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NCT ID: NCT04259281 Active, not recruiting - Angelman Syndrome Clinical Trials

A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome

Start date: February 24, 2020
Phase: Phase 1/Phase 2
Study type: Interventional

The primary objective of the study is to evaluate the safety and tolerability of multiple-ascending doses of GTX-102 administered by intrathecal (IT) injection to participants with Angelman Syndrome (AS).

NCT ID: NCT04259138 Active, not recruiting - Colitis, Ulcerative Clinical Trials

Determination of the Optimal Treatment Target in Ulcerative Colitis

VERDICT
Start date: February 18, 2020
Phase: Phase 4
Study type: Interventional

Disease activity and response to therapy in ulcerative colitis (UC) can be assessed by a range of endpoints including symptoms, endoscopic mucosal activity, histological disease activity, and biomarkers. This study aims to determine the optimal treatment target, which is a research priority for the management of UC both to inform clinical practice and to help inform regulatory endpoints and targets for drug development. Participants with active UC will be randomized in a 5:4:1 (initially 2:3:5) ratio to 1 of 3 groups, each with a different treatment target. Treatment targets will be defined as: - Group 1: corticosteroid-free symptomatic remission - Group 2: corticosteroid-free endoscopic + symptomatic remission - Group 3: corticosteroid-free histological + endoscopic + symptomatic remission An interim analysis was performed to assess the proportion of subjects that reached their assigned treatment target after 50 subjects in each group had reached the first 32-week assessment. The interim analysis and projections made based on target achievement rates for all subjects included in the interim analysis resulted in a recommendation to adjust the randomization ratio from 2:3:5 to 5:4:1 for Groups 1, 2 and 3 respectively as of May 5th, 2023. This change was necessary in order to complete the study with approximately 100 subjects achieving treatment target within each group.

NCT ID: NCT04256772 Active, not recruiting - Cystic Fibrosis Clinical Trials

Anti-IL5 and Other Biotherapies in Cystic Fibrosis

MAB-CF
Start date: July 1, 2019
Phase:
Study type: Observational

Our project is to describe retrospectively and prospectively CF patients treated with biotherapy in French CF centers. Main objective: To describe the clinical and paraclinical course of CF patients before and after treatment with anti-IL5 and other biotherapies since 2019. Secondary objective: To describe adverse events potentially related to the biotherapies.

NCT ID: NCT04256213 Active, not recruiting - Cervix Cancer Clinical Trials

COL Immunotherapy Before Radiochimio + Ipilimumab

COLIBRI
Start date: July 2, 2020
Phase: N/A
Study type: Interventional

This is a multicenter, single arm pilot study evaluating the biological impact of "Nivolumab + Ipilimumab" in patients with cervical squamous cell carcinoma requiring RT-CT as initial therapy

NCT ID: NCT04255433 Active, not recruiting - Clinical trials for Type 2 Diabetes Mellitus

A Study of Tirzepatide (LY3298176) Compared With Dulaglutide on Major Cardiovascular Events in Participants With Type 2 Diabetes

SURPASS-CVOT
Start date: May 29, 2020
Phase: Phase 3
Study type: Interventional

The purpose of the trial is to assess the efficacy and safety of tirzepatide to dulaglutide in participants with type 2 diabetes and increased cardiovascular risk.

NCT ID: NCT04254094 Active, not recruiting - Dementia Clinical Trials

A Cohort Study of Early Onset Neurodegenerative Dementias Prognostic Factors

NEODEM
Start date: May 20, 2020
Phase:
Study type: Observational [Patient Registry]

NEODEM is a multicenter cohort study of patients with early-onset degenerative dementia (before age 65), the main objective of which is to study behavioral disorders and in particular depression as functional prognostic factors at 3 years.

NCT ID: NCT04253496 Active, not recruiting - Clinical trials for Primary Central Nervous System Lymphoma (PCNSL)

Mathematic Modeling at Micro and Macroscopic Level of Primary Central Nervous System Lymphomas (PCNSL)

LOC-MODEL
Start date: May 12, 2020
Phase:
Study type: Observational

We plan to analyze 100 PCNSL homogenously treated with high-dose methotrexate based chemotherapy using NGS of PCNSL samples. We will perform DNA-seq and RNA-seq from tumor samples. This data will be combined with their magnetic resonance imaging (MRI) at different time points: at diagnosis, at the end of the treatment and at disease progression. Among the 100 PCNSL that will be included, 70 will be from a retrospective (training set) from patients included in the French National PCNSL dataset (LOC cohort) and 30 PCNSL from a prospective cohort from patients included in a phase III clinical trial (BLOCAGE, PHRC 2014). On the one hand, we will perform a radiomics analysis (quantitative imaging) using 3D tumor and edema segmentation. This analysis will help us to elucidate the potential correlation of MRI phenotypes and genotype (using high-throughput data). In addition, we will use the radiomics data combined with in vitro and in vivo data (using a mouse model of PCNSL) as well as immunohistochemistry data to obtain a multidimensional mathematical modeling of PCNSL clinical evolution that will allow us to better predict the clinical course of this rare subtype of brain tumor.

NCT ID: NCT04253184 Active, not recruiting - Bradycardia Clinical Trials

Micra AV Transcatheter Pacing System Post-Approval Registry

Micra AV PAS
Start date: February 8, 2020
Phase:
Study type: Observational [Patient Registry]

Medtronic is sponsoring the Micra AV Registry using the Micra AV system for continued surveillance of chronic atrioventricular synchronous pacing as intended, through the collection of data based on routine clinical care practice, following commercial release. The Micra AV Registry is conducted within Medtronic's Product Surveillance Registry (PSR) platform.

NCT ID: NCT04253106 Active, not recruiting - Clinical trials for Hereditary Diffuse Gastric Cancer

Liquid Biopsies for the Personalized Management of Patients With Hereditary Diffuse Gastric Cancer

LISA-HDGC
Start date: November 26, 2020
Phase: N/A
Study type: Interventional

Activating somatic mutations and methylation profiles identified by liquid biopsies could identify CDH1 and CTNNA1 pathogenic variants carriers with invasive diffuse gastric cancer undetectable by upper G-I endoscopy.

NCT ID: NCT04251533 Active, not recruiting - Clinical trials for Triple Negative Breast Neoplasms

Study Assessing the Efficacy and Safety of Alpelisib + Nab-paclitaxel in Subjects With Advanced TNBC Who Carry Either a PIK3CA Mutation or Have PTEN Loss

EPIK-B3
Start date: June 8, 2020
Phase: Phase 3
Study type: Interventional

The purpose of this study is to determine whether treatment with alpelisib in combination with nab-paclitaxel is safe and effective in subjects with advanced triple negative breast cancer (aTNBC) who carry either a PIK3CA mutation (Study Part A) or have PTEN loss (Study Part B1) or PTEN loss without PIK3CA mutation (Study Part B2)