Efficacy and Safety of BMY 21,502 in Alzheimer Disease

1996 ◽  
Vol 30 (12) ◽  
pp. 1376-1380 ◽  
Author(s):  
Rajesh C Shrotriya ◽  
Neal R Cutler ◽  
John J Sramek ◽  
Amy E Veroff ◽  
Denise Y Hironaka
Keyword(s):  
Placebo Group ◽  
Alzheimer Disease ◽  
Disease Assessment ◽  
Efficacy And Safety ◽  
Double Blind ◽  
Nootropic Agent ◽  
Moderate Dementia ◽  
Study Population ◽  
The Difference ◽  
State Examination

OBJECTIVE: To assess the efficacy and safety of BMY 21,502, a nootropic agent, in patients with mild-to-moderate Alzheimer disease. DESIGN AND PARTICIPANTS: Sixty-nine patients with Alzheimer disease (28 men, 41 women, mean age 72 y, range 54–92, mean Mini-Mental State Examination [MMSE] score 23.5) were randomized to receive either BMY 21,502 (n = 34) or placebo (n = 35) for 12 weeks of double-blind treatment followed by a 4-week placebo washout period. SETTING: Outpatient research facility. MEASUREMENTS: Primary efficacy assessments were the Alzheimer's Disease Assessment Scale (ADAS) and the Clinical Global Impressions Scale. The Computerized Neurological Test Battery and MMSE were performed as secondary efficacy measurements. RESULTS: Although overall effects were not statistically significant (p > 0.05), patients taking BMY 21,502 showed a mean change in the ADAS cognitive score of −1.5 points at week 12, compared with −0.5 in patients who received placebo. Patients with moderate dementia (MMSE ≤20) showed a greater change at week 12 with BMY 21,502 (−2.7 points) compared with placebo (+0.3 points), but the difference was not statistically significant. Although BMY 21,502 was well tolerated in general, patients treated with BMY 21,502 experienced higher rates of abnormal liver enzyme concentrations and nausea than did those in the placebo group. There was also a higher rate of discontinuations in the BMY 21,502 group, with 12 of 34 (35%) patients in the BMY 21,502 group discontinuing, compared with 3 of 35 (9%) in the placebo group (p < 0.05). CONCLUSIONS: In this pilot study, BMY 21,502 was not found to be significantly superior to placebo during the treatment period. The compound was generally well tolerated, although 8 of 34 (24%) patients discontinued active drug treatment. Further evaluation of BMY 21,502 in a larger study population may be warranted.

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 A 36-week multicenter, randomized, double-blind, placebo-controlled, parallel-group, phase 3 clinical trial of sodium oligomannate for mild-to-moderate Alzheimer’s dementia

2021 ◽  
Vol 13 (1) ◽  
Author(s):  
Shifu Xiao ◽  
Piu Chan ◽  
Tao Wang ◽  
Zhen Hong ◽  
Shuzhen Wang ◽  
...  
Keyword(s):  
Alzheimer’S Disease ◽  
Alzheimer's Disease ◽  
Clinical Trial ◽  
Controlled Trial ◽  
Drug Effects ◽  
Disease Assessment ◽  
Phase 3 ◽  
Double Blind ◽  
Double Blind Placebo ◽  
The Difference

Abstract Background New therapies are urgently needed for Alzheimer’s disease (AD). Sodium oligomannate (GV-971) is a marine-derived oligosaccharide with a novel proposed mechanism of action. The first phase 3 clinical trial of GV-971 has been completed in China. Methods We conducted a phase 3, double-blind, placebo-controlled trial in participants with mild-to-moderate AD to assess GV-971 efficacy and safety. Participants were randomized to placebo or GV-971 (900 mg) for 36 weeks. The primary outcome was the drug-placebo difference in change from baseline on the 12-item cognitive subscale of the Alzheimer’s Disease Assessment Scale (ADAS-cog12). Secondary endpoints were drug-placebo differences on the Clinician’s Interview-Based Impression of Change with caregiver input (CIBIC+), Alzheimer’s Disease Cooperative Study-Activities of Daily Living (ADCS-ADL) scale, and Neuropsychiatric Inventory (NPI). Safety and tolerability were monitored. Results A total of 818 participants were randomized: 408 to GV-971 and 410 to placebo. A significant drug-placebo difference on the ADAS-Cog12 favoring GV-971 was present at each measurement time point, measurable at the week 4 visit and continuing throughout the trial. The difference between the groups in change from baseline was − 2.15 points (95% confidence interval, − 3.07 to − 1.23; p < 0.0001; effect size 0.531) after 36 weeks of treatment. Treatment-emergent adverse event incidence was comparable between active treatment and placebo (73.9%, 75.4%). Two deaths determined to be unrelated to drug effects occurred in the GV-971 group. Conclusions GV-971 demonstrated significant efficacy in improving cognition with sustained improvement across all observation periods of a 36-week trial. GV-971 was safe and well-tolerated. Trial registration ClinicalTrials.gov, NCT02293915. Registered on November 19, 2014

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.

A Double-Blind Trial of Bradilan (Tetranicotinoylfructose) in Perniosis in General Practice

1974 ◽  
Vol 2 (1) ◽  
pp. 56-58 ◽  
Author(s):  
F de S Donnan
Keyword(s):  
General Practice ◽  
Placebo Group ◽  
Comparative Study ◽  
Effective Treatment ◽  
Double Blind Trial ◽  
Double Blind ◽  
Blind Trial ◽  
The Difference

An initial double-blind cross-over study and a subsequent double-blind comparative study against placebo tablets has shown Bradilan (tetranicotinoylfructose) to be an effective treatment of chilblains. The difference between the active and placebo group was highly significant statistically with p < ·001

Heparin and Infusion Phlebitis: A Prospective Study

1992 ◽  
Vol 26 (10) ◽  
pp. 1211-1214 ◽  
Author(s):  
Jose A. Nieto-Rodriguez ◽  
M.A. Garcìa-Martìn ◽  
M.D. Barreda-Hernandez ◽  
M.J. Hervàs ◽  
O. Cano-Real
Keyword(s):  
Placebo Group ◽  
Confidence Interval ◽  
Venous Catheter ◽  
Final Concentration ◽  
Percent Confidence Interval ◽  
Heparin Group ◽  
Double Blind ◽  
Heparin Sodium ◽  
The Difference ◽  
Infusion Fluids

OBJECTIVE: To determine whether heparin sodium, when added to fluids infused through a peripheral catheter, reduces some local catheter-related problems (i.e., phlebitis, catheter obstruction, and extravasation of fluid) and extends the catheter's useful life. DESIGN: Prospective, double-blind, placebo-controlled. SETTING: Internal medicine ward of a general hospital with 400 beds. PATIENTS: Eighty-one consecutive acute collaborating patients who had a venous catheter in the forearm, inserted by nurses with more than one year of professional experience. INTERVENTION: Heparin sodium 1000 units/mL or NaCl 0.9% was added to compatible infusion fluids just before they were given to the patients. The final concentration of heparin was 1 unit/mL. MEASUREMENTS: Length of time until catheters were removed and reasons for their removal. RESULTS: The heparin group had a lower rate of local catheter-related problems than the placebo group (34.3 vs. 61.5 percent, respectively, p<0.05; 95 percent confidence interval for the difference 4.3 to 50.1 percent). The catheter's mean life in the heparin group was longer than that of the placebo group (98.9 ± 55.2 vs. 66.2 ± 47.2 h, respectively, p<0.05; 95 percent confidence interval for the difference 9.15 to 56.25 h). This effect was mainly attributable to prevention of noninflammatory catheter-related problems (e.g., obstruction, extravasation) and to a delay in the appearance of phlebitis. CONCLUSIONS: Heparin sodium, when added to infusion fluids to a final concentration of 1 unit/mL, diminishes local intravenous catheter-related problems and extends the catheter's life.

Efficacy and safety of sorafenib in patients with advanced clear-cell renal cell carcinoma (RCC) with diabetes: Results from the phase III TARGET study

2009 ◽  
Vol 27 (15_suppl) ◽  
pp. e16099-e16099
Author(s):  
S. Oudard ◽  
T. Eisen ◽  
C. Szczylik ◽  
M. Siebels ◽  
S. Negrier ◽  
...  
Keyword(s):  
Clear Cell ◽  
Formal Analysis ◽  
Phase Iii ◽  
Controlled Study ◽  
Efficacy And Safety ◽  
Cell Renal Cell Carcinoma ◽  
Double Blind ◽  
Diabetes Status ◽  
Study Population ◽  
Clear Cell Rcc

e16099 Background: Results of the phase III TARGET trial, a randomized, double-blind, placebo-controlled study of sorafenib (SOR) treatment in pts with clear-cell RCC in whom 1 prior systemic therapy had failed, indicated that SOR is effective and safe for pts with advanced RCC, leading to the approval of SOR for the treatment of advanced RCC. Diabetes can be associated with increased morbidity during treatment in a variety of malignancies. Therefore, an exploratory subset analysis was performed to evaluate the efficacy and safety of SOR in pts enrolled in TARGET with or without diabetes at baseline. Methods: Pts (N=903) with advanced clear-cell RCC, ECOG PS 0–2, and low- or intermediate-risk MSKCC score were randomized 1:1 to SOR 400 mg BID or placebo (PBO). End points included OS, PFS, and safety. A planned independently-assessed formal analysis of PFS showed significant benefit for SOR over PBO; consequently, pts assigned to PBO were able to cross over to SOR. Results: Pt demographics were similar for all subsets. Pre- crossover data by subset are shown in the table . The incidence of drug-related adverse events (AEs) across subgroups was consistent with that for the overall population. In pts with vs without diabetes, treatment with SOR was not associated with increased hyperglycemia (1 pt/arm in the without diabetes subgroups only) or hypertension. Conclusions: The safety profile of SOR in pts with diabetes was comparable with that for the overall study population. SOR was well tolerated and AEs were manageable. Trends in improved PFS were observed for SOR regardless of baseline diabetes status; however, the small diabetic subset limits interpretation of a SOR OS benefit in this subpopulation. *Final PFS of overall study population based on independent review from Jan 2005; all other data from May 2005 database [Table: see text] [Table: see text]

scholarly journals Efficacy and Safety of Human Placental Extract Solution on Fatigue: A Double-Blind, Randomized, Placebo-Controlled Study

2012 ◽  
Vol 2012 ◽  
pp. 1-6 ◽  
Author(s):  
Kang-Kon Lee ◽  
Whan-Seok Choi ◽  
Keun-Sang Yum ◽  
Sang-Wook Song ◽  
Sun-Myeong Ock ◽  
...  
Keyword(s):  
Placebo Group ◽  
Controlled Study ◽  
Common Symptom ◽  
Efficacy And Safety ◽  
Double Blind ◽  
Extract Solution ◽  
Group 2 ◽  
Fatigue Recovery ◽  
Placental Extract ◽  
Group 1

Introduction. Fatigue is a common symptom, but only a few effective treatments are available. This study was conducted to assess the efficacy and safety of the human placental extract solution, which has been known to have a fatigue recovery effect.Methods. A total of 315 subjects were randomly assigned to three groups: group 1 (with Unicenta solution administration), group 2 (with exclusively human placental extract administration, excluding other ingredients from the Unicenta solution), and the placebo group. Subsequently, solutions were administered for four weeks.Results. The fatigue recovery rate was 71.00% in group 1, 71.72% in group 2, and 44.21% in the placebo group, which show statistically significant differences between the group 1 and the placebo group (Pvalue = 0.0002), and between group 2 and the placebo group (Pvalue = 0.0001).Conclusion. The human placental extract solution was effective in the improvement of fatigue.

scholarly journals MERIDIAN: A phase 2, randomized, double-blind, placebo-controlled, multicenter study to evaluate the efficacy and safety of pegcetacoplan in patients with amyotrophic lateral sclerosis

2021 ◽  
Author(s):  
Frederico Mennucci de Haidar Jorge ◽  
Angela Genge ◽  
Ammar Al- Chalabi ◽  
Orla Hardiman ◽  
Alice Shen ◽  
...  
Keyword(s):  
Amyotrophic Lateral Sclerosis ◽  
Rating Scale ◽  
Phase 2 ◽  
Efficacy And Safety ◽  
Open Label ◽  
Double Blind ◽  
Double Blind Placebo ◽  
Sporadic Als ◽  
The Difference ◽  
Lateral Sclerosis

Introduction: Inflammation underlies the pathogenesis of numerous neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS). In ALS, the complement system has been implicated in the neuropathology of disease and disease progression. Pegcetacoplan, a subcutaneously administered C3 complement inhibitor, is being investigated in hematology, nephrology, and neurology. The current clinical study (NCT04579666) is investigating whether pegcetacoplan can improve survival and function in people diagnosed with apparent sporadic ALS. Objectives and Methodology: Evaluate the efficacy and safety of pegcetacoplan compared to placebo among people diagnosed with ALS in a global, multicenter, randomized, double-blind, placebo-controlled, phase 2 study. Approximately 228 patients diagnosed with apparent sporadic ALS, ≥18 years of age and with an ALS Functional Rating Scale-Revised (ALSFRS-R) score ≥30, slow vital capacity (SVC) ≥60% of the predicted value at screening, and with symptom onset within 72 weeks before screening, are eligible for enrollment. After screening, patients will be randomized 2:1 to treatment groups receiving either subcutaneous pegcetacoplan (1080 mg) or placebo twice weekly for a duration of 52 weeks. The primary efficacy endpoint is the difference in the Combined Assessment of Function and Survival (CAFS) ranked score at 52 weeks after treatment initiation. Additional, secondary functional efficacy (ALSFRS-R, percent SVC, muscle strength, quality of life, and caregiver burden) and safety endpoints will be analyzed at 52 weeks. After the placebo-controlled period, all patients will have the option to receive pegcetacoplan in an open-label period for an additional 52 weeks. Results: This ongoing study is currently enrolling participants. Conclusions: Results of this study will determine the role of complement and C3 inhibition in patients with ALS.

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