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2/2 Pomalidomide for Bleeding in Patients with Hereditary Hemorrhagic Telangiectasia (HHT) Bleeding Data Coordinating Center

Award Number: U24HL140090
ORGANIZATION: NATIONAL HEART, LUNG, & BLOOD INSTITUTE
OPDIV: NIH
AWARD CLASS: COOPERATIVE AGREEMENT
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

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Project Summary Hereditary hemorrhagic telangiectasia (HHT) is an inherited disorder characterized by diffuse arteriovenous malformations (AVMs) and telangiectasia that affect many organs. The most common manifestations of HHT are epistaxis and GI bleeding; however AVMs that involve the lungs, brain, or liver can also lead to devastating cardiovascular complications including congestive heart failure, pulmonary hemorrhage, and hemorrhagic stroke. Estimates suggest a prevalence of more than 100,000 cases of HHT in the United States. In addition to bleeding and cardiovascular manifestations, the morbidity of HHT leads to a high incidence of anxiety and depression that greatly affects social function. There is no broadly accepted, effective therapy for HHT. Ablation of HHT lesions via interventional approaches is commonly used but provides only transient benefit. Medical approaches have included anti-fibrinolytic therapy, estrogen, and octreotide, among others; however, these are not effective in most patients. A recent study demonstrated that nasal sprays containing tranexamic acid, estriol, or bevacizumab were no more effective than placebo in treating HHT-associated epistaxis. The current study builds on results of several small studies that suggest efficacy of thalidomide in HHT; however, thalidomide is not available for clinical trials in the United States. Thus, we propose to study pomalidomide, a third-generation thalidomide analog with similar anti-angiogenic activity but less toxicity than thalidomide. A small, industry-funded pilot study by the PI has demonstrated efficacy and safety of pomalidomide in HHT, and FDA has issued an IND to expand these observations. This resubmission proposes a randomized, placebo- controlled study developed with support from an NHLBI U34 Clinical Trial Planning Grant and U24 Clinical Trials Development Resource for Hematologic Disorders by a team of HHT experts, a patient advocate, and experienced clinical trial statisticians. The same team that designed the study will conduct it, using an sIRB at the Cleveland Clinic. We propose to (1) determine the efficacy and safety of pomalidomide in patients with HHT and epistaxis requiring periodic iron infusion or blood transfusion, (2) determine the effect of pomalidomide on quality of life in patients with HHT using NIH PROMIS tools and an HHT-specific questionnaire, and (3) create a biorepository of samples from study patients for future biomarker and mechanistic studies. This study may validate pomalidomide as a new therapeutic option and change the paradigm for treatment of HHT.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2024 ( Subtotal = $0 )
2024	2023	RESEARCH TRIANGLE INSTITUTE	3040 E CORNWALLIS RD	RESEARCH TRIANGLE PARK	NC	27709	DURHAM	USA	Blood Diseases and Resources Research	000	5	12/15/2023	NON-COMPETING CONTINUATION	$0
														Subtotal = $0

Issue Date FY: 2023 ( Subtotal = $398,234 )
2023	2023	RESEARCH TRIANGLE INSTITUTE	3040 E CORNWALLIS RD	RESEARCH TRIANGLE PARK	NC	27709	DURHAM	USA	Blood Diseases and Resources Research	000	5	3/2/2023	NON-COMPETING CONTINUATION	$398,234
2023	2023	RESEARCH TRIANGLE INSTITUTE	3040 E CORNWALLIS RD	RESEARCH TRIANGLE PARK	NC	27709	DURHAM	USA	Blood Diseases and Resources Research	001	5	9/8/2023	NON-COMPETING CONTINUATION	$0
														Subtotal = $398,234

Issue Date FY: 2022 ( Subtotal = $395,332 )
2022	2022	RESEARCH TRIANGLE INSTITUTE	3040 E CORNWALLIS RD	RESEARCH TRIANGLE PARK	NC	27709	DURHAM	USA	Blood Diseases and Resources Research	000	4	3/1/2022	NON-COMPETING CONTINUATION	$395,332
														Subtotal = $395,332

Issue Date FY: 2021 ( Subtotal = $394,666 )
2021	2021	RESEARCH TRIANGLE INSTITUTE	3040 E CORNWALLIS RD	RESEARCH TRIANGLE PARK	NC	27709	DURHAM	USA	Blood Diseases and Resources Research	000	3	4/6/2021	NON-COMPETING CONTINUATION	$394,666
														Subtotal = $394,666

Issue Date FY: 2020 ( Subtotal = $425,631 )
2020	2020	RESEARCH TRIANGLE INSTITUTE	3040 E CORNWALLIS RD	RESEARCH TRIANGLE PARK	NC	27709	DURHAM	USA	Blood Diseases and Resources Research	001	2	3/6/2020	NON-COMPETING CONTINUATION	$425,631
2020	2020	RESEARCH TRIANGLE INSTITUTE	3040 E CORNWALLIS RD	RESEARCH TRIANGLE PARK	NC	27709	DURHAM	USA	Blood Diseases and Resources Research	002	2	6/10/2020	NON-COMPETING CONTINUATION	$0
2020	2019	RESEARCH TRIANGLE INSTITUTE	3040 E CORNWALLIS RD	RESEARCH TRIANGLE PARK	NC	27709	DURHAM	USA	Blood Diseases and Resources Research	000	1	2/20/2020	NEW	$0
														Subtotal = $425,631

Issue Date FY: 2019 ( Subtotal = $527,881 )
2019	2019	RESEARCH TRIANGLE INSTITUTE	3040 E CORNWALLIS RD	RESEARCH TRIANGLE PARK	NC	27709	DURHAM	USA	Blood Diseases and Resources Research	000	1	3/5/2019	NEW	$527,881
														Subtotal = $527,881

Grand Total All Awards = $2,141,744

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2/2 Pomalidomide for Bleeding in Patients with Hereditary Hemorrhagic Telangiectasia (HHT) Bleeding Data Coordinating Center

Award Number: U24HL140090

ORGANIZATION: NATIONAL HEART, LUNG, & BLOOD INSTITUTE

OPDIV: NIH

AWARD CLASS: COOPERATIVE AGREEMENT

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

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