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Telangiectasis clinical trials

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NCT ID: NCT04976036 Recruiting - Clinical trials for Telangiectasia, Hereditary Hemorrhagic

Efficacy of Nintedanib for Treatment of Epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT) Patients

EPISTOP
Start date: May 5, 2022
Phase: Phase 2
Study type: Interventional

Patients affected by hereditary hemorrhagic telangiectasia (HHT) very often suffer from recurrent nosebleeds called epistaxis. There is no treatment currently available to reduce the frequency or severity of epistaxis. This research project will examine the effect of nintedanib, a capsule to be taken twice a day, on the frequency and severity of epistaxis in HHT. The study will take place at the Respiratory medicine department of the Lausanne University Hospital (Centre hospitalier universitaire vaudois, CHUV). The investigators will recruit about 48 participants with HHT, who will be divided in 2 groups. Each group will perform the same examinations and follow-up visits. The study will begin with 2 months of observation during which subjects will be asked to fill a diary to record the number and duration of epistaxis episodes. The diary will be filled daily for the entire duration of the study, i.e. 8 months. After 2 months of observation, the treatment phase will begin. Participants will take a capsule (nintedanib 150 mg or placebo) once a day for 2 weeks, then twice a day for 14 weeks. In case of intolerance at the dose of 2 capsules per day, the treatment may be reduced to 1 capsule per day. Subjects will also have to mention on the diary any blood transfusion, iron perfusion, and any symptoms they may be experiencing. Following the 16 weeks of treatment, an 8-week follow-up period will allow to observe the effects of nintedanib after the end of the treatment period, and to monitor any unexpected adverse events.

NCT ID: NCT04717583 Recruiting - Clinical trials for Facial Telangiectasia in Patients With Systemic Lupus Erythematosus

Efficacy of Intense Pulsed Light in the Treatment of Facial Telangiectasia in Systemic Lupus Erythematosus

Start date: February 1, 2021
Phase: N/A
Study type: Interventional

This clinical trial intends to evaluate the efficacy and safety of intense pulsed light (IPL) in the treatment of facial telangiectasia in patients with systemic lupus erythematosus (SLE).

NCT ID: NCT04469517 Recruiting - Clinical trials for Hereditary Hemorrhagic Telangiectasia

Influence of Hypoxic Induced Factors in Patients With Hereditary Hemorrhagic Telangiectasia

Start date: August 10, 2020
Phase:
Study type: Observational [Patient Registry]

Patients with hereditary hemorrhagic telangiectasia (HHT) suffer from an inherited disorder leading to systemic vascular malformations. Mutations in several genes of the transforming growth factor (TGF)-β superfamily pathway influence angiogenesis in patients with HHT. The genetic loss of ALK1 or ENG alone are not sufficient to induce AVMs; environmental insults that could trigger angiogenesis (e.g. wounding) are also needed. In this study it will be analyzed if hypoxic induced factors could have an influence on the disease HHT.

NCT ID: NCT04404881 Recruiting - Clinical trials for Hereditary Hemorrhagic Telangiectasia

Bevacizumab In Hereditary Hemorrhagic Telangiectasia

Start date: November 23, 2020
Phase: Phase 2
Study type: Interventional

This research study is studying to see whether bevacizumab may treat chronic bleeding and iron deficiency anemia in Hereditary Hemorrhagic Telangiectasia (HHT). Hereditary Hemorrhagic Telangiectasia (HHT) is a disorder that causes abnormal blood vessel formation. In HHT, there is a mutation in the TGF-β pathway, which results in an increase of vascular endothelial growth factor (VEGF) levels. An increase in VEGF levels can result in poorly formed blood vessels that have a higher rate of bleeding than normal blood vessels. Bevacizumab is designed to block VEGF activity. It is believed that targeting increased VEGF levels may be able to treat HHT. This research study involves the following study drug: - Bevacizumab

NCT ID: NCT04037189 Recruiting - Lymphoma Clinical Trials

Treatment of Leukemia and Lymphoma in Children With Ataxia Telangiectasia

Start date: July 28, 2019
Phase:
Study type: Observational

Ataxia telangiectasia (A-T) is a multisystem disease with diverse manifestations, including progressive neurodegeneration, immunodeficiency, respiratory disease, and genomic instability. One of the most important features of A-T is the increased predisposition to cancer, especially to lymphoid malignancies. Patients with A-T are generally excluded from collaborative clinical trials, their treatment outcomes and toxicity profiles have rarely been reported, and little is currently known concerning the treatment intensity required to provide a reasonable balance between efficacy and toxicity. The aims of this study are to build a large international de-identified database of children with A-T treated for leukemia and lymphoma, to investigate epidemiology and outcome of treatment, toxicity profiles and risk factors which impact outcome, in order to eventually enable the generation of data-based treatment recommendations for this population.

NCT ID: NCT03981562 Recruiting - Clinical trials for Hereditary Haemorrhagic Telangiectasia

Vitamin D and Hereditary Haemorrhagic Telangiectasia

Start date: July 16, 2018
Phase: Phase 2
Study type: Interventional

The study aims to assess whether supplementing vitamin D in patients diagnosed with Hereditary Haemorrhagic Telangiectasia (HHT) will decrease the frequency and severity of nosebleeds these patients experience. It is hypothesized that the larger the dose of daily vitamin D given to the patients, the less frequent and less severe the nosebleeds will be.

NCT ID: NCT03850964 Recruiting - Anemia Clinical Trials

Effects of Pazopanib on Hereditary Hemorrhagic Telangiectasia Related Epistaxis and Anemia (Paz)

Paz
Start date: May 8, 2023
Phase: Phase 2/Phase 3
Study type: Interventional

During the Efficacy Study (Part B), the investigators will study whether Pazopanib, taken daily for 24 weeks, will reduce the severity of nose bleeds in patients with hereditary hemorrhagic telangiectasia (HHT). Patients will either be provided active drug or a placebo [sugar - inactive pill], and be tested for nose bleed severity throughout the trial, including particularly nose bleed duration. Investigators will also test for blood loss, as well as for safety. This study is funded by the US Department of Defense USAMRAA and FDA/OOPD.

NCT ID: NCT03845049 Recruiting - Clinical trials for Macular Telangiectasia

Comparative Multicenter Randomized Study of Aflibercept Versus Placebo in Macular Telangiectasia Type 1

TELeMAC
Start date: July 3, 2019
Phase: Phase 3
Study type: Interventional

Idiopathic juxtafoveal telangiectasia type 1 is a rare unilateral disease that mostly affects men before 50 years of age. Mac Tel 1 are characterized by microvascular telangiectasia and increased tortuosity of the macular capillary network on the temporal part of the fovea that can be identified on fundus examination. It can be associated with peripheral vascular changes, similar to manifestations of Coats' disease. It can be complicated by macular edema due to leakage from microvascular ectasia. When associated with visual loss, macular edema can be treated with different strategies although there is no consensus about the best approach. Laser can be performed on leaky aneurysms with questionable long term efficacy and potential irreversible adverse effects. Recently, anti-VEGF agents have been put forward as particularly good candidates to treat this macular edema, as observed in vein occlusion or diabetic macular edema. Indeed, in limited case series, the first anti-VEGF agents (ranibizumab and bevacizumab) showed mitigated results. More recently, authors have reported some favorable results with aflibercept in patients refractory to other anti-VEGF agents. Indeed a recent study reported both good anatomical and functional results in macular edema due to Mac Tel 1 in a non-comparative study that included 8 patients and carried out a concomitant quantification of growth factors. As an explanation, the authors found that levels of placental growth factor (PlGF), which is targeted by aflibercept but not by other anti-VEGF agents, were decreased after treatment. Moreover, PlGF correlated with capillary plexus densities assessed by OCTA. The aim of this study is thus to assess the efficacy of a 6 months treatment by aflibercept compared to placebo in macular edema linked to Mac Tel 1 with a multicenter double-blind randomized clinical trial.

NCT ID: NCT03841422 Recruiting - Clinical trials for Hereditary Haemorrhagic Telangiectasia

Evaluation of Video-assisted Instructions of Nasal Self-packing in Patients With HHT

Start date: January 30, 2019
Phase:
Study type: Observational

Inserting nasal self-packings is a secure method leaving patients more self-confident. We provide a video on https://www.youtube.com/watch?v=LaioLYfSJ-E demonstrating how to use nasal self-packings.

NCT ID: NCT03759678 Recruiting - Clinical trials for Ataxia Telangiectasia

N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T)

Start date: January 8, 2020
Phase: Phase 2
Study type: Interventional

This is a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of Ataxia-Telangiectasia (A-T). There are two phases to this study: the Parent Study, and the Extension Phase. The Parent Study evaluates the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the symptomatic treatment of A-T. The Extension Phase evaluates the long-term safety and efficacy of IB1001 for the neuroprotective, disease-modifying treatment of A-T.