scholarly journals AbobotulinumtoxinA (Dysport®) Improves Function According to Goal Attainment in Children With Dynamic Equinus Due to Cerebral Palsy

2017 ◽  
Vol 32 (5) ◽  
pp. 482-487 ◽  
Author(s):  
Ann Tilton ◽  
Barry Russman ◽  
Resa Aydin ◽  
Umit Dincer ◽  
Raul G. Escobar ◽  
...  
Keyword(s):  
Cerebral Palsy ◽  
Goal Attainment ◽  
Secondary Analysis ◽  
Standard Of Care ◽  
Placebo Treatment ◽  
Goal Attainment Scaling ◽  
Double Blind Study ◽  
Double Blind ◽  
Large N ◽  
Common Goals

This secondary analysis of a large (n = 241), randomized, double-blind study evaluated the efficacy of 2 doses of abobotulinumtoxinA + standard of care (SOC) versus placebo + SOC in enabling children with dynamic equinus due to cerebral palsy to achieve their functional goals using Goal Attainment Scaling. Most parents/caregivers selected goals targeting aspects of gait improvement as most relevant. Mean (95% confidence interval) Goal Attainment Scaling T scores at week 4 were higher for both abobotulinumtoxinA groups versus placebo (treatment difference vs placebo: 10 U/kg/leg: 5.32 [2.31, 8.32], P = .0006, and 15 U/kg/leg 4.65 [1.59, 7.71], P = .0031). Superiority of both abobotulinumtoxinA doses versus placebo was maintained at week 12. Best goal attainment T scores were higher in the abobotulinumtoxinA groups versus placebo for the common goals of improved walking pattern, decreased falling, decreased tripping, and improved endurance. These findings demonstrate that single injections of abobotulinumtoxinA (10 and 15 U/kg/leg) significantly improved the ability of pediatric cerebral palsy patients to achieve their functional goals.

scholarly journals Auditory stimulation improves motor function and caretaker burden in children with cerebral palsy- A randomized double blind study

PLoS ONE ◽  
2018 ◽  
Vol 13 (12) ◽  
pp. e0208792 ◽  
Author(s):  
Hilla Ben-Pazi ◽  
Adi Aran ◽  
Anand Pandyan ◽  
Nava Gelkop ◽  
Gary Ginsberg ◽  
...  
Keyword(s):  
Cerebral Palsy ◽  
Motor Function ◽  
Auditory Stimulation ◽  
Blind Study ◽  
Double Blind Study ◽  
Double Blind ◽  
Children With Cerebral Palsy

scholarly journals Randomized phase 2 study of FcRn antagonist efgartigimod in generalized myasthenia gravis

Neurology ◽  
2019 ◽  
Vol 92 (23) ◽  
pp. e2661-e2673 ◽  
Author(s):  
James F. Howard ◽  
Vera Bril ◽  
Ted M. Burns ◽  
Renato Mantegazza ◽  
Malgorzata Bilinska ◽  
...  
Keyword(s):  
Adverse Events ◽  
Myasthenia Gravis ◽  
Vital Signs ◽  
Standard Of Care ◽  
Placebo Treatment ◽  
Phase 2 ◽  
Double Blind ◽  
Severity Scores ◽  
Quality Of Life Scale ◽  
Life Scale

ObjectiveTo investigate safety and explore efficacy of efgartigimod (ARGX-113), an anti-neonatal Fc receptor immunoglobulin G1 Fc fragment, in patients with generalized myasthenia gravis (gMG) with a history of anti-acetylcholine receptor (AChR) autoantibodies, who were on stable standard-of-care myasthenia gravis (MG) treatment.MethodsA phase 2, exploratory, randomized, double-blind, placebo-controlled, 15-center study is described. Eligible patients were randomly assigned (1:1) to receive 4 doses over a 3-week period of either 10 mg/kg IV efgartigimod or matched placebo combined with their standard-of-care therapy. Primary endpoints were safety and tolerability. Secondary endpoints included efficacy (change from baseline to week 11 of Myasthenia Gravis Activities of Daily Living, Quantitative Myasthenia Gravis, and Myasthenia Gravis Composite disease severity scores, and of the revised 15-item Myasthenia Gravis Quality of Life scale), pharmacokinetics, pharmacodynamics, and immunogenicity.ResultsOf the 35 screened patients, 24 were enrolled and randomized: 12 received efgartigimod and 12 placebo. Efgartigimod was well-tolerated in all patients, with no serious or severe adverse events reported, no relevant changes in vital signs or ECG findings observed, and no difference in adverse events between efgartigimod and placebo treatment. All patients treated with efgartigimod showed a rapid decrease in total immunoglobulin G (IgG) and anti-AChR autoantibody levels, and assessment using all 4 efficacy scales consistently demonstrated that 75% showed a rapid and long-lasting disease improvement.ConclusionsEfgartigimod was safe and well-tolerated. The correlation between reduction of levels of pathogenic IgG autoantibodies and disease improvement suggests that reducing pathogenic autoantibodies with efgartigimod may offer an innovative approach to treat MG.Classification of evidenceThis study provides Class I evidence that efgartigimod is safe and well-tolerated in patients with gMG.

Comparison of a New Hypnotic (Finorgal) with Placebo in a Double-Blind Trial

1979 ◽  
Vol 7 (5) ◽  
pp. 387-390
Author(s):  
Gianni Baiotti
Keyword(s):  
Placebo Treatment ◽  
Blind Study ◽  
Double Blind Study ◽  
Double Blind Trial ◽  
Double Blind ◽  
Blind Trial ◽  
The Mean ◽  
Mean Time

The hypnotic effects of Finorgal (ethchlorvynol with diphenhydramine) were compared with those of placebo in a double-blind study with crossover of treatments in thirty-five hospital in-patients. During the four-week period of Finorgal treatment there was a significant reduction in the mean time elapsing between the administration of the hypnotic and the onset of sleep, and a significant increase in the duration of sleep, compared with the four weeks of placebo treatment. There was also a significant increase in the proportion of nights when the patients felt they had slept well, and in the incidence of morning ‘hangover’ and nocturnal confusion during the Finorgal treatment periods. Patients had to be actively woken in the morning significantly more often following Finorgal administration. In patients experiencing pain in the night there was a significant reduction in the occurrence of pain during the nights when Finorgal had been given.

scholarly journals Rifabutin-Containing Triple Therapy (RHB-105) for Eradication of Helicobacter pylori: Randomized ERADICATE Hp Trial

Antibiotics ◽  
2020 ◽  
Vol 9 (10) ◽  
pp. 685 ◽  
Author(s):  
Ira N. Kalfus ◽  
David Y. Graham ◽  
Dennis S. Riff ◽  
Raymond M. Panas
Keyword(s):  
Helicobacter Pylori ◽  
Eradication Rate ◽  
Study Drug ◽  
Standard Of Care ◽  
Empiric Therapy ◽  
World Health ◽  
Double Blind Study ◽  
Double Blind ◽  
Treatment Naïve ◽  
H Pylori

Due to increasing resistance to commonly used antibiotics, the World Health Organization and Food and Drug Administration have advocated the development of new therapeutic regimens for Helicobacter pylori (H. pylori). This phase three, double-blind study (ERADICATE Hp) randomized (2:1) treatment-naïve adults with H. pylori infection and dyspepsia to RHB-105 (an all-in-one combination of omeprazole 40 mg, amoxicillin 1000 mg, and rifabutin 50 mg) or an identically-appearing placebo, both administered every 8 h for 14 days. The H. pylori eradication rate with RHB-105, using a modified intent-to-treat (mITT) population of subjects who received ≥1 dose of study drug and had test-of-eradication performed 28–35 days post-completion of therapy, was compared (one-sample Z-test) to a literature-derived comparator rate of 70% and success rate with physician-selected standard-of-care given to placebo failures. The mITT H. pylori eradication rate (95% CI) with RHB-105 of 89.4% (82.0–96.8%) was greater than both the literature-derived comparator rate (P < 0.001) and the standard-of-care rate of 63.0% (44.8–81.1%) (P = 0.006). Adverse events with an incidence ≥5% for RHB-105 were diarrhea (12.7%), headache (11.9%), chromaturia (9.3%), abdominal tenderness (6.8%), and dizziness (5.1%). No leukopenia was noted. RHB-105 (Talicia®) proved to be a safe and effective empiric therapy for H. pylori eradication.

scholarly journals Safety and Efficacy of Repeat Open-Label AbobotulinumtoxinA Treatment in Pediatric Cerebral Palsy

2017 ◽  
Vol 32 (13) ◽  
pp. 1058-1064 ◽  
Author(s):  
Mauricio R. Delgado ◽  
Marcin Bonikowski ◽  
Jorge Carranza ◽  
Edward Dabrowski ◽  
Dennis Matthews ◽  
...  
Keyword(s):  
Adverse Events ◽  
Goal Attainment ◽  
Muscle Tone ◽  
Cumulative Effect ◽  
Double Blind Study ◽  
Open Label ◽  
Double Blind ◽  
Childhood Infections ◽  
Limb Spasticity ◽  
Goal Attainment Scale

This was a prospective, repeat-treatment, open-label study (NCT01251380) of abobotulinumtoxinA for the management of lower limb spasticity in children who had completed a double-blind study. Children (2-17 years) received injections into the gastrocnemius-soleus complex, and other distal and proximal muscles as required (maximum total dose per injection cycle: 30 U/kg or 1000U). A total of 216 of the 241 double-blind patients entered the extension study and 207 received ≥1 open label injection into the gastrocnemius-soleus; 17-24% of patients also had injections into the hamstrings. The most frequent adverse events were related to common childhood infections and the most frequent treatment-related adverse event was injection site pain (n = 10). There was no evidence of a cumulative effect on adverse events. Sustained significant clinical improvements in muscle tone (Modified Ashworth Scale), spasticity (Tardieu Scale), overall clinical benefit (Physicians Global Assessment), and goal attainment (Goal Attainment Scale) were also observed across treatment cycles.

scholarly journals Efficacy and safety of inhaled ENaC inhibitor BI 1265162 in patients with cystic fibrosis: BALANCE–CF™ 1 – a randomised, Phase II study

2021 ◽  
pp. 2100746
Author(s):  
Christopher H. Goss ◽  
Isabelle Fajac ◽  
Raksha Jain ◽  
Wolfgang Seibold ◽  
Abhya Gupta ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Phase Ii ◽  
Clinical Effect ◽  
Quality Criteria ◽  
Standard Of Care ◽  
Double Blind Study ◽  
Efficacy And Safety ◽  
Double Blind ◽  
Dose Levels ◽  
Epithelial Sodium Channel Enac

BackgroundInhibition of the epithelial sodium channel (ENaC) in cystic fibrosis (CF) airways provides a mutation-agnostic approach that could improve mucociliary clearance in all CF patients. BI 1265162 is an ENaC inhibitor with demonstrated preclinical efficacy and safety already demonstrated in humans.ObjectiveWe present results from BALANCE-CF™ 1, a Phase II, placebo-controlled, randomised, double-blind study of four dose levels of BI 1265162 versus placebo for 4 weeks on top of standard of care in adults and adolescents with CF.ResultsInitially, 28 randomised subjects (n=14 each BI 1265162 200 µg BID, placebo BID) were assessed at an interim futility analysis. Compared with placebo, numerical changes of –0.8% (95%CI –6.6, 4.9) in ppFEV1 and +2.1 units (95%CI –2.4, 6.5) in LCI were observed in the active group, meeting a predefined stopping rule; accordingly, the study was terminated. Recruitment had continued during the interim analysis and pending results; 24 patients were added across three dose levels and placebo. The final results including these patients (+1.5% ppFEV1, 200 µg BID dose versus placebo) were not supportive of relevant clinical effect. LCI change was also not supportive, although interpretation was limited due to insufficient traces meeting quality criteria. A 9.4-point improvement in CFQ-R Respiratory Domain was observed in the 200 µg BID dose group versus placebo. BI 1265162 up to 200 µg BID was safe and well-tolerated. Pharmacokinetics were similar to those in healthy volunteers.ConclusionBI 1265162 was safe, but did not demonstrate a potential for clinical benefit. Development has been terminated.

Phase II randomized, double-blind, study of mFOLFIRINOX plus ramucirumab versus mFOLFIRINOX plus placebo in advanced pancreatic cancer patients hcrn GI14-198.

2019 ◽  
Vol 37 (4_suppl) ◽  
pp. TPS475-TPS475
Author(s):  
Walid Labib Shaib ◽  
Bert O'Neil ◽  
Bassel F. El-Rayes ◽  
Steven J. Cohen ◽  
Tina Ashley Khair ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Phase Ii ◽  
De Novo ◽  
Advanced Pancreatic Cancer ◽  
Standard Of Care ◽  
Double Blind Study ◽  
Current Standard ◽  
Double Blind ◽  
Angiogenic Therapy ◽  
Pancreas Adenocarcinoma

TPS475 Background: The prognosis of pancreas adenocarcinoma (PCA) remains poor. A chemotherapy backbone is the current standard of care in PCA. The choice of a chemotherapeutic backbone may impact the efficacy of anti-angiogenic therapy in PCA. Ramucirumab has increased activity with fluoropyrimidines (5FU) because 5FU increases VEGF expression. Methods: This is a double-blind, placebo controlled Phase II study. Subjects will receive either Ramucirumab or a placebo followed by mFOLFIRINOX every two weeks of a 28 day cycle until progression or discontinuation for other reasons. The primary endpoint of this clinical trial is nine month PFS defined as the time from enrollment to the time of progression or death. Among the key inclusion criteria, subjects must have recurrent or metastatic pancreas adenocarcinoma (PCA) with no prior first-line systemic treatment, ECOG PS 0-1, adequate organ function, no DVT, PE or other thromboembolism within three months of randomization. Total number of patient enrolled as of September 19, 2018 is 48 of 85 at eight sites; 27 male (56%), 42 Caucasians (87.5%), three African American (6.2%), one Asian (2%). Median age is 63 (40 - 76). Majority of patients (41) had de novo metastatic disease and six with recurrent disease after surgery. Regimen has been tolerated well with no unanticipated events. Clinical trial information: NCT02581215.

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