scholarly journals Long-Term (1 Year) Safety and Efficacy of Methylphenidate Modified-Release Long-Acting Formulation (MPH-LA) in Adults with Attention-Deficit Hyperactivity Disorder: A 26-Week, Flexible-Dose, Open-Label Extension to a 40-Week, Double-Blind, Randomised, Placebo-Controlled Core Study

CNS Drugs ◽  
2014 ◽  
Vol 28 (10) ◽  
pp. 951-962 ◽  
Author(s):  
Y. Ginsberg ◽  
T. Arngrim ◽  
A. Philipsen ◽  
P. Gandhi ◽  
C.-W. Chen ◽  
...  
Keyword(s):  
Modified Release ◽  
Open Label ◽  
Double Blind ◽  
Safety And Efficacy ◽  
Hyperactivity Disorder ◽  
Open Label Extension ◽  
Core Study ◽  
Long Acting ◽  
Flexible Dose

One-Year Open-Label Safety and Efficacy Study of Paliperidone Extended-Release Tablets in Patients With Schizophrenia

CNS Spectrums ◽  
2010 ◽  
Vol 15 (8) ◽  
pp. 506-514 ◽  
Author(s):  
Michelle Kramer ◽  
George Simpson ◽  
Valentinas Maciulis ◽  
Stuart Kushner ◽  
Yanning Liu ◽  
...  
Keyword(s):  
Adverse Events ◽  
Extended Release ◽  
Tolerability Profile ◽  
Open Label ◽  
Double Blind ◽  
Safety And Efficacy ◽  
Stabilization Phase ◽  
Open Label Extension ◽  
Paliperidone Extended Release

ABSTRACTIntroduction: This 52-week open-label extension (OLE) to a double-blind placebo-controlled recurrence prevention study examined the long-term safety and efficacy of flexibly-dosed paliperidone extended-release (ER) tablets in patients with schizophrenia.Methods: Patients entering the OLE either entered from the double-blind phase (placebo or paliperidone ER treatment) or entered directly from the run-in or stabilization phase (paliperidone ER) of the earlier study. During the OLE, patients were treated with flexibly-dosed paliperidone ER (3–15 mg/day; 9 mg starting dose). Safety and tolerability assessments included incidence of adverse events and extrapyramidal symptoms. Efficacy was also assessed.Results: The study population (n=235) was predominantly men (66%), 18–58 years of age. Twelve patients (5%) experienced an adverse event requiring treatment discontinuation. One or more serious treatment-emergent adverse events were reported in 13 patients (6%). There was one death. The mean Positive and Negative Syndrome Scale total score decreased from open-label baseline to endpoint for all groups, regardless of previous double-blind treatment (placebo or paliperidone ER).Conclusion: This year-long OLE provides information on the long-term safety and tolerability of paliperidone ER in patients with schizophrenia. The resulting safety and tolerability profile was similar to that seen in earlier short-term studies.

Long-term safety and efficacy of erenumab in patients with chronic migraine: Results from a 52-week, open-label extension study

Cephalalgia ◽  
2020 ◽  
Vol 40 (6) ◽  
pp. 543-553 ◽  
Author(s):  
Stewart J Tepper ◽  
Messoud Ashina ◽  
Uwe Reuter ◽  
Jan Lewis Brandes ◽  
David Doležil ◽  
...  
Keyword(s):  
Adverse Event ◽  
Chronic Migraine ◽  
Treatment Phase ◽  
Extension Study ◽  
Open Label ◽  
Double Blind ◽  
Safety And Efficacy ◽  
Open Label Extension ◽  
Event Rates

Background This study reports the long-term safety and efficacy of erenumab in chronic migraine patients. Methods This was a 52-week open-label extension study of a 12-week double-blind treatment phase study. During the double-blind treatment phase, patients received placebo or once-monthly erenumab 70 mg or 140 mg. During the open-label treatment phase, the initial monthly dose was erenumab 70 mg. Following protocol amendment, patients continued to receive erenumab 70 mg if they had already completed their Week 28 visit, otherwise, patients switched from 70 mg to 140 mg; if enrolled after the amendment, patients received 140 mg monthly throughout. Results In all, 451/609 (74.1%) enrolled patients completed the study. The exposure-adjusted patient incidence rate for any adverse event was 126.3/100 patient-years for the overall erenumab group. Overall, the adverse event profile was similar to that observed in the double-blind treatment phase. Adverse event incidence rates did not increase with long-term erenumab treatment compared with the double-blind treatment phase, and no new serious or treatment-emergent events were seen. Efficacy was sustained throughout the 52 weeks. Clinically significant reductions from double-blind treatment phase baseline (about half) were observed for monthly migraine days and migraine-specific medication days. Achievement of ≥50%, ≥75% and 100% reductions from the double-blind treatment phase baseline in monthly migraine days at Week 52 were reported by 59.0%, 33.2% and 8.9% of patients, respectively, for the combined dose group. A numerically greater benefit was observed with 140 mg compared with 70 mg at Weeks 40 and 52. Conclusions Sustained efficacy of long-term erenumab treatment in patients with chronic migraine is demonstrated, with safety results consistent with the known safety profile of erenumab and adverse event rates comparable to placebo adverse event rates in the double-blind treatment phase. Trial registration: This study is registered at ClinicalTrials.gov (NCT02174861)

scholarly journals Long-term safety and efficacy of opicapone in Japanese Parkinson’s patients with motor fluctuations

2021 ◽  
Author(s):  
Atsushi Takeda ◽  
Ryosuke Takahashi ◽  
Yoshio Tsuboi ◽  
Masahiro Nomoto ◽  
Tetsuya Maeda ◽  
...  
Keyword(s):  
Parkinson’S Disease ◽  
Parkinson's Disease ◽  
Adverse Events ◽  
Motor Fluctuations ◽  
Open Label ◽  
Double Blind ◽  
Safety And Efficacy ◽  
Open Label Extension ◽  
Off Time

AbstractThe double-blind part of the COMFORT-PD (COMt-inhibitor Findings from Opicapone Repeated Treatment for Parkinson’s Disease) study in Japanese levodopa-treated patients with Parkinson’s disease and motor fluctuations found that both opicapone 25 and 50 mg were significantly more effective than placebo. This 52-week open-label extension study evaluated the long-term safety and efficacy of opicapone 50 mg tablets in patients who completed the double-blind part of the COMFORT-PD study. Safety was monitored via adverse events, laboratory testing, and physical, cardiovascular and neurological examinations. Efficacy was primarily assessed by change in OFF-time. Secondary efficacy measures included: ON-time, percentage of OFF/ON-time responders, other outcomes from the double-blind part. 391/437 patients were transferred to the open-label extension period and included in the safety analysis set (full analysis set, n = 387; open-label completers, n = 316). Adverse events were frequently reported (n = 338, 86.4%), but < 50% were considered drug-related (39.9%) and few were considered serious (2.6%) or led to discontinuation (2.8%). Decreased OFF-time was consistently observed over the open-label period regardless of initial randomization. Change [LSM (SE)] in OFF-time from the open-label baseline to the last visit showed a persistent effect in patients initially randomized to opicapone 25 mg [− 0.37 (0.20) h, P = 0.0689] and opicapone 50 mg [− 0.07 (0.21) h, P = 0.6913] whereas opicapone 50 mg led to a statistically significant reduction in the previous placebo group [− 1.26 (0.19) h, P < 0.05]. Once-daily opicapone 50 mg was generally well tolerated and consistently reduced OFF-time over 52 weeks in Japanese levodopa-treated patients with motor fluctuations.Trial registration JapicCTI-153112; date of registration: December 25, 2015.

Long-term safety and efficacy of sarilumab over 5 years in patients with rheumatoid arthritis refractory to TNF inhibitors

Rheumatology ◽  
2021 ◽  
Author(s):  
Roy Fleischmann ◽  
Mark C Genovese ◽  
Karina Maslova ◽  
Henry Leher ◽  
Amy Praestgaard ◽  
...  
Keyword(s):  
Controlled Trial ◽  
Tnf Inhibitors ◽  
Open Label ◽  
Double Blind ◽  
Safety And Efficacy ◽  
Serious Infection ◽  
Open Label Extension ◽  
Post Marketing ◽  
Grade 3

Abstract Objective To evaluate long-term safety and efficacy of sarilumab over 5 years in patients with RA refractory to TNF inhibitors (TNFi). Methods Patients in the 24-week randomized controlled trial (RCT) TARGET (NCT01709578) whoreceived double-blind placebo or sarilumab 150 or 200 mg every 2 weeks (q2w), plus conventionalsynthetic DMARDs (csDMARDs), were eligible to receive open-label sarilumab 200 mg q2w pluscsDMARDs in the open-label extension (OLE), EXTEND (NCT01146652). OLE dose reduction to 150 mg q2w was permitted per investigators’ judgement or protocol-mandated safety concerns. Safety and efficacy were assessed through treatment-emergent adverse events (AEs), laboratoryabnormalities and clinical disease activity scores. All statistics are descriptive. Results Of 546 patients, 454 (83%) were treated with sarilumab in the OLE. Cumulative observation period was 1654.8 patient-years (PY; n = 521); 268 patients (51%) had ≥4 years’ exposure. Incidencerates per 100 PY of AEs, AEs leading to discontinuation, infection and serious infection were 160.4, 8.1, 57.8 and 3.9, respectively. Neutropenia was the most common AE (15.3 per 100 PY). Absoluteneutrophil count &lt;1000 cells/mm3 (Grade 3/4 neutropenia) was observed in 74 patients (14.2%) and normalized on treatment in 48. Clinical efficacy was sustained through 5 years’ follow-up. Efficacy was similar for patients with 1 and &gt;1 TNFi failure, and between patients who either remained on 200mg or reduced to 150 mg. Conclusion In patients with RA refractory to TNFi, sarilumab’s long-term term safety profile was consistent with previous clinical studies and post-marketing reports. Efficacy was sustained over 5years.

scholarly journals Long-term efficacy and safety of oral semaglutide and the effect of switching from sitagliptin to oral semaglutide in patients with type 2 diabetes: a 52-week, randomized, open-label extension of the PIONEER 7 trial

2020 ◽  
Vol 8 (2) ◽  
pp. e001649
Author(s):  
John B Buse ◽  
Bruce W Bode ◽  
Ann Mertens ◽  
Young Min Cho ◽  
Erik Christiansen ◽  
...  
Keyword(s):  
Type 2 Diabetes ◽  
Body Weight ◽  
Dose Adjustment ◽  
Main Phase ◽  
Open Label ◽  
Open Label Extension ◽  
Registration Number ◽  
Flexible Dose

IntroductionThe PIONEER 7 trial demonstrated superior glycemic control and weight loss with once-daily oral semaglutide with flexible dose adjustment versus sitagliptin 100 mg in type 2 diabetes. This 52-week extension evaluated long-term oral semaglutide treatment and switching from sitagliptin to oral semaglutide.Research design and methodsA 52-week, open-label extension commenced after the 52-week main phase. Patients on oral semaglutide in the main phase continued treatment (n=184; durability part); those on sitagliptin were rerandomized to continued sitagliptin (n=98) or oral semaglutide (n=100; initiated at 3 mg) (switch part). Oral semaglutide was dose-adjusted (3, 7, or 14 mg) every 8 weeks based on glycated hemoglobin (HbA1c) (target <7.0% (<53 mmol/mol)) and tolerability. Secondary endpoints (no primary) included changes in HbA1c and body weight.ResultsIn the durability part, mean (SD) changes in HbA1c and body weight from week 0 were –1.5% (0.8) and –1.3% (1.0) and –2.8 kg (3.8) and –3.7 kg (5.2) at weeks 52 and 104, respectively. In the switch part, mean changes in HbA1c from week 52 to week 104 were –0.2% for oral semaglutide and 0.1% for sitagliptin (difference –0.3% (95% CI –0.6 to 0.0); p=0.0791 (superiority not confirmed)). More patients achieved HbA1c <7.0% with oral semaglutide (52.6%) than sitagliptin (28.6%; p=0.0011) and fewer received rescue medication (9% vs 23.5%). Respective mean changes in body weight were –2.4 kg and –0.9 kg (difference –1.5 kg (95% CI –2.8 to –0.1); p=0.0321). Gastrointestinal adverse events were the most commonly reported with oral semaglutide.ConclusionsLong-term oral semaglutide with flexible dose adjustment maintained HbA1c reductions, with additional body weight reductions, and was well tolerated. Switching from sitagliptin to flexibly dosed oral semaglutide maintained HbA1c reductions, helped more patients achieve HbA1c targets with less use of additional glucose-lowering medication, and offers the potential for additional reductions in body weight.Trial registration numberNCT02849080.

scholarly journals Long‐term safety and efficacy of add‐on cannabidiol in patients with Lennox–Gastaut syndrome: Results of a long‐term open‐label extension trial

Epilepsia ◽  
2021 ◽  
Author(s):  
Anup D. Patel ◽  
Maria Mazurkiewicz‐Bełdzińska ◽  
Richard F. Chin ◽  
Antonio Gil‐Nagel ◽  
Boudewijn Gunning ◽  
...  
Keyword(s):  
Open Label ◽  
Safety And Efficacy ◽  
Open Label Extension ◽  
Extension Trial
Sign in / Sign up

Export Citation Format

Share Document