scholarly journals Combining Lovastatin and Minocycline for the Treatment of Fragile X Syndrome: Results From the LovaMiX Clinical Trial

2022 ◽  
Vol 12 ◽  
Author(s):  
Camille Champigny ◽  
Florence Morin-Parent ◽  
Laurence Bellehumeur-Lefebvre ◽  
Artuela Çaku ◽  
Jean-François Lepage ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Fragile X Syndrome ◽  
Outcome Measures ◽  
Primary Outcome ◽  
Outcome Measure ◽  
Fragile X ◽  
Combined Therapy ◽  
Primary Outcome Measure ◽  
Secondary Outcome ◽  
Global Score

Background: Limited success of previous clinical trials for Fragile X syndrome (FXS) has led researchers to consider combining different drugs to correct the pleiotropic consequences caused by the absence of the Fragile X mental retardation protein (FMRP). Here, we report the results of the LovaMiX clinical trial, the first trial for FXS combining two disease-modifying drugs, lovastatin, and minocycline, which have both shown positive effects when used independently.Aim: The main goals of the study were to assess the safety and efficacy of a treatment combining lovastatin and minocycline for patients with FXS.Design: Pilot Phase II open-label clinical trial. Patients with a molecular diagnostic of FXS were first randomized to receive, in two-step titration either lovastatin or minocycline for 8 weeks, followed by dual treatment with lovastatin 40 mg and minocycline 100 mg for 2 weeks. Clinical assessments were performed at the beginning, after 8 weeks of monotherapy, and at week 20 (12 weeks of combined therapy).Outcome Measures: The primary outcome measure was the Aberrant Behavior Checklist-Community (ABC-C) global score. Secondary outcome measures included subscales of the FXS specific ABC-C (ABC-CFX), the Anxiety, Depression, and Mood Scale (ADAMS), the Social Responsiveness Scale (SRS), the Behavior Rating Inventory of Executive Functions (BRIEF), and the Vineland Adaptive Behavior Scale second edition (VABS-II).Results: Twenty-one individuals out of 22 completed the trial. There were no serious adverse events related to the use of either drugs alone or in combination, suggesting good tolerability and safety profile of the combined therapy. Significant improvement was noted on the primary outcome measure with a 40% decrease on ABC-C global score with the combined therapy. Several outcome measures also showed significance.Conclusion: The combination of lovastatin and minocycline is safe in patients for FXS individuals and appears to improve several elements of the behavior. These results set the stage for a larger, placebo-controlled double-blind clinical trial to confirm the beneficial effects of the combined therapy.

scholarly journals 188 The Use of a Group Based Upper Limb Programme in Improving Outcome in Acute Stroke Rehabilitation

2019 ◽  
Vol 48 (Supplement_3) ◽  
pp. iii17-iii65
Author(s):  
Kerri Donnelly ◽  
Enya Mulcahy ◽  
Catherine Merrick ◽  
Orla Fitzgerald
Keyword(s):  
Acute Stroke ◽  
Grip Strength ◽  
Outcome Measures ◽  
Upper Limb ◽  
Primary Outcome ◽  
Outcome Measure ◽  
Primary Outcome Measure ◽  
Secondary Outcome ◽  
Dominant Hand ◽  
Nine Hole Peg Test

Abstract Background After a stroke 85% of patients experience altered arm function. Current research demonstrates that increasing upper limb rehabilitation results in improved outcomes (Ward et al., 2019). The Graded Repetitive Arm Supplementary Programme (GRASP) group is an established adjunctive therapy in the stroke service. The group provides additional therapy for suitable patients with upper extremity deficits. The group is run jointly by a physiotherapist and an occupational therapist. Patients attend twice weekly for a one hour period in addition to their regular therapy. The GRASP group consists of 3 levels of varying abilities and patients are categorised by their Fugl-Meyer score. Methods A prospective audit was completed in 2019 and data was collected using a word document and excel spread sheet. Standardised outcome measures were completed on admission and discharge to establish upper limb ability. The primary outcome measure was the Fugl-Meyer and the secondary outcome measures were the nine hole peg test and grip strength using the Dynamometer. Results 12 patients attended the GRASP group over this period. 75% were males. The average age was 76 years with the age range from 48-95 years. 58% of patients experienced upper limb weakness in their non-dominant hand. Post intervention data was not obtained for 5 patients due to unforeseeable discharge from the acute setting. Preliminary data to date shows that our primary outcome measure improved in 86% of patients with increases ranging from 2-11 points on the Fugl-Meyer score (Minimally Clinical Important Difference =5points). Our secondary outcome measures demonstrated patients had no change (28%) or an improvement (72%) in grip strength and 100% of patients improved on the time taken to complete the nine hole peg test. Conclusion The GRASP group was found to be effective in improving upper limb outcome measures in an acute stroke inpatient hospital setting.

scholarly journals Efficacy of a strategy-based intervention on text-level reading comprehension in persons with aphasia: a study protocol for a repeated measures study

BMJ Open ◽  
2021 ◽  
Vol 11 (7) ◽  
pp. e048126
Author(s):  
Sarah-Maria Thumbeck ◽  
Philipp Schmid ◽  
Sophie Chesneau ◽  
Frank Domahs
Keyword(s):  
Reading Comprehension ◽  
Outcome Measures ◽  
Repeated Measures ◽  
Primary Outcome ◽  
Outcome Measure ◽  
Primary Outcome Measure ◽  
Primary Objective ◽  
Secondary Outcome ◽  
Waiting Period ◽  
Reading Activities

IntroductionAt least 68% of persons with aphasia (PWA) experience reading difficulties. Even though strategy-based interventions are a promising treatment approach for text level reading comprehension deficits in PWA, empirical evidence for their efficacy remains rare. The primary objective of this study is the analysis of the efficacy of a strategy-based intervention on text-level reading comprehension and on reading activities in PWA.Methods and analysisIn a repeated measures trial, 24 PWA will first participate in a waiting period and then in a strategy-based intervention (14 face-to-face-sessions, 60 min each). We will apply two combinations of strategies to treat either the microstructure or the macrostructure, respectively. Participants will be randomly allocated to two parallel groups that will receive these combinations in interchanged sequences. Assessments will be implemented before and after each period as well as 3 and 6 months after the intervention. The primary outcome measure is text-level reading comprehension measured with a German version of the Test de Compréhension de Textes (TCT-D) and represented by the score TCT-D Total . A non-blinded and a blinded rater will evaluate the primary outcome measure. Secondary outcome measures will address specific reading functions, reading activities and cognitive functions. The sample size was determined with an a priori power analysis. For statistical analysis, we will use contrast analyses within repeated measures analysis of variance models. We expect significant improvements in primary and secondary outcome measures during the intervention as compared with changes during the waiting period.Ethics and disseminationThis study was approved by the ethics committee of Deutscher Bundesverband für akademische Sprachtherapie und Logopädie (20–10074-KA-MunmErw+Ko). Results and relevant data will be disseminated in peer-reviewed journals, at conferences and on the Open Science Framework.Trial registration numberDRKS00021411 (see Supplementary Table 1).

scholarly journals The Addition of MRI to CT Based Stroke and TIA Evaluation Does Not Impact One year Outcomes

2013 ◽  
Vol 7 (1) ◽  
pp. 17-22 ◽  
Author(s):  
Hebah Hefzy ◽  
Elizabeth Neil ◽  
Patricia Penstone ◽  
Meredith Mahan ◽  
Panayiotis Mitsias ◽  
...  
Keyword(s):  
Outcome Measures ◽  
Primary Outcome ◽  
Outcome Measure ◽  
Recurrent Stroke ◽  
Multiple Logistic Regression Analysis ◽  
Primary Outcome Measure ◽  
Secondary Outcome ◽  
Ct Angiogram ◽  
Primary Stroke Center ◽  
One Year

Background: The 2010 American Academy of Neurology guideline for the diagnosis of acute ischemic stroke recommends MRI with diffusion weighted imaging (DWI) over noncontrast head CT. No studies have evaluated the influence of imaging choice on patient outcome. We sought to evaluate the variables that influenced one-year outcomes of stroke and TIA patients, including the type of imaging utilized. Methods: Patients were identified from a prospectively collected stroke and TIA database at a single primary stroke center during a one-year period. Data were abstracted from patient electronic medical records. The primary outcome measure was death, myocardial infarction, or recurrent stroke within the following year. Secondary outcome measures included predictors of getting an MRI study. Results: 727 consecutive patients with a discharge diagnosis of stroke or TIA were identified (616 and 111 respectively); 536 had CT and MRI, 161 had CT alone, 29 had MRI alone, and one had no neuroimaging. On multiple logistic regression analysis, there were no differences in primary or secondary outcome measures among different imaging strategies. Predictors of the primary outcome measure included age and NIHSS, while performance of a CT angiogram (CTA) predicted a decreased odds of death, stroke, or MI. The strongest predictor of having an MRI was admission to a stroke unit. Conclusions: These results suggest that long-term (one-year) patient outcomes may not be influenced by imaging strategy. Performance of a CTA was protective in this cohort. A randomized trial of different imaging modalities should be considered.

scholarly journals Vibration therapy versus standard treatment for tennis elbow: A randomized controlled study

2018 ◽  
Vol 26 (3) ◽  
pp. 230949901879274 ◽  
Author(s):  
Nicholas Duncan Furness ◽  
Alistair Phillips ◽  
Sian Gallacher ◽  
James Charles Sherard Beazley ◽  
Jonathan Peter Evans ◽  
...  
Keyword(s):  
Outcome Measures ◽  
Primary Outcome ◽  
Standard Treatment ◽  
Outcome Measure ◽  
Tennis Elbow ◽  
Dropout Rate ◽  
Primary Outcome Measure ◽  
Secondary Outcome ◽  
Vibration Therapy ◽  
Outcome Scores

Aim: To determine whether a mechanical, high-frequency vibration device (Tenease™) can improve pain and function for the treatment of tennis elbow (TE), compared with standard treatment. Methods: Adults presenting to an elbow clinic with a clinical diagnosis of TE were randomized to standard treatment with physiotherapy, activity modification and analgesia or standard treatment plus Tenease therapy. Tenease therapy consisted of a 6-week period of treatment using the Tenease device with three 10-min episodes each day. The primary outcome measure was the quick Disabilities of the Arm, Shoulder and Hand score at 6 months, with scores also taken at 6 weeks. Secondary outcome measures were the Patient Rated Tennis Elbow Evaluation Score and EuroQol 5-Dimension Visual Analogue Scale at the same time points. Results: Fifty-four patients were recruited into the study. Following randomization and initial dropout, 18 patients were included in the standard group and 27 in the Tenease group. Both groups reported improvements in primary outcome measure scores. The control group had a mean score of 44.3 (standard deviation (SD) = 18.8) at baseline, which dropped to 31.2 (SD = 17.2) at 6 months ( p = 0.002). The Tenease group had a mean score of 43.2 (SD = 22.7) at baseline, which dropped to 23.4 (SD = 15.0) at 6 months ( p = 0.064). Similar improvements were seen in secondary outcome measures with none reaching statistical significance. There were no statistically significant differences seen between the primary outcome scores at 6 weeks ( p = 0.9) or 6 months ( p = 0.5). No complications were noted in either group. Conclusions: Vibration therapy did not result in any statistically significant improvement in functional outcome scores compared to standard treatment for TE. It is important to note that this was a relatively small cohort and a high dropout rate was observed.

Intervention Outcomes of Two Treatments for Muscle Tension Dysphonia: A Randomized Controlled Trial

2019 ◽  
Vol 62 (2) ◽  
pp. 272-282 ◽  
Author(s):  
Christopher R. Watts ◽  
Amy Hamilton ◽  
Laura Toles ◽  
Lesley Childs ◽  
Ted Mau
Keyword(s):  
Outcome Measures ◽  
Primary Outcome ◽  
Outcome Measure ◽  
Muscle Tension ◽  
Primary Outcome Measure ◽  
Voice Therapy ◽  
Secondary Outcome ◽  
Treatment Groups ◽  
Perceptual Evaluation ◽  
Muscle Tension Dysphonia

Purpose The aim of this study was to test the hypothesis that stretch-and-flow voice therapy (SnF) is noninferior to resonant voice therapy (RVT) for speakers with muscle tension dysphonia. Method Participants with primary muscle tension dysphonia were randomly assigned to 1 of 2 treatment groups. Participants received 6 sessions of either SnF or RVT for 6 weeks (1 session per week). Pretreatment and posttreatment audio recordings of sustained vowels and connected speech were acquired. Response to treatment was assessed using the voice handicap index (VHI) as the primary outcome measure. Secondary outcome measures included the acoustic voice quality index, the smoothed cepstral peak prominence, and scales from the Consensus Auditory-Perceptual Evaluation of Voice instrument. Data were analyzed for 21 participants who completed the study (12 in the SnF group, 9 in the RVT group). Results Direction of change for the primary outcome measure and all 3 secondary outcome measures at posttreatment was in the direction of improvement for both SnF and RVT. Confidence intervals for VHI measures did not cross the null effect line on forest plots, suggesting significant effects for both treatments on the primary outcome measure. The effect sizes for pretreatment to posttreatment changes in VHI were large for both treatment groups. Similar results were found for the secondary acoustic outcome measures. There were statistically significant pretreatment to posttreatment changes in the primary and secondary outcome measures for patients receiving both treatments, indicating significant improvement in response to both RVT and SnF. There were no statistically significant differences in pretreatment to posttreatment changes in the primary outcome measure or any secondary outcome measure between the two groups. The within-group pretreatment to posttreatment changes in Consensus Auditory-Perceptual Evaluation of Voice scales did not reach statistical significance for either RVT or SnF. Conclusions Both SnF and RVT produced positive treatment response in speakers with muscle tension dysphonia, with no statistically significant difference in the outcome measures between the two treatments. This suggests that SnF is noninferior to RVT and that both are effective options for treating vocal hyperfunction. Results from this study also support previous findings documenting the sensitivity of multidimensional acoustic measurements to treatment response.

A phase III trial of intramuscular recombinant interferon beta as treatment for exacerbating-remitting multiple sclerosis: design and conduct of study and baseline characteristics of patients

1995 ◽  
Vol 1 (2) ◽  
pp. 118-135 ◽  
Author(s):  
LD Jacobs ◽  
DL Cookfair ◽  
RA Rudick ◽  
RM Herndon ◽  
J R Richert ◽  
...  
Keyword(s):  
Primary Outcome ◽  
Interferon Beta ◽  
Outcome Measure ◽  
Primary Outcome Measure ◽  
Phase Iii ◽  
Secondary Outcome ◽  
Recombinant Interferon ◽  
Study Population ◽  
The Mean ◽  
Baseline Characteristics

The design and conduct of a randomized, double-blinded, placebo-controlled, multicenter, phase III study of recombinant interferon beta-1a (IFN-β-1a) as treatment for exacerbating-remitting MS are described, as are baseline characteristics of the study population. The purpose of the study was to determine if 6.0 × 106 IU (30 μg) of IFN-β-1a, administered by weekly intramuscular (i.m.) injections, was effective in delaying the onset of sustained disability. The primary outcome measure was time to onset of treatment failure, defined as a worsening on the Kurtzke Expanded Disability Status Scale (EDSS) of greater than or equal to 1.0 point compared with baseline, persisting for at least 6 months. An intent-to-treat design was used. The primary outcome measure was analyzed using the Mantel-Cox log-rank statistic and Kaplan-Meier survival curves. Secondary outcomes included quantitative measures of upper and lower extremity function, neuropsychological test performance, functional and quality of life assessments and several measures derived from annual brain MRI studies. Entry criteria included prestudy exacerbation rates of at least 0.67 per year and EDSS scores of 1.0–3.5. A total of 301 MS patients were randomly assigned to receive weekly i.m. injections of IFN-β-1a or placebo. The average age of the study population at entry was 37 years; 92% were Caucasian and 73% were women. The mean prestudy disease duration was 6.5 years, mean prestudy exacerbation rate was 1.2 per year and the mean EDSS score was 2.3. The randomization yielded well-balanced treatment arms. Various aspects of the study are discussed, including: (1) the decision to focus study design on sustained disability; (2) the rationale for the treatment regimen; (3) measures taken to assure the reliability of the primary outcome measure; and (4) a description of the secondary outcome measures.

scholarly journals Association between workarounds and medication administration errors in bar-code-assisted medication administration in hospitals

2017 ◽  
Vol 25 (4) ◽  
pp. 385-392 ◽  
Author(s):  
Willem van der Veen ◽  
Patricia MLA van den Bemt ◽  
Hans Wouters ◽  
David W Bates ◽  
Jos WR Twisk ◽  
...  
Keyword(s):  
Prospective Observational Study ◽  
Primary Outcome ◽  
Outcome Measure ◽  
Medication Administration ◽  
Primary Outcome Measure ◽  
Secondary Outcome ◽  
Bar Code ◽  
Implementation Evaluation ◽  
Medication Administration Errors ◽  
Large Numbers

Abstract Objective To study the association of workarounds with medication administration errors using barcode-assisted medication administration (BCMA), and to determine the frequency and types of workarounds and medication administration errors. Materials and Methods A prospective observational study in Dutch hospitals using BCMA to administer medication. Direct observation was used to collect data. Primary outcome measure was the proportion of medication administrations with one or more medication administration errors. Secondary outcome was the frequency and types of workarounds and medication administration errors. Univariate and multivariate multilevel logistic regression analysis were used to assess the association between workarounds and medication administration errors. Descriptive statistics were used for the secondary outcomes. Results We included 5793 medication administrations for 1230 inpatients. Workarounds were associated with medication administration errors (adjusted odds ratio 3.06 [95% CI: 2.49-3.78]). Most commonly, procedural workarounds were observed, such as not scanning at all (36%), not scanning patients because they did not wear a wristband (28%), incorrect medication scanning, multiple medication scanning, and ignoring alert signals (11%). Common types of medication administration errors were omissions (78%), administration of non-ordered drugs (8.0%), and wrong doses given (6.0%). Discussion Workarounds are associated with medication administration errors in hospitals using BCMA. These data suggest that BCMA needs more post-implementation evaluation if it is to achieve the intended benefits for medication safety. Conclusion In hospitals using barcode-assisted medication administration, workarounds occurred in 66% of medication administrations and were associated with large numbers of medication administration errors.

scholarly journals Day hospital versus intensive out-patient mentalisation-based treatment for borderline personality disorder: multicentre randomised clinical trial

2019 ◽  
Vol 216 (2) ◽  
pp. 79-84 ◽  
Author(s):  
Maaike L. Smits ◽  
Dine J. Feenstra ◽  
Hester V. Eeren ◽  
Dawn L. Bales ◽  
Elisabeth M. P. Laurenssen ◽  
...  
Keyword(s):  
Borderline Personality Disorder ◽  
Personality Disorder ◽  
Outcome Measures ◽  
Primary Outcome ◽  
Outcome Measure ◽  
Primary Outcome Measure ◽  
Borderline Personality ◽  
Treatment Intensity ◽  
Day Hospital ◽  
Secondary Outcomes

BackgroundTwo types of mentalisation-based treatment (MBT) have been developed and empirically evaluated for borderline personality disorder (BPD): day hospital MBT (MBT-DH) and intensive out-patient MBT (MBT-IOP). No trial has yet compared their efficacy.AimsTo compare the efficacy of MBT-DH and MBT-IOP 18 months after start of treatment. MBT-DH was hypothesised to be superior to MBT-IOP because of its higher treatment intensity.MethodIn a multicentre randomised controlled trial (Nederlands Trial Register: NTR2292) conducted at three sites in the Netherlands, patients with BPD were randomly assigned to MBT-DH (n = 70) or MBT-IOP (n = 44). The primary outcome was symptom severity (Brief Symptom Inventory). Secondary outcome measures included borderline symptomatology, personality functioning, interpersonal functioning, quality of life and self-harm. Patients were assessed every 6 months from baseline to 18 months after start of treatment. Data were analysed using multilevel modelling based on intention-to-treat principles.ResultsSignificant improvements were found on all outcome measures, with moderate to very large effect sizes for both groups. MBT-DH was not superior to MBT-IOP on the primary outcome measure, but MBT-DH showed a clear tendency towards superiority on secondary outcomes.ConclusionsAlthough MBT-DH was not superior to MBT-IOP on the primary outcome measure despite its greater treatment intensity, MBT-DH showed a tendency to be more effective on secondary outcomes, particularly in terms of relational functioning. Patients receiving MBT-DH and MBT-IOP, thus, seem to follow different trajectories of change, which may have important implications for clinical decision-making. Longer-term follow-up and cost-effectiveness considerations may ultimately determine the optimal intensity of specialised treatments such as MBT for patients with BPD.

scholarly journals Diagnosis and outcome measures in trials for neoplastic meningitis: a review of the literature and clinical experience

1998 ◽  
Vol 4 (6) ◽  
pp. E6 ◽  
Author(s):  
Michael J. Glantz ◽  
Marc C. Chamberlin ◽  
Beverly C. Walters
Keyword(s):  
Cerebrospinal Fluid ◽  
Outcome Measures ◽  
Primary Outcome ◽  
Randomized Clinical Trials ◽  
Outcome Measure ◽  
Clinical Care ◽  
Primary Outcome Measure ◽  
Neoplastic Meningitis ◽  
Review Of The Literature ◽  
Cytological Examination

Innovative approaches to the treatment of neoplastic meningitis are being widely tested. Unfortunately, research on diagnostic strategies and outcome measures on which any advances in treatment ultimately depend, has not been avidly pursued. A critical review of the literature on neoplastic meningitis published since 1978 was undertaken by using MEDLINE and other English language databases. All articles addressing the issues of diagnostic or response criteria were included. Randomized clinical trials (RCTs) were emphasized. Prospectively collected data from the authors' institution correlating the results of cerebrospinal fluid (CSF) cytological examinations with Karnofsky Performance Scale (KPS) score are also discussed. Twenty-six studies (representing 1208 patients) fulfilled search criteria. Only three were RCTs. Cerebrospinal fluid cytology was the sole diagnostic criterion in two-thirds of studies. The results of CSF cytological examination alone or in combination with other clinical or laboratory endpoints constituted the primary outcome measure in 85%. Few studies attempted to address known deficiencies in the reliability and validity of these measures, and correlation between measures was poor. Quality of life was never used as a primary outcome measure. All currently available measurements, including CSF cytology, biochemistry, immunological, and molecular markers, neuroimaging studies, clinical examination, and survival, suffer from poor sensitivity and/or specificity, and often correlate poorly with each other. Although CSF cytological examination, performed according to a rigorous, research-supported protocol, may be the optimum diagnostic and outcome measure at this time, additional research is a prerequisite for any further advances in the clinical care of patients with neoplastic meningitis.

Statistical analysis, trial design and duration in Alzheimer's disease clinical trials: a review

2011 ◽  
Vol 24 (5) ◽  
pp. 689-697 ◽  
Author(s):  
P. A. Thompson ◽  
D. E. Wright ◽  
C. E. Counsell ◽  
J. Zajicek
Keyword(s):  
Alzheimer’S Disease ◽  
Alzheimer's Disease ◽  
Clinical Trials ◽  
Statistical Analysis ◽  
Statistical Methods ◽  
Outcome Measures ◽  
Primary Outcome ◽  
Outcome Measure ◽  
Primary Outcome Measure ◽  
Primary Analysis

ABSTRACTBackground: The social and economic burden of Alzheimer's disease (AD) and its increasing prevalence has led to much work on new treatment strategies and clinical trials. The search for surrogate markers of disease progression continues but traditional parallel group trial designs that use well-established, but often insensitive, clinical outcome measures predominate.Methods: We performed a systematic search across the Cochrane Library and PubMed abstracts published between January 2004 and August 2009. Information regarding the clinical trial methodology, outcome measures, intervention type and primary statistical analysis techniques was extracted and categorized, according to a standard protocol.Results: We identified 149 papers describing results from clinical trials in AD containing sufficient detail for our purposes. The largest proportion (38%) presented results of trials based on tests of cognition as the primary outcome measure. The primary analysis in most papers (85%) was a univariate significance test of a single primary outcome measure.Conclusions: The majority of trials reported a comparison of baseline and end-point assessment over relatively short patient follow-up periods, using univariate statistical methods to compare differences between intervention and control groups in the primary analysis. There is considerable scope to introduce newer statistical methods and trial designs in treatment evaluations in AD.

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