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NCT ID: NCT04912869 Recruiting - Sickle Cell Disease Clinical Trials

A Study Evaluating the Safety, Pharmacokinetics, Pharmacodynamics and Efficacy of Crovalimab for the Management of Acute Uncomplicated Vaso-Occlusive Episodes (VOE) in Participants With Sickle Cell Disease (SCD).

CROSSWALK-a
Start date: March 26, 2022
Phase: Phase 1
Study type: Interventional

The purpose of this study is to evaluate crovalimab for the treatment of a sickle cell pain crisis (also known as a VOE) that requires hospitalisation in adult and adolescent participants with SCD. The primary objective of this study is safety and will additionally evaluate pharmacokinetics (how crovalimab is processed by your body), pharmacodynamics (how your body reacts to crovalimab) and the preliminary efficacy of crovalimab compared with placebo.

NCT ID: NCT04910776 Recruiting - Clinical trials for Glycogen Storage Disease Type II

Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa

Baby-COMET
Start date: September 1, 2021
Phase: Phase 3
Study type: Interventional

This is a single group, treatment, Phase 3, open-label study to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in treatment-naïve male and female participants with IOPD. Study details include: - Study duration: Screening - up to 4 weeks; - Primary Analysis Period (PAP) - 52 weeks; - Extended Treatment Period (ETP) - 52 weeks; - Extended Long term Treatment Period (ELTP) - 104 weeks; 4-week follow-up period for a total study duration - up to 4.08 years. - Treatment duration: Up to 4 years - Visit frequency: every other week and potentially every week

NCT ID: NCT04910685 Recruiting - Clinical trials for Indolent Systemic Mastocytosis

(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis

Start date: November 30, 2021
Phase: Phase 2/Phase 3
Study type: Interventional

This is a randomized, double-blind, placebo-controlled, Phase 2/3 study comparing the efficacy and safety of BLU-263 + best supportive care (BSC) with placebo + BSC in patients with indolent systemic mastocytosis (ISM) whose symptoms are not adequately controlled by BSC. Parts 1 and 2 will enroll patients with ISM. Patients enrolled in Part 1 or Part 2 will roll over onto Part 3 to receive treatment with BLU-263 in an open-label fashion following completion of the earlier Part. Part M will enroll patients with monoclonal mast cell activation syndrome (mMCAS). The study also includes PK groups that will enroll patients with ISM.

NCT ID: NCT04910568 Recruiting - Multiple Myeloma Clinical Trials

A Study Evaluating the Safety, Pharmacokinetics, and Activity of Cevostamab in Participants With Relapsed or Refractory Multiple Myeloma

CAMMA 1
Start date: July 26, 2021
Phase: Phase 1
Study type: Interventional

This Phase Ib, multicenter, open-label study will evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of cevostamab monotherapy, cevostamab plus pomalidomide and dexamethasone (Pd) or cevostamab plus daratumumab and dexamethasone (Dd) which will be administered to participants with relapsed or refractory multiple myeloma (R/R MM) via intravenous (IV) infusion.

NCT ID: NCT04907240 Recruiting - Clinical trials for Peripheral Artery Disease

Observational GORE® VIABAHN® Endoprosthesis With PROPATEN Bioactive Surface Global Registry

Start date: October 9, 2021
Phase:
Study type: Observational [Patient Registry]

Collect real-world post-market clinical follow-up data on patients treated with the GORE® VIABAHN® Endoprosthesis with PROPATEN Bioactive Surface (VSX)

NCT ID: NCT04906551 Recruiting - Endometrium Clinical Trials

Measuring Endometrial Blood Flow Using Laser Doppler: Acceptability & Reproducibility

EMBeD
Start date: June 7, 2021
Phase:
Study type: Observational [Patient Registry]

35 participants will be recruited from a list of patients who are scheduled, as part of their routine NHS care, to have a clinically-indicated outpatient hysteroscopy for assessment of their uterine cavity. The participants will be women from the age of 18 up to the age of 40 who are undergoing hysteroscopy investigation. All participants included in the study require at least one ultrasound scan, within the preceding 5 years, demonstrating the presence of a normal uterine cavity devoid of uterine anomalies such as a septate uterus or intrauterine fibroids (which may affect endometrial blood flow measurements). It is usual for a patients to have an ultrasound assessment prior to hysteroscopy therefore we do not anticipate this requirement limiting the number of eligible participants available.

NCT ID: NCT04905069 Recruiting - Prostate Cancer Clinical Trials

Effectiveness of the SpaceOAR Vue System in Subjects With Prostate Cancer Being Treated With Stereotactic Body Radiotherapy

SABRE
Start date: December 21, 2021
Phase: N/A
Study type: Interventional

To demonstrate the effectiveness of the SpaceOAR Vue System in reducing late gastrointestinal (GI) toxicity in subjects undergoing Stereotactic Body Radiotherapy (SBRT) to treat prostate cancer.

NCT ID: NCT04904523 Recruiting - Covid19 Clinical Trials

Blood Markers for Identifying & Investigating Infection With COVID19 & Sepsis in Children.

pSeP
Start date: December 16, 2020
Phase:
Study type: Observational

Corona Virus Disease or COVID-19 is caused by SARS-CoV-2 virus. There have been reports of serious COVID-19 illness in children. In addition it has been linked to clusters of children presenting with high fevers and inflammation. Sepsis, also called blood poisoning, is an abnormal response of the body to some infections. We aim to conduct research in children under 18 admitted to intensive care unit. Using a systems approach towards mapping the body's immune and metabolic responses to COVID-19 or related illnesses and compare with other sepsis like illnesses.

NCT ID: NCT04903236 Recruiting - Cancer Clinical Trials

MR-BIO: A Study to Evaluate Changes in MR Imaging and Biological Parameters

MR-BIO
Start date: October 20, 2020
Phase:
Study type: Observational

The MR BIO study aims to understand the changes in the tumour and normal tissues during a course of radiotherapy. This is accomplished by studying the MR images taken during each treatment session on the MR Linear accelerator (MR Linac). The overarching hypothesis is that changes in MR imaging and biological parameters from blood, tissue, or urine biomarkers can be measured during radiotherapy and associated with clinical outcome. The MR Linac is a new radiotherapy machine with an on board MR scanner. This enables us to take images with high resolution and target the tumours more precisely and also reduce the dose to normal tissues. All patients undergoing treatment in the MR Linac at the Christie hospital will be considered for enrolment regardless of tumour site being treated. The study participants will receive the standard of care treatment for their disease condition. In addition, they will be requested to give weekly blood and urine samples during the course of radiotherapy and at first follow up. On completion of radiotherapy treatment, the participants will continue to be on standard of care follow up protocol with the treating oncologist. A small cohort of ten healthy volunteers will also be recruited to the study to develop and select some of the MR sequences only; they will not provide blood or urine samples. The healthy volunteers will be scanned for no more than one hour per session for a maximum of two sessions in total. These optimised sequences can then be used in the patient cohort.

NCT ID: NCT04903067 Recruiting - Clinical trials for Juvenile Idiopathic Arthritis

An Investigation Into the Relationship Between Dietary Intake and Health-related Quality of Life in Children and Young People With Juvenile Idiopathic Arthritis

JIA
Start date: May 1, 2021
Phase:
Study type: Observational

"Physical activity and diet in children and young people with arthritis" A qualitative study of exploring stake holder's experiences. Juvenile Idiopathic Arthritis (JIA) is the most common type of arthritis in children under the age of 16. The disease and its therapeutic management can cause serious long-term complications, which affect general activities and quality of life. The lack of specific guidelines for safe physical activity and appropriate management of any nutritional deficit aiming our study to find out your views and opinions about the needs of children and young people with juvenile idiopathic arthritis. We want to improve our knowledge about the impact of physical activity and eating habit on juvenile idiopathic arthritis and we want to develop a tool to help evaluate care. Few studies targeting quality of life and wellbeing in children adolescent populations have adopted the diet and physical activity perspective or approaches, consequently, this research project will help to address this gap through: 1. Interview: to look at young people's current experiences with JIA as well as their parents/caregivers and health care professionals. Study findings will provide a snapshot of the current experiences of participants, helping to improve our knowledge about JIA, physical activity, and diet. Qualitative studies exploring people perspectives on their experiences, when collected systematically, adds valuable depth, insight and understanding into the issues related to JIA not possible through quantitative methodologies. This study uses a qualitative approach known as framework methodology to understand stakeholder's experience of what helps and what hinders improving the quality of life in children and young adult with JIA. 21-30 stakeholders will be recruited in Oxford UK, to take part in individual semi-structured guided interviews lasting approximately one hour. Participant responses will be transcribed by the chief investigator and analysed to extract themes that will answer the research question. 2. Delphi study: which aims to develop a diet and physical activity intervention for children and young adult with JIA.