Anti-Estrogen Therapy for Hereditary Hemorrhagic Telangiectasia A Double-Blind Placebo-Controlled...
Hereditary Hemorrhagic TelangiectasiaHereditary hemorrhagic telangiectasia (HHT) is associated with recurrent epistaxis in 90% of cases. Good response to hormone treatment has been documented, although its use remains controversial. A double-blind placebo-controlled trial with estrogen treatment did not show any benefit over placebo. The aim of this study is to examine the efficacy of an antiestrogenic agent, TAMOXIFEN, in the treatment of HHT-associated epistaxis. The study will include up to 60 patients, over 18 years old.
Pomalidomide in Hereditary Hemorrhagic Telangiectasia and Transfusion-Dependent Vascular Ectasia:...
Hereditary Hemorrhagic TelangiectasiaIdiopathic Vascular EctasiaThis study will evaluate patients > 18 years of age with transfusion-dependent gastrointestinal bleeding due to documented gastrointestinal vascular ectasia with or without concurrent hereditary hemorrhagic telangiectasia (HHT). This study will focus on documented bleeding sites in the small bowel, including the duodenum, jejunum and ileum. Eligible patients will have endoscopically-documented sites of vascular ectasia and will have required at least 4 units of blood transfusion or episodes of intravenous iron administration over the preceding four months.
The ELLIPSE Study: A Phase-1 Study Evaluating the Tolerance of Bevacizumab Nasal Spray to Treat...
Rendu Osler DiseaseTelangiectasia1 moreAntiangiogenic drugs, such as bevacizumab, are a new treatment strategy in Hereditary Hemorrhagic Telangiectasia (HHT). Its systemic administration in patients with HHT improves liver damage-related symptoms and epistaxis (cases reported and on-going study-ClinicalTrials.gov Identifier #NCT00843440-). To limit the systemic adverse effects of bevacizumab and to ease administration, a local administration seems suitable. A clinical case recently showed the benefits of bevacizumab nasal spray in these patients. Its results were confirmed in a characterization study on bevacizumab transport through porcine nasal mucosa (in press). It seems necessary to assess the tolerance and efficacy of bevacizumab nasal spray in humans for the treatment of epistaxis in HHT with a prospective phase 1 study. The primary objective of the study is to evaluate the tolerance of increasing doses of bevacizumab administered as a nasal spray in patients with HHT-related epistaxis. This phase-1, randomized, double-blind, placebo-controlled, monocentric study is to be carried out sequentially (dose escalation) on 5 groups of 8 patients. Each group is made up of 6 verum and 2 placebos.
A Study to Determine the Safety and Efficacy of NT-501 in Macular Telangiectasia Type 2 - Protocol...
Macular Telangiectasia Type 2This study is a phase 3, randomized, multi-center study to evaluate the efficacy and safety of the NT-501 implants in participants with macular telangiectasia type 2.
Extension Study of NT-501 Ciliary Neurotrophic Factor (CNTF) Implant for Macular Telangiectasia...
Macular TelangiectasiaThis study is a prospective, phase 2 extension study of participants previously enrolled in NTMT-01 and NTMT-02. This study is designed to evaluate long term safety and efficacy of the NT-501 implant in participants previously enrolled in the NTMT-01 and NTMT-02 protocols.
Response of Individuals With Ataxia-Telangiectasia to Metformin and Pioglitazone
Ataxia-TelangiectasiaThis study aims to investigate the link between the Ataxia Telangiectasia Mutated (ATM) gene and metformin response. This link has been identified from large studies of the human genome, and this study aims to confirm this link in a clinical study. The ATM gene is involved in DNA repair - if a person inherits a "faulty" copy of this gene from both their parents, they have a genetic condition called Ataxia-telangiectasia (A-T). A-T is associated with, among other things, a resistance to insulin, which causes fatty liver and diabetes. This study will recruit people who have A-T, but have not developed diabetes, and compare this group to "healthy" controls, i.e. people who do not have A-T or diabetes. The study will compare how the groups respond to two drugs used to treat diabetes (metformin and pioglitazone), with the intention that this will guide the management of diabetes in A-T. This is an, open label unblinded study recruiting 15 people with A-T and 15 age and gender matched controls. Each participant will have three study visits to the Clinical Research Centre at Ninewells hospital in Dundee - one at baseline, a second after 8 weeks of metformin and the final visit after eight weeks of pioglitazone. During each visit we will carry out a number of investigations to study the insulin resistance of A-T and how it responds to metformin and pioglitazone.
Topical Brimonidine to Reduce Inflammation After IPL-treatment in Patients With Facial Telangiectasias...
TelangiectasiasThe aim of the study is to investigate whether brimonidine cream can reduce IPL-induced inflammation in terms of redness, swelling and pain in patients with facial vascular lesions (telangiectasias). Furthermore, the effect of brimonidine cream on IPL-efficacy is evaluated one month after final IPL-treatment. The hypothesis is that brimonidine, which has been proved effective in reduction of symptomatic erythema in patients with rosacea, also may have the ability to reduce IPL-induced erythema. Since the potential reduction in erythema is caused by vasoconstriction, brimonidine may further reduce IPL-induced oedema and pain.
Efficacy of Nintedanib Per os as a Treatment for Epistaxis in HHT Disease.
TelangiectasiaHereditary Hemorrhagic1 moreThe recognized manifestations of HHT are all due to abnormalities in vascular structure. Epistaxis are spontaneous, very variable, may occur as often as several times every day, and are recurrent in 90% of patients and associated with chronic and severe anemia in 2-10%. They also significantly reduce quality of life. Blood transfusions are sometimes required in 10-30% of patients. Previous studies showed that antiangiogenic treatments such as anti-VEGF treatment (bevacizumab) administered intravenously was efficient on epistaxis and dramatically reduced nosebleeds. Tyrosine kinase inhibitors are anti-angiogenic molecules which are available orally and could therefore overcome the difficulties encountered with bevacizumab. The investigator hypothesized that nintedanib, acting by indirect inhibition of the VEGF receptor should allow a reduction of epistaxis in HHT patient. Nintedanib has been used in one HHT patient following the diagnosis of Insterstitial Pulmonary Fibrosis (published case report in 2017, Kovacs et al) with encouraging results. The aim is to evaluate efficacy of nintedanib for the treatment of epistaxis in HHT patients
Pilot Study to Evaluate the Interest of PDL in the Management of Telangiectasia of the Face and...
TelangiectasiaThis single-center, prospective, open-label, quasi-experimental, intra-individual comparative study will include a consecutive cohort of 21 patients with diffuse or limited, minimally active scleroderma with 3 to 30 years of evolution. Patients will have 4 sessions of pulsed dye laser 595 nm spaced 8 weeks apart. The final quadruple evaluation by several evaluators will be 2 months after the last session, on the following criteria: evolution of the number of telangiectasia; subjective improvement score (LINKERT scale); impact on quality of life (SKINDEX score); visual analog pain scale (VAS); adverse events (AEs), including discontinuation of treatment due to post-session purpura (AT-PPS); patient satisfaction (yes or no).
Clinical Observation of Pulsed Dye Laser and Intense Pulsed Light in Treating Facial Telangiectasia...
TelangiectasiaFacial telangiectasia is one of the common skin vascular lesions characterized by dilated cutaneous vasculatures in the skin surface or mucous membranes. Lesions are generally sensitive to exposure of cold, heat and sun. Those distinct small dilated blood vessels are cosmetically disfiguring for patients which require an effective solution. The traditional management options for facial telangiectasia include cryotherapy, CO2 laser, topical agent, oral estrogens, electrosurgery, and radioactive treatment. However, inadequate outcomes and severe adversaries are the major concerns to patients and physicians. Pulsed dye laser (PDL) is effective for vasodilatory diseases, especially for the superficial to middle layers of the dermis. The intense pulsed light source is an alternative or supplement to the already existing laser devices that are part of the laser surgeon's repertoire. The broad wavelength spectrum and variable pulse duration allow greater penetration depths to be reached without damaging surrounding tissue and thus enhance the versatility of this system. This study aims to compare the clearance efficacy of facial telangiectasia using PDL (595nm) with IPL configured by various wavelength bands, including M22 vascular filter (530-650nm and 900-1200nm), M22 560 (560-1200nm), M22 590 (590-1200nm) .