Vivek N. Iyer, M.D., M.P.H.

The purpose of this study with a 28-week Open-label Extension of BLU-5937 in adult (18 to 80 years of age) participants who have had persistent cough for ≥ 1 year prior to Screening, with a diagnosis of RCC defined as:

• insufficient improvement in cough after treatment for the underlying condition(s) causing the cough; OR
• unexplained cough for which an underlying condition has not been determined despite adequate investigation with diagnostic tests and trials of therapy.

The objective of Part A of this study is to assess the pharmacokinetics (PK) of pazopanib in Hereditary Hemorrhagic Telangiectasia (HHT) participants.  The objective of Parts B and C of this study is to assess the effects of up to 24 weeks of pazopanib treatment on duration of epistaxis or level of serum hemoglobin in the Severe Cohort.

The purpose of this study is to evaluate the effectiveness of GDC-6599, as compared with placebo, in patients with RCC with asthma or UCC. Also, to evaluate mannitol-induced cough response as a measure of TRPA1 airway activity and treatment benefit to GDC-6599, and to evaluate mannitol-induced cough response as a measure of TRPA1 airway activity and treatment benefit to GDC-6599, as compared with placebo. 

The purpose of this study is to evaluate the effectiveness of two escalating doses of Duloxetine & Amitriptyline in reducing awake objective cough frequency (at 4 & 8 weeks).

The purpose of this study is to evaluate the effectiveness of two escalating doses of Duloxetine & Amitriptyline in reducing awake objective cough frequency (at 4 & 8 weeks).

The purpose of this study is to develop a data collection registry with a large cohort of Hereditary Hemorrhagic Telangiectasia (HHT) patients with a large cohort of HHT patients that will be followed over time.  

The purpose of this study is to assess the safety and tolerability of escalating doses of NAL ER in patients with IPF. Also, to evaluate the pharmacodynamic (PD) effect of escalating doses of NAL ER and nalbuphine plasma concentrations on Minute Ventilation (MV), determined by ExSpiron®.

The purpose of this study is to assess the effectiveness and safety of AF-219 given to subjects with a treatment resistant chronic cough.

The purpose of this study is to evaluate the dose-response of bradanicline in reducing awake objective cough frequency.

The purpose of this study is to determine the effectiveness of three doses of orvepitant, taken once a day, in the treatment of chronic refractory cough.

ORV-PF-01 is a two way, placebo controlled, cross-over study, to evaluate the effect of two doses of orvepitant on cough in patients with Idiopathic Pulmonary Fibrosis (IPF).

The purpose of this study is to evaluate the safety, tolerability, and effectiveness of ADX-629 300 mg administered orally BID for 14 days to approximately 50 adult subjects with refractory or unexplained chronic cough.

The purpose of this study is to develop a repository of blood and tissue from patients with epistaxis and GI bleeding secondary to the conditions described above (HHT, GAVE, SBA and OGIB).

The purpose of this study is to evaluate the sasfety and effectiveness of MK-7264 in reducing cough frequency as measured over a 24-hour period.

This study is aimed at understanding whether oral nitric oxide levels can be used as a non-invasive marker for the diagnosis and management of patient with eosinophilic esophagitis (EoE).

The purpose of this study is to assess the safety and effectiveness of AF-219 in patients with idiopathic pulmonary fibrosis with persistent cough.

Study of the efficacy, safety, and tolerability of serlopitant for the treatment of refractory chronic cough.

The purpose of this study is to demonstrate the effectiveness and safety of multiple doses of BLU-5937 in adults suffering from RCC. The study will help determine the optimal BLU-5937 dose for Phase 3 studies.

The purpose of this study is to establish whether therapeutic-dose parenteral anticoagulation improves outcomes (reduces intubation or mortality) by 30 days after randomization.

This study is one of the three projects of an NIH Rare Disease Clinical Research Consortium. A "consortium" is a group of centres sharing information and resources to perform research. The consortium research focuses on brain blood vessel malformations in three different rare diseases.

The focus of this specific study is on Hemorrhagic Telangiectasia (HHT).

HHT is a condition characterized by blood vessel malformations, called telangiectasia and arteriovenous malformations (AVMs), occurring in the brain, nose, lungs, stomach, bowels and liver. Brain AVMs (BAVMs) in HHT are difficult to study because they are rare, affecting approximately 10% of people with HHT. While other types of BAVMs have been studied in depth, studies in the HHT population have been very small. Here, we propose the first large-scale collaboration by joining with 12 HHT Centers of Excellence in North America to perform a large study of risk factors for bleeding from BAVMs, called intracranial hemorrhage (ICH) in HHT patients.

The current standard of clinical practice across North America, is to screen all HHT patients for BAVMs with magnetic resonance imaging (MRI). If BAVMs are detected, patients are referred to a multidisciplinary neurovascular team for consideration for treatment. Treatment decisions are made on a case by case basis, balancing risks of complications from the BAVM with risks of therapy, but are limited by the few studies available in HHT. We hope that the knowledge we obtain about the risk factors for intracranial bleeding in these patients from this larger study will help us to improve the care of HHT patients.

We plan to study risk factors for rupture of BAVMs, including primarily genetics and imaging characteristics of the BAVMs. Knowledge about risk factors will help in the care and management of HHT patients. This will be achieved through the collection of health information to construct a HHT database, blood sampling and banking (through the National Institute of Neurological Disorders and Stroke [NINDS]), and through genetic analysis at the University of California San Francisco.

The purpose of this study is to evaluate pomalidomide vs. placebo in patients with hereditary hemorrhagic telangiectasia (HHT) with moderate-to-severe epistaxis who require parenteral iron infusions or blood transfusions. A total of 159 patients will be randomized 2:1 to treatment with oral pomalidomide or matching placebo for 24 weeks. Mean change from baseline to 24 weeks in the Epistaxis Severity Score (ESS) will be compared between treatment groups to determine pomalidomide efficacy.

This study aims to evaluate each agent with respect to speed of recovery, mortality, illness severity, and hospital resource utilization. Each agent will be evaluated as add-on therapy to the standard of care (SoC) in accordance with national or local guidelines, which may include remdesivir. 

The purpose of this study is to evaluate Inhaled RVT-1601 (formerly, PA101B), a new inhalation formulation of cromolyn sodium delivered via the eFlow® Closed System (CS) nebulizer, for the treatment of persistent cough in patients with idiopathic pulmonary fibrosis (IPF).

The study aims to identify predictors of brain outcomes in Hereditary Hemorrhagic Telangiectasia (HHT) patients. The study will use the modified Rankin Score (mRS). Furthermore to define a severe bleeding phenotype in HHT for clinical trial readiness.