scholarly journals Treatment of Barth Syndrome by Cardiolipin Manipulation (CARDIOMAN) With Bezafibrate: Protocol for a Randomized Placebo-Controlled Pilot Trial Conducted in the Nationally Commissioned Barth Syndrome Service (Preprint)

2020 ◽  
Author(s):  
Lucy Dabner ◽  
Guido E Pieles ◽  
Colin G Steward ◽  
Julian P Hamilton-Shield ◽  
Andrew R Ness ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Pilot Trial ◽  
Barth Syndrome ◽  
Peak Oxygen Consumption ◽  
Lipid Lowering ◽  
Definitive Treatment ◽  
Resonance Spectroscopy ◽  
Potential Treatment ◽  
Randomized Controlled

BACKGROUND Barth syndrome is a rare, life-threatening, X-linked recessive genetic disease that predominantly affects young males and is caused by abnormal mitochondrial lipid metabolism. Currently, there is no definitive treatment for Barth syndrome other than interventions to ameliorate acute symptoms, such as heart failure, cardiac arrhythmias, neutropenia, and severe muscle fatigue. Previous mechanistic studies have identified the lipid-lowering drug bezafibrate as a promising potential treatment; however, to date, no human trials have been performed in this population. OBJECTIVE The aim of this study is to determine whether bezafibrate (and resveratrol in vitro) will increase mitochondrial biogenesis and potentially modify the cellular ratio of monolysocardiolipin (MLCL) to tetralinoleoyl-cardiolipin (L4-CL), ameliorating the disease phenotype in those living with the disease. METHODS The CARDIOMAN (Cardiolipin Manipulation) study is a UK single-center, double-blinded, randomized, placebo-controlled crossover study investigating the efficacy of bezafibrate in participants with Barth syndrome. Treatment was administered in two 15-week phases with a minimum washout period of 1 month between the phases where no treatment was administered. The primary outcome is peak oxygen consumption (VO<sub>2</sub> peak). Secondary outcomes include MLCL/L4-CL ratio and CL profile in blood cells, amino acid expression, phosphocreatine to adenosine triphosphate ratio in cardiac muscle and skeletal muscle oxidative function on phosphorus-31 magnetic resonance spectroscopy, quality of life using the Pediatric Quality of Life Inventory questionnaire, absolute neutrophil count, cardiac function and rhythm profiles at rest and during exercise, and mitochondrial organization and function assessments. Outcomes were assessed at baseline and during the final week of each treatment phase. RESULTS A total of 12 patients were scheduled to participate across three consecutive research clinics between March and April 2019. In total, 11 participants were recruited, and the follow-up was completed in January 2020. Data analysis is ongoing, with publication expected in 2021. CONCLUSIONS This trial was approved by the United Kingdom National Research Ethics Service Committee and the Medicines and Healthcare products Regulatory Agency. The feasibility of the CARDIOMAN study will help to inform the future conduct of randomized controlled trials in rare disease populations as well as testing the efficacy of bezafibrate as a potential treatment for the disease and advancing the mechanistic understanding of Barth syndrome. CLINICALTRIAL International Standard Randomized Controlled Trial Number (ISRCTN): 58006579; https://www.isrctn.com/ISRCTN58006579 INTERNATIONAL REGISTERED REPORT DERR1-10.2196/22533

scholarly journals Treatment of Barth Syndrome by Cardiolipin Manipulation (CARDIOMAN) With Bezafibrate: Protocol for a Randomized Placebo-Controlled Pilot Trial Conducted in the Nationally Commissioned Barth Syndrome Service

10.2196/22533 ◽  
2021 ◽  
Vol 10 (5) ◽  
pp. e22533
Author(s):  
Lucy Dabner ◽  
Guido E Pieles ◽  
Colin G Steward ◽  
Julian P Hamilton-Shield ◽  
Andrew R Ness ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Pilot Trial ◽  
Barth Syndrome ◽  
Peak Oxygen Consumption ◽  
Lipid Lowering ◽  
Definitive Treatment ◽  
Resonance Spectroscopy ◽  
Potential Treatment ◽  
Randomized Controlled

Background Barth syndrome is a rare, life-threatening, X-linked recessive genetic disease that predominantly affects young males and is caused by abnormal mitochondrial lipid metabolism. Currently, there is no definitive treatment for Barth syndrome other than interventions to ameliorate acute symptoms, such as heart failure, cardiac arrhythmias, neutropenia, and severe muscle fatigue. Previous mechanistic studies have identified the lipid-lowering drug bezafibrate as a promising potential treatment; however, to date, no human trials have been performed in this population. Objective The aim of this study is to determine whether bezafibrate (and resveratrol in vitro) will increase mitochondrial biogenesis and potentially modify the cellular ratio of monolysocardiolipin (MLCL) to tetralinoleoyl-cardiolipin (L4-CL), ameliorating the disease phenotype in those living with the disease. Methods The CARDIOMAN (Cardiolipin Manipulation) study is a UK single-center, double-blinded, randomized, placebo-controlled crossover study investigating the efficacy of bezafibrate in participants with Barth syndrome. Treatment was administered in two 15-week phases with a minimum washout period of 1 month between the phases where no treatment was administered. The primary outcome is peak oxygen consumption (VO2 peak). Secondary outcomes include MLCL/L4-CL ratio and CL profile in blood cells, amino acid expression, phosphocreatine to adenosine triphosphate ratio in cardiac muscle and skeletal muscle oxidative function on phosphorus-31 magnetic resonance spectroscopy, quality of life using the Pediatric Quality of Life Inventory questionnaire, absolute neutrophil count, cardiac function and rhythm profiles at rest and during exercise, and mitochondrial organization and function assessments. Outcomes were assessed at baseline and during the final week of each treatment phase. Results A total of 12 patients were scheduled to participate across three consecutive research clinics between March and April 2019. In total, 11 participants were recruited, and the follow-up was completed in January 2020. Data analysis is ongoing, with publication expected in 2021. Conclusions This trial was approved by the United Kingdom National Research Ethics Service Committee and the Medicines and Healthcare products Regulatory Agency. The feasibility of the CARDIOMAN study will help to inform the future conduct of randomized controlled trials in rare disease populations as well as testing the efficacy of bezafibrate as a potential treatment for the disease and advancing the mechanistic understanding of Barth syndrome. Trial Registration International Standard Randomized Controlled Trial Number (ISRCTN): 58006579; https://www.isrctn.com/ISRCTN58006579 International Registered Report Identifier (IRRID) DERR1-10.2196/22533

scholarly journals Effects of visualization of successful revascularization on chest pain and quality of life in chronic coronary syndrome: study protocol for the multi-center, randomized, controlled PLA-pCi-EBO-pilot-trial

Trials ◽  
2020 ◽  
Vol 21 (1) ◽  
Author(s):  
Michael Wester ◽  
Franziska Koll ◽  
Florian Zeman ◽  
Astrid Dempfle ◽  
Michael Koller ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Pilot Trial ◽  
Intervention Group ◽  
Control Group ◽  
Daily Routine ◽  
Coronary Syndrome ◽  
Randomized Controlled ◽  
Before And After ◽  
Stable Cad

Abstract Background Stable coronary artery disease (CAD), recently termed chronic coronary syndrome (CCS), is a highly prevalent disease. Current treatment strategies often include a relevant placebo effect. The hypothesis is that visual angiographic demonstration of the coronary arteries before and after successful percutaneous coronary intervention (PCI) by itself reduces the symptom burden of stable CAD/CCS. Design and methods The PLA-pCi-EBO-pilot-trial is a prospective, multi-center, randomized, controlled investigator-initiated pilot trial to study the effect of visual demonstration of successful PCI on quality of life (QoL) and angina pectoris (AP) in patients with symptomatic stable CAD/CCS. All patients with stable CAD/CCS and successful PCI will be screened. One hundred forty four patients with a frequency of AP ≥ 2/week will be randomized 1:1 stratified for AP frequency > 1/day. The control group will receive the common written procedural report on the procedure. Patients in the intervention group will additionally be given a printout picture of their coronary angiogram both before and after PCI. Primary endpoints are change in the Seattle Angina Questionnaire (SAQ)-derived QoL score 1 and 6 months after PCI. Secondary endpoints are changes in other SAQ-derived scores and dyspnea (NYHA score) 1 and 6 months after PCI. Discussion The PLA-pCi-EBO-pilot-trial evaluates the effect of visual angiographic result demonstration on disease symptoms and QoL in patients with stable CAD/CCS on top of PCI. A positive outcome of our study would encourage the routine use of angiographic picture demonstration and has thus the potential to change daily routine in the catheterization laboratory. Trial registration German Clinical Trials Register DRKS00017524. Registered on 5 July 2019

scholarly journals Randomized controlled pilot trial of yoga in overweight and obese breast cancer survivors: effects on quality of life and anthropometric measures

2011 ◽  
Vol 20 (2) ◽  
pp. 267-277 ◽  
Author(s):  
Alyson J. Littman ◽  
Lisa Cadmus Bertram ◽  
Rachel Ceballos ◽  
Cornelia M. Ulrich ◽  
Jaya Ramaprasad ◽  
...  
Keyword(s):  
Breast Cancer ◽  
Quality Of Life ◽  
Cancer Survivors ◽  
Breast Cancer Survivors ◽  
Pilot Trial ◽  
Anthropometric Measures ◽  
Randomized Controlled

Effects of active/passive interventions on pain, anxiety, and quality of life in women with fibromyalgia: Randomized controlled pilot trial

2016 ◽  
Vol 57 (1) ◽  
pp. 88-107 ◽  
Author(s):  
Gamze Ekici ◽  
Edibe Unal ◽  
Turkan Akbayrak ◽  
Naciye Vardar-Yagli ◽  
Yavuz Yakut ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Pilot Trial ◽  
Pain Anxiety ◽  
Randomized Controlled

scholarly journals Internet-Based Cognitive Behavioral Therapy for Psychological Distress Associated With the COVID-19 Pandemic: A Pilot Randomized Controlled Trial

2021 ◽  
Vol 12 ◽  
Author(s):  
Victoria Aminoff ◽  
Malin Sellén ◽  
Elise Sörliden ◽  
Mikael Ludvigsson ◽  
Matilda Berg ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Psychological Distress ◽  
Alcohol Use ◽  
Psychological Treatment ◽  
Controlled Trial ◽  
Pilot Trial ◽  
Secondary Outcome ◽  
Randomized Controlled ◽  
Symptom Profile

Background: The COVID-19 pandemic has been associated with various negative psychological consequences. This is a challenge for the society as regular psychological services cannot be offered to the same extent as before the pandemic. In addition to the requirement of social distancing, there is a need to adjust psychological treatment components like exposure to avoid increasing the spread of the infection. Internet-delivered cognitive behavior therapy (ICBT) has an established evidence base for a range of psychiatric problems and has been suggested as one possible approach to deal with the situation. This study aimed to conduct a randomized controlled pilot trial during the summer of 2020 with a broad focus on psychological distress and a treatment approach that tailors the intervention based on symptom profile and preferences.Methods: Following the advertisement and interview, we included 52 participants with elevated levels of psychological distress. They were randomly allocated to either a 7-week-long individually tailored ICBT (n = 26) or a wait-list control condition (n = 26). Measures of depression and quality of life were used as primary outcomes. We also included secondary outcome measures of anxiety, insomnia, trauma, stress, anger, and alcohol use. For screening, we used the CoRonavIruS Health Impact Survey (CRISIS).Results: Overall moderate to large between-group effects were found at post-treatment in favor of the treatment on measures of both depression [Beck Depression Inventory (BDI); Cohens d = 0.63; Patient Health Questionnaire (PHQ-9): d = 0.62] and anxiety [Generalized Anxiety Disorder-7-item scale (GAD-7); d = 0.82]. This was also observed for stress symptoms [Perceived Stress Scale (PSS-14); d = 1.04]. No effects were seen on measures of quality of life, insomnia, symptoms of post-traumatic stress, and anger. There was an effect on alcohol use [Alcohol Use Disorder Identification Test (AUDIT); d = 0.54], which was not of clinical relevance.Conclusion: Individually tailored ICBT shows initial promise as a way to reduce psychological problems in association with the COVID-19 pandemic. A possible limitation was that the trial was conducted when the effects of the pandemic were decreasing and when fewer people were affected by the restrictions (e.g., the summer of 2020).

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