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NCT ID: NCT05035030 Active, not recruiting - Alagille Syndrome Clinical Trials

Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome

ASSERT-EXT
Start date: September 3, 2021
Phase: Phase 3
Study type: Interventional

An Open Label Study to Evaluate the Long-term Safety and Efficacy of Odevixibat (A4250) in Patients with Alagille Syndrome (ASSERT-EXT)

NCT ID: NCT05032066 Active, not recruiting - Clinical trials for Idiopathic Pulmonary Fibrosis

A Multicenter Trial to Evaluate the Efficacy, Safety and Tolerability of HZN-825 in Subjects With Idiopathic Pulmonary Fibrosis

Start date: August 25, 2021
Phase: Phase 2
Study type: Interventional

HZNP-HZN-825-303 (HARBOR) comprises of 2 parts. Part 1 (Core Phase) is a randomized, double-blind, placebo-controlled, repeat-dose, multicenter trial to evaluate the efficacy, safety and tolerability of HZN-825 in participants with Idiopathic Pulmonary Fibrosis (IPF). Part 2 (Extension Phase) is an optional, open-label, repeat-dose, multicenter extension of the Core Phase. The trial will include up to an 8-week Screening Period and a 52-week Double-blind Treatment Period in the Core Phase and 52 weeks of open-label HZN-825 treatment in the Extension Phase. During the Core Phase, participants will be screened within 8 weeks prior to the baseline (Day 1) Visit. Approximately 135 participants who meet the trial eligibility criteria will be randomly assigned in a 1:1:1 ratio on Day 1 to receive HZN-825 300 mg QD, HZN-825 300 mg BID or matching placebo orally for 52 weeks using the following 2 stratification factors: 1. Concomitant use of approved IPF therapy (i.e., nintedanib or pirfenidone): yes or no 2. Forced vital capacity (FVC) % predicted at Baseline: ≥70% or <70% Participants who complete the 52-week Double blind Treatment Period of the Core Phase of the trial will be invited to extend their participation in the 52-week Extension Phase of the trial.

NCT ID: NCT05030428 Active, not recruiting - Clinical trials for Atherosclerotic Cardiovascular Disease

Study of Inclisiran to Prevent Cardiovascular (CV) Events in Participants With Established Cardiovascular Disease

VICTORION-2P
Start date: November 23, 2021
Phase: Phase 3
Study type: Interventional

Study CKJX839B12302 is a pivotal Phase III trial to evaluate the benefits of inclisiran on major adverse cardiovascular (MACE) events in participants with established cardiovascular disease (CVD).

NCT ID: NCT05027802 Active, not recruiting - Clinical trials for Fibrodysplasia Ossificans Progressiva (FOP)

A Rollover Study to Further Evaluate the Safety and Efficacy of Palovarotene Capsules in Male and Female Participants Aged ≥14 Years With Fibrodysplasia Ossificans Progressiva (FOP) Who Have Completed the Relevant Parent Studies.

PIVOINE
Start date: March 14, 2022
Phase: Phase 3
Study type: Interventional

The main objective of this study is to further evaluate the safety and efficacy of palovarotene in adult and paediatric participants with FOP. The aim of the study is also to ensure treatment continuity to participants who have completed one of the parent studies (Study PVO-1A-301, Study PVO-1A-202 and Study PVO-1A-204) and who, in the investigator's judgement, may benefit from palovarotene therapy.

NCT ID: NCT05027074 Active, not recruiting - Clinical trials for Kidney Failure, Chronic

Global Study of MK-2060 (Anti-Factor XI Monoclonal Antibody) in Participants With End Stage Renal Disease Receiving Hemodialysis (FXI Hemodialysis Study) (MK-2060-007)

Start date: September 17, 2021
Phase: Phase 2
Study type: Interventional

The purpose of this study is to evaluate the efficacy and safety of two different doses of MK-2060 (a monoclonal antibody against Factor XI) in end stage renal disease (ESRD) participants receiving hemodialysis via an arteriovenous graft (AVG). Data from this study will be used to aid dose selection of MK-2060 in future studies. The primary hypothesis is that at least one of the MK-2060 doses is superior to placebo in increasing the time to first occurrence of AVG event.

NCT ID: NCT05024045 Active, not recruiting - Multiple Myeloma Clinical Trials

Study of Oral LOXO-338 in Patients With Advanced Blood Cancers

Start date: September 30, 2021
Phase: Phase 1
Study type: Interventional

The purpose of this study is to find out whether the study drug, LOXO-338, is safe and effective in patients with advanced blood cancer. Patients must have already received standard therapy. The study may last up to approximately 3 years.

NCT ID: NCT05023980 Active, not recruiting - Clinical trials for Chronic Lymphocytic Leukemia

A Study of Pirtobrutinib (LOXO-305) Versus Bendamustine Plus Rituximab (BR) in Untreated Patients With Chronic Lymphocytic Leukemia (CLL)/Small Lymphocytic Lymphoma (SLL)

BRUIN CLL-313
Start date: September 23, 2021
Phase: Phase 3
Study type: Interventional

The purpose of this study is to compare the efficacy and safety of pirtobrutinib (LOXO-305; Arm A) compared to BR (Arm B) in patients with CLL/SLL who have not been treated. Participation could last up to five years.

NCT ID: NCT05021536 Active, not recruiting - Clinical trials for Amyotrophic Lateral Sclerosis

Phase III Trial of AMX0035 for Amyotrophic Lateral Sclerosis Treatment

Phoenix
Start date: October 28, 2021
Phase: Phase 3
Study type: Interventional

The Phoenix Trial is a randomized double blind placebo controlled Phase III trial to evaluate the safety and efficacy of AMX0035 for treatment of ALS

NCT ID: NCT05019521 Active, not recruiting - Geographic Atrophy Clinical Trials

A Study of Danicopan in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration

Start date: August 23, 2021
Phase: Phase 2
Study type: Interventional

This is a dose finding study designed to evaluate the efficacy, safety, and pharmacokinetics of danicopan in participants with GA secondary to AMD. The study consists of a Screening Period of up to 6 weeks, a 104-week masked Treatment Period, followed by a 30-day Follow-up after the last dose. This study will have 4 treatments arms: 100 milligrams (mg) twice daily (bid), 200 mg bid, 400 mg once daily (qd), and matching placebo.

NCT ID: NCT05019313 Active, not recruiting - COVID-19 Pneumonia Clinical Trials

Diaphragm Ultrasound Evaluation During Weaning From Mechanical Ventilation in the Positive COVID-19 Patient

Start date: April 1, 2020
Phase:
Study type: Observational

Hypoxemic acute respiratory failure is one of the main COVID-19 patients complication that lead to in intensive care hospitalization. This complication determines a variable mortality from 25 to 30%. To correct hypoxemia (often severe) is often needed non-invasive or invasive mechanical ventilation. Mechanical ventilation is not a therapeutic strategy, but it allows to extend the time-to-recovery necessary to solve COVID-19 respiratory failure cause. Calibration of ventilatory support is essential to ensure adequate time-to-recovery without contributing to onset lung and / or diaphragmatic damage. Basal diaphragmatic activity assessment, device for administering the oxygenation support choice and setting ventilatory support parameters are decisive. Ultrasound is the best method for measuring diaphragmatic work. The aim of this study is to evaluate the diaphragmatic thickening fraction in COVID-19 patients admitted to Intensive Care Unit (ICU) for acute respiratory failure and to record its function on weaning.