scholarly journals Effect of topiramate on eating behaviours in Prader-Willi syndrome: TOPRADER double-blind randomised placebo-controlled study

2019 ◽  
Vol 9 (1) ◽  
Author(s):  
Angèle Consoli ◽  
Sophie Çabal Berthoumieu ◽  
Marie Raffin ◽  
Denise Thuilleaux ◽  
Christine Poitou ◽  
...  
Keyword(s):  
Eating Disorders ◽  
Primary Outcome ◽  
Dose Effect ◽  
Secondary Outcome ◽  
Controlled Study ◽  
Prader Willi Syndrome ◽  
Eating Behaviours ◽  
Behavioural Disorders ◽  
Double Blind ◽  
Effect Relationship

Abstract Prader–Willi Syndrome (PWS) is a rare genetic syndrome leading to severe behavioural disorders and mild cognitive impairment. The objective of this double-blind randomised placebo-controlled trial was to study the efficacy and tolerance of topiramate on behavioural disorders in patients with PWS. Participants (aged 12–45 years) had genetically confirmed PWS and severe irritability/impulsivity, eating disorders and/or obesity, and skin picking. Thirty-two participants received a placebo (PBO), and 30 participants received topiramate (TOP) (50–200 mg/day) for 8 weeks. The primary outcome was the rate of responders using the Clinical Global Impression-Improvement (CGI-I) scale. The secondary outcome measures included the Aberrant Behaviour Checklist, the Dykens Hyperphagia Questionnaire (DHK), the Self-Injurious Behaviour Scale (SIBS) and the body mass index (BMI). We found no significant difference in the primary outcome (the CGI-I): 9 (30%) patients were very much or much improved in the TOP group compared to 7 (22.6%) patients in the PBO group. However, the DHK behaviour and severity scores improved significantly more over time in patients treated with topiramate versus those receiving a placebo, with a significant dose–effect relationship. DHK scores were also significantly associated with genetic subtypes and hospitalisation status. The effects of topiramate on eating behaviours remained significant after adjusting for genetic subtype and hospitalisation. Topiramate had therefore a significant effect on eating disorders, with a dose–effect relationship. Given the burden of eating disorders in PWS, we believe that topiramate may become the first psychotropic option within the global care of obesity in individuals with PWS.

Continuous bilateral thoracic paravertebral blockade for analgesia after cardiac surgery: a randomised, controlled trial

Perfusion ◽  
2017 ◽  
Vol 32 (7) ◽  
pp. 591-597 ◽  
Author(s):  
Geoff G. Lockwood ◽  
Leilani Cabreros ◽  
Dorota Banach ◽  
Prakash P. Punjabi
Keyword(s):  
Cardiac Surgery ◽  
Primary Outcome ◽  
Controlled Trial ◽  
Morphine Consumption ◽  
Controlled Study ◽  
Double Blind ◽  
Clinical Trial Registration ◽  
Subcutaneous Infusion ◽  
Randomised Controlled Study ◽  
Randomised Controlled

Background: Continuous bilateral thoracic paravertebral blockade has been used for analgesia after cardiac surgery, but its efficacy has never been formally tested. Method: Fifty adult patients were enrolled in a double-blind, randomised, controlled study of continuous bilateral thoracic paravertebral infusion of 0.5% lidocaine (1 mg.kg-1.hr-1) for analgesia after coronary surgery. Control patients received a subcutaneous infusion of lidocaine at the same rate through catheters inserted at the same locations as the study group. The primary outcome was morphine consumption at 48 hours using patient-controlled analgesia (PCA). Secondary outcomes included pain, respiratory function, nausea and vomiting. Serum lidocaine concentrations were measured on the first two post-operative days. Results: There was no difference in morphine consumption or in any other outcome measure between the groups. Serum lidocaine concentrations increased during the study, with a maximum of 5.9 mg.l-1. There were no adverse events as a consequence of the study. Conclusion: Bilateral paravertebral infusion of lidocaine confers no advantage over systemic lidocaine infusion after cardiac surgery. Clinical trial registration: ISRCTN13424423 ( https://www.isrctn.com )

scholarly journals Association between Age, Weight, and Dose and Clinical Response to Probiotics in Children with Acute Gastroenteritis

2020 ◽  
Vol 151 (1) ◽  
pp. 65-72
Author(s):  
David Schnadower ◽  
Robert E Sapien ◽  
T Charles Casper ◽  
Cheryl Vance ◽  
Phillip I Tarr ◽  
...  
Keyword(s):  
Acute Gastroenteritis ◽  
Primary Outcome ◽  
Controlled Trial ◽  
Interaction Effects ◽  
Secondary Outcome ◽  
Z Score ◽  
Double Blind ◽  
Multicenter Randomized Controlled Trial ◽  
Weight Percentile ◽  
Age And Sex

ABSTRACT Background Gastroenteritis is a common and impactful disease in childhood. Probiotics are often used to treat acute gastroenteritis (AGE); however, in a large multicenter randomized controlled trial (RCT) in 971 children, Lactobacillus rhamnosus GG (LGG) was no better than placebo in improving patient outcomes. Objectives We sought to determine whether the effect of LGG is associated with age, weight z score and weight percentile adjusted for age and sex, or dose per kilogram administered. Methods This was a preplanned secondary analysis of a multicenter double-blind RCT of LGG 1 × 1010 CFU twice daily for 5 d or placebo in children 3–48 mo of age with AGE. Our primary outcome was moderate to severe gastroenteritis. Secondary outcomes included diarrhea and vomiting frequency and duration, chronic diarrhea, and side effects. We used multivariable linear and nonlinear models testing for interaction effects to assess outcomes by age, weight z score and weight percentile adjusted for age and sex, and dose per kilogram of LGG received. Results A total of 813 children (84%) were included in the analysis; 413 received placebo and 400 LGG. Baseline characteristics were similar between treatment groups. There were no differential interaction effects across ranges of age (P-interaction = 0.32), adjusted weight z score (P-interaction = 0.43), adjusted weight percentile (P-interaction = 0.45), or dose per kilogram of LGG received (P-interaction = 0.28) for the primary outcome. Whereas we found a statistical association favoring placebo at the extremes of adjusted weight z scores for the number of vomiting episodes (P-interaction = 0.02) and vomiting duration (P-interaction = 0.0475), there were no statistically significant differences in other secondary outcome measures (all P-interactions > 0.05). Conclusions LGG does not improve outcomes in children with AGE regardless of the age, adjusted weight z score, and adjusted weight percentile of participants, or the probiotic dose per kilogram received. These results further strengthen the conclusions of low risk of bias clinical trials which demonstrate that LGG provides no clinical benefit in children with AGE. This trial was registered at clinicaltrials.gov as NCT01773967.

scholarly journals Efficacy of faecal microbiota transplantation for patients with irritable bowel syndrome in a randomised, double-blind, placebo-controlled study

Gut ◽  
2019 ◽  
Vol 69 (5) ◽  
pp. 859-867 ◽  
Author(s):  
Magdy El-Salhy ◽  
Jan Gunnar Hatlebakk ◽  
Odd Helge Gilja ◽  
Anja Bråthen Kristoffersen ◽  
Trygve Hausken
Keyword(s):  
Irritable Bowel Syndrome ◽  
Secondary Outcome ◽  
Controlled Study ◽  
Rrna Gene ◽  
Faecal Microbiota ◽  
Double Blind ◽  
Double Blind Placebo ◽  
Faecal Microbiota Transplantation ◽  
Link Type ◽  
Irritable Bowel

ObjectiveFaecal microbiota transplantation (FMT) from healthy donors to patients with irritable bowel syndrome (IBS) has been attempted in two previous double-blind, placebo-controlled studies. While one of those studies found improvement of the IBS symptoms, the other found no effect. The present study was conducted to clarify these contradictory findings.DesignThis randomised, double-blind, placebo-controlled study randomised 165 patients with IBS to placebo (own faeces), 30 g FMT or 60 g FMT at a ratio of 1:1:1. The material for FMT was obtained from one healthy, well-characterised donor, frozen and administered via gastroscope. The primary outcome was a reduction in the IBS symptoms at 3 months after FMT (response). A response was defined as a decrease of 50 or more points in the total IBS symptom score. The secondary outcome was a reduction in the dysbiosis index (DI) and a change in the intestinal bacterial profile, analysed by 16S rRNA gene sequencing, at 1 month following FMT.ResultsResponses occurred in 23.6%, 76.9% (p<0.0001) and 89.1% (p<00.0001) of the patients who received placebo, 30 g FMT and 60 g FMT, respectively. These were accompanied by significant improvements in fatigue and the quality of life in patients who received FMT. The intestinal bacterial profiles changed also significantly in the groups received FMT. The FMT adverse events were mild self-limiting gastrointestinal symptoms.ConclusionsFMT is an effective treatment for patients with IBS. Utilising a well-defined donor with a normal DI and favourable specific microbial signature is essential for successful FMT. The response to FMT increases with the dose.Trial registrationwww.clinicaltrials.gov (NCT03822299) and www.cristin.no (ID657402).

scholarly journals Efficacy and safety of galcanezumab for prevention of migraine headache in Japanese patients with episodic migraine: A phase 2 randomized controlled clinical trial

2020 ◽  
Vol 3 ◽  
pp. 251581632093257 ◽  
Author(s):  
Fumihiko Sakai ◽  
Akichika Ozeki ◽  
Vladimir Skljarevski
Keyword(s):  
Migraine Headache ◽  
Japanese Patients ◽  
Episodic Migraine ◽  
Secondary Outcome ◽  
Controlled Study ◽  
Controlled Clinical Trial ◽  
Life Questionnaire ◽  
Efficacy And Safety ◽  
Double Blind ◽  
Secondary Endpoints

Objective: This study was designed to assess the efficacy and safety of galcanezumab in comparison with placebo for the prevention of migraine in Japanese patients with episodic migraine. Methods: In this double-blind, placebo-controlled study, which was conducted over 6 months, randomized adult patients received subcutaneous injections of galcanezumab (120 mg n = 115, 240 mg n = 114) or placebo ( n = 230) once monthly. The primary endpoint was the overall mean change from baseline in the number of monthly migraine headache days. The key secondary outcome measures were response rates (≥50%, ≥75%, and 100%); the Migraine-Specific Quality-of-Life Questionnaire Role Function-Restrictive score; monthly migraine headache days requiring acute treatment; and Patient Global Impression of Severity (PGI-S). Results: The mean change from baseline in monthly migraine headache days over months 1–6 was significantly ( p < 0.001) greater for the 120-mg galcanezumab dose (−3.60 days) and the 240-mg galcanezumab dose (−3.36 days) compared with placebo (−0.59 days). Both the 120-mg and 240-mg doses of galcanezumab were superior compared with placebo for each of the key secondary endpoints except for PGI-S (only the 240-mg dose was superior). The most commonly reported treatment-emergent adverse events were local injection-site reactions; erythema, swelling, pruritus, and pain were more commonly reported by patients who were treated with galcanezumab than those treated with placebo. Conclusion: The number of monthly migraine headache days was reduced with both doses of galcanezumab, and both doses were safe and well tolerated in Japanese patients with episodic migraine.

scholarly journals Efficacy of ethyl-eicosapentaenoic acid in bipolar depression: Randomised double-blind placebo-controlled study

2006 ◽  
Vol 188 (1) ◽  
pp. 46-50 ◽  
Author(s):  
Sophia Frangou ◽  
Michael Lewis ◽  
Paul McCrone
Keyword(s):  
Eicosapentaenoic Acid ◽  
Rating Scale ◽  
Bipolar Depression ◽  
Unipolar Depression ◽  
Adjunctive Treatment ◽  
Secondary Outcome ◽  
Controlled Study ◽  
Double Blind Study ◽  
Young Mania ◽  
Double Blind

BackgroundEpidemiological and clinical studies suggest that increased intake of eicosapentaenoic acid (EPA) alleviates unipolar depression.AimsTo examine the efficacy of EPA in treating depression in bipolar disorder.MethodIn a 12-week, double-blind study individuals with bipolar depression were randomly assigned to adjunctive treatment with placebo (n=26) or with 1g/day (n=24) or 2 g/day (n=25) of ethyl-EPA. Primary efficacy was assessed by the Hamilton Rating Scale for Depression (HRSD), with changes in the Young Mania Rating Scale and Clinical Global Impression Scale (CGI) as secondary outcome measures.ResultsThere was no apparent benefit of 2g over 1g ethyl-EPA daily. Significant improvement was noted with ethyl-EPA treatment compared with placebo in the HRSD (P=0.04) and the CGI (P=0.004) scores. Both doses were well tolerated.ConclusionsAdjunctive ethyl-EPA is an effective and well-tolerated intervention in bipolar depression.

scholarly journals Phase III, Randomized, Double-Blind, Placebo-Controlled Study of Long-Acting Methylphenidate for Cancer-Related Fatigue: North Central Cancer Treatment Group NCCTG-N05C7 Trial

2010 ◽  
Vol 28 (23) ◽  
pp. 3673-3679 ◽  
Author(s):  
Amanda R. Moraska ◽  
Amit Sood ◽  
Shaker R. Dakhil ◽  
Jeff A. Sloan ◽  
Debra Barton ◽  
...  
Keyword(s):  
Short Form ◽  
Phase Iii ◽  
Secondary Outcome ◽  
Controlled Study ◽  
Symptom Experience ◽  
Double Blind ◽  
Time Curve ◽  
Cancer Related Fatigue ◽  
Significant Difference ◽  
Long Acting

Purpose Fatigue is one of the most common symptoms experienced by patients with cancer. This trial was developed to evaluate the efficacy of long-acting methylphenidate for improving cancer-related fatigue and to assess its toxicities. Patients and Methods Adults with cancer were randomly assigned in a double-blinded manner to receive methylphenidate (target dose, 54 mg/d) or placebo for 4 weeks. The Brief Fatigue Inventory was the primary outcome measure, while secondary outcome measures included a Symptom Experience Diary (SED), the Short Form-36 (SF-36) Vitality Subscale, a linear analog self-assessment, the Pittsburgh Sleep Quality Index, and the Subject Global Impression of Change. Results In total, 148 patients were enrolled. Using an area under the serum concentration-time curve analysis, there was no evidence that methylphenidate, as compared with placebo, improved the primary end point of cancer-related fatigue in this patient population (P = .35). Comparisons of secondary end points, including clinically significant changes in quality-of-life variables and cancer-related fatigue change from baseline, were similarly negative. However, a subset analysis suggested that patients with more severe fatigue and/or with more advanced disease did have some fatigue improvement with methylphenidate (eg, in patients with stage III or IV disease, the mean improvement in usual fatigue was 19.7 with methylphenidate v 2.1 with placebo; P = .02). There was a significant difference in self-reported toxicities (SED), with increased levels of nervousness and appetite loss in the methylphenidate arm. Conclusion This clinical trial was unable to support the primary prestudy hypothesis that the chosen long-acting methylphenidate product would decrease cancer-related fatigue.

HAGIL (Hamburg Vigil Study): a randomized placebo-controlled double-blind study with modafinil for treatment of fatigue in patients with multiple sclerosis

2011 ◽  
Vol 17 (8) ◽  
pp. 1002-1009 ◽  
Author(s):  
F Möller ◽  
J Poettgen ◽  
F Broemel ◽  
A Neuhaus ◽  
M Daumer ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Cognitive Impairment ◽  
Safety Concern ◽  
Dropout Rate ◽  
Secondary Outcome ◽  
Controlled Study ◽  
Double Blind Study ◽  
Double Blind ◽  
Neuropsychological Measures ◽  
Intention To Treat

Objective: To reassess the effect of modafinil, a wakefulness-promoting artificial psychostimulant, on fatigue and neuropsychological measures in patients with multiple sclerosis. Methods: Multiple sclerosis (MS) patients with a baseline score of ≥4 on the Fatigue Severity Scale (FSS) and an Expanded Disability Status Scale score <7 were eligible for the 8-week randomized, double-blind, placebo-controlled study. Modafinil was dosed up to 200 mg/day within 1 week. Assessments were performed at baseline and after 4 and 8 weeks. The primary outcome parameter was the mean change of the FSS mean score. Secondary outcome variables were other questionnaires covering fatigue, daytime sleepiness and sleep quality. Cognitive impairment was assessed by the oral version of the Symbol Digit Modalities Test (SDMT) and the Paced Auditory Serial Addition Test (PASAT). Results: The study included 121 MS patients. Dropout rate was 9%. Both treatment groups showed improvements through time. While mean FSS at 8 weeks showed a trend difference between groups in the intention-to-treat analysis, the primary endpoint was not met. Assessment of cognitive impairment by SDMT and PASAT showed contradictory results. All other secondary endpoints were not met. There was no major safety concern. Conclusions: In general, the study does not support modafinil as an effective treatment for MS fatigue. However, the study shows the need for new study designs and endpoints in MS fatigue studies.

Dose-remission of pulsating electromagnetic fields as augmentation in therapy-resistant depression: a randomized, double-blind controlled study

2014 ◽  
Vol 26 (5) ◽  
pp. 272-279 ◽  
Author(s):  
Birgit Straasø ◽  
Lise Lauritzen ◽  
Marianne Lunde ◽  
Maj Vinberg ◽  
Lone Lindberg ◽  
...  
Keyword(s):  
Electromagnetic Fields ◽  
Depression Scale ◽  
Well Being ◽  
Dose Effect ◽  
Controlled Study ◽  
Hamilton Depression Scale ◽  
Self Administration ◽  
Double Blind ◽  
Resistant Depression ◽  
Active Dose

ObjectiveTo evaluate to what extent a twice daily dose of Transcranial Pulsating ElectroMagnetic Fields (T-PEMF) was superior to once daily in patients with treatment-resistant depression as to obtaining symptom remission after 8 weeks of augmentation therapy.MethodsA self-treatment set-up of the T-PEMF device was used allowing self-administration by patients in own homes. All patients were treated for 30 min per T-PEMF session. The antidepressant medication the patients were receiving at baseline remained unchanged during the trial. The patients were randomised to either one T-PEMF dose (active dose in the morning and sham in the afternoon) or two T-PEMF doses (active dose both morning and afternoon) in a double-blind procedure. A score of 7 or less on the Hamilton Depression Scale (HAM-D17) was the criterion of remission.ResultsIn total 34 patients received active T-PEMF once a day and 31 patients twice daily. After 5 weeks of therapy remission was obtained in 26.5% and 32.3% on one dose and two doses of T-PEMF, respectively. After 8 weeks the rate of remission was 73.5% and 67.7%, respectively. The side effects as measured by the Udvalget for Kliniske Undersøgelser scale showed a better toleration of the antidepresssive medication in both treatment groups, which was reflected by the WHO-5 well-being scale with increased scores in both groups of patients.ConclusionThe high remission rate obtained by the T-PEMF augmentation was not a dose effect (one versus two daily T-PEMF sessions) but was explained by the extension of the treatment period from 5 to 8 weeks.

scholarly journals Safety and Efficacy of the Combination of BCc1 and Hep-S Nanochelating-Based Medicines in Hospitalized COVID-19 Adult Patients: A Randomized, Double-Blind, Placebo-Controlled Clinical Trial

2021 ◽  
Author(s):  
Maryam Hafizi ◽  
Somayeh Kalanaky ◽  
Saideh Fakharzadeh ◽  
Atefeh Fakharian ◽  
Somayeh Lookzadeh ◽  
...  
Keyword(s):  
Treatment Group ◽  
Inflammatory Cytokines ◽  
Clinical Symptoms ◽  
Secondary Outcome ◽  
Controlled Study ◽  
Controlled Clinical Trial ◽  
Double Blind ◽  
Double Blind Placebo ◽  
Reduce Mortality Rate ◽  
Tnf Α

Abstract Background: The mortality and morbidity of COVID‐19 disease as well as the lack of a proper medication has forced researchers and clinicians to employ urgent efficient technologies to overcome this current pandemic. In the severe forms of COVID-19, the patients develop a cytokine storm syndrome (CSS) where pro-inflammatory cytokines such as IL-6 and TNF-α play a key role in the development of this serious process. The efficiency of nanomedicines - as efficient immunomodulators - that are synthesized based on nanochelating technology have been proved in the previous studies. In the present study, the therapeutic effect of the combination of BCc1 and Hep-S nanomedicines on hospitalized COVID-19 patients was evaluated.Method: Laboratory-confirmed moderate COVID-19 patients at Masih Daneshvari Hospital were enrolled to participate in a randomized, double-blind, placebo-controlled study in two separate groups: combination of BCc1 and Hep-S (N=62) (treatment) or placebo (N=60) (placebo). The primary outcome of the study was evaluating the safety of the nanomedicines combination and its effect on the number of deceased patients, while the secondary outcome was decrease in inflammatory cytokines.Results: The evaluation of blood biochemical indices as well as clinical symptoms showed that adding the combination of BCc1 and Hep-S nanomedicines to the standard protocol of the treatment caused no adverse effects. The results analysis revealed that 28-day consumption of the nanomedicines led to a significant decrease in the mean level of IL-6 cytokine of the patients in the treatment group (p < 0.05). In addition, the patients in the treatment group had lower TNF-α levels compared to those in the control (p > 0.05) and they also showed less need for oxygen therapy. Finally, the number of the deceased patients in the treatment group was 30% lower than that of the control (p > 0.05).Conclusion: The combination of BCc1 and Hep-S, as safe nanomedicines, inhibits IL-6 as a highly important and well-known cytokine in COVID-19 pathophysiology, and presents a promising view for immunomodulation that can manage CSS and reduce mortality rate in COVID19 patients.Trial registration IRCTID, IRCT20170731035423N2. Registered 12 Jun 2020, http://www.irct.ir/ IRCT20170731035423N2.

scholarly journals Treatment of Menopausal Vasomotor Symptoms With Fezolinetant, a Neurokinin 3 Receptor Antagonist: A Phase 2a Trial

2019 ◽  
Vol 104 (12) ◽  
pp. 5893-5905 ◽  
Author(s):  
Herman Depypere ◽  
Dirk Timmerman ◽  
Gilbert Donders ◽  
Peter Sieprath ◽  
Steven Ramael ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Primary Outcome ◽  
Gastrointestinal Disorder ◽  
Controlled Study ◽  
Vasomotor Symptoms ◽  
Electronic Diary ◽  
Double Blind ◽  
Menopausal Women ◽  
Neurokinin 3 Receptor

Abstract Context The thermoregulatory center in the hypothalamus is stimulated by neurokinin 3 receptor (NK3R) activation and inhibited by estrogen-negative feedback. This balance is disrupted in menopause, producing vasomotor symptoms (VMSs). Objective To evaluate safety and efficacy of the NK3R antagonist fezolinetant in menopausal VMSs. Design Twelve-week, double-blind, randomized, placebo-controlled study. Setting Eight Belgian centers from September 2015 to October 2016. Participants Generally healthy menopausal women aged 40 to 65 years with moderate/severe VMSs. Interventions Subjects were randomized (1:1) to 90 mg of fezolinetant twice daily or placebo for 12 weeks. Main Outcome Measures Subjects captured VMS severity and frequency using an electronic diary. The primary outcome was change from baseline to week 12 in total VMS score with fezolinetant vs placebo. Secondary outcomes included timing of changes in frequency and severity of moderate/severe VMSs and quality-of-life assessments at weeks 4, 8, and 12. Pharmacodynamic and pharmacokinetic effects were assessed, as were safety and tolerability. Results Of 122 subjects screened, 87 were randomized and 80 (92%) completed the study. At week 12, fezolinetant significantly reduced total VMS score vs placebo (−26.5 vs −12.2, P < 0.001) and decreased mean frequency of moderate/severe VMSs by five episodes per day vs placebo. Severity and frequency of moderate/severe VMSs were reduced from the first day of treatment. Improvements were achieved in all quality-of-life measures. Fezolinetant was well tolerated. The most common fezolinetant-related adverse event was gastrointestinal disorder (n = 6). Conclusions Fezolinetant rapidly and significantly reduced moderate/severe VMSs, supporting its potential as an effective nonhormonal treatment option for menopausal women.

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