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NCT ID: NCT04850339 Completed - Healthy Clinical Trials

First in Human Study of ANXV (Recombinant Human Annexin A5) in Healthy Volunteers

Start date: December 21, 2020
Phase: Phase 1
Study type: Interventional

This is an adaptive, randomised, double-blind, single-centre, placebo-controlled phase I, First in Human study designed to evaluate the safety, tolerability and pharmacokinetics of single and multiple intravenous dosing of ANXV in healthy male subjects.

NCT ID: NCT04849741 Recruiting - Alexander Disease Clinical Trials

A Study to Evaluate the Safety and Efficacy of ION373 in Patients With Alexander Disease (AxD)

Start date: June 1, 2021
Phase: Phase 3
Study type: Interventional

The purpose of this study is to evaluate the safety and efficacy of zilganersen (ION373) in improving or stabilizing gross motor function across the full range of affected domains in patients with AxD.

NCT ID: NCT04849728 Recruiting - Clinical trials for NASH - Nonalcoholic Steatohepatitis

A Phase 3 Study Evaluating Efficacy and Safety of Lanifibranor Followed by an Active Treatment Extension in Adult Patients With (NASH) and Fibrosis Stages F2 and F3 ( NATiV3 )

NATiV3
Start date: August 19, 2021
Phase: Phase 3
Study type: Interventional

This Phase 3 study is conducted to evaluate lanifibranor in adults with NASH and liver fibrosis histological stage F2 or F3

NCT ID: NCT04849390 Completed - Clinical trials for Eosinophilic Esophagitis

A Study to Investigate the Efficacy and Tolerability of ESO-101 in Patients With Eosinophilic Esophagitis

Start date: June 29, 2021
Phase: Phase 2
Study type: Interventional

This is a randomized, placebo-controlled, double-blind trial to evaluate the efficacy, tolerability, and safety of ESO-101 in adult patients with active eosinophilic esophagitis (EoE). Patients will be screened at 2 visits (Visit 1 and Visit 2) during which their eligibility will be assessed based on endoscopy-independent criteria (Visit 1) and based on the histologic assessment of esophageal biopsy samples taken during the screening endoscopy (Visit 2). Eligible patients will be randomized 2:1 to once-daily treatment with ESO-101 or placebo and treated for 28 days starting on Day 0. Further clinic visits will be performed at Day 14 (Visit 4) and Day 28 (Visit 5, end of treatment) to assess the efficacy, tolerability, and safety. In addition, a safety follow-up call will be scheduled 2 weeks after the end of treatment (Day 42, Visit 6).

NCT ID: NCT04848779 Recruiting - Clinical trials for Glycogen Storage Disease Type II

A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)

Start date: June 10, 2021
Phase:
Study type: Observational [Patient Registry]

Primary Objective: To describe the effect of routine practice with alglucosidase alfa in patients with IOPD ≤6 months of age, on invasive ventilation-free survival after 52 weeks of treatment. Secondary Objectives: - To describe the effect of routine practice with alglucosidase alfa on invasive ventilation-free survival and survival at 12 and 18 months of age, as well as on change in left ventricular mass (LVM) Z score, Alberta Infant Motor Scale (AIMS) score, body weight, body length, and head circumference Z scores, and urinary glucose tetrasaccharide (Hex4), at Week 52 of treatment. - To describe the safety, tolerability, and immunogenicity of alglucosidase alfa in the routine practice of IOPD treatment.

NCT ID: NCT04848558 Completed - Healthy Clinical Trials

A Study of Nipocalimab in Healthy Male and Female Participants

Start date: May 25, 2021
Phase: Phase 1
Study type: Interventional

The purpose of this study is to evaluate the safety and tolerability of single and multiple doses of nipocalimab following subcutaneous (SC) administration compared with intravenous (IV) administration in healthy participants.

NCT ID: NCT04848506 Enrolling by invitation - Clinical trials for Symptomatic Hypertrophic Cardiomyopathy (HCM)

Open-label Extension Study to Evaluate the Long-term Safety and Tolerability of Aficamten in Adults With HCM

FOREST-HCM
Start date: May 6, 2021
Phase: Phase 2
Study type: Interventional

The purpose of this study is to collect long-term safety and tolerability data for aficamten (CK-3773274)

NCT ID: NCT04848480 Completed - Clinical trials for Diabetes Mellitus, Type 1

A Research Study to Compare a New Weekly Insulin, Insulin Icodec, and an Available Daily Insulin, Insulin Degludec, Both in Combination With Mealtime Insulin in People With Type 1 Diabetes (ONWARDS 6)

ONWARDS 6
Start date: April 30, 2021
Phase: Phase 3
Study type: Interventional

This study compares insulin icodec (a new insulin) to insulin degludec (an insulin already available on the market) in people with type 1 diabetes. The study will look at how well insulin icodec taken weekly controls blood sugar compared to insulin degludec taken daily. Participants will either get insulin icodec that participants will have to inject once a week on the same day of the week, or insulin degludec that participants will have to inject once a day at the same time every day. Which treatment participants get is decided at random. Participants will also get a mealtime insulin. The insulin is injected with a needle in a skin fold in the thigh, upper arm or stomach. The study will last for about 1 year and 2 months. Participants will have 28 clinic visits and 28 phone calls with the study doctor. At 11 clinic visits participants will have blood samples taken. At 6 clinic visits participants cannot eat or drink (except for water) for 8 hours before the visit. Participants will be asked to wear a sensor that measures your blood sugar all the time. Participants will be asked to wear it for a total of 57 weeks (around 1 year). Women cannot take part if pregnant, breast-feeding or plan to become pregnant during the study period.

NCT ID: NCT04848220 Terminated - Clinical trials for Microvascular Obstruction

A Study Evaluating the Safety, Tolerability, and Effect on Microvascular Obstruction of Intravenous Temanogrel in Adult Participants Undergoing Percutaneous Coronary Intervention

Start date: May 20, 2021
Phase: Phase 2
Study type: Interventional

The purpose of this study is to determine whether intravenous temanogrel is a safe and effective treatment for microvascular obstruction (MVO) in adult participants undergoing percutaneous coronary intervention (PCI).

NCT ID: NCT04848077 Recruiting - Parkinson Disease Clinical Trials

STEPWISE Parkinson: A Smartphone Based Exercise Solution for Patients With Parkinson's Disease

STEPWISE
Start date: May 18, 2021
Phase: N/A
Study type: Interventional

The aim of this study is to investigate whether a smartphone app can increase physical activity in patients with Parkinson's Disease in daily life for a long period of time (12 months).