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NCT ID: NCT05121376 Active, not recruiting - Clinical trials for Hereditary Angioedema

A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema

HAErmony-1
Start date: February 15, 2022
Phase: Phase 1/Phase 2
Study type: Interventional

This is a Phase 1/2, single-arm, open-label, dose-escalation and dose-expansion study of BMN 331 for the treatment of hereditary angioedema (HAE) due to C1 Esterase Inhibitor (C1-INH) protein deficiency. The study drug BMN 331is identified as AAV5 hSERPING1, an adeno-associated virus (AAV5)-based gene therapy vector that expresses wild-type human C1 Esterase Inhibitor (hC1-INH), under the control of a liver-selective promoter, and is being developed for the treatment of HAE with C1-INH deficiency. The pharmaceutical form of BMN 331 is a solution for intravenous infusion.

NCT ID: NCT05121246 Completed - Healthy Volunteers Clinical Trials

Pharmacokinetic Study Comparing MB05 (Proposed Palivizumab Biosimilar), EU-sourced Synagis® and US-sourced Synagis® in Healthy Volunteers.

Start date: June 15, 2022
Phase: Phase 1
Study type: Interventional

Randomized, double blind, parallel group, single dose, 3 arm study to investigate and compare the pharmacokinetics (PK), safety, immunogenicity and tolerability of MB05 with US and EU Synagis® in healthy subjects. During the course of the study, the similarity in pharmacokinetics will be assessed by sampling the levels of drug in the blood, and by comparing these levels among the different administration arms. Safety, tolerability, and immunologic response to the administered drugs will also be evaluated throughout.

NCT ID: NCT05120856 Completed - Clinical trials for Alcohol Use Disorder (AUD)

AAT-App Outpatient Trial

AAT-App
Start date: May 5, 2022
Phase: N/A
Study type: Interventional

Cognitive biases contribute to the difficulty experienced by heavy drinkers wishing to reduce their alcohol use. Recent interventions designed to reduce cognitive biases demonstrate efficacy for Approach Bias Modification (ApBM). Reductions in the likelihood of relapse have been found after ApBM in Alcohol Use Disorder (AUD) patients during residential treatment. Current methods of ApBM are usually delivered by computer and joystick and come with several limitations, including accessibility. If ApBM could be shown to be feasible in other settings, such as outpatient treatment, it could benefit a much larger population with AUD. This randomised controlled trial will test the efficacy of a recently-developed ApBM smartphone app called "AAT-App" ("Alcohol Avoidance Training App"). We aim to test whether AAT-App, relative to a minimal version of the app which excludes ApBM training, is effective at reducing alcohol use, cravings, severity of dependence, and approach bias (a measure of a person's automatic tendency to automatically approach alcohol-related stimuli), and to explore user experiences of AAT-App to guide future improvements to the app and its implementation.

NCT ID: NCT05120830 Active, not recruiting - Clinical trials for Hereditary Angioedema

NTLA-2002 in Adults With Hereditary Angioedema (HAE)

NTLA-2002
Start date: December 10, 2021
Phase: Phase 1/Phase 2
Study type: Interventional

This study will be conducted to evaluate the safety, tolerability, activity, pharmacokinetics, and pharmacodynamics of NTLA-2002 in adults with Hereditary Angioedema (HAE).

NCT ID: NCT05119686 Completed - Gout Clinical Trials

Phase 2b Evaluation of Efficacy and Safety of AR882 in Gout Patients

Start date: November 16, 2021
Phase: Phase 2
Study type: Interventional

This study will assess the serum uric acid lowering effect and safety of AR882 in gout patients at two doses compared to placebo over 12 weeks.

NCT ID: NCT05118789 Recruiting - Clinical trials for Metastatic Solid Tumor

A Study of NVL-520 in Patients With Advanced NSCLC and Other Solid Tumors Harboring ROS1 Rearrangement (ARROS-1)

Start date: January 4, 2022
Phase: Phase 1/Phase 2
Study type: Interventional

Phase 1/2, dose escalation and expansion study designed to evaluate the safety and tolerability of NVL-520, determine the recommended phase 2 dose (RP2D), and evaluate the antitumor activity in patients with advanced ROS1-positive (ROS1+) NSCLC and other advanced ROS1-positive solid tumors. Phase 1 will determine the RP2D and, if applicable, the maximum tolerated dose (MTD) of NVL-520 in patients with advanced ROS1-positive solid tumors. Phase 2 will determine the objective response rate (ORR) as assessed by Blinded Independent Central Review (BICR) of NVL-520 at the RP2D. Secondary objectives will include the duration of response (DOR), time to response (TTR), progression-free survival (PFS), overall survival (OS), and clinical benefit rate (CBR) of NVL-520 in patients with advanced ROS1-positive NSCLC and other solid tumors.

NCT ID: NCT05117723 Recruiting - IPMN Clinical Trials

Intraductal Papillary Mucinous Neoplasm (IPMN) Database - A Tool to Predict Pancreatic Cancer

MAPS
Start date: August 26, 2021
Phase:
Study type: Observational [Patient Registry]

Pancreatic cancer is the 5th leading cause of cancer death in Australia. Surgery remains the most effective treatment for early pancreatic cancer and currently the only potential for cure. Unfortunately, many patients present with advanced disease and are not suitable for surgery. Therefore, it is vital to detect these cancers early. In the absence of significant data from prospective studies, all of the guidelines are based on a critical review of available data and consensus of experts. The primary aim is to delineate the progression of IPMN to pancreatic malignancy as confirmed by surgical pathology, radiology and biochemical diagnosis. The secondary aims are (i) To outline the management of IPMNs for those who have progressed straight to surgery or surveillance by endoscopic ultrasound (EUS) (ii)To validate the International consensus guidelines for management of IPMN - Fukuoka consensus guidelines and tertiary aim to identify potential risk factors, if any that increase risk of malignancy within the IPMNs.

NCT ID: NCT05117476 Recruiting - Clinical trials for Advanced Solid Tumor

A Study of CLN-619 Alone and in Combination With Pembrolizumab in Advanced Solid Tumors

Start date: October 29, 2021
Phase: Phase 1
Study type: Interventional

CLN-619-001 is a Phase 1, open-label, multi-center study of CLN-619 alone and in combination with pembrolizumab in patients with advanced solid tumors.

NCT ID: NCT05117060 Completed - Atopic Dermatitis Clinical Trials

Efficacy and Safety of LEO 152020 Tablets for the Treatment of Adults With Moderate to Severe Atopic Dermatitis

Start date: December 13, 2021
Phase: Phase 2
Study type: Interventional

This is an up to 22-week clinical study in adult participants with moderate to severe atopic dermatitis (AD). The purpose of the study is to test a new tablet (LEO 152020) to see if it improves AD and what the side effects are when compared with a placebo tablet with no medical ingredient. During the study, there will be a 16-week treatment period during which the participants will be asked to take the tablets. The participants will regularly visit the clinic for tests and the study doctor will evaluate their AD. The participants will also be asked to answer questions about their AD symptoms, itch, sleep, and quality of life.

NCT ID: NCT05116865 Completed - Clinical trials for COVID-19 Respiratory Infection

A Study to Assess the Safety, Tolerability and Pharmacokinetics of Inhaled HH-120 Aerosol in Healthy Volunteers

Start date: November 1, 2021
Phase: Phase 1
Study type: Interventional

This is a Phase 1, randomized, double-blinded, placebo controlled, dose escalation study of HH-120 in healthy adult volunteers. HH-120 is a novel inhalable biologic being developed for COVID-19 treatment. The study aims to evaluate the safety, tolerability and pharmacokinetic profile of HH-120 administered by aerosol inhalation after single and multiple ascending doses.