scholarly journals Prevention of acute respiratory viral infections and influenza in children during the peaks of seasonal morbidity: the results of the international double-blind placebo-controlled randomized clinical trial

2020 ◽  
Vol 65 (3) ◽  
pp. 109-120
Author(s):  
N. A. Geppe ◽  
A. V. Gorelov ◽  
O. V. Shamsheva ◽  
I. G. Sitnikov ◽  
E. P. Sitnikova ◽  
...  
Keyword(s):  
Placebo Group ◽  
Bacterial Infections ◽  
Viral Infections ◽  
Group Versus ◽  
Study Drug ◽  
Preventive Therapy ◽  
Efficacy And Safety ◽  
Double Blind ◽  
Double Blind Placebo ◽  
Respiratory Viral Infections

The authors conducted an international multicenter double-blind, placebo-controlled clinical study to obtain additional data on the efficacy and safety of a 12-week course of Anaferon for children for the prevention of acute respiratory viral infections (ARVI), including influenza, in children during the rise in seasonal morbidity (RCT of the Ministry of Health of the Russian Federation: №356 dated June 29, 2017; ClinicalTrials.gov Identifier: NCT03301155).Materials and methods. The study involved 1,036 children (1 month – 6 years 11 months 29 days) during two epidemiological seasons. The patients were divided into 2 groups: 528 patients received Anaferon for children 1 tablet a day for 12 weeks, 508 patients received placebo according to Anaferon for children scheme. The primary end point was the duration of the period from the first dose of the drug until manifestation of ARVI/influenza. Additional end points were percentage of children not falling with ARVI/influenza during 4-, 8- and 12-week course of preventive therapy; percentage of children with respiratory or ear-nose-throat bacterial infections requiring antibiotics within 12 week; percentage of children hospitalized with ARVI/influenza or their complications within 12 week. To assess safety, the authors analyzed the presence and nature of the adverse events (AEs), their severity, connection with the medication, outcome. The authors used the following statistical methods: calculation of hazard ratio, median time to the manifestation of symptoms of ARVI / influenza, 95% confidence intervals.Results. The Intention-to-treat (ITT) and Per Protocol [РР] analysis included the data of 1,021 [975] patients: 520 [494] – Anaferon for children group and 501 [481] –Placebo group. The average duration of the period from the first dose of the drug to the development of ARVI/influenza symptoms obtained as a result of the analysis of the statistical model was 428.8 [434.1] days for Anaferon for children group, that is 1.5 times higher than in Placebo group (275.8 [274.9] days; p=0.001 [p=0.0009]). The percentage of children without ARVI/influenza was 99.2% [99.2%] in Anaferon for children group (versus 90.2% [90.0%] in Placebo group; p=0.0003 [p=0.0003]) within 4 weeks, 92.7% [92.3%] (versus 82.8% [82.7%]; p=0.0003 [p=0.0003]) within 8 weeks, and 81.5% [81.8%] (versus 73.4% [73.4%], respectively; p=0.0021 [p=0.0021]). None of the patients was hospitalized for ARVI/influenza or complications. The frequency of AEs in Anaferon for children and Placebo groups had no differ. No one AE definitely related to the study drug was registered.Conclusion. The results confirm the efficacy and safety of a 12-week course of Anaferon for children to prevent ARVI and influenza during seasonal rise of morbidity in children.

Treatment of acute respiratory viral infections: results of a multicenter, double-blind, placebo-controlled, randomized clinical trial

2020 ◽  
Vol 18 (3) ◽  
pp. 178-189
Author(s):  
V.V. Rafalsky ◽  
◽  
R.F. Khamitov ◽  
T.I. Martynenko ◽  
M.V. Chernogorova ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Randomized Clinical Trial ◽  
Viral Infections ◽  
Wilcoxon Test ◽  
Controlled Study ◽  
Control Group ◽  
Efficacy And Safety ◽  
Double Blind ◽  
Double Blind Placebo ◽  
Respiratory Viral Infections

This multicenter, double-blind, placebo-controlled clinical trial was conducted to obtain additional data on the efficacy and safety of Anaferon for the treatment of acute respiratory viral infections (ARVI) during seasonal increase in their incidence (RCT of the Ministry of Health of Russia No 356 dated 24.07.2018; ClinicalTrials.gov Identifier: NCT03707912). Patients and methods. Between October 2018 and March 2019, a total of 204 patients aged 18 to 70 years with ARVI symptoms were included in this study within the first 24 hours of symptom onset. Patients were randomized into 2 groups: 104 individuals received oral Anaferon (should be kept in the mouth until completely dissolved and without food) according to the following scheme: 1 tablet every 30 minutes during the first 2 hours; then 3 more doses at regular intervals during the first day; then 1 tablet 3 times a day on days 2–5; 100 individuals received placebo according to the same scheme. The primary endpoint was time to resolution of symptoms of clinically diagnosed and/or PCR (polymerase chain reaction) – confirmed ARVI. Addithional endpoints included: time to resolution of symptoms of ARVI confirmed by PCR; proportion of patients with resolution of symptoms of clinically diagnosed and/or PCR-confirmed ARVI and separately PCR-confirmed ARVI; severity of clinically diagnosed and/or PCR-confirmed ARVI (assessed by ‘area under the curve’ for the total severity index); the number of antipyretic doses taken according to indications on days 1–3 of treatment (checked in the patient's diary); proportion of patients who required antibiotic treatment on days 4–7 of follow-up. To assess safety, we analyzed the incidence and type of adverse events (AEs), their severity, association with drug use, and treatment outcome. The following statistical methods were used: Fisher's exact test, Cochran–Mantel–Haenszel test, Wilcoxon test, and repeated measures ANOVA, PROC MIXED. Results. A total of 203 patients were included in the intention-to-treat (ITT) and per protocol (РР) analysis: 103 [95] individuals in the Anaferon arm and 100 [93] individuals in the Placebo arm. Patients receiving Anaferon had significantly shorter time to resolution of all ARVI symptoms than patients receiving placebo: 4.1 ± 1.6 days vs 4.5 ± 1.5 days (p = 0.032). The disease was on average 1 day shorter in patients from the experimental group compared to controls: 3.6 ± 1.5 days vs 4.6 ± 1.5 days (p = 0.007). The proportion of patients who had resolution of symptoms of clinically diagnosed and/or PCR-confirmed ARVI was significantly higher in the Anaferon arm compared to Placebo arm (p = 0.0012). Among patients with PCR-verified ARVI, treatment with Anaferon resulted in twice as frequent recovery as in the control group on day 4 (53.7% vs 26.3%) and day 7 (70.7% vs 36.8%). In the Anaferon arm, we observed shorter disease duration and higher proportion of patients recovered compared to the Placebo arm; however, patients in both groups had a similar need for antipyretic drugs on days 1–3 of treatment, as well as for antibiotic therapy. The incidence of AEs in the Anaferon and Placebo groups did not vary significantly. No AEs with a reliable association with Anaferon were registered. Conclusion. Our findings suggest high efficacy and safety of Anaferon in patients with ARVI. The best results were obtained in patients with PCR-verified diagnosis, which can be attributed to the involvement of the interferon system in the action of the drug. The results of this RCT confirm the data obtained in previous studies and long-term clinical experience of using Anaferon. Key words: acute respiratory viral infections, ARVI, treatment, effective therapy, placebo-controlled study, Anaferon, randomized clinical trial, comprehensive therapy, efficacy, safety

scholarly journals Treatment of influenza and other acute respiratory viral infections in children: multicenter double-blind placebo-controlled randomized comparative clinical trial

2021 ◽  
Vol 66 (1) ◽  
pp. 131-139
Author(s):  
L. V. Lukashova ◽  
O. I. Afanasyeva ◽  
E. V. Portnyagina ◽  
A. N. Khmeleva ◽  
N. P. Chernyshova
Keyword(s):  
Clinical Trial ◽  
Placebo Group ◽  
Body Temperature ◽  
Adverse Events ◽  
Viral Infections ◽  
Group Versus ◽  
Adoptive Parents ◽  
Efficacy And Safety ◽  
Number Of Patients ◽  
Respiratory Viral Infections

The article presents the main results of a randomized clinical trial of the efficacy and safety of Panavir®, rectal suppositories 100 μg, in children with influenza and other acute respiratory viral infections (ARVI).Characteristics of children and research methods. The trial included 40 children from 12 to 17 years old, the children were randomized into 2 groups: 20 children were treated with Panavir®, 1 rectal suppository per day for 7days, 20 children received placebo according to Panavir® regimen on the background of standard symptomatic therapy. The scientists monitored the efficacy criteria: the primary criteria: recovery time and the number of patients with successful treatment (normalization of axillary body temperature no later than 48 hours of therapy); the main secondary criteria: the timing of the normalization of body temperature, the disappearance of intoxication and catarrhal symptoms and satisfaction with the results of treatment by the parents /adoptive parents of patients on the IMPSS scale.Results. In the Group of Panavir®, the authors registered shorter mean recovery periods (4.5 days versus 7.4 days in the Group of Placebo), normalization of body temperature (39.1 hours versus 76.1 hours) and regression of intoxication manifestations (2.9 days versus 4.3 days) and catarrhal (3.4 days versus 5.6 days) syndromes (with statistical significance of intergroup differences). The treatment success was achieved in 85% of cases in the Panavir® Group versus 30% of cases in the Placebo Group; according to this criterion Panavir® exceeded placebo by at least 24.7%. At the end of the therapy course, 80% of the parents /adoptive parents were satisfied with the treatment results in the Panavir® Group versus 25% of cases in the Placebo Group. The authors determined a comparable incidence of adverse events in the Panavir® and Placebo Groups. There were no adverse events associated with the administration of Panavir®.Conclusion. This study established the efficacy and safety of Panavir® in the treatment of children of 12-17 years old with influenza and other acute respiratory viral infections. This drug with a combined antiviral and immunomodulatory effect is promising for pediatric practice.

scholarly journals Efficacy and Safety of Ergoferon in Children from 6 Months to 6 Years Old with Acute Respiratory Viral Infections in Contemporary Outpatient Practice: A Multicenter, Double-Blind, Placebo-Controlled Randomized Trial

2021 ◽  
Vol 2021 ◽  
pp. 1-9
Author(s):  
N. A. Geppe ◽  
B. M. Blokhin ◽  
O. V. Shamsheva ◽  
S. T. Abdrakhmanova ◽  
K. A. Alikhanova ◽  
...  
Keyword(s):  
Placebo Group ◽  
Viral Infections ◽  
Respiratory Infections ◽  
Symptomatic Therapy ◽  
Efficacy And Safety ◽  
Double Blind ◽  
Double Blind Placebo ◽  
Intention To Treat ◽  
Therapy Effectiveness ◽  
Outpatient Practice

To evaluate the efficacy and safety of Ergoferon in combination with symptomatic therapy in children from 6 months to 6 years old with acute respiratory infections (ARI) in contemporary outpatient practice, an international, multicenter, double-blind, placebo-controlled, randomized, parallel-group clinical trial was performed. Derived by technological treatment of antibodies to interferon gamma, histamine, and CD4, Ergoferon was previously proved to modulate its molecular targets promoting effective antiviral protection. The data of 282 patients with oral temperature ≥38.0°C plus mild to moderate severity of flu-like nonspecific and nasal/throat/chest symptoms were included in intention-to-treat analysis (n = 140, Ergoferon group; n = 142, placebo group). Time to alleviation of all ARI symptoms was the primary endpoint, and 8 outcome measures were estimated as the secondary endpoints. Respiratory viruses were confirmed in 57.1% (Ergoferon) and 54.9% (Placebo) of patients. Compared to placebo, Ergoferon reduced time to alleviation of all ARI symptoms (4.5 ± 1.7 versus 5.2 ± 2.2 days in placebo; p = 0.026 ) including fever (2.8 ± 1.5 vs 3.4 ± 2.0; p = 0.031 ), flu-like nonspecific (4.0 ± 1.8 vs 4.7 ± 2.2, p = 0.022 ), and nasal/throat/chest (4.3 ± 2.0 versus 5.0 ± 2.3; p = 0.024 ) symptoms. Ergoferon add-on therapy decreased the mean total symptom severity score (according to 4-point scale for each symptom), ARI severity, frequency of antipyretic use, and percentage of complication requiring antibiotics and increased the percentage of recovered patients. The incidence of adverse events (AEs) in the Ergoferon group was significantly lower compared to the placebo group (7.0% versus 18.8%; p = 0.004 ) including infectious diseases (3.5% vs 12.5%; p = 0.008 ). In the Ergoferon group, AEs were mild or moderate. In 8 (57.1%) cases, AEs were unrelated to Ergoferon, in 5 (35.7%), the relationship was uncertain, and in 1 (7.1%), it was possible (mild rash on the face). Ergoferon treatment is beneficial for infants and young children with ARI in contemporary outpatient practice. Being well-tolerated, Ergoferon increases the symptomatic therapy effectiveness and improves the patient condition and disease outcomes.

scholarly journals Clinical Efficacy and Safety of Pentanedioic Acid Imidazolyl Ethanamide in Patients Aged 3 to 6 years with Influenza and other Acute Respiratory Viral Infections Based on the Results of Double-Blind Randomized Placebo-Controlled Multicenter Study

2021 ◽  
Vol 65 (6) ◽  
pp. 166-174
Author(s):  
N. A. Geppe ◽  
E. G. Kondyurina ◽  
N. G. Kolosova ◽  
E. A. Jablokova
Keyword(s):  
Placebo Group ◽  
Body Temperature ◽  
Adverse Events ◽  
Viral Infections ◽  
The Body ◽  
Efficacy And Safety ◽  
Double Blind ◽  
Respiratory Viral Infections ◽  
The Individual ◽  
Pentanedioic Acid

Study Goals. Assessment of the efficacy and safety of the Pentanedioic Acid Imidazolyl Ethanamide (IPA) in the treatment of influenza and other acute respiratory viral infections (ARVI) in children aged 3–6 years. Children Characteristics and Study Methods. The randomized double-blind placebo-controlled multicenter clinical study involving 190 patients aged 3–6 years was carried out as follows: 95 patients received IPA at 30 mg/day dose once for 5 days and 95 patients – placebo using the same pattern. The therapy efficacy was assessed by the body temperature, dynamics of the individual symptoms of the disease as per the Severity Scale for the patients with influenza and other acute respiratory viral infections (Scale) and the incidence rate of complications of the acute respiratory viral infections. The primary endpoint is the period of the score reduction on the Scale to 2 points, providing that there is not more than 1 score on the individual subscales with the body temperature normalization from the start of treatment. The safety analysis was carried out using the assessment of the nature and incidence rate of the adverse events. Results. The IPA use at 30 mg/day dose significantly promotes the achievement of goals for the primary endpoint – the average value in the IPA group is 91.79 h (95% confidence interval – CI from 87.45 to 96.13), in the placebo group – 100.12 h (95% CI from 96.73 to 103.51). In the IPA group, the body temperature returned to normal by 18.56 h faster in average compared with the placebo. The regression of the catarrhal and intoxication symptoms was significantly promoted with the IPA use as follows: by the 3rd day of the therapy, the average score on the Scale was 5.22 points for IPA, and it was statistically significantly lower than that in the placebo group – 6.21 points. The comparative analysis of the incidence rate of adverse events did not reveal the statistically significant differences between IPA and placebo. None of the adverse events recorded was clinically significant, and none caused the cancellation or change in the dosage of the study drug. Conclusions. IPA broad-spectrum antiviral drug at 30 mg/day dose has demonstrated the high efficacy in the treatment of the acute respiratory viral infections of various etiologies in children aged 3–6 years. The IPA use compared with the placebo causes the significant reduction of the fever period, accelerates the relief of the intoxication and catarrhal symptoms, and decreases the recovery time. The IPA is characterized by high tolerability and high safety, which allows to recommend the drug for the treatment of influenza and ARVI (acute respiratory viral infections) in children from 3 years of age.

scholarly journals A 12-Week, Multicenter, Randomized, Double-Blind, Placebo-Controlled Clinical Trial for Evaluation of the Efficacy and Safety of DKB114 on Reduction of Uric Acid in Serum

Nutrients ◽  
2020 ◽  
Vol 12 (12) ◽  
pp. 3794
Author(s):  
Yu Hwa Park ◽  
Do Hoon Kim ◽  
Jung Suk Lee ◽  
Hyun Il Jeong ◽  
Kye Wan Lee ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Uric Acid ◽  
Placebo Group ◽  
Serum Uric Acid ◽  
Controlled Study ◽  
Efficacy And Safety ◽  
Food Ingredient ◽  
Double Blind ◽  
Double Blind Placebo ◽  
Significant Difference

This study sought to investigate the antihyperuricemia efficacy and safety of DKB114 (a mixture of Chrysanthemum indicum Linn flower extract and Cinnamomum cassia extract) to evaluate its potential as a dietary supplement ingredient. This clinical trial was a randomized, 12-week, double-blind, placebo-controlled study. A total of 80 subjects (40 subjects with an intake of DKB114 and 40 subjects with that of placebo) who had asymptomatic hyperuricemia (7.0–9.0 mg/dL with serum uric acid) was randomly assigned. No significant difference between the DKB114 and placebo groups was observed in the amount of uric acid in serum after six weeks of intake. However, after 12 weeks of intake, the uric acid level in serum of subjects in the DKB114 group decreased by 0.58 ± 0.86 mg/dL and was 7.37 ± 0.92 mg/dL, whereas that in the placebo group decreased by 0.02 ± 0.93 mg/dL and was 7.67 ± 0.89 mg/dL, a significant difference (p = 0.0229). In the analysis of C-reactive protein (CRP) change, after 12 weeks of administration, the DKB114 group showed an increase of 0.05 ± 0.27 mg/dL (p = 0.3187), while the placebo group showed an increase of 0.10 ± 0.21 mg/dL (p = 0.0324), a statistically significant difference (p = 0.0443). In the analysis of amount of change in apoprotein B, after 12 weeks of administration, the DKB114 group decreased by 4.75 ± 16.69 mg/dL (p = 0.1175), and the placebo group increased by 3.13 ± 12.64 mg/dL (p = 0.2187), a statistically significant difference between the administration groups (p = 0.0189). In the clinical pathology test, vital signs and weight measurement, and electrocardiogram test conducted for safety evaluation, no clinically significant difference was found between the ingestion groups, confirming the safety of DKB114. Therefore, it may have potential as a treatment for hyperuricemia and gout. We suggest that DKB114 as a beneficial and safe food ingredient for individuals with high serum uric acid. Trial registration (CRIS.NIH. go. Kr): KCT0002840.

scholarly journals Raloxifene and body composition and muscle strength in postmenopausal women: a randomized, double-blind, placebo-controlled trial

2010 ◽  
Vol 162 (2) ◽  
pp. 371-376 ◽  
Author(s):  
Didy E Jacobsen ◽  
Monique M Samson ◽  
Marielle H Emmelot-Vonk ◽  
Harald J J Verhaar
Keyword(s):  
Body Composition ◽  
Placebo Group ◽  
Muscle Strength ◽  
Postmenopausal Women ◽  
Controlled Trial ◽  
Group Versus ◽  
The Body ◽  
Fat Free Mass ◽  
Double Blind ◽  
Double Blind Placebo

ObjectiveTo compare the effects of raloxifene and placebo on body composition and muscle strength.DesignRandomized, double-blind, placebo-controlled trial involving 198 healthy women aged 70 years or older conducted between July 2003 and January 2008 at the University Medical Centre, Utrecht, The Netherlands.MethodsParticipants were randomly assigned to receive raloxifene 60 mg or placebo daily for 12 months. Measurements were taken at baseline, 3, 6, and 12 months, and change from baseline was calculated. Main outcome measures were body composition (bioelectrical impedance analysis), muscle strength, and muscle power (maximum voluntary isometric knee extension strength, explosive leg extensor power, and handgrip strength).ResultsAt 12 months, the body composition of women taking raloxifene was significantly different from that of women taking placebo: fat-free mass (FFM) had increased by a mean of 0.83 (2.4) kg in the raloxifene group versus 0.03 (1.5) kg in the placebo group (P=0.05), and total body water had increased by a mean of 0.6 (1.8) litres in the raloxifene group versus a decrease of 0.06 (1.1) litres in the placebo group (P=0.02). Muscle strength and power were not significantly different.ConclusionRaloxifene significantly changed body composition (increased FFM; increased water content) compared with placebo in postmenopausal women.

scholarly journals Efficacy and Safety of Yokukansan in Treatment-Resistant Schizophrenia: A Randomized, Multicenter, Double-Blind, Placebo-Controlled Trial

2015 ◽  
Vol 2015 ◽  
pp. 1-11 ◽  
Author(s):  
Tsuyoshi Miyaoka ◽  
Motohide Furuya ◽  
Jun Horiguchi ◽  
Rei Wake ◽  
Sadayuki Hashioka ◽  
...  
Keyword(s):  
Placebo Group ◽  
Controlled Trial ◽  
Controlled Study ◽  
Efficacy And Safety ◽  
Treatment Resistant ◽  
Double Blind ◽  
Double Blind Placebo ◽  
Intention To Treat ◽  
The Difference ◽  
The Individual

Objectives. We aimed at evaluating both the efficacy and safety of TJ-54 (Yokukansan) in patients with treatment-resistant schizophrenia. This randomized, multicenter, double-blind, placebo-controlled study was conducted.Methods. One hundred and twenty antipsychotic-treated inpatients were included. Patients were randomized to adjuvant treatment with TJ-54 or placebo. During a 4-week follow-up, psychopathology was assessed using the Positive and Negative Syndrome Scale (PANSS).Results. TJ-54 showed a tendency of being superior to placebo in reduction total, positive, and general PANSS scores in treatment-resistant schizophrenia, but the difference was not statistically significant in both per-protocol set (PPS) and intention-to-treat (ITT). However, in PPS analysis, compared to the placebo group, the TJ-54 group showed statistically significant improvements in the individual PANSS subscale scores for lack of spontaneity and flow of conversation (TJ-54:−0.23±0.08; placebo:−0.03±0.08,P<0.018), tension (TJ-54:−0.42±0.09; placebo:−0.18±0.09,P<0.045), and poor impulse control (TJ-54:−0.39±0.10; placebo:−0.07±0.10,P<0.037).Conclusions. The results of the present study indicate that TJ-54 showed a tendency of being superior to placebo in reduction PANSS scores in treatment-resistant schizophrenia, but the difference was not statistically significant. However, compared to the placebo group, TJ-54 group showed statistically significant improvements in the individual PANSS subscale scores.

scholarly journals Efficacy and Safety of MLC601 in the Treatment of Mild Cognitive Impairment: A Pilot, Randomized, Double-Blind, Placebo-Controlled Study

2017 ◽  
Vol 7 (1) ◽  
pp. 136-142 ◽  
Author(s):  
Hossein Pakdaman ◽  
Ali Amini Harandi ◽  
Mehdi Abbasi ◽  
Hosein Delavar Kasmaei ◽  
Farzad Ashrafi ◽  
...  
Keyword(s):  
Cognitive Impairment ◽  
Placebo Group ◽  
Mild Cognitive Impairment ◽  
Cognitive Function ◽  
Disease Assessment ◽  
Controlled Study ◽  
Efficacy And Safety ◽  
Double Blind ◽  
Double Blind Placebo ◽  
Global Cognitive Function

Background and Aim: Mild cognitive impairment (MCI) is characterized by declined cognitive function greater than that expected for a person’s age. The clinical significance of this condition is its possible progression to dementia. MLC601 is a natural neuroprotective medication that has shown promising effects in Alzheimer disease. Accordingly, we conducted this randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of MLC601 in MCI patients. Methods: Seventy-two patients with a diagnosis of MCI were recruited. The included participants were randomly assigned to groups to receive either MLC601 or placebo. An evaluation of global cognitive function was performed at baseline as well as at 3-month and 6-month follow-up visits. Global cognitive function was assessed by Mini-Mental State Examination (MMSE) and Alzheimer’s Disease Assessment Scale-cognitive subscale (ADAS-cog) scores. Efficacy was evaluated by comparing global function scores between the 2 groups during the study period. Safety assessment included adverse events (AEs) and abnormal laboratory results. Results: Seventy patients completed the study, 34 in the MLC601 group and 36 in the placebo group. The mean changes (±SD) in cognition scores over 6 months in the MLC601 group were –2.26 (±3.42) for the MMSE and 3.82 (±6.16) for the ADAS-cog; in the placebo group, they were –2.66 (±3.43) for the MMSE and 4.41 (±6.66) for the ADAS-cog. The cognition changes based on both MMSE and ADAS-cog scores were statistically significant between the placebo and the MLC601 group (p < 0.001). Only 5 patients (14.7%) reported minor AEs in the MLC601 group, the most commonly reported of which were gastrointestinal, none of them leading to patient withdrawal. Conclusion: MLC601 has shown promising efficacy and acceptable AEs in MCI patients.

scholarly journals Efficacy and safety of calcium, magnesium, potassium, and sodium oxybates (lower-sodium oxybate [LXB]; JZP-258) in a placebo-controlled, double-blind, randomized withdrawal study in adults with narcolepsy with cataplexy

SLEEP ◽  
2020 ◽  
Author(s):  
Richard K Bogan ◽  
Michael J Thorpy ◽  
Yves Dauvilliers ◽  
Markku Partinen ◽  
Rafael Del Rio Villegas ◽  
...  
Keyword(s):  
Placebo Group ◽  
Group Versus ◽  
Sodium Oxybate ◽  
Efficacy And Safety ◽  
Open Label ◽  
Double Blind ◽  
Clinical Trial Registration ◽  
Titration Period ◽  
Secondary Efficacy Endpoint ◽  
Optimized Treatment

Abstract Study Objectives Evaluate efficacy and safety of lower-sodium oxybate (LXB), a novel oxybate medication with 92% less sodium than sodium oxybate (SXB). Methods Adults aged 18–70 years with narcolepsy with cataplexy were eligible. The study included a ≤30-day screening period; a 12-week, open-label, optimized treatment and titration period to transition to LXB from previous medications for the treatment of cataplexy; a 2-week stable-dose period (SDP); a 2-week, double-blind, randomized withdrawal period (DBRWP); and a 2-week safety follow-up. During DBRWP, participants were randomized 1:1 to placebo or to continue LXB treatment. Results Efficacy was assessed in 134 participants who received randomized treatment, and safety was assessed in all enrolled participants (N = 201). Statistically significant worsening of symptoms was observed in participants randomized to placebo, with median (first quartile [Q1], third quartile [Q3]) change in weekly number of cataplexy attacks from SDP to DBRWP (primary efficacy endpoint) in the placebo group of 2.35 (0.00, 11.61) versus 0.00 (−0.49, 1.75) in the LXB group (p &lt; 0.0001; mean [standard deviation, SD] change: 11.46 [24.751] vs 0.12 [5.772]), and median (Q1, Q3) change in Epworth Sleepiness Scale score (key secondary efficacy endpoint) of 2.0 (0.0, 5.0) in the placebo group versus 0.0 (−1.0, 1.0) in the LXB group (p &lt; 0.0001; mean [SD] change: 3.0 [4.68] vs 0.0 [2.90]). The most common treatment-emergent adverse events with LXB were headache (20.4%), nausea (12.9%), and dizziness (10.4%). Conclusions Efficacy of LXB for the treatment of cataplexy and excessive daytime sleepiness was demonstrated. The safety profile of LXB was consistent with SXB. Clinical trial registration NCT03030599.

scholarly journals Efficacy and safety of Kami-guibi-tang for mild cognitive impairment: a pilot, randomized, double-blind, placebo-controlled trial

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Hee-Yeon Shin ◽  
Ha-Ri Kim ◽  
Geon-Ho Jahng ◽  
Chul Jin ◽  
Seungwon Kwon ◽  
...  
Keyword(s):  
Cognitive Impairment ◽  
Placebo Group ◽  
Mild Cognitive Impairment ◽  
Adverse Events ◽  
Vital Signs ◽  
Efficacy And Safety ◽  
Mri Findings ◽  
Double Blind ◽  
Double Blind Placebo ◽  
Significant Difference

Abstract Background Mild cognitive impairment (MCI) is considered an intermediate phase between normal aging and dementia. As the majority of cases of amnestic MCI (aMCI) progress to Alzheimer’s disease (AD), it is considered the prodromal stage of AD, and a treatment target for prevention of further cognitive decline. However, no medications have been shown to have symptomatic or preventive benefits in MCI. Kami-guibi-tang (KGT) is a traditional herbal formula used in Korean medicine to treat amnesia, which is reported to increase acetylcholine levels via activation of choline acetyltransferase. The objective of this study was to evaluate the efficacy and safety of KGT in patients with aMCI. Methods This study was designed as a single-center, randomized, double-blind, placebo-controlled pilot study. Participants diagnosed with aMCI were randomized to receive either KGT or placebo granules for 24 weeks. The efficacy measure was a change in the Seoul Neuropsychological Screening Battery (SNSB) score. The safety measures included the occurrence of adverse events and abnormalities in vital signs and blood chemistry, electrocardiogram (ECG), and brain magnetic resonance imaging (MRI) findings. Results A total of 16 patients in the KGT group and 14 patients in the placebo group were investigated in the study. The mean score of Clinical Dementia Rating-Sum of Boxes (CDR-SB) significantly improved from 1.53 (0.64) points to 1.13 (0.62) points in the KGT group (p = 0.010), whereas it worsened from 1.61 (0.88) points to 1.75 (0.94) points in the placebo group. There was a significant difference in the CDR-SB scores between the two groups after the intervention (p = 0.045). The total SNSB-D scores and the scores in the memory domain after the treatment were significantly higher than the baseline values in the KGT group, but not in the placebo group. The frequency of adverse events was not significantly different between the two groups, and there were no abnormalities in vital signs or blood test, ECG, and brain MRI findings after the intervention. Conclusions KGT may provide a safe and effective treatment option for patients with aMCI. Further studies with a larger sample size are needed to validate the findings. Trial registration Korean Clinical Trial Registry, ID: KCT0002407; Registered on March 30, 2017, http://cris.nih.go.kr/

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