Pilot, open non-controlled trial to assess the feasibility of implementing objective parameters as primary endpoints in a clinical trial with patients affected by knee osteoarthritis. (Preprint)

2019 ◽  
Author(s):  
Bogdan Corneliu Andor ◽  
Dionisio Franco Barattini ◽  
Dumitru Emanuel Dogaru ◽  
Simone Guadagna ◽  
Serban Rosu
Keyword(s):  
Chondroitin Sulphate ◽  
Controlled Trial ◽  
Subjective Evaluation ◽  
Pilot Trial ◽  
Final Visit ◽  
Life Questionnaire ◽  
Quality Of Data ◽  
Open Label ◽  
Objective Measurements

BACKGROUND Osteoarthritis (OA) is one of the top five most disabling conditions and it affects more than one third of persons over 65 years of age. Currently 80% of persons affected by OA already report having some movement limitation, 20% of people are not be able to perform major activities of daily living, and about 11% of the total affected population need of personal care. On 2014 the European Society for Clinical and Economic Aspects of Osteoporosis and Osteoarthritis (ESCEO) suggested as first step of pharmacological treatment for knee OA a background therapy with chronic symptomatic slow-acting drugs for osteoarthritis (SYSADOAs), such as glucosamine sulphate, chondroitin sulphate and hyaluronic acid (HA). In studies with oral HA, symptoms of OA are often measured using subjective parameters such as the visual analog scale (VAS) or the quality of life questionnaire (QoL) and objective measurements as ultrasonography (US) or range of motion (ROM) are employed in very few trials. This affects the quality of data in the literature. OBJECTIVE The primary objective of this work is to assess the feasibility of implementing US and ROM as objective measurements to correlate the improvement of knee mobility with pain reduction, evaluated using a subjective scale (VAS) in patients assuming a nutraceutical containing HA. The secondary objective is to evaluate the enrollment rate in one month to verify the feasibility for time and budget of the planned future main study. The explorative objective of the trial is to obtain preliminary data on efficacy of the tested product. METHODS This open-label pilot trial is performed in an orthopedic clinic (Timisoara, Romania). Male and female subjects (from 50 to 70 years) diagnosed with symptomatic OA of the knee with mild joint discomfort for at least 6 months are included. Following protocol, 8 patients are administered for 8 weeks Syalox® 300 Plus (River Pharma, Italy), a product based on HA of high molecular weight. Baseline and final visit assessments include orthopedic assessment, US, Knee injury and Osteoarthritis Outcome Score (KOOS) questionnaire, VAS and ROM of knee. RESULTS Data collection occurred between February 2018 and June 2018. All results are expected to be available by the end of 2018. CONCLUSIONS This pilot trial will be the first study to analyze the potential correlation between subjective evaluation (VAS, KOOS questionnaire) and objective measurements (US, ROM and actigraphy). The data from this study will assess the feasibility of the planned monthly recruitment rate and the necessary time and budget, and should provide preliminary information on efficacy of the tested product. CLINICALTRIAL ClinicalTrials.gov (NCT number: NCT03421054).

scholarly journals Effects of a probiotic treatment (Enterococcus faecalis) and open-label placebo on symptoms of allergic rhinitis: study protocol for a randomised controlled trial

BMJ Open ◽  
2019 ◽  
Vol 9 (10) ◽  
pp. e031339 ◽  
Author(s):  
Michael Schaefer ◽  
Paul Enck
Keyword(s):  
Quality Of Life ◽  
Allergic Rhinitis ◽  
Study Design ◽  
Controlled Trial ◽  
Life Questionnaire ◽  
Open Label ◽  
Quality Of Life Questionnaire ◽  
Probiotic Treatment ◽  
Randomised Controlled

IntroductionSeveral studies suggest that gut microbiota may play an important role in allergic diseases. The present trial aims to examine effects of the probioticEnterococcus faecalison symptoms of allergic rhinitis in patients. Effects of this probiotic on the immune system have been reported by several studies, but the majority of the previous trials were animal studies. In addition, it is well known that symptoms in allergic rhinitis are prone to exhibit high placebo responses. Moreover, recent studies report that even placebos without deception (open-label placebos) are highly effective in reducing symptoms of allergic rhinitis. Our study design combines both new approaches to assess effects on allergic symptoms in patients. The objective of this study is to compare the effects of a probiotic treatment (E. faecalis) with effects seen by open-label placebo, concealed placebo treatment and no treatment control.Methods and analysisA total of 120 patients with allergic rhinitis will be randomly assigned to one of four different groups: a double-blind probiotic/placebo group (groups 1 and 2), an open-label placebo group (group 3) and a no-treatment group (group 4) to control for spontaneous variation of symptoms. The primary outcome is the evaluation of allergic symptoms using the Combined Symptoms Medication Score. Furthermore, health-related quality of life is examined (Rhinitis Quality of Life Questionnaire). Secondary outcomes include a visual analogue scale on allergic burden and a second quality of life questionnaire. This report describes the study design of the randomised controlled trial.Ethics and disseminationThe study design was approved by the ethical committee of the UKT Department of Psychosomatic Medicine and Psychotherapy, Tübingen, Germany. The trial is registered at the German Clinical Trials Register (www.drks.de, DRKS00015804). The trial results will be published in peer-reviewed journals and at conferences.Trial registration numberGerman Clinical Trials Register (www.drks.de, DRKS00015804); Pre-results.

scholarly journals Pilot, Open and Non-controlled Trial to Assess the Objective Parameters which Correlate Knee Mobility with Pain Reduction in Patients Affected by Knee Osteoarthritis and Treated with Oral Hyaluronic Acid

2019 ◽  
Vol 70 (9) ◽  
pp. 3364-3371
Author(s):  
Bogdan Corneliu Andor ◽  
Simona Cerbu ◽  
Dionisio Franco Barattini ◽  
Dumitru Emanuel Dogaru ◽  
Ovidiu Dulgheru ◽  
...  
Keyword(s):  
Hyaluronic Acid ◽  
Knee Osteoarthritis ◽  
Preliminary Data ◽  
Controlled Trial ◽  
Pilot Trial ◽  
Sample Size Calculation ◽  
Pain Reduction ◽  
Main Study ◽  
Open Label ◽  
Objective Measurements

The study was in preparation for a future randomized clinical trial (RCT) with oral hyaluronic acid (HA) in knee osteoarthritis. Its purpose was to evaluate the feasibility of i) ultrasonography (US) and range of motion (ROM) parameters as objective measurements to correlate the improvement of knee mobility with its pain reduction; ii) the planned recruitment monthly rate to estimate the resources for the main study. In addition, it should give by US, ROM, visual analogue scale (VAS) and KOOS questionnaire, preliminary data on efficacy. This open-label pilot trial was performed in an orthopedic clinic (Timisoara, Romania). Male and female subjects (from 50 to 70 years) diagnosed with symptomatic OA of the knee with i) mild joint discomfort for at least 6 months, ii) Kellgren/Lawrence score 2 by X-ray, iii) pain for at least 15 of the 30 previous days, were included. Exclusion criteria were: inflammatory arthritic condition; oral corticosteroids within 4 weeks; intra-articular injections of HA or corticosteroids within 3 months; anti-inflammatory or chondroprotective drugs within 2 weeks. Following protocol, 8 patients administered for 8 weeks Syalox� 300 Plus (River Pharma, Italy) a product based on HA of high molecular weight. US parameters improved from baseline, even if no statistically significant differences were found. ROM and VAS (at rest, on moving and on pressing) values improved significantly at week 4 and 8 in comparison with baseline. The correlation between objective improvement of knee mobility and subjective pain reduction was documented. The enrolment rate was 8 patients/month. The KOOS subscales scores evidenced statistically significant differences during the study. No adverse event. US and ROM can be used as objective measurements to correlate improvement of knee mobility with pain reduction. The recruitment capability evidenced a realistic estimation of time and budget for the main study. Preliminary data on efficacy using objective measurements (US, ROM) and subjective parameters (VAS, KOOS questionnaire) showed significant improvements and will be used for sample size calculation in the main study.

scholarly journals Randomized controlled trial of convalescent plasma therapy against standard therapy in patients with severe COVID-19 disease

2021 ◽  
Vol 11 (1) ◽  
Author(s):  
Manaf AlQahtani ◽  
Abdulkarim Abdulrahman ◽  
Abdulrahman Almadani ◽  
Salman Yousif Alali ◽  
Alaa Mahmood Al Zamrooni ◽  
...  
Keyword(s):  
Standard Therapy ◽  
Primary Outcome ◽  
Controlled Trial ◽  
Severe Disease ◽  
Pilot Trial ◽  
Standard Of Care ◽  
Primary Outcome Measure ◽  
Secondary Outcome ◽  
Open Label ◽  
Plasma Therapy

AbstractConvalescent plasma (CP) therapy in COVID-19 disease may improve clinical outcome in severe disease. This pilot study was undertaken to inform feasibility and safety of further definitive studies. This was a prospective, interventional and randomized open label pilot trial in patients with severe COVID-19. Twenty COVID-19 patients received two 200 ml transfusions of convalescent patient CP over 24-h compared with 20 who received standard of care. The primary outcome was the requirement for ventilation (non-invasive or mechanical ventilation). The secondary outcomes were biochemical parameters and mortality at 28 days. The CP group were a higher risk group with higher ferritin levels (p < 0.05) though respiratory indices did not differ. The primary outcome measure was required in 6 controls and 4 patients on CP (risk ratio 0.67, 95% CI 0.22–2.0, p = 0.72); mean time on ventilation (NIV or MV) did not differ. There were no differences in secondary measures at the end of the study. Two patients died in the control and one patient in the CP arm. There were no significant differences in the primary or secondary outcome measures between CP and standard therapy, although a larger definitive study is needed for confirmation. However, the study did show that CP therapy appears to be safe in hospitalized COVID-19 patients with hypoxia.Clinical trials registration NCT04356534: 22/04/2020.

An Open-Label, Analgesic Efficacy and Safety of Pituitary Radiosurgery for Patients With Opioid-Refractory Pain: Study Protocol for a Randomized Controlled Trial

Neurosurgery ◽  
2017 ◽  
Vol 83 (1) ◽  
pp. 146-153 ◽  
Author(s):  
Pierre-Yves Borius ◽  
Stéphanie Ranque Garnier ◽  
Karine Baumstarck ◽  
Frédéric Castinetti ◽  
Anne Donnet ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Pain Relief ◽  
Cancer Pain ◽  
Controlled Trial ◽  
Analgesic Efficacy ◽  
Standards Of Care ◽  
Control Group ◽  
Efficacy And Safety ◽  
Open Label

Abstract BACKGROUND Hypophysectomy performed by craniotomy or percutaneous techniques leads to complete pain relief in more than 70% to 80% of cases for opioid refractory cancer pain. Radiosurgery could be an interesting alternative approach to reduce complications. OBJECTIVE To assess the analgesic efficacy compared with standard of care is the primary goal. The secondary objectives are to assess ophthalmic and endocrine tolerance, drug consumption, quality of life, and mechanisms of analgesic action. METHODS The trial is multicenter, randomized, prospective, and open-label with 2 parallel groups. This concerns patients in palliative care suffering from nociceptive or mixed cancer pain, refractory to standard opioid therapy. Participants will be randomly assigned to the control group receiving standards of care for pain according to recommendations, or to the experimental group receiving a pituitary GammaKnife (Elekta, Stockholm, Sweden) radiosurgery (160 Gy delivered in pituitary gland) associated with standards of care. Evaluation assessments will be taken at baseline, day0, day4, day7, day14, day28, day45, month3, and month6. EXPECTED OUTCOMES We could expect pain improvement in 70% to 90% of cases at day4. In addition we will assess the safety of pituitary radiosurgery in a vulnerable population. The secondary endpoints could show decay of opioid consumption, good patient satisfaction, and improvement of the quality of life. DISCUSSION The design of this study is potentially the most appropriate to demonstrate the efficacy and safety of radiosurgery for this new indication. New recommendations could be obtained in order to improve pain relief and quality of life.

scholarly journals Efficacy and safety of electro-acupuncture (EA) on insomnia in patients with lung cancer: study protocol of a randomized controlled trial

Trials ◽  
2020 ◽  
Vol 21 (1) ◽  
Author(s):  
Hongyu Yue ◽  
Shuang Zhou ◽  
Huangan Wu ◽  
Xuan Yin ◽  
Shanshan Li ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Lung Cancer ◽  
Cancer Survivors ◽  
Controlled Trial ◽  
Control Group ◽  
Life Questionnaire ◽  
Efficacy And Safety ◽  
Symptom Scale ◽  
Significance Level

Abstract Background Cancer-related insomnia (CRI) is one of the most prevalent complaints among cancer survivors and severely impairs patients’ quality of life. As a popular non-pharmacological alternative treatment, acupuncture provides a good clinical curative effect on insomnia. The aim of this trial is to evaluate efficacy and safety of electro-acupuncture on insomnia in patients with lung cancer. Method This is a protocol for a multicenter randomized single-blinded sham-controlled trial. We will randomly assign 252 eligible patients with lung cancer-related insomnia into two groups at a ratio of 1:1, the treatment group (EA) and the control group (sham EA). All treatment will be given 3 times per week for 8 weeks, and a 12-week follow-up will be conducted. The primary outcome will be measured by the Pittsburgh Sleep Quality Index (PSQI). The secondary outcomes will include sleep parameters recorded from the actigraphy, scores from Quality of Life Questionnaire Core-30 (QLQ-C30), and Patient Health Questionnaire-9 (PHQ-9). All adverse effects during the trial will be assessed by the Treatment Emergent Symptom Scale (TESS). All analyses will be based on ITT principle and performed with the statistical software SPSS (version 24.0) by t test, rank-sum test, chi-square, and so on. A two-sided significance level will be set at 5%. Discussion This large-sample trial protocol will evaluate the efficacy of electro-acupuncture on insomnia in patients with lung cancer. This protocol, if proven to be effective, will contribute to filling the gap in treatment options in the CRI field and provide a promising intervention for insomnia in lung cancer survivors. Trial registration ChiCTR ChiCTR1900026395. Registered on 8 October 2019, http://www.chictr.org.cn/showproj.aspx?proj=44068

scholarly journals Early specialist palliative care on quality of life for malignant pleural mesothelioma: a randomised controlled trial

Thorax ◽  
2019 ◽  
Vol 74 (4) ◽  
pp. 354-361 ◽  
Author(s):  
Fraser Brims ◽  
Samal Gunatilake ◽  
Iain Lawrie ◽  
Laura Marshall ◽  
Carole Fogg ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Palliative Care ◽  
Malignant Pleural Mesothelioma ◽  
Controlled Trial ◽  
Mean Difference ◽  
Pleural Mesothelioma ◽  
Life Questionnaire ◽  
Specialist Palliative Care ◽  
European Organization

PurposeMalignant pleural mesothelioma (MPM) has a high symptom burden and poor survival. Evidence from other cancer types suggests some benefit in health-related quality of life (HRQoL) with early specialist palliative care (SPC) integrated with oncological services, but the certainty of evidence is low.MethodsWe performed a multicentre, randomised, parallel group controlled trial comparing early referral to SPC versus standard care across 19 hospital sites in the UK and one large site in Western Australia. Participants had newly diagnosed MPM; main carers were additionally recruited. Intervention: review by SPC within 3 weeks of allocation and every 4 weeks throughout the study. HRQoL was assessed at baseline and every 4 weeks with the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire Core 30. Primary outcome: change in EORTC C30 Global Health Status 12 weeks after randomisation.ResultsBetween April 2014 and October 2016, 174 participants were randomised. There was no significant between group difference in HRQoL score at 12 weeks (mean difference 1.8 (95% CI −4.9 to 8.5; p=0.59)). HRQoL did not differ at 24 weeks (mean difference −2.0 (95% CI −8.6 to 4.6; p=0.54)). There was no difference in depression/anxiety scores at 12 weeks or 24 weeks. In carers, there was no difference in HRQoL or mood at 12 weeks or 24 weeks, although there was a consistent preference for care, favouring the intervention arm.ConclusionThere is no role for routine referral to SPC soon after diagnosis of MPM for patients who are cared for in centres with good access to SPC when required.Trial registration numberISRCTN18955704.

Comparison of the effects of Buteyko and pranayama breathing techniques on quality of life in patients with asthma – a randomized controlled trial

2012 ◽  
Vol 27 (2) ◽  
pp. 133-141 ◽  
Author(s):  
Venkatesan Prem ◽  
Ramesh Chandra Sahoo ◽  
Prabha Adhikari
Keyword(s):  
Quality Of Life ◽  
Controlled Trial ◽  
Control Group ◽  
Life Questionnaire ◽  
Control Groups ◽  
Quality Of Life Questionnaire ◽  
Randomized Controlled ◽  
Questionnaire Score ◽  
And Control

Objective: To compare two breathing exercises (Buteyko and pranayama) with a control group in patients with asthma. Design: Randomized controlled trial. Subjects: One hundred and twenty subjects were randomized to three groups through block randomization. Subjects with an Asthma Quality of Life Questionnaire score <5.5 participated in the study. Setting: Outpatient pulmonary medicine department. Interventions: Subjects in the Buteyko and pranayama groups were trained for 3–5 days and instructed to practise the exercises for 15 minutes twice daily, and for three months duration. The control group underwent routine pharmacological management during the study period. Outcome measures: Asthma Quality of Life Questionnaire, Asthma Control Questionnaire and pulmonary function test. Results: The baseline characteristics were similar in all three groups. Post intervention, the Buteyko group showed better trends of improvement (mean (95% confidence interval), P-value) in total Asthma Quality of Life Questionnaire score than the pranayama (0.47 (–0.008–0.95), P = 0.056) and control groups (0.97 (0.48–1.46), P = 0.0001). In comparison between the pranayama and control groups, pranayama showed significant improvement (0.50 (0.01–0.98), P = 0.042) in total Asthma Quality of Life Questionnaire score. Conclusion: The Buteyko group showed better trends of improvement in quality of life and asthma control than the group performing the pranayama breathing exercise.

The Effect of Aromatherapy on Sleep and Quality of Life in Menopausal Women with Sleeping Problems: A Non-Randomized, Placebo-Controlled Trial

2020 ◽  
Vol 27 (6) ◽  
pp. 421-430
Author(s):  
Meryem Gürler ◽  
Aynur Kızılırmak ◽  
Mürüvvet Baser
Keyword(s):  
Quality Of Life ◽  
Placebo Group ◽  
Sleep Quality ◽  
Controlled Trial ◽  
Intervention Group ◽  
Placebo Control ◽  
Life Questionnaire ◽  
Menopausal Women ◽  
Sleeping Problems

<b><i>Introduction:</i></b> Menopause is the termination of menstruation and fertility. Women commonly experience sleeping problems during the menopausal period. Aromatherapy is among the complementary therapies used to remedy sleeping problems. <b><i>Methods:</i></b>This study aims to investigate the effects of lavender oil on sleep and quality of life of menopausal women through steam inhalation. This study was quasi-experimental with pre-test/post-test placebo control groups. It was conducted with 57 women, 27 of whom were subject to aromatherapy and 30 to a placebo. Data were collected using the Questionnaire Form, the Pittsburgh Sleep Quality Index (PSQI) and the Menopause-Specific Quality of Life Questionnaire (MENQOL). <b><i>Results:</i></b> For the intervention group, the PSQI median scores after the administration of aromatherapy were found to be significantly lower than those before the administration (<i>p</i> &#x3c; 0.001) and those of the placebo group (<i>p</i> &#x3c; 0.001). Similarly, for the intervention group, the total median MENQOL scores after the administration of the aromatherapy were found to be significantly lower than the scores prior to the administration (<i>p</i> &#x3c; 0.001) as well as the scores of the placebo group (<i>p</i> &#x3c; 0.001). <b><i>Conclusion:</i></b> It was found that aromatherapy involving lavender-scented steam inhalation increased sleep quality and quality of life in women with sleep deprivation problems during menopause.

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