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NCT ID: NCT04040049 Terminated - Fabry Disease Clinical Trials

A Fabry Disease Gene Therapy Study

MARVEL1
Start date: July 8, 2019
Phase: Phase 1/Phase 2
Study type: Interventional

This is a multinational, open-label study to assess the safety and efficacy of FLT190 in up to 15 adult male participants with classical Fabry disease.

NCT ID: NCT04039633 Recruiting - Erythromelalgia Clinical Trials

Spinal Cord Stimulation for Refractory Pain in Erythromelalgia

Start date: August 26, 2019
Phase: N/A
Study type: Interventional

Erythromelalgia is a rare disorder characterized by red, warm, and painful extremities, which is often precipitated by warm conditions. The pathophysiology is incompletely understood. The management of pain in erythromelalgia is challenging as no single therapy has been found to be effective. Response to pharmacotherapy varies, meaning that the physician has to take a stepwise trial and error approach with each patient. Consequently, this disorder is often associated with poorer health-related quality of life. There is currently no consensus or guideline on management of pain in erythromelalgia. Spinal cord stimulation is a widely applied therapy to treat severe chronic pain of various origin. Case reports and anecdotal evidence suggest that this therapy might alleviate refractory pain in patients with erythromelalgia. The aim of this trial is to evaluate the efficacy of spinal cord stimulation for refractory pain in erythromelalgia.

NCT ID: NCT04038177 Completed - Sedentary Lifestyle Clinical Trials

Superset Strength Training for Time-efficiency

Start date: August 5, 2019
Phase: N/A
Study type: Interventional

Physical inactivity is a global challenge and there is an urgent need to find strategies to increases people's activity levels. Strength training is one of the activities that is recommended to engage in regularly by both the American College of Sports Medicine and the World Health Organization. Understanding how strength training can be done effectively without spending much time could potentially increase people's involvement in strength training, as lack of time often is reported as barrier to training. Superset strength training can potentially be a time-efficient way of strength training, as this training method has been found to take half the time of the traditional ways of training. The aim of the present study is therefore to compare the effects of superset strength training and traditional strength training on muscular strength, body composition and fatigue.

NCT ID: NCT04035811 Recruiting - Achondroplasia Clinical Trials

Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)

Start date: August 12, 2019
Phase:
Study type: Observational

This is a long-term, multi-center, observational study in children 2.5 to <17 years with achondroplasia (ACH). The objective is to evaluate growth, ACH-related medical complications, assessments of health-related quality of life, body pain, functional abilities, cognitive functions, and treatments of study participants. No study medication will be administered.

NCT ID: NCT04034680 Completed - Dementia Clinical Trials

Targeted Interdisciplinary Model for Evaluation and Treatment of Neuropsychiatric Symptoms in Home Care Services

Start date: February 1, 2019
Phase: N/A
Study type: Interventional

Continuity of care in home care services is necessary to facilitate the assessment and treatment of persons with dementia and cognitive failure. TIME (Targeted Interdisciplinary Model for Evaluation and Treatment of Neuropsychiatric Symptoms) is a multi-component intervention with a biopsychosocial approach, based on the theoretical framework of cognitive behavioral therapy and person-centred care. The trial is designed to assess the effects of TIME on Neuropsychiatric symptoms for persons (NPS) with dementia in home care services.

NCT ID: NCT04034485 Completed - Clinical trials for Homozygous Familial Hypercholesterolemia

Phase 3 Study to Evaluate the Efficacy and Safety of LIB003 With Evolocumab in HoFH

Start date: December 7, 2019
Phase: Phase 3
Study type: Interventional

To compare the safety, tolerability and LDL-C response after 24 Weeks of monthly (every 4 weeks [Q4W]) subcutaneous (SC) dosing of LIB003 300 mg with monthly (Q4W) SC dosing of 420 mg evolocumab (Repatha®) in patients with HoFH on stable diet and oral LDL-C-lowering drug therapy

NCT ID: NCT04031092 Completed - Chronic Pain Clinical Trials

Use of Wearable Tech to Increase Physical Activity in Inpatient Rehabilitation for Patients With Chronic Pain

Start date: August 8, 2019
Phase: N/A
Study type: Interventional

The aim of this project is to evaluate whether use of wearable tech increases levels of physical activity in patients participating in inpatient rehabilitation for chronic pain.

NCT ID: NCT04031079 Completed - Obesity Clinical Trials

Use of Wearable Tech to Increase Physical Activity in Inpatient Rehabilitation for Overweight and Obese Patients

Start date: August 12, 2019
Phase: N/A
Study type: Interventional

The aim of this project is to evaluate whether use of wearable tech increases physical activity in patients participating in inpatient rehabilitation for overweight and obesity.

NCT ID: NCT04030169 Completed - PTSD Clinical Trials

Open Label Study of MDMA-assisted Psychotherapy for Treatment of PTSD With Optional fMRI Sub-Study

Start date: June 24, 2020
Phase: Phase 2
Study type: Interventional

This open-label, lead-in Phase 2 study is intended to gather supportive data on the safety and effectiveness of manualized MDMA-assisted psychotherapy as a treatment for PTSD. This will be the first study of MDMA-assisted psychotherapy in Europe using the CAPS-5 as a primary outcome measure to confirm assumptions made for statistical power calculations using the Clinician-Administered PTSD Scale for DSM-4 (CAPS-4) which support planned Phase 3 clinical trials. This study will gather supportive data on the safety and effectiveness of manualized MDMA-assisted psychotherapy as a treatment for PTSD and provide clinical supervision to planned Phase 3 therapy teams. This study will also be the first multi-site study of MDMA-assisted psychotherapy for PTSD in Europe and will explore reproducibility of findings from FDA-regulated trials in a multi-site format to further confirm the Phase 3 study design. This study will compare the effects of two open-label manualized Experimental Sessions of psychotherapy assisted by flexible doses of MDMA. Initial doses per Experimental Session include 80 mg or 120 mg of MDMA compounded with lactose, followed 1.5 to 2 hours later by a supplemental half-dose (40 mg or 60 mg). Total amounts of MDMA to be administered per Experimental Session range from 80 mg to 180 mg.

NCT ID: NCT04027309 Active, not recruiting - Clinical trials for Acute Myeloid Leukemia

A Study of Gilteritinib Versus Midostaurin in Combination With Induction and Consolidation Therapy Followed by One-year Maintenance in Patients With Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndromes With Excess Blasts-2 With FLT3 Mutations Eligible for Intensive Chemotherapy

HOVON 156 AML
Start date: December 20, 2019
Phase: Phase 3
Study type: Interventional

Activating mutations in the fms like tyrosine kinase 3 (FLT3) gene are observed in approximately 30% of patients with newly diagnosed acute myeloid leukemia (AML). Addition of the multitargeted kinase inhibitor midostaurin to standard chemotherapy prolongs event-free survival (EFS) and overall survival (OS) in patients with a FLT3 mutation. Gilteritinib is a more potent and more specific inhibitor of mutant FLT3 in comparison to midostaurin and has shown promising clinical activity in AML.