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NCT ID: NCT04977986 Recruiting - Fragile X Syndrome Clinical Trials

Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults With Fragile X Syndrome

RECONNECT
Start date: September 13, 2021
Phase: Phase 3
Study type: Interventional

This is a randomized, double-blind, placebo-controlled, multiple-center study, to assess the efficacy and safety of Cannabidiol administered as ZYN002 for the treatment of children, adolescent, and young adult patients with Fragile X Syndrome (FXS). Eligible participants will participate in up to an 18-week treatment period, where all participants will receive placebo or active study drug. Patients ages 3 to < 23 years will be eligible to participate.

NCT ID: NCT04976153 Recruiting - Fecal Incontinence Clinical Trials

Skeletal Muscle-derived Cell Implantation for Treatment of Fecal Incontinence

Fidelia
Start date: May 11, 2022
Phase: Phase 3
Study type: Interventional

The objective of this study is the final assessment of clinical safety and efficacy of autologous autologous skeletal muscle derived cells for patients with urge fecal incontinence due to external anal sphincter dysfunction caused by its disruption and/or weakness.

NCT ID: NCT04975997 Recruiting - Multiple Myeloma Clinical Trials

Open-label Study Comparing Iberdomide, Daratumumab and Dexamethasone (IberDd) Versus Daratumumab, Bortezomib, and Dexamethasone (DVd) in Participants With Relapsed or Refractory Multiple Myeloma (RRMM)

EXCALIBER-RRMM
Start date: June 23, 2022
Phase: Phase 3
Study type: Interventional

This is a multicenter, two-stage, randomized, controlled, open-label, Phase 3 study comparing the efficacy and safety of iberdomide in combination with dexamethasone and daratumumab (IberDd) versus daratumumab, bortezomib, and dexamethasone (DVd) in participants with relapsed or refractory multiple myeloma (RRMM).

NCT ID: NCT04975685 Recruiting - Multiple Sclerosis Clinical Trials

Strengthening Mental Abilities With Relational Training (SMART) in Multiple Sclerosis (MS): A Feasibility Trial

SMART_MS
Start date: February 7, 2022
Phase: N/A
Study type: Interventional

Background: Multiple Sclerosis (MS) is a chronic condition of the central nervous system; around 1 in 600 people in the United Kingdom have MS. Many people with MS (70%) have cognitive difficulties, which they experience as distressing and disabling, and there is currently a lack of treatment options to improve these difficulties. SMART (Strengthening Mental Abilities with Relational Training) - a theory-based online cognitive training programme, which has been shown to improve general cognitive abilities - has not been tested with people who have MS. Aims: To conduct a feasibility study to inform development of a definitive trial of SMART for improving cognitive functioning in people with MS. The investigators will assess: 1. Acceptability to participants of the intervention, delivery format, inclusion/exclusion criteria, baseline and outcome measures, randomisation protocol, and study procedures 2. The framework for a cost-effectiveness analysis alongside a definitive trial 3. Participant recruitment and retention rates 4. Sample-size needed for fully powered trial 5. Signal of efficacy Plan: To address Aims 1-5, the investigators will recruit 60 adults with MS who are experiencing cognitive difficulties, identified from MS clinics. Participants will complete baseline assessments of their cognitive abilities and answer questionnaires about their cognitive difficulties, personal priorities, mood, fatigue, self-efficacy, quality of life, and healthcare services used. Assessments will be administered by a researcher, face-to-face or remotely. Participants will be randomly allocated to one of three arms (20 per group): Group 1: Receives SMART intervention online - plus usual care (MS Nurse support). SMART intervention involves completing a series of logic problems, which are designed to train skills that scaffold complex cognition. Group 2: Receives usual care alone. Group 3: Receives a 'control' intervention online - plus usual care. Baseline measures will be re-administered at three- and six-months post-randomisation. Researchers and patient-partners (people with personal experience of MS, who will act as co-researchers) will also interview 30 participants about their experience of the study and treatment. All qualitative data will be transcribed and thematically analysed in terms of a priori feasibility aims. Quantitative data will enable sample-size calculation for a definitive study and determine signal of efficacy.

NCT ID: NCT04975581 Recruiting - Rotator Cuff Tears Clinical Trials

Rotator Cuff Tears Repair With or Without Dermal Patch Augmentation

CUFFPATCH
Start date: December 3, 2021
Phase: Phase 4
Study type: Interventional

The Investigators are planning to conduct a Pilot study of 40 patients of Pragmatic randomised controlled trial comparing rotator cuff tendons repair (small tendons around the shoulder ) with or without augmentative patch. It includes patients between age groups 50 to 75 years with large (≥ 3cm and < 5 cm ) rotator cuff tear that can be fully repaired using open or mini open repair with or without an augmentation with a human dermal matrix allograft (processed skin graft from a human donor ). The main aim is to examine whether the addition of the patch helps to reduce the rate of re-tear of the rotator cuff tendons at one year following surgery. Patients are randomized to receive either a repair with addition of an augmentative patch or a repair without a patch and have an identical follow-up after surgery. Patients are followed up in outpatient at 6 weeks , 3 months and 12 months after randomization and receive an Magnetic Resonant Imaging (MRI) scan 12 months after surgery. The primary outcome measure is to assess the Re-tear rate of rotator cuff at 12 months after surgery. The secondary outcome measure is to assess functional scores of the shoulder at 12 months . The tertiary outcome measures are to check the cost effectiveness of each procedure.

NCT ID: NCT04974294 Recruiting - Clinical trials for Streptococcus Pneumonia

PREVENTING PNEUMOcoccal Disease Through Vaccination (Study 2)

Pneumo 2
Start date: July 28, 2021
Phase: Phase 4
Study type: Interventional

To determine the effect of PCV-13 and PPV-23 vaccination versus control on experimental pneumococcal colonisation of 2 clades of serotypes 3 and 6B at 1 month and 6 months post vaccination respectively, using the EHPC model.

NCT ID: NCT04974242 Recruiting - Rotator Cuff Tears Clinical Trials

Physiotherapy for Patients Awaiting Rotator Cuff Repair

POWER
Start date: September 29, 2021
Phase: N/A
Study type: Interventional

POWER is a pragmatic multi-centre, external pilot randomised controlled trial with feasibility objectives using a parallel group design with 1:1 allocation ratio and integrated qualitative study. The study aims to answer the question: In adult patients diagnosed with tears of the rotator cuff and awaiting elective surgical repair, is it feasible to conduct a future, fully powered, multi-site RCT to test the hypothesis that physiotherapist-led exercise is superior to waiting-list control in terms of clinical and cost-effectiveness?

NCT ID: NCT04973800 Recruiting - Depression Clinical Trials

Simvastatin and Emotional Processing (OxSTEP)

OxSTEP
Start date: July 5, 2021
Phase: N/A
Study type: Interventional

Simvastatin is being employed because it is a 'statin'. As a drug class, statins have broad anti-inflammatory properties. Low-level inflammation is thought to be a potentially important mediator of the effects of psychosocial stress (including loneliness) on affect and vulnerability to depression. In this study we are using statins as an experimental tool to investigate this relationship further. Statins are widely prescribed agents that are regarded as very safe and so are suitable tools in this context. We have selected simvastatin because it is one of the most widely used statins and has an excellent safety profile, being also available 'over the counter'.

NCT ID: NCT04973657 Recruiting - Clinical trials for Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD)

A Study to Assess the Pharmacodynamics of VK0214 in Male Subjects With AMN

Start date: June 22, 2021
Phase: Phase 1
Study type: Interventional

Subjects will be administered multiple doses of VK0214 in a cohort dose escalation process. A total of up to 36 subjects will be enrolled in the study, into one of three dose cohorts. For each cohort, up to 12 subjects will be randomized to receive VK0214 or placebo in a 3:1 ratio so that there will be a total of up to 9 subjects for each of the active doses and up to 3 subjects dosed with placebo in each cohort.

NCT ID: NCT04973605 Recruiting - Clinical trials for Relapsed/Refractory Multiple Myeloma

A Phase 1b/2 Study of BGB-11417in Monotherapy and in Various Combinations With Dexamethasone and Carfilzomib in Multiple Myeloma

Start date: September 16, 2021
Phase: Phase 1/Phase 2
Study type: Interventional

Study consists of two parts, a part 1 dose escalation and a part 2 cohort expansion in combination with dexamethasone and carfilzomib intravenously across two cohorts with a monotherapy component as well.