A safety, tolerability and biomarker update from an ongoing open-label extension study of RG6042 in adults with early manifest Huntington’s disease

2019 ◽  
Vol 405 ◽  
pp. 26-27
Author(s):  
B. Leavitt ◽  
S. Tabrizi ◽  
P. Sanwald Ducray ◽  
E. Wild ◽  
V. Schlegel ◽  
...  
Keyword(s):  
Huntington's Disease ◽  
Huntington’S Disease ◽  
Extension Study ◽  
Open Label ◽  
Open Label Extension

Safety and Exploratory Efficacy at 36 Months in Open-HART, an Open-Label Extension Study of Pridopidine in Huntington’s Disease

2017 ◽  
Vol 6 (3) ◽  
pp. 189-199 ◽  
Author(s):  
Andrew McGarry ◽  
Karl Kieburtz ◽  
Victor Abler ◽  
Igor D. Grachev ◽  
Sanjay Gandhi ◽  
...  
Keyword(s):  
Huntington's Disease ◽  
Huntington’S Disease ◽  
Extension Study ◽  
Open Label ◽  
Open Label Extension

scholarly journals Evaluation of the Safety of Deutetrabenazine at Higher Doses to Treat Chorea in Huntington’s Disease

CNS Spectrums ◽  
2021 ◽  
Vol 26 (2) ◽  
pp. 162-163
Author(s):  
Samuel Frank ◽  
Christina Vaughan ◽  
David Stamler ◽  
David Oakes ◽  
Mat D. Davis ◽  
...  
Keyword(s):  
Huntington's Disease ◽  
Huntington’S Disease ◽  
Drug Exposure ◽  
Open Label ◽  
Titration Period ◽  
Open Label Extension ◽  
Pharmaceutical Industries ◽  
Post Hoc ◽  
Higher Doses

AbstractBackgroundIn the First-HD pivotal trial, the maximum deutetrabenazine dose evaluated to treat chorea associated with Huntington’s disease (HD chorea) was 48 mg/d, which is the approved maximum dose for this population. In ARC-HD, an open-label extension study evaluating the long-term efficacy and safety of deutetrabenazine to treat HD chorea, dosage ranged from 6 mg/d to 72 mg/d, with doses ≥12 mg/d administered twice daily. Doses in ARC-HD were increased by 6 mg/d per week in a response-driven manner based on efficacy and tolerability until 48 mg/d (Week 8). At the investigator’s discretion, further increases were permitted by 12 mg/d per week to a maximum of 72 mg/d. This post-hoc analysis evaluates the safety and tolerability of deutetrabenazine >48 mg/d compared to ≤48 mg/d to treat HD chorea in ARC-HD.MethodsPatient counts and safety assessments were attributed to patients when they received a dose of either ≤48 mg/d or >48 mg/d. For 9 selected adverse events (AEs), we compared AE rates adjusted for duration of drug exposure (as number of AEs/year) at ≤48 mg/d or >48 mg/d. The AE rates were determined after titration when participants were on stable doses of deutetrabenazine.ResultsAll 113 patients were exposed to doses ≤48 mg/d (177.1 patient-years) and 49 patients were ever exposed to doses >48 mg/d (74.1 patient-years). In patients taking deutetrabenazine >48 mg/d compared to ≤48 mg/d after the titration period, there were no apparent differences in exposure-adjusted AE rates.ConclusionsBased on clinical experience, some patients with HD may benefit from doses higher than 48 mg/d to adequately control chorea. These doses were tolerated without apparent increase in the exposure-adjusted rates of selected AEs after titration. This analysis does not address the occurrence of other AEs or whether adequate efficacy was achieved at lower doses, factors that may have influenced dose increases.FundingTeva Pharmaceutical Industries Ltd., Petach Tikva, Israel

Q07 Long-term safety and tolerability of pridopidine in patients with huntington's disease (HD): results of the mermaihd study open-label extension

2012 ◽  
Vol 83 (Suppl 1) ◽  
pp. A56.3-A57
Author(s):  
F Squitieri ◽  
B Landwehrmeyer ◽  
R Reilmann ◽  
A Rosser ◽  
J Garcia de Yebenes ◽  
...  
Keyword(s):  
Huntington's Disease ◽  
Huntington’S Disease ◽  
Open Label ◽  
Open Label Extension

scholarly journals Intravitreal conbercept for diabetic macular oedema: 2-year results from a randomised controlled trial and open-label extension study

2021 ◽  
pp. bjophthalmol-2020-318690
Author(s):  
Kun Liu ◽  
Hanying Wang ◽  
Wei He ◽  
Jian Ye ◽  
Yanping Song ◽  
...  
Keyword(s):  
Macular Oedema ◽  
Laser Photocoagulation ◽  
Sham Group ◽  
Controlled Trial ◽  
Diabetic Macular Oedema ◽  
Intravitreal Injections ◽  
Phase Iii ◽  
Extension Study ◽  
Open Label ◽  
Open Label Extension

BackgroundTo demonstrate the efficacy and safety of intravitreal injections of conbercept versus laser photocoagulation in the treatment of diabetic macular oedema (DME).MethodsA 12-month multicentre, randomised, double-masked, double-sham, parallel controlled, phase III trial (Sailing Study), followed by a 12-month open-label extension study. Patients with centre-involved DME were randomly assigned to receive either laser photocoagulation followed by pro re nata (PRN) sham intravitreal injections (laser/sham) or sham laser photocoagulation followed by PRN 0.5 mg conbercept intravitreal injections (sham/conbercept). Patients who entered the extension study received PRN conbercept treatment. The primary endpoint was the changes in best-corrected visual acuity (BCVA) from baseline.ResultsA total of 248 eyes were included in the full analysis set and 157 eyes continued in the extension study. Significant improvement in mean change in BCVA from baseline to month 12 was observed in the sham/conbercept group (8.2±9.5 letters), whereas no improvement was observed in the laser/sham group (0.3±12.0 letters). Patients in the laser/sham group showed a marked improvement in BCVA after the switch to conbercept in the extension study, and there was no difference in BCVA between the two groups at the end of the extension study.ConclusionThe use of a conbercept PRN intravitreal injection regimen improved the BCVA of patients with DME, and its efficacy was better than that of laser photocoagulations, and the same efficacy was observed when the eyes treated with laser alone were switched to conbercept.Trial registration numberNCT02194634.

scholarly journals Long-Term Efficacy and Safety of Deutetrabenazine for Chorea in Huntington’s Disease: Results From the ARC-HD Open-label Study

CNS Spectrums ◽  
2021 ◽  
Vol 26 (2) ◽  
pp. 164-165
Author(s):  
Samuel Frank ◽  
Claudia M. Testa ◽  
David Stamler ◽  
Elise Kayson ◽  
David Oakes ◽  
...  
Keyword(s):  
Huntington's Disease ◽  
Huntington’S Disease ◽  
Rating Scale ◽  
Study Drug ◽  
Motor Dysfunction ◽  
Open Label ◽  
Entire Treatment Period ◽  
End Of Treatment ◽  
Pharmaceutical Industries

AbstractBackgroundChorea is a prominent motor dysfunction in Huntington’s disease (HD). Deutetrabenazine, a vesicular monoamine transporter 2 (VMAT2) inhibitor, is FDA-approved for the treatment of chorea in HD. In the pivotal, 12-week First-HD trial, deutetrabenazine treatment reduced the Unified Huntington’s Disease Rating Scale (UHDRS) total maximal chorea (TMC) score versus placebo. ARC-HD, an open-label extension study, evaluated long-term safety and efficacy of deutetrabenazine dosed in a response-driven manner for treatment of HD chorea.MethodsPatients who completed First-HD (Rollover) and patients who converted overnight from a stable dose of tetrabenazine (Switch) were included. Safety was assessed over the entire treatment period; exposure-adjusted incidence rates (EAIRs; adverse events [AEs] per person-year) were calculated. A stable, post-titration time point of 8 weeks was chosen for efficacy analyses.ResultsOf 119 patients enrolled (Rollover, n=82; Switch, n=37), 100 (84%) completed ≥1 year of treatment (mean [SD] follow-up, 119 [48] weeks). End of study EAIRs for patients in the Rollover and Switch cohorts, respectively, were: any AE, 2.6 and 4.3; serious AEs, 0.13 and 0.14; AEs leading to dose suspension, 0.05 and 0.04. Overall, 68% and 73% of patients in Rollover and Switch, respectively, experienced a study drug–related AE. Most common AEs possibly related to study drug were somnolence (17% Rollover; 27% Switch), depression (23%; 19%), anxiety (9%; 11%), insomnia (10%; 8%), and akathisia (9%; 14%). Rates of AEs of interest include suicidality (9%; 3%) and parkinsonism (6%; 11%). In both cohorts, mean UHDRS TMC score and total motor score (TMS) decreased from baseline to Week 8; mean (SD) change in TMC score (units) was –4.4 (3.1) and –2.1 (3.3) and change in TMS was –7.1 (7.3) and –2.4 (8.7) in Rollover and Switch, respectively. While receiving stable dosing from Week 8 to 132 (or end of treatment), patients showed minimal change in TMC score (0.9 [5.0]), but TMS increased compared to Week 8 (9.0 [11.3]). Upon drug withdrawal, there were no remarkable AEs and TMC scores increased 4.4 (3.7) units compared to end of treatment.ConclusionsThe type and severity of AEs observed in long-term deutetrabenazine exposure are consistent with the previous study. Efficacy in reducing chorea persisted over time. There was no unexpected worsening of HD or chorea associated with HD upon deutetrabenazine withdrawal.FundingTeva Pharmaceutical Industries Ltd., Petach Tikva, Israel

scholarly journals Oxcarbazepine adjunctive therapy for partial seizures in Japanese pediatric patients: a randomized double-blind placebo-controlled study and open-label extension study

2019 ◽  
Vol 11 (1) ◽  
pp. 30-45
Author(s):  
Tateki Fujiwara ◽  
Rie Teshima ◽  
Ikuo Sugiyama ◽  
Ken Tsuchida ◽  
Yoko Ohtsuka
Keyword(s):  
Adjunctive Therapy ◽  
Pediatric Patients ◽  
Extension Study ◽  
Controlled Study ◽  
Partial Seizures ◽  
Open Label ◽  
Double Blind ◽  
Double Blind Placebo ◽  
Open Label Extension
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