Active clinical trials for "Telangiectasia, Hereditary Hemorrhagic"

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This study will investigate whether pazopanib can reduce epistaxis and improve anaemia in subjects with hereditary haemorrhagic telangiectasia (HHT) at a dose that is well tolerated. The study will have 2 parts. Part A will be an open label, dose-escalation study in which up to 4 cohorts of approximately 6 subjects each will receive increasing doses of pazopanib for a maximum of 12 weeks. The dose in the first cohort will be 50mg per day and the maximum dose in a cohort will be 400 mg per day. Dose escalation will not occur as planned if the predefined safety stopping criteria are met or at least 4 subjects in a cohort have demonstrated efficacy (as measured by epistaxis, haemoglobin, transfusion or iron infusion requirements). If efficacy is demonstrated in Part A with an acceptable safety profile, Part B will be initiated to further define the optimal dose(s) including dose duration/schedule and to provide further support for the proof of mechanism. Approximately 15 subjects will participate and will be randomised to active or placebo in a ratio of 3:2. This part of the study will be double-blind.

Hereditary Hemorrhagic Telangiectasia (HHT) is a rare (~ 1/6000) but ubiquitous genetic disease. It is associated with abnormal angiogenesis and autosomal dominant inheritance, leading to telangiectasias and arteriovenous fistulae. More than 95% of patients are concerned by epistaxis (nosebleeds). These events are spontaneous, repeated, irregular, both diurnal and nocturnal, a source of anemia, disabling and very socially embarrassing. Anti-angiogenic treatments, including bevacizumab, are a new therapeutic option in HHT. The aim of this study is to evaluate 3 months after the end of the treatment the efficacy on the duration of the nosebleeds with 3 different doses (25, 50 and 75 mg) of bevacizumab administered as a nasal spray in a repeated manner (3 administrations) in patients with Hereditary Hemorrhagic Telangiectasia complicated by nosebleeds. This randomized, double-blind, placebo-controlled, seamless phase II/III study is to be carried out on 4 groups of 20 patients for first step and 2 groups of 20 to 40 patients for second step

Hereditary Haemorrhagic Telangiectasia (HHT, also known as Osler-Weber-Rendu Syndrome) is an inherited vascular disease that leads to the development of dilated and fragile blood vessels. The study goal is to culture white blood cells that express the proteins mutated in HHT and examine in the laboratory to explain aspects of the HHT disease phenotype.

Managing iron deficiency is important for more than 1 billion individuals worldwide, to avoid blood transfusions, or excessive strain on vital organs that depend on iron-containing haemoglobin to deliver oxygen to the tissues. Iron deficiency is a particular problem for people with the inherited condition hereditary haemorrhagic telangiectasia (HHT). Their iron deficiency and anaemia results from blood losses, especially from the nose (nosebleeds, and they often need additional iron to replace that lost through bleeding. Our goal is to stratify HHT patients into high/low absorbers of iron; to define what extra iron they need to adjust for their current and likely future blood losses; and to work out how to achieve this most safely for each individual to improve their later health. We will test the hypothesis that informed assessment of iron intake and post absorption cellular profiles changes the recommendations for iron intake for HHT patients.

The purpose of the study is to evaluate the safety and tolerability of pegylated interferon alpha-2b (PEG-Intron) in patients with severe complications related to Hereditary hemorrhagic telangiectasia (HHT). Funding Source - FDA Office of Orphan Products Development (OOPD)

This is a randomized, controlled, double-blind, placebo-controlled trial of intranasal Avastin (bevacizumab) injection versus saline control for control of HHT-related epistaxis when used in conjunction with bipolar electrocautery.

Terence M. Davidson, MD is conducting a research study to find out more about the topical application of Avastin (proper chemical name is bevacizumab) in the treatment of epistaxis (nose bleeding) in patients with Hereditary Hemorrhagic Telangiectasia (HHT).

In a case series intranasal submucosal bevacizumab has been shown to reduce epistaxis in patients suffering from Hereditary Haemorrhagic Telangiectasia together with KTP Laser therapy. The aim of this study is to evaluate the effectiveness of submucosal intranasal bevacizumab compared to placebo in a randomized double blind trial setting.

The purpose of this study is to determine whether Thalidomide is effective in the treatment of arteriovenous malformations in the gastrointestinal tract.