MO253A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED DOSE-RANGING STUDY (LUMINA-1 STUDY) TO EVALUATE THE SAFETY AND EFFICACY OF CCX140 IN SUBJECTS WITH FOCAL SEGMENTAL GLOMERULOSCLEROISS (FSGS)*

2021 ◽  
Vol 36 (Supplement_1) ◽  
Author(s):  
Frank Cortazar ◽  
John L Niles ◽  
Thomas J Schall ◽  
Peter Staehr
Keyword(s):  
Placebo Group ◽  
Adverse Events ◽  
Geometric Mean ◽  
The United States ◽  
Double Blind ◽  
Safety And Efficacy ◽  
Double Blind Placebo ◽  
Treatment Groups ◽  
Median Reduction ◽  
Dose Ranging

Abstract Background and Aims Primary Focal Segmental Glomerulosclerosis (FSGS) is the most common primary glomerular disease in patients with end stage renal disease in the United States. Current treatment regimens target reduction in proteinuria, but may have limited response or exhibit disease recurrence. CCX140 is an orally-administered selective small molecule inhibitor of the C-C chemokine receptor 2 (CCR2) under investigation for the treatment reduction of proteinuria in patients with FSGS. The primary objectives of this study were to evaluate the safety and efficacy of CCX140 in patients with FSGS and a urine protein to creatinine ratio (UPCR) of ≥1 g/g. Efficacy was assessed by the change in UPCR. Method Forty-six adult patients with primary or genetic FSGS were randomized into a double-blind, placebo-controlled Phase 2 dose-ranging study designed to evaluate the safety and efficacy of CCX140. Changes of urinary protein excretion estimation (UPCR) from baseline to Week 12 were measured in four blinded treatment groups (three active CCX140 doses of 5 mg once daily, 10 mg and 15 mg twice-daily (BID) vs placebo). Starting at Week 12, all subjects including those in the placebo group received the CCX140, 15 mg BID for an additional 12 weeks, and UPCR changes from Week 12 to Week 24 were assessed. There was a 4-week follow-up period from Week 24 to Week 28 where no CCX140 was administered. Results In the intent to treat (ITT) analysis of UPCR changes at Week 12 relative to baseline, the 15 mg BID CCX140 group exhibited the greatest reduction of UPCR (median reduction from baseline 0.9 g/g or approximately 30%, and approximately 25% reduction from baseline for the geometric mean), but that did not differ significantly from the placebo group (median reduction from baseline 0.45 g/g; or approximately 22%, and approximately 23% reduction from baseline for the geometric mean). Also, after crossover of the blinded portion of the trial to 15 mg BID active dosing, the previous placebo group did not appear to exhibit an additional reduction of UPCR. CCX140 at all doses was well-tolerated, with no serious adverse events (SAEs) during the blinded trial and a numerically lower rate of treatment-emergent adverse events in the CCX140 treatment groups. Conclusion In the study, CCX140 did not demonstrate a therapeutically meaningful reduction in proteinuria relative to the control group after 12 weeks of blinded treatment. The study provides insights into the natural disease progression of patients with primary or genetic FSGS as part of a clinical trial setting.

scholarly journals Safety and Efficacy of Tien-Hsien Liquid Practical in Patients with Refractory Metastatic Breast Cancer: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Phase IIa Trial

2012 ◽  
Vol 2012 ◽  
pp. 1-8 ◽  
Author(s):  
Wen-Hung Kuo ◽  
Chien-An Yao ◽  
Chih Hui Lin ◽  
King-Jen Chang
Keyword(s):  
Breast Cancer ◽  
Placebo Group ◽  
Metastatic Breast Cancer ◽  
Adverse Events ◽  
Pilot Trial ◽  
Metastatic Breast ◽  
Double Blind ◽  
Safety And Efficacy ◽  
Double Blind Placebo ◽  
Parallel Group

To evaluate the safety and efficacy of Tien-Hsien Liquid Practical (THL-P), a Chinese herbal mixture, in patients with refractory metastatic breast cancer, we performed a randomized, double-blind, placebo-controlled, parallel-group, phase IIa pilot trial. Patients were randomly assigned to either receive THL-P or matching placebo and followed up every 4 weeks for 24 weeks. The primary endpoint was changes in the global health status/quality of life (GHS/QOL) scale. The secondary endpoints were changes in functional and symptom scales, immunomodulating effects, and adverse events. Sixty-three patients were enrolled between June 2009 and June 2011. The intent-to-treat population included 28 patients in the THL-P group and 11 patients in the placebo group. Compared to the placebo group, the THL-P group had significant improvement from baseline to last visit in GHS/QOL (41.7 versus −33.3;P<0.05), CD3, CD4/CD8, CD19, CD16+56 positive cells (P<0.05), and higher levels of physical, role, emotional, and cognitive functioning, as well as decreased fatigue and systemic side effects. Treatment-related adverse events were mild constipation and localized itching, and no serious adverse events were reported. THL-P appears to be a safe alternative adjuvant treatment for patients with refractory metastatic breast cancer, as it effectively improves QOL and palliates cancer-related symptoms.

scholarly journals Efficacy of two siddha polyherbal decoctions, Nilavembu Kudineer and Kaba Sura Kudineer, along with standard allopathy treatment in the management of mild to moderate symptomatic COVID-19 patients—a double-blind, placebo-controlled, clinical trial

Trials ◽  
2021 ◽  
Vol 22 (1) ◽  
Author(s):  
Anurag Srivastava ◽  
Manickavasagam Rengaraju ◽  
Saurabh Srivastava ◽  
Vimal Narayanan ◽  
Vivek Gupta ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Placebo Group ◽  
Viral Load ◽  
Adverse Events ◽  
Hospital Stay ◽  
Double Blind ◽  
Double Blind Placebo ◽  
Function Test ◽  
Stay Time ◽  
The Mean

Abstract Background and aim Globally, the ongoing pursuit in exploring an effective drug to combat severe acute respiratory syndrome coronavirus-2 (SARS-CoV-2) virus has not met with significant success to date. Indian traditional medicines, especially polyherbal formulations like Nilavembu Kudineer (NVK) and Kaba Sura Kudineer (KSK) of the Siddha system of medicine, have been used as public health interventions for controlling viral epidemics like dengue and Chikungunya. These traditional therapies have been found safe, effective, and widely accepted. The current study evaluates the comparative efficacy of NVK and KSK as opposed to the placebo, in the management of mild to moderate COVID-19 disease. Methods The study was a double-blind, placebo-controlled comparative clinical trial, with the primary objective of determining the efficacy of KSK and NVK. Patients (n=125) diagnosed with mild to moderate COVID-19 symptoms were enrolled in the study over a period of 4 months (Aug 2020—Dec 2020). Participants were randomized into 3 arms; placebo-decaffeinated tea in Arm I, NVK in Arm II, and KSK in Arm III. Each arm received 60 ml of the respective treatment twice a day, post morning and evening meals, along with standard allopathy treatment for a maximum of 10 days. The main outcome measures of the study were the reduction in SARS-CoV-2 viral load, hospital stay, and time taken by the patients to become asymptomatic from symptomatic. Efficacy assessments included clinical symptoms (fever, cough, and breathlessness) each day and real-time reverse transcription-polymerase chain reaction (RT-PCR), liver function test (LFT), renal function test (RFT), and electrolytes and electrocardiogram (ECG) at baseline (day 0) and days 3, 6, and 10. Post-treatment, participants were followed up for 30 days via phone for adverse effects if any. Effects of drugs on inflammatory markers (IL6) at the end of treatment were also recorded. Adverse events (AE) were monitored throughout the study. Results The results revealed that when compared to patients in the placebo arm, those in NVK and KSK arms showed a statistically significant reduction in hospital stay time, reduction in viral load of SARS-CoV-2, and the time taken to become symptomatic from asymptomatic. Out of 125 COVID-19 patients recruited, 120 completed the study; two from the placebo group developed severe symptoms and were shifted to the intensive care unit (ICU) and three patients from Arms II and III withdrew from the study. The mean age of females (n=60) and males (n=60) enrolled was between 40.2 and 44.3 years, respectively. Results were more promising for all the patients in NVK and KSK arms as all enrolled participants (100%) under this group got discharged by day 6 as compared to only 42.5% (n=17) from the placebo group on that day. The hospital stay time for patients in Arm I was significantly longer (mean [SD]=8.4 [2.0] days) as compared to the Arms II and III (mean [SD]=4.7 [1.5] and 4.2 [1.5] days, respectively (Kruskal-Wallis test, P=0.0001). Patients in the three groups took a significantly different number of days to become asymptomatic. While Arm II and III patients took mean of 2.5 and 1.7 days, respectively, Arm I, patients took a mean of 4.2 days (Kruskal-Wallis test, P=0.0001). In all, two adverse events were recorded, one for vomiting and one for diarrhea lasting a day in Arm I and Arm II, respectively. The mean value of interleukin-6 (IL6) was significantly different in comparison to the placebo-decaffeinated tea arm (NVK=2.6 and KSK=2.2, placebo=4.0, P=0.02). The other blood biochemical parameters like C-reactive protein (CRP), lactate dehydrogenase (LDH), ferritin, and D-dimer that were analyzed at the baseline and at the time of discharge from the hospital, were not significantly different in the three arms. Conclusion NVK and KSK arms showed a statistically significant reduction in hospital stay time, reduction in viral load of SARS-CoV-2, and time taken for patients to become asymptomatic from symptomatic, when compared to the placebo (decaffeinated tea). The primary outcome measures of the KSK arm were significantly better than those in the NVK arm.

Acamprosate for the Treatment of Alcohol Dependence: A Review of Double-Blind, Placebo-Controlled Trials

CNS Spectrums ◽  
2000 ◽  
Vol 5 (2) ◽  
pp. 58-69 ◽  
Author(s):  
Barbara J. Mason ◽  
Raymond L. Ownby
Keyword(s):  
Clinical Trials ◽  
Alcohol Dependence ◽  
Latin American ◽  
The United States ◽  
Concomitant Treatment ◽  
Controlled Clinical Trials ◽  
Double Blind ◽  
Safety And Efficacy ◽  
Double Blind Placebo ◽  
Alcohol Dependent

AbstractAcamprosate (calcium acetyl-homotaurine) is a synthetic compound that crosses the blood-brain barrier and has a chemical structure similar to that of the naturally occurring amino acid neuromediators, homotaurine and γ-aminobu-tyric acid (GABA). Acamprosate appears to act primarily by restoring normal N-methyl-D-aspartate (NMDA) receptor tone in the glutamate system, and has been shown to have a specific dose-dependent effect on decreasing voluntary alcohol intake in animals with no effects on food and water consumption. The safety and efficacy of acamprosate in alcohol-dependent outpatients is currently under evaluation in the United States. Acamprosate has been available by prescription since 1989 in France and more recently in most European and Latin American coutries as well as Australia, South Africa, and Hong Kong. More than 4 million people have been treated with acamprosate since it became commercially available.The purpose of this article is to review all available double-blind, placebo-controlled clinical trials evaluating the safety and efficacy of acamprosate treatment of alcohol dependence. This work encompasses 16 controlled clinical trials conducted across 11 European countries and involves more than 4,500 outpatients with alcohol dependence. Fourteen of 16 studies found alcohol-dependent patients treated with acamprosate had a significantly greater rate of treatment completion, time to first drink, abstinence rate, and/or cumulative abstinence duration than patients treated with placebo. Additionally, a multinational open-label study of acamprosate in 1,281 patients with alcohol dependence found acamprosate to be equally effective across four major psychosocial concomitant treatment programs in maintaining abstinence and reducing consumption during any periods of relapse. An absence of known strong predictors of response to acamprosate, in conjunction with a modest but consistent effect on prolonging abstinence, and an excellent safety profile, lend support to the use of acamprosate across a broad range of patients with alcohol dependence.

scholarly journals Results of a multicentre double-blind placebo-controlled randomized trial of the liquid form of Anaferon for children in the treatment of acute upper respiratory tract infections

2019 ◽  
Vol 64 (4) ◽  
pp. 105-113
Author(s):  
B. M. Blokhin ◽  
O. V. Shamsheva ◽  
N. L. Chernaya ◽  
I. G. Sitnikov ◽  
S. G. Lazareva ◽  
...  
Keyword(s):  
Placebo Group ◽  
Adverse Events ◽  
Respiratory Tract ◽  
Average Duration ◽  
Upper Respiratory Tract ◽  
Double Blind ◽  
Double Blind Placebo ◽  
Randomized Clinical Study ◽  
Upper Respiratory ◽  
Tract Infections

Acute respiratory infections (ARI) are major causes of morbidity in children. Symptomatic treatment is insufficiently effective and requires additional drugs with etiotropic action. This multi-center double blind placebo-controlled randomized clinical study with superiority model design considers a liquid formulation of Anaferon for children.Methods. The study included 142 children from 1 month to 3 years 11 month 29 days old with ARI of the upper respiratory tract. They were randomized into Anaferon for children and Placebo groups (71 in each group) via interactive voice randomization system. The treatment period was 5 days, observation period – 14 days. The average duration of the symptoms and the severity of the respiratory disease were used as primary efficacy endpoints.Results. All 142 patients were randomized and included into ITT (Intention to treat) analysis. 140 patients (70 patients in each group) were included into PP-analysis. The patients in the Anaferon for children group had better results with average duration of ARI as compared to the Placebo group (87.7±31.5h vs. 103.3±19.4h; p =0.007). The authors marked efficacy of Anaferon for children in such parameters as disease severity (ITT-analysis: p=0.0004), total severity of disease symptoms (ITT-analysis: ANOVA: “Group” factor p=0.0004; “Day” factor p<0.0001), and percentage of recovered patients (Log-rank test, p=0.035). There were no discrepancies between the groups in concomitant therapy (ITT-analysis: ANOVA “Group-Day” factor p=0.88), bacterial complications frequency (p=1.0), adverse effects (Fisher criteria: p=0.5321). The authors registered 14 cases of adverse events (10 cases in Anaferon group and 4 cases in Placebo group) in 11 patients. None of the adverse events had a certain or probable relation to the studied drug.Conclusion. Anaferon for children proved their efficiency and safety in young children with ARI. 

scholarly journals Treatment of acute bronchitis in adults with extract of Pelargonium Sidoides: a randomised, double-blind, placebo controlled trial

2007 ◽  
pp. 49-55
Author(s):  
A. G. Chuchalin ◽  
B. Berman ◽  
V. Lemakher
Keyword(s):  
Placebo Group ◽  
Adverse Events ◽  
Controlled Trial ◽  
Alternative Therapy ◽  
Acute Bronchitis ◽  
Complete Recovery ◽  
Primary Outcome Measure ◽  
Double Blind ◽  
Double Blind Placebo ◽  
Pelargonium Sidoides

Acute bronchitis is a wide-spread disease. Although it is generally caused by viruses, antibiotics are used for its treatment too much often. So it is very important to evaluate alternative therapy of acute bronchitis. The aim of the trial was to assess efficacy and safety of Pelargonium Sidoides (EPs 7630 is a registered trademark of Dr. Willmar Schwabe GmbH & Co, Karlsruhe, Germany) compared to placebo in patients with acute bronchitis. Study design: randomised, double-blind, placebo controlled trial with planned interim analysis. The trial centres: 6 outpatient medical centres. Patients: 124 adult patients with acute bronchitis, onset of the disease ≤ 48 h, and severity of symptoms ≥ 5 according to BSS scale gave written informed concert. EPs 7630 or placebo were administered in the dose of 30 drops t.i.d. during 7 days. The primary outcome measure was BSS scoring at the 7th day of the treatment. BSS scoring decreased by 7.2 ± 3.1 in the EPs 7630 group (n = 64) vs 4.9 ± 2.7 in the placebo group (n = 60). 95 % confidence interval (CI, 1.21, 3.56) for difference of the effects between two groups demonstrated significant improvement in the EPs 7630 in 7 days of the treatment compared to the placebo group (p < 0.0001). The EPs 7630 patients had parameters of complete recovery for every of 5 individual symptoms reliably higher compared to placebo patients. During the first 4 days of the treatment therapeutic effect was noted in 68.8 % of the EPs 7630 group vs 33.3 % of the placebo group (p < 0.0001). Health-related quality of life improved better in the EPs 7630 patients compared to the placebo group. Adverse events were found in 25 of 124 patients: 15 / 64 in the EPs 7630 group and 10 / 60 in the placebo group. All the adverse events were considered as non-serious. The efficacy of EPs 7630 in treatment of adults with acute bronchitis was higher compared to placebo. It could be an efficient alternative drug in therapy of acute bronchitis at the absence of indications for antibiotics administration.

scholarly journals The Safety and Efficacy of an Enzyme Combination in Managing Knee Osteoarthritis Pain in Adults: A Randomized, Double-Blind, Placebo-Controlled Trial

Arthritis ◽  
2015 ◽  
Vol 2015 ◽  
pp. 1-7 ◽  
Author(s):  
Wolfgang W. Bolten ◽  
Michael J. Glade ◽  
Sonja Raum ◽  
Barry W. Ritz
Keyword(s):  
Adverse Events ◽  
Controlled Trial ◽  
Median Number ◽  
Secondary Outcome ◽  
Secondary Outcome Measure ◽  
Double Blind ◽  
Safety And Efficacy ◽  
Double Blind Placebo ◽  
Knee Oa ◽  
Enzyme Combination

This randomized, double-blind, placebo-controlled, and comparator-controlled trial evaluated the safety and efficacy of an enzyme combination, as Wobenzym, in adults with moderate-to-severe osteoarthritis (OA) of the knee. Adults (n=150) received Wobenzym, diclofenac (a nonsteroidal anti-inflammatory drug, NSAID), or placebo for 12 weeks. Improvement in pain scores (Lequesne Functional Index) did not differ between subjects treated with Wobenzym or diclofenac, and both treatment groups improved compared to placebo (P<0.05). Reduction in total WOMAC scores (secondary outcome measure) did not differ between Wobenzym and diclofenac, although only diclofenac emerged as different from placebo (P<0.05). The median number of rescue medication (paracetamol) tablets consumed was less in the Wobenzym group compared to placebo (P<0.05), while there was no difference between diclofenac and placebo. Adverse events were similar in frequency in Wobenzym and placebo groups (7.2% and 9.1% of subjects, resp.) and higher in diclofenac group (15.6%). Wobenzym is comparable to the NSAID diclofenac in relieving pain and increasing function in adults with moderate-to-severe painful knee OA and reduces reliance on analgesic medication. Wobenzym is associated with fewer adverse events and, therefore, may be appropriate for long-term use.

Double-blind, placebo-controlled trial of venlafaxine to treat behavioural disorders in cats: a pilot study

2021 ◽  
pp. 1098612X2110367
Author(s):  
Delphine Metz ◽  
Tiphaine Medam ◽  
Sylvia Masson
Keyword(s):  
Placebo Group ◽  
Pilot Study ◽  
Adverse Effects ◽  
Controlled Trial ◽  
Specific Inhibitor ◽  
Human Medicine ◽  
Behavioural Disorders ◽  
Double Blind ◽  
Double Blind Placebo ◽  
Treatment Groups

Objectives Venlafaxine, a specific inhibitor of both noradrenaline and serotonin, is commonly used in human medicine to treat depression, anxiety and social phobia. Its formulation in small granules renders it interesting to test on cats, which are usually reluctant to take medication. Venlafaxine was administered at 1 mg/kg for 60 days, using a double-blind, placebo-controlled protocol, to cats aged ⩾6 months exhibiting aggressiveness, fear or house-soiling. Methods After one cat’s withdrawal, 21 cats were included in the study: 11 in the venlafaxine group and 10 in the placebo group. Three consultations were conducted, on day 0, day 30 and day 60. Each visit consisted of (1) veterinarian- and owner-based scoring of the cat’s behavioural improvement; (2) scoring of the cat’s compliance with removal from its carrier and compliance with manipulation; and (3) owner scoring of the ease of administration and recording of the potential adverse effects of the treatment. Results Improvement was significantly higher in the venlafaxine group; as early as day 30, according to the veterinarian scoring, and at day 60, according to both the veterinarian and owner scoring. In contrast, neither the removal nor the manipulation scores were significantly different between the two treatment groups. Venlafaxine seemed to improve all three studied signs, as early as day 30 for fear and aggressiveness, and at day 60 for house-soiling. The adverse effects of venlafaxine were limited to drowsiness in one cat. All cats tolerated the treatment well during the 60-day period. Conclusions and relevance These results suggest that venlafaxine is efficient in treating several behavioural problems and is easy to administer. More studies should be conducted to explore its effects at different dosages on specific diagnoses.

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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