasal) within previous 6 weeks*
- Prior treatment with pazopanib within previous 6 weeks*
Show all 16Show fewer
- The use of octreotide or oral estrogens within the previous month*
- History of prior unprovoked thromboembolism confirmed by venous ultrasound or other imaging modalities
- Peripheral neuropathy, confirmed by neurologic consultation
- Known underlying hypoproliferative anemia (i.e. myelodysplasia, aplastic anemia)
- Currently enrolled in other interventional 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 Family Member 4 (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
- * Use of these treatments is not permitted during study participation.
Trial locations and contacts
14 sites in 1 country.
14 sites in 1 country
United States14 sites
Johns Hopkins Hospital
Baltimore, Maryland, 21287
University of Alabama at Birmingham
Birmingham, Alabama, 35233
Massachussets General Hospital
Boston, Massachusetts, 02114
University of North Carolina
Chapel Hill, North Carolina, 27517
Cleveland Clinic
Cleveland, Ohio, 44195
University of Florida
Gainesville, Florida, 32610
Baylor College of Medicine, Texas Children's Hospital
Houston, Texas, 77030
Medical College of Wiconsin
Milwaukee, Wisconsin, 53226
University of Minnesota Health Clinical Research Unit
Minneapolis, Minnesota, 55455
University of Pennsylvania Perelman School of Medicine
Philadelphia, Pennsylvania, 19104
Mayo Clinic
Rochester, Minnesota, 55905
University of Utah Healthcare
Salt Lake City, Utah, 84132
UCSD Hemophilia and Thrombosis Treatment Center
San Diego, California, 92121
UCSF Outpatient Hematology Clinic
San Francisco, California, 94143
About this study
Official title: Pomalidomide for the Treatment of Bleeding in Hereditary Hemorrhagic Telangiectasia
This is a Phase II placebo-controlled double-blind study of pomalidomide in patients with hereditary hemorrhagic telangiectasia (HHT) with moderate to severe epistaxis who have anemia and/or require parenteral iron infusions or blood transfusions. A total of 159 patients will be randomized 2:1 to treatment with oral pomalidomide or matching placebo for 24 weeks. Mean change from baseline to 24 weeks in the Epistaxis Severity Score (ESS) will be compared between treatment groups to determine pomalidomide efficacy.
Read the detailed description
HHT is associated with substantial morbidity, leading to a reduced quality of life, decreased rate of employment and a high incidence of depression. There currently exists no medical therapy recognized as consistently efficacious in HHT. Reports of the efficacy of thalidomide in HHT, as well as interim results of a pilot trial of pomalidomide in HHT provide evidence of efficacy with minimal toxicity. The favorable efficacy:toxicity ratio of pomalidomide suggest that it may benefit patients with HHT.
This study is designed as a Phase II placebo-controlled double-blind study of pomalidomide in HHT patients with moderate to severe epistaxis who have anemia and/or require parenteral iron infusions or blood transfusions. A total of 159 patients will be randomized 2:1 to treatment with oral pomalidomide or matching placebo for 24 weeks.
Primary Objective: To determine efficacy of pomalidomide compared to placebo for the reduction in severity of epistaxis after 24 weeks of treatment.
Secondary Objectives: To determine the safety and tolerability of pomalidomide for the treatment of HHT; to determine if pomalidomide treatment improves quality of life in HHT; to determine whether a continued response to pomalidomide is evident 4 weeks after treatment discontinuation; to develop a biorepository for future studies to define biomarkers predictive of pomalidomide response and allow investigations into the biology of HHT and mechanisms of pomalidomide.
- Conditions
- Telangiectasia, Hereditary Hemorrhagic
- Being tested
- Pomalidomide Oral Product, Placebo oral capsule
- Keywords
- Epistaxis, pomalidomide, blood transfusion
- Medical subject headings
- Telangiectasia, Hereditary Hemorrhagic, Epistaxis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Pomalidomide Oral ProductDrug
Also called POMALYST
Pomalidomide, a third generation derivative of thalidomide, given orally at a starting dose of 4 mg/day for days 1-28 of six 28-day cycles. The dose may be reduced to 3 or 2 mg/day based on specific adverse event (AE) criteria.
Placebo oral capsuleDrug
Matching placebo will be given.
| Study group | Role | What they receive |
|---|---|---|
| Pomalidomide | Experimental | Oral Pomalidomide will be provided as a capsule at 4 mg/day dose. There will be 6 treatment cycles of 28 days (4 weeks) each. Total treatment phase duration will be 24 weeks. Receives: Drug: Pomalidomide Oral Product |
| Placebo | Placebo comparator | A placebo matching the study drug will be provided as a capsule. There will be 6 treatment cycles of 28 days (4 weeks) each. Total treatment phase duration will be 24 weeks. Receives: Drug: Placebo oral capsule |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline Epistaxis Severity Score
The primary outcome measure is the change from baseline in Epistaxis Severity Score (ESS) after 6 months of treatment administration to compare the outcomes of Pomalidomide versus Placebo. The ESS ranges from 0-10 with higher scores indicating worse condition in the prior 4 weeks. The minimal important difference is 0.71
Measured: 4, 8, 12, 16, 20, and 24 Weeks and 4 weeks post treatment
Secondary outcomes (12)
Average Total Daily Duration of Nosebleeds - Change From Baseline
The daily epistaxis duration is calculated as the total duration of all reported nose bleeding events in a day, averaged across all days reported in a 4-week smartphone diary. The outcome is the change between the baseline diary on the specified timepoint
Measured: After 12 and 24 weeks of treatment, and 4 weeks post-treatment
Weighted Average Total Daily Duration of Nosebleeds - Change From Baseline
The daily epistaxis duration is calculated as the total duration of all reported nose bleeding events in a day, averaged across all days reported in a 4-week smartphone diary, weighted by the intensity, with 90%% winsorization. The outcome is the change between the baseline diary on the specified timepoint
Measured: After 12 and 24 weeks of treatment, and 4 weeks post-treatment
Total Iron Infused
Total iron infused (mg) is calculated as the total in 4 weeks, averaged across all visits reported through the 24-week treatment period. Patients with no infusions have a value of zero.
Measured: Baseline through 24 Weeks
Total Iron Infused
Total iron infused (mg) is calculated as the total in 4 weeks, averaged across all visits reported through the first 12 weeks of the treatment period. Patients with no infusions have a value of zero.
Measured: Baseline through 12 Weeks
Total Iron Infused
Total iron infused (mg) is calculated as the total in 4 weeks, averaged across all visits reported through the second 12 weeks of the treatment period. Patients with no infusions have a value of zero.
Measured: 12 through 24 Weeks
Patients With Any Packed Red Blood Cells Transfusion Through 24 Weeks
Patients with any packed red blood cells transfusion through the 24-week treatment period
Measured: Baseline through 24 Weeks
Patients With Any Packed Red Blood Cells Transfusion Through 12 Weeks
Patients with any packed red blood cells transfusion through the first 12 weeks of the treatment period
Measured: Baseline through 12 Weeks
Patients With Any Packed Red Blood Cells Transfusion 12-24 Weeks
Patients with any packed red blood cells transfusion through the second 12 weeks of the treatment period
Measured: 12 through 24 Weeks
Neuro-QoL - Satisfaction With Social Roles and Activities - T Score
The outcome measure is the Neuro-QoL Satisfaction with Social Roles and Activities T-Score to compare the outcomes of Pomalidomide versus Placebo. The Neuro-QoL Satisfaction with Social Roles and Activities Short Form (V1.1) T-score has a value of 50 representing the average general US population, with a standard deviation of 10, and with higher scores indicating more satisfaction. The minimal detectable change is 3.7 T score points
Measured: Baseline, after 12 and 24 weeks of treatment, and 4 weeks post-treatment
Patient Reported Outcomes Measurement Information System (PROMIS) - Emotional Distress - Depression - T Score
The outcome measure is the PROMIS Emotional Distress - Depression T-Score to compare the outcomes of Pomalidomide versus Placebo. The PROMIS Emotional Distress-Depression Short Form (V1.0) T-score has a value of 50 representing the average general US population, with a standard deviation of 10, and with higher scores indicating more depression
Measured: Baseline, after 12 and 24 weeks of treatment, and 4 weeks post-treatment
PROMIS - Fatigue - T Score
The outcome measure is the PROMIS Fatigue T-Score to compare the outcomes of Pomalidomide versus Placebo. The PROMIS® Fatigue Short Form (V1.0) T-score has a value of 50 representing the average general US population, with a standard deviation of 10, and with higher scores indicating more fatigue
Measured: Baseline, after 12 and 24 weeks of treatment, and 4 weeks post-treatment
HHT-Specific QOL Questionnaire - Score
The outcome measure is the HHT-Specific QOL Questionnaire - Score to compare the outcomes of Pomalidomide versus Placebo. The HHT-specific QOL score ranges from 0 to 16 with higher scores indicating more limitations due to HHT in the prior 4 weeks
Measured: Baseline, after 12 and 24 weeks of treatment, and 4 weeks post-treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Apr 2019
- Study start17 Oct 2019
- Primary completion8 Sep 2023
- Study completion8 Sep 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Telangiectasia, Hereditary HemorrhagicStudies of the same condition, open and under way.
- More trials run by The Cleveland ClinicThe 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 145 participants (actual)
Sponsor and investigators
- Lead sponsor
- The Cleveland Clinic
- Sponsor type
- University, hospital or other
- Collaborators
- RTI International
- Responsible party
- Principal investigator, Keith McCrae, The Cleveland Clinic
- Investigators
- Keith McCrae, MD, Principal investigator, The Cleveland Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03910244
- Sponsor's protocol ID
- 133646
- Acronym
- PATH-HHT
- Status last verified
- Sep 2024
- First submitted
- 8 Apr 2019
- Last updated
- 23 Oct 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: The Data Coordinating Center (DCC) will prepare de-identified data files for sharing. A final data sharing plan will be developed with NHLBI and the study Steering Committee (SC). The data can be provided in Statistical Analysis System (SAS) data sets and export files and documentation will be in portable document format (PDF). The first level of data sharing will be with trial investigators. In addition, the DCC will be responsible for sharing study data with outside researchers, if approved by NHLBI and the SC.
Publications
McDonald J, Wooderchak-Donahue W, VanSant Webb C, Whitehead K, Stevenson DA, Bayrak-Toydemir P. Hereditary hemorrhagic telangiectasia: genetics and molecular diagnostics in a new era. Front Genet. 2015 Jan 26;6:1. doi: 10.3389/fgene.2015.00001. eCollection 2015.
PubMed 25674101Hoag JB, Terry P, Mitchell S, Reh D, Merlo CA. An epistaxis severity score for hereditary hemorrhagic telangiectasia. Laryngoscope. 2010 Apr;120(4):838-43. doi: 10.1002/lary.20818.
PubMed 20087969Chaturvedi S, Clancy M, Schaefer N, Oluwole O, McCrae KR. Depression and post-traumatic stress disorder in individuals with hereditary hemorrhagic telangiectasia: A cross-sectional survey. Thromb Res. 2017 May;153:14-18. doi: 10.1016/j.thromres.2017.03.003. Epub 2017 Mar 9.
PubMed 28314138Al-Samkari H, Thomas SM, Marsh D, Kasthuri RS, Iyer VN, Pishko AM, Decker JE, Weiss CR, Whitehead KJ, Conrad MB, Zumberg MS, Zhou JY, Parambil J, Carper B, Clancy M, McCrae KR. Characteristics associated with clinical response to pomalidomide in hereditary hemorrhagic telangiectasia. Blood Adv. 2026 May 12;10(9):2967-2976. doi: 10.1182/bloodadvances.2025019484.
PubMed 41719457Al-Samkari H, Kasthuri RS, Iyer VN, Pishko AM, Decker JE, Weiss CR, Whitehead KJ, Conrad MB, Zumberg MS, Zhou JY, Parambil J, Marsh D, Clancy M, Bradley L, Wisniewski L, Carper BA, Thomas SM, McCrae KR. Pomalidomide for Epistaxis in Hereditary Hemorrhagic Telangiectasia. N Engl J Med. 2024 Sep 19;391(11):1015-1027. doi: 10.1056/NEJMoa2312749.
PubMed 39292928
Study at a glance
From the registry record, last updated by the sponsor on 23 Oct 2024. 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
- ClinicalTrials.gov ID
- NCT03910244
- Acronym / sponsor ID
- PATH-HHT / 133646
- Conditions
- Telangiectasia, Hereditary Hemorrhagic
- Keywords
- Epistaxis, pomalidomide, blood transfusion
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 145 participants (actual)
- Lead sponsor
- The Cleveland ClinicUniversity, hospital or other
- Collaborators
- RTI International
- Study start
- 17 Oct 2019 (actual)
- Primary completion
- 8 Sep 2023 (actual)
- Study completion
- 8 Sep 2023 (actual)
- First posted
- 10 Apr 2019 (actual)
- Results
- Posted 23 Oct 2024
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 Phase II placebo-controlled double-blind study of pomalidomide in patients with hereditary hemorrhagic telangiectasia (HHT) with moderate to severe epistaxis who have anemia and/or require parenteral iron infusions or blood transfusions. A total of 159 patients will be randomized 2:1 to treatment with oral pomalidomide or matching placebo for 24 weeks. Mean change from baseline to 24 weeks in the Epistaxis Severity Score (ESS) will be compared between treatment groups to determine pomalidomide efficacy.
Detailed description
HHT is associated with substantial morbidity, leading to a reduced quality of life, decreased rate of employment and a high incidence of depression. There currently exists no medical therapy recognized as consistently efficacious in HHT. Reports of the efficacy of thalidomide in HHT, as well as interim results of a pilot trial of pomalidomide in HHT provide evidence of efficacy with minimal toxicity. The favorable efficacy:toxicity ratio of pomalidomide suggest that it may benefit patients with HHT.
This study is designed as a Phase II placebo-controlled double-blind study of pomalidomide in HHT patients with moderate to severe epistaxis who have anemia and/or require parenteral iron infusions or blood transfusions. A total of 159 patients will be randomized 2:1 to treatment with oral pomalidomide or matching placebo for 24 weeks.
Primary Objective: To determine efficacy of pomalidomide compared to placebo for the reduction in severity of epistaxis after 24 weeks of treatment.
Secondary Objectives: To determine the safety and tolerability of pomalidomide for the treatment of HHT; to determine if pomalidomide treatment improves quality of life in HHT; to determine whether a continued response to pomalidomide is evident 4 weeks after treatment discontinuation; to develop a biorepository for future studies to define biomarkers predictive of pomalidomide response and allow investigations into the biology of HHT and mechanisms of pomalidomide.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Pomalidomide | Experimental | Drug: Pomalidomide Oral ProductOral Pomalidomide will be provided as a capsule at 4 mg/day dose. There will be 6 treatment cycles of 28 days (4 weeks) each. Total treatment phase duration will be 24 weeks. |
| Placebo | Placebo comparator | Drug: Placebo oral capsuleA placebo matching the study drug will be provided as a capsule. There will be 6 treatment cycles of 28 days (4 weeks) each. Total treatment phase duration will be 24 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- A clinical diagnosis of HHT as defined by the Curacao criteria
- Age ≥ 18 years
- Platelet count ≥ 100,000/µl
- White Blood Count (WBC) ≥ 2,500/µl
- International Normalized Ratio (INR) ≤ 1.4 and normal ± 2 sec activated partial thromboplastin time (aPTT or partial thromboplastin time (PTT) per local laboratory designation) by local laboratory criteria (except for patients on a stable dose of warfarin or direct oral anticoagulants)
Key exclusion criteria (16 listed)
- Women currently breast feeding
- 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 Immunomodulatory imide drugs 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 |
|---|---|---|
| Pomalidomide Oral ProductAlso: POMALYSTPomalidomide, a third generation derivative of thalidomide, given orally at a starting dose of 4 mg/day for days 1-28 of six 28-day cycles. The dose may be reduced to 3 or 2 mg/day based on specific adverse event (AE) criteria. | Drug | Pomalidomide |
| Placebo oral capsuleMatching placebo will be given. | Drug | Placebo |
Primary outcome
Change From Baseline Epistaxis Severity Score
Time frame: 4, 8, 12, 16, 20, and 24 Weeks and 4 weeks post treatment
The primary outcome measure is the change from baseline in Epistaxis Severity Score (ESS) after 6 months of treatment administration to compare the outcomes of Pomalidomide versus Placebo. The ESS ranges from 0-10 with higher scores indicating worse condition in the prior 4 weeks. The minimal important difference is 0.71
12 secondary outcomes
Average Total Daily Duration of Nosebleeds - Change From Baseline
Time frame: After 12 and 24 weeks of treatment, and 4 weeks post-treatment
Weighted Average Total Daily Duration of Nosebleeds - Change From Baseline
Time frame: After 12 and 24 weeks of treatment, and 4 weeks post-treatment
Total Iron Infused
Time frame: Baseline through 24 Weeks
Total Iron Infused
Time frame: Baseline through 12 Weeks
Total Iron Infused
Time frame: 12 through 24 Weeks
Patients With Any Packed Red Blood Cells Transfusion Through 24 Weeks
Time frame: Baseline through 24 Weeks
Patients With Any Packed Red Blood Cells Transfusion Through 12 Weeks
Time frame: Baseline through 12 Weeks
Patients With Any Packed Red Blood Cells Transfusion 12-24 Weeks
Time frame: 12 through 24 Weeks
Neuro-QoL - Satisfaction With Social Roles and Activities - T Score
Time frame: Baseline, after 12 and 24 weeks of treatment, and 4 weeks post-treatment
Patient Reported Outcomes Measurement Information System (PROMIS) - Emotional Distress - Depression - T Score
Time frame: Baseline, after 12 and 24 weeks of treatment, and 4 weeks post-treatment
PROMIS - Fatigue - T Score
Time frame: Baseline, after 12 and 24 weeks of treatment, and 4 weeks post-treatment
HHT-Specific QOL Questionnaire - Score
Time frame: Baseline, after 12 and 24 weeks of treatment, and 4 weeks post-treatment
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Oct 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 25674101McDonald J, Wooderchak-Donahue W, VanSant Webb C, Whitehead K, Stevenson DA, Bayrak-Toydemir P. Hereditary hemorrhagic telangiectasia: genetics and molecular diagnostics in a new era. Front Genet. 2015 Jan 26;6:1. doi: 10.3389/fgene.2015.00001. eCollection 2015.
- Background: PubMed 20087969Hoag JB, Terry P, Mitchell S, Reh D, Merlo CA. An epistaxis severity score for hereditary hemorrhagic telangiectasia. Laryngoscope. 2010 Apr;120(4):838-43. doi: 10.1002/lary.20818.
- Background: PubMed 28314138Chaturvedi S, Clancy M, Schaefer N, Oluwole O, McCrae KR. Depression and post-traumatic stress disorder in individuals with hereditary hemorrhagic telangiectasia: A cross-sectional survey. Thromb Res. 2017 May;153:14-18. doi: 10.1016/j.thromres.2017.03.003. Epub 2017 Mar 9.
- Derived from this study: PubMed 41719457Al-Samkari H, Thomas SM, Marsh D, Kasthuri RS, Iyer VN, Pishko AM, Decker JE, Weiss CR, Whitehead KJ, Conrad MB, Zumberg MS, Zhou JY, Parambil J, Carper B, Clancy M, McCrae KR. Characteristics associated with clinical response to pomalidomide in hereditary hemorrhagic telangiectasia. Blood Adv. 2026 May 12;10(9):2967-2976. doi: 10.1182/bloodadvances.2025019484.
- Derived from this study: PubMed 39292928Al-Samkari H, Kasthuri RS, Iyer VN, Pishko AM, Decker JE, Weiss CR, Whitehead KJ, Conrad MB, Zumberg MS, Zhou JY, Parambil J, Marsh D, Clancy M, Bradley L, Wisniewski L, Carper BA, Thomas SM, McCrae KR. Pomalidomide for Epistaxis in Hereditary Hemorrhagic Telangiectasia. N Engl J Med. 2024 Sep 19;391(11):1015-1027. doi: 10.1056/NEJMoa2312749.
- Individual participant dataSharing planned: Yes. The Data Coordinating Center (DCC) will prepare de-identified data files for sharing. A final data sharing plan will be developed with NHLBI and the study Steering Committee (SC). The data can be provided in Statistical Analysis System (SAS) data sets and export files and documentation will be in portable document format (PDF). The first level of data sharing will be with trial investigators. In addition, the DCC will be responsible for sharing study data with outside researchers, if approved by NHLBI and the SC.
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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.
Keith McCrae, MD principal investigator
The Cleveland Clinic
14 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Pomalidomide Oral Product in people with Telangiectasia, Hereditary Hemorrhagic, run by The Cleveland Clinic at 14 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 10 inclusion and 16 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 23 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 23 Oct 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Change From Baseline Epistaxis Severity Score (time frame: 4, 8, 12, 16, 20, and 24 Weeks and 4 weeks post treatment). 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 NCT03910244, which also lists 5 linked publications.
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 23 Oct 2024.