EDS in Ataxia Telangiectasia Patients
Nervous System DiseaseGenetic SyndromeThis is an international, multi-center, one-year, randomized, prospective, double-blind, placebo-controlled, phase III study, designed to assess the effect of two non-overlapping dose ranges of EDS EP, administered by IV infusion once per month, on neurological symptoms of patients with Ataxia Telangiectasia.
MacTel Laser Study
Macular TelangiectasiaThis is a two armed Single Masked Pilot Study enrolling participants with Macular Telangiectasia Type 2 who will be randomized to undergo either a subthreshold photothermal treatment or a sham procedure to one eye. The participants will be followed for one year with visits at 1 month, 3 months, 6 months and 12 months post-treatment (laser or sham). Due to the small number of participants enrolled in this study the data will be analyzed by descriptive statistics and exploratory figures. Summary statistics will be produced for study and fellow eyes.
Intranasal Bevacizumab for HHT-Related Epistaxis
HHTHereditary Hemorrhagic Telangiectasia3 moreThis 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.
A Phase 2 Multicenter Randomized Clinical Trial of CNTF for MacTel
Macular Telangiectasia Type 2This study is a phase 2, randomized, multi-center, single-masked study to evaluate the efficacy and safety of the NT-501 implants in participants with Mactel.
Study of the 532 nm KTP Laser for the Treatment of Poikiloderma of Civatte
Poikiloderma of CivatteDyschromia3 moreThis study will evaluate the safety and efficacy of the Excel V 532 nm KTP laser for the treatment of dyschromia of the neck and/or chest (poikiloderma of Civatte).
Topical Bevacizumab for the Management of Recurrent Epistaxis in Patients With Hereditary Hemorrhagic...
Hereditary Hemorrhagic Telangiectasia (HHT)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).
Efficacy and Safety of Bevacizumab for the Treatment Hemorrhagic Hereditary Telangiectasia (HHT)...
Hemorrhagic Hereditary TelangiectasiaThe efficacy of anti-VEGF treatments such as Bevacizumab in cases of HHT can be considered because of the molecular mechanisms implied in angiogenesis and HHT, as well as the mechanisms of action of this type of treatment. Two articles that have recently reported spectacular improvement thanks to Bevacizumab in patients with HHT complicated with severe liver involvement and cardiac effects support us in this sense. Up to now, the only treatment recommended in the severe hepatic forms of HHT is a liver transplant, the disadvantages of which are both multiple and well known: long waiting lists, surgical morbidity and mortality, immunosuppressive treatment for life. Furthermore, treatment with Bevacizumab is not a contraindication, should the drug be ineffective, for a subsequent liver transplant if necessary.
Ranibizumab to Treat Type 2 Idiopathic Macular Telangiectasia (RAMA-Trial)
Type 2 Idiopathic Macular TelangiectasiaThe purpose of this study is to investigate if a new drug called ranibizumab is effective to treat a rare bilateral disease of the macula: type 2 idiopathic macular telangiectasia (type 2 IMT). 10 patients will receive monthly injections of the drug into one eye over a period of one year.
Intranasal Submucosal Bevacizumab for Epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT)
HHTMorbus Osler1 moreIn 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.
Thalidomide Reduces Arteriovenous Malformation Related Gastrointestinal Bleeding
Arteriovenous MalformationHereditary Hemorrhagic Telangiectasia2 moreThe purpose of this study is to determine whether Thalidomide is effective in the treatment of arteriovenous malformations in the gastrointestinal tract.