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NCT ID: NCT04050436 Active, not recruiting - Clinical trials for Cutaneous Squamous Cell Carcinoma

Study Evaluating Cemiplimab Alone and Combined With RP1 in Treating Advanced Squamous Skin Cancer

CERPASS
Start date: October 8, 2019
Phase: Phase 2
Study type: Interventional

To estimate the clinical benefit of cemiplimab monotherapy versus cemiplimab in combination with RP1 for patients with locally advanced or metastatic CSCC, as assessed by overall response rate (ORR) and complete response rate (CRR) according to blinded independent review.

NCT ID: NCT04048369 Active, not recruiting - Influenza Clinical Trials

Impact of Influenza/RSV PCR Point-of-care Testing in the Emergency Medical Service.

Start date: January 17, 2019
Phase: N/A
Study type: Interventional

The purpose of the study is to compare Emergency Department patients who undergo influenza and RSV PCR testing using an FDA-approved point-of-care device (Cepheid Xpert® Xpress Flu/RSV) located in the ED, to patients who undergo influenza and RSV PCR testing at the core laboratory. The principal purpose is to determine if the time spent in the ED is different in the group undergoing POC influenza testing compared to those undergoing laboratory-based influenza testing.

NCT ID: NCT04045158 Active, not recruiting - Clinical trials for Muscular Dystrophies

Diaphragm Ultrasound in Neuromuscular Disorders

Start date: May 15, 2019
Phase:
Study type: Observational

Muscular dystrophies are neurologic disease with higher morbidity and mortality. Cardiac function and respiratory muscles can be affected, in addition with skeletal muscle deficiency . Lung function is classically assessed with functionnal pulmonary tests (FPT) . Ultrasound recently emerged as a non-invasive tool to assess diaphragm function. The aims of the study are: - to provide the spectrum of diaphragm ultrasound in patients with muscular dystrophies - to assess the correlation between diaphragm ultrasound and FPT - to assess the correlation between diaphragm ultrasound and cardiac function

NCT ID: NCT04044690 Active, not recruiting - Dermatomyositis Clinical Trials

A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of IgPro20 in Adults With Dermatomyositis (DM)

RECLAIIM
Start date: October 21, 2019
Phase: Phase 3
Study type: Interventional

This is a phase 3, multicenter, randomized, placebo-controlled, double-blind study of IgPro20 (subcutaneous Ig) treatment in adult subjects with dermatomyositis (DM). The primary objective of this study is to assess the efficacy of IgPro20 subcutaneous (SC) doses in comparison to placebo in adult subjects with DM, as measured by responder status based on Total Improvement Score (TIS) assessments.

NCT ID: NCT04042025 Active, not recruiting - SMA Clinical Trials

Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi

Start date: February 10, 2020
Phase: Phase 3
Study type: Interventional

This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi. Participants will roll over from their respective previous (parent) study into this long-term study for continuous monitoring of safety as well as monitoring of continued efficacy and durability of response to onasemnogene abeparvovec-xioi treatment.

NCT ID: NCT04041050 Active, not recruiting - Clinical trials for Myeloproliferative Neoplasm

A Study Evaluating Safety and Tolerability, and Pharmacokinetics of Navitoclax Monotherapy and in Combination With Ruxolitinib in Participants With Myeloproliferative Neoplasm

Start date: November 8, 2019
Phase: Phase 1
Study type: Interventional

There are 5 parts to this study for which the primary objectives are to evaluate safety, tolerability, and pharmacokinetics (PK) of navitoclax when administered alone (Part 1) or when administered in combination with ruxolitinib (Part 2). In Part 2, participants must have been receiving a stable dose of ruxolitinib therapy for at least 12 weeks prior to study enrollment. In Part 3, all eligible participants will receive navitoclax, with the primary objective being to evaluate potential navitoclax effect on QTc prolongation. In Part 4, effect of navitoclax is evaluated on the PK, safety, and tolerability of a single dose of celecoxib. In Part 5, all eligible participants will receive ruxolitinib twice daily and navitoclax once daily for drug-drug interaction (DDI) assessment, followed by continued administration of navitoclax in combination with ruxolitinib.

NCT ID: NCT04039607 Active, not recruiting - Clinical trials for Hepatocellular Carcinoma

A Study of Nivolumab in Combination With Ipilimumab in Participants With Advanced Hepatocellular Carcinoma

CheckMate 9DW
Start date: September 30, 2019
Phase: Phase 3
Study type: Interventional

The main purpose of this study is to compare the overall survival (OS) of nivolumab plus ipilimumab versus standard of care (SOC) (sorafenib or lenvatinib) in all randomized participants with advanced hepatocellular carcinoma (HCC) who have not received prior systemic therapy.

NCT ID: NCT04038138 Active, not recruiting - Muscular Dystrophy Clinical Trials

Clinical Trial Readiness Network FSHD France: Prospective 24 Months MRI Study

ReSOLVE_France
Start date: September 16, 2019
Phase: N/A
Study type: Interventional

The overall aim of this study is to hasten drug development for facioscapulohumeral muscular dystrophy (FSHD). Recent breakthroughs in FSHD research have identified the primary disease mechanism as the aberrant expression of a normally silenced gene, DUX4, resulting in a toxic gain-of-function. This disease mechanism is particularly amenable to knock-down of DUX4 using epigenetic strategies or RNA therapies, as well as to other interventions targeting the downstream effects of DUX4 expression. There are many drug companies actively working towards disease-targeted therapies, and two clinical trials either under way now, or planned to start in early Fall 2016. However, meetings with industry, advocacy groups, and FSHD researchers have identified several gaps in the clinical trial arsenal, and clinical trial planning as a major goal for the community. Consequently, there is an urgent need to establish the tools necessary for the conduct of currently planned and expected therapeutic trials in FSHD. To this end, the researchers propose to develop two novel clinical outcome assessments (COA), a composite functional outcome measure (FSH-COM) and skeletal muscle biomarker, electrical impedance myography (EIM). In addition there is broad consensus a better understanding of the relationship of genetic and demographic features to disease progression will be necessary for enumerating eligibility criteria. The specific aims are to: 1. Determine the multi-site validity of the COAs, 2. Compare the responsiveness of new COAs to other FSHD outcomes and determine the minimal clinically meaningful changes, and 3. establish FSHD cohort characteristics useful for determining clinical trial eligibility criteria. To achieve these aims, the Nice University Hospital is conducting a monocentric, prospective, 18 month study on 30 subjects.

NCT ID: NCT04036864 Active, not recruiting - Dysodia Clinical Trials

Singing-voice Disorders and Aerodynamic Profiles in Dysodic Singers

AeroDysodiaPro
Start date: March 1, 2019
Phase:
Study type: Observational

Singers are over-represented in phoniatric consultation; they present increased risk of developing voice disorders. They represent a specific population among phoniatric patients (specific singing-voice complaints, increased sensitivity to voice). Singing-voice disorder is called dysodia in the same way as dysphonia, which refers to speaking-voice disorders. The diagnosis of dysodia is made after a laryngeal examination and a voice assessment to identify patient's speech and singing difficulties. Most studies on prevalence of voice disorders in singers come from the American continent. Very few descriptions of this population have been made in France. Voice assessment of dysodic singers includes aerodynamic voice measurements. The use of aerodynamic measurements for the diagnosis of voice pathologies is now widely demonstrated. These parameters include measurements of estimated subglottal pressure (ESGP), phonation threshold pressure (PTP) (minimum pressure to initiate a sound) and oral airflow (OAF). They depend on the level of training of singers and the type of vocal dysfunction. Aerodynamic behaviours of dysodic singers have not yet been described, although they can help to better identify the singer's vocal difficulties and could be essential parameters for targeting more precisely rehabilitation exercises proposed in voice therapy. This study aims to describe the characteristics of a French population of singers consulting in phoniatrics and their aerodynamic behaviours. Do the singers' aerodynamic parameters (pressure, flow) reflect the dysfunctions in singing voice? Based on results of the literature on speaking and singing non-pathological voice, the investigators hypothesize that during voice assessment, ESGP values will be increased and OAF ones will be decreased over the entire vocal range of the singer in case of voice pathology.

NCT ID: NCT04036461 Active, not recruiting - Multiple Myeloma Clinical Trials

A Study of CC-99712, a BCMA Antibody-Drug Conjugate, in Participants With Relapsed and Refractory Multiple Myeloma

Start date: August 26, 2019
Phase: Phase 1
Study type: Interventional

Study CC-99712-MM-001 is an open-label, Phase 1, dose escalation (Part A) and expansion (Part B), First-in-Human (FIH) clinical study of CC-99712 in monotherapy or combination with BMS-986405 in participants with relapsed and refractory multiple myeloma (MM). The dose escalation part (Part A) of the study will evaluate the safety and tolerability of escalating doses of CC-99712, administered intravenously (IV) in monotherapy (Arm 1) or combination with BMS-986405 (Arm 2), to determine the maximum tolerated dose (MTD) of CC-99712 guided by a Bayesian logistic regression model (BLRM). A modified accelerated titration design will also be used for Arm 1 and Arm 2. The MTD may be established separately for CC-99712 administered at Q3W and/ or Q4W schedules. The expansion part (Part B) will further evaluate the safety and efficacy of CC-99712 in monotherapy (Arm 1) or combination (Arm 2) administered at or below the MTD in selected expansion cohorts in order to determine the RP2D. One or more doses or dosing regimens may be selected for cohort expansion. All participants will be treated until confirmed disease progression per IMWG criteria, unacceptable toxicity, or participants//Investigator decision to withdraw.