A Phase 2 Clinical Study in Children With HeFH Aged 6 to 17 Treated Once Daily With Bempedoic Acid...
HypercholesterolemiaMultiple-dose study to measure PK, PD and safety of bempedoic acid in pediatric patients 6 to 17 years of age with HeFH.
A Prospective, One-arm and Open Clinical Study of CM313 in the Treatment of Immune Thrombocytopenia...
Immune ThrombocytopeniaTreatmentTo evaluate the safety and efficacy of CM313 in the treatment of immune thrombocytopenia in patients who have not responded adequately or relapsed after first-line treatment and at least one second-line therapy including rituximab and/or TPO-RA.
Clinical Feasibility & Validation of the Virtual Reality GlenxRose Speech-Language Therapies
Vocal Cord DysfunctionSpeech DisordersFactors related to successful rehabilitation are often directly related to adherence; for instance, dosage, frequency, and intensity can burden the patient regarding time and motivational factors. Furthermore, surrounding salience, patients may lose interest or find an intervention boring after a few sessions. It is well documented that nonadherence not only impacts rehabilitation for the patient but can also further prolong treatment, and increase hospital and clinician costs, in addition to a higher prevalence of future comorbidities. Therefore, strategies that improve patient adherence can significantly help optimize patient care and treatment outcomes. One avenue to increase patient adherence is through the gamification of rehabilitation therapies using virtual reality (VR). Gamification of rehabilitation therapy can make mass practice required in rehabilitation therapies seemingly fun and more personally engaging for the patient. Additionally, the immersive experience achieved through VR can further promote salience and be customizable to individual patient requirements. As VR systems are now highly portable and relatively simple to utilize, they can provide an excellent opportunity to continue rehabilitation practice on the home front. Overall, the VR gamification of rehabilitation may increase adherence by shifting patients' perspectives of therapy as tedious, boring, or a hassle, to a fun and engaging game that ultimately helps their recovery processes. The GlenXRose VR-delivered speech-language therapies (Cognitive Projections Lab, University of Alberta) have been developed and piloted in collaboration with the Glenrose Rehabilitation Hospital with the overall goal of increasing patient adherence, treatment outcomes, and satisfaction with vocal therapy. The proposed studies are to investigate the feasibility of implementing this technology in routine clinical care (specific to voice disorders), obtaining clinician feedback, examining associated financial costs, and continuing to examine the effect of the GlenXRose VR speech-language therapies on patient adherence and clinical outcomes, compared to traditional clinical care.
Study of SFA002 in Patients With Mild to Moderate Psoriasis Plaques
PsoriasisThe purpose of this study is to determine the safety, metabolism and potential effect of drug product SFA004 on mild to moderate chronic plaque psoriasis. Psoriasis is a common chronic skin disorder that affects over 4 million people. There is no cure for psoriasis and treatment is directed at controlling patients' symptoms.
Dupilumab Step-down Strategy to Maintain Remission in Adult and Adolescents Patients With Atopic...
Atopic DermatitisEczema1 moreThe goal of this clinical trial is to compare a step-down strategy of spacing dupilumab injections with a standard maintenance treatment in adolescents and adults with controlled Atopic dermatitis (AD) for at least six months. The impact of dosage reduction strategies will be assessed with an innovative primary endpoint: the area under the curve of the weekly ADCT assessment.
A Study of Suvecaltamide in Adults With Moderate to Severe Residual Tremor in Parkinson's Disease...
Parkinson DiseaseTremorThis is a 17-week double-blind, placebo-controlled, randomized, flexible-dosing, parallel-group, multicenter study designed to evaluate the efficacy and safety of suvecaltamide for the treatment of moderate to severe residual tremor in adult participants with Parkinson's disease (PD). The target population represents participants who have tremor that is not adequately controlled by PD medications and that interferes with their activities of daily living (ADL) and/or with their performance of tasks.
Focused Ultrasound Modulation of the Globus Pallidus Interna in Schizophrenia
SchizophreniaSchizophrenia SchizoaffectiveThis is a single-site, phase 2, sham-controlled random-order cross-over pilot trial of PLIFUS targeting the right GPi in individuals with schizophrenia. Twelve individuals with schizophrenia who report continuous hallucinations or delusions of mild or greater severity will receive one session of PLIFUS and one session of sham PLIFUS in random order, one week apart. If no effect of PLIFUS is detected on measures of functional connectivity or psychotic symptoms in the first four completers, the trial will be changed to 3 sessions of PLIFUS or sham administered over 5 days.
Self-determination Plus Instant Messaging for Smokers With Cancer
SmokingCancerObjective: To test the efficacy of a self-determination theory-based intervention plus instant messaging to help smokers with cancer quit smoking. Hypothesis to be tested: Subjects who are allowed to choose their quit schedule and receive regular instant messaging about smoking cessation will show a significantly higher biochemically validated quit rate than those who receive only brief advice to quit immediately. Design and Subjects: An RCT will be conducted to 1448 smokers with cancer attending the outpatient clinics of five major acute care hospitals in Hong Kong for medical follow-up. Instruments: A structured questionnaire will be used to assess subjects' smoking history and demographic and clinical characteristics. EQ-5D-5L will be used to measure subjects' quality of life. Interventions: The intervention group will receive brief advice and will be invited to choose their own quit schedules (immediate or progressive) in the outpatient clinics. They will receive instant messaging about smoking cessation during the first 6-month follow-up period. The control group will receive brief advice to quit smoking immediately in the outpatient clinics, and will receive a placebo intervention during the first 6-month follow-up period. Subjects in both groups will receive leaflets on smoking cessation.
Hyperbaric Oxygen Therapy for Post-Concussion Syndrome
Post-Concussion SyndromeThe purpose of this study is to decrease symptom burden, improve cognitive function, and improve quality of life outcomes in subjects with mild TBI and persisting post-concussion syndrome using Hyperbaric Oxygen Treatment compared to a sham intervention. Specific Aims: Evaluate the efficacy of hyperbaric oxygen treatment to improve outcomes for adults with persisting post-concussion syndrome. Specifically, the investigators hypothesize that a prescribed course of hyperbaric oxygen treatments (HBOT) will improve outcomes and quality of life in adults with persisting symptoms >3 months after injury. Decrease symptom burden as measured by the Rivermead Post-Concussion Symptoms Questionnaire (RPQ). Improve cognitive function as measured by the National Institutes of Health (NIH) Toolbox Cognition Battery. Improve quality of life as measured by the 36-Item Short Form Survey (SF-36). Assess the safety and tolerability of hyperbaric oxygen treatments and compliance with treatment in adults with persisting post-concussion syndrome.
Study to Assess the Safety and Efficacy of Tenapanor for the Treatment of IBS-C
Irritable Bowel Syndrome With Constipation (IBS-C)This is a randomized, double-blind, placebo-controlled study to assess the efficacy, safety and tolerability of tenapanor (25 mg and 50 mg) in pediatric patients (≥12 and <18 years old) with IBS-C when administered twice daily (BID) for 12 consecutive weeks.