scholarly journals HAPPi Kneecaps! A double-blind, randomised, parallel group superiority trial investigating the effects of sHoe inserts for adolescents with patellofemoral PaIn: phase II feasibility study

2021 ◽  
Vol 14 (1) ◽  
Author(s):  
Isobel C. O’Sullivan ◽  
Kay M. Crossley ◽  
Steven J. Kamper ◽  
Marienke van Middelkoop ◽  
Bill Vicenzino ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Recruitment Rate ◽  
Full Scale ◽  
Trial Registration ◽  
Foot Orthoses ◽  
Double Blind ◽  
Parallel Group ◽  
Patient Reported ◽  
To Receive

Abstract Background Patellofemoral pain (PFP) affects one-third of adolescents and can persist into adulthood, negatively impacting health and quality of life. Foot orthoses are a recommended treatment for adults with PFP, but have not been evaluated in adolescents. The primary objective was to determine the feasibility of conducting a full-scale randomised controlled trial (RCT) evaluating effects of contoured, prefabricated foot orthoses on knee pain severity and patient-perceived global change, compared to flat insoles. The secondary objective was to describe outcomes on a range of patient-reported outcome measures. Methods We recruited adolescents aged 12–18 years with PFP of ≥2 months duration into a double-blind, randomised, parallel-group feasibility trial. Participants were randomised to receive prefabricated contoured foot orthoses or flat shoe insoles, and followed for 3 months. Participants and outcome assessors were blinded to group allocation. Primary outcomes were feasibility of a full-scale RCT (number of eligible/enrolled volunteers; recruitment rate; adherence with the intervention and logbook completion; adverse effects; success of blinding; drop-out rate), and credibility and expectancy of interventions. Secondary outcomes were patient-reported measures of pain, symptoms, function, quality of life, global rating of change, patient acceptable symptom state, and use of co-interventions. Results 36 out of 279 (12.9%) volunteers (27 female, mean (SD) age 15 (2) years, body mass 60 (13) kg) were eligible and enrolled, at a recruitment rate of 1.2 participants/week. 17 participants were randomised to receive foot orthoses, and 19 to flat insoles. 15 participants returned logbooks; 7/15 (47%) adhered to the intervention. No serious adverse events were reported. 28% (10/36, 4 pandemic-related) of participants dropped out before 3 months. Blinding was successful. Both groups found the inserts to be credible. Conclusions Based on a priori criteria for feasibility, findings suggest that a full-scale RCT comparing contoured foot orthoses to flat insoles in adolescents with PFP would not be feasible using the current protocol. Prior to conducting a full-scale RCT, feasibility issues should be addressed, with protocol modifications to facilitate participant retention, logbook completion and shoe insert wear. Trial registration Australian New Zealand Clinical Trials Registry (ANZCTR): ACTRN12619000957190. Date registered: 8/07/2019.

scholarly journals Happi Kneecaps! A Double-Blind, Randomised, Parallel Group Superiority Trial Investigating the Effects of Shoe Inserts for Adolescents with Patellofemoral Pain: Phase II Feasibility Study

2021 ◽  
Author(s):  
Isobel C O'Sullivan ◽  
Kay M Crossley ◽  
Steven J Kamper ◽  
Marienke van Middelkoop ◽  
Bill Vicenzino ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Patellofemoral Pain ◽  
Recruitment Rate ◽  
Full Scale ◽  
Foot Orthoses ◽  
Double Blind ◽  
Parallel Group ◽  
Patient Reported ◽  
To Receive

Abstract Background: Patellofemoral pain (PFP) affects one-third of adolescents and can persist into adulthood, negatively impacting health and quality of life. Foot orthoses are a recommended treatment for adults with PFP, but have not been evaluated in adolescents. The primary objective was to determine the feasibility of conducting a full-scale randomised controlled trial (RCT) evaluating effects of contoured, prefabricated foot orthoses on knee pain severity and patient-perceived global change, compared to flat insoles. The secondary objective was to describe outcomes on a range of patient-reported outcome measures. Methods: We recruited adolescents aged 12-18 years with PFP of ³2 months duration into a double-blind, randomised, parallel-group feasibility trial. Participants were randomised to receive prefabricated contoured foot orthoses or flat shoe insoles, and followed for 3 months. Participants and outcome assessors were blinded to group allocation. Primary outcomes were feasibility of a full-scale RCT (number of eligible/enrolled volunteers; recruitment rate; adherence with the intervention and logbook completion; adverse effects; success of blinding; drop-out rate), and credibility and expectancy of interventions. Secondary outcomes were patient-reported measures of pain, symptoms, function, quality of life, global rating of change, patient acceptable symptom state, and use of co-interventions.Results: 36 out of 279 (12.9%) volunteers (27 female, mean (SD) age 15 (2) years, body mass 60 (13) kg) were eligible and enrolled, at a recruitment rate of 1.2 participants/week. 17 participants were randomised to receive foot orthoses, and 19 to flat insoles. 15 participants returned logbooks; 7/15 (47%) adhered to the intervention. No serious adverse events were reported. 28% (10/36, 4 pandemic-related) of participants dropped out before 3 months. Blinding was successful. Both groups found the inserts to be credible. Conclusions: Based on a priori criteria for feasibility, findings suggest that a full-scale RCT comparing contoured foot orthoses to flat insoles in adolescents with PFP would not be feasible using the current protocol. Prior to conducting a full-scale RCT, further feasibility studies are needed, with protocol modifications to facilitate participant retention, logbook completion and shoe insert wear. Trial registration: Australian New Zealand Clinical Trials Registry (ANZCTR): ACTRN12619000957190. Date registered: 8/07/2019.

scholarly journals DOP24 Patient-reported health-related quality-of-life outcomes with vedolizumab vs. adalimumab treatment of ulcerative colitis: Results of the VARSITY trial

2020 ◽  
Vol 14 (Supplement_1) ◽  
pp. S063-S063
Author(s):  
E V Loftus ◽  
S W Schreiber ◽  
S Danese ◽  
L Peyrin-Biroulet ◽  
J F Colombel ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Ulcerative Colitis ◽  
Randomised Trial ◽  
Double Blind ◽  
Specific Domain ◽  
Faecal Calprotectin ◽  
Meaningful Improvement ◽  
Treatment Difference ◽  
Patient Reported

Abstract Background Patients with ulcerative colitis (UC) experience substantial impairment in quality of life (QOL). Patient QOL endpoints are important measures of treatment outcome. We evaluated the effects of intravenous vedolizumab vs. adalimumab on QOL in VARSITY, the first head-to-head trial comparing the efficacy and safety of biologics in patients with moderately to severely active UC. Methods VARSITY was a phase 3b, double-blind, double-dummy, randomised trial (NCT02497469; EudraCT 2015-000939-33). QOL was assessed using the inflammatory bowel disease questionnaire (IBDQ) at baseline, Week (Week) 30, and Week 52. Endpoints included clinically meaningful IBDQ improvement (defined as an increase in total score of ≥16 points from baseline to Week 52), IBDQ remission (defined as a total score of >170 points at Week 52) and change from baseline in IBDQ-specific domain scores (bowel symptoms, systemic symptoms, emotional function, and social function) at Week 30 and Week 52. Serum C-reactive protein (CRP) and faecal calprotectin (FCP) were also assessed as indicators of disease activity. Results Among randomised patients, 383 (vedolizumab) and 386 (adalimumab) patients received ≥1 dose of study drug (N=769). At Week 52, clinically meaningful IBDQ improvement was observed in 52.0% (vedolizumab) vs. 42.2% (adalimumab) of patients (treatment difference 9.7%; 95% confidence interval [CI], 2.7% to 16.7%), while IBDQ remission was achieved by 50.1% (vedolizumab) vs. 40.4% (adalimumab) of patients (treatment difference 9.6%; 95% CI, 2.8% to 16.5%). Mean (standard deviation [SD]) changes in IBDQ total score from baseline for observed cases favoured vedolizumab over adalimumab (Week 30: 61.3 [39.8] vs. 52.6 [42.8]; Week 52: 66.1 [41.8] vs. 60.4 [42.2]; Figure 1). IBDQ subscores showed similar favourable trends for vedolizumab (Figure 2). At Week 52, mean (SD) changes from baseline in CRP for patients treated with vedolizumab vs. adalimumab were –50.9 (174.8) nmol/l vs. –37.2 (169.2) nmol/l and for FCP were –2187.3 (7440.4) µg/g vs. –1846.6 (4560.6) µg/g (Figure 3). Among patients with FCP >250 µg/g at baseline, the proportion of patients achieving FCP ≤250 µg/g was 33.9% vs. 24.5% at Week 30 and 35.2% vs. 28.9% at Week 52 for patients treated with vedolizumab vs. adalimumab, respectively. Conclusion Based on IBDQ total score and subscores, more patients with UC treated with vedolizumab than with adalimumab achieved clinically meaningful improvement and clinical remission. Reduced inflammation, as indicated by improvements in CRP and FCP, was consistent with improvements in QOL.

Randomized, Controlled, Double-Blind, Cross-Over Trial Assessing Treatment Preference for Pazopanib Versus Sunitinib in Patients With Metastatic Renal Cell Carcinoma: PISCES Study

2014 ◽  
Vol 32 (14) ◽  
pp. 1412-1418 ◽  
Author(s):  
Bernard Escudier ◽  
Camillo Porta ◽  
Petri Bono ◽  
Thomas Powles ◽  
Tim Eisen ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Renal Cell Carcinoma ◽  
Cell Carcinoma ◽  
Metastatic Renal Cell Carcinoma ◽  
Patient Preference ◽  
Renal Cell ◽  
Double Blind ◽  
Patient Reported ◽  
Intent To Treat

Purpose Patient-reported outcomes may help inform treatment choice in advanced/metastatic renal cell carcinoma (RCC), particularly between approved targeted therapies with similar efficacy. This double-blind cross-over study evaluated patient preference for pazopanib or sunitinib and the influence of health-related quality of life (HRQoL) and safety factors on their stated preference. Patients and Methods Patients with metastatic RCC were randomly assigned to pazopanib 800 mg per day for 10 weeks, a 2-week washout, and then sunitinib 50 mg per day (4 weeks on, 2 weeks off, 4 weeks on) for 10 weeks, or the reverse sequence. The primary end point, patient preference for a specific treatment, was assessed by questionnaire at the end of the two treatment periods. Other end points and analyses included reasons for preference, physician preference, safety, and HRQoL. Results Of 169 randomly assigned patients, 114 met the following prespecified modified intent-to-treat criteria for the primary analysis: exposure to both treatments, no disease progression before cross over, and completion of the preference questionnaire. Significantly more patients preferred pazopanib (70%) over sunitinib (22%); 8% expressed no preference (P < .001). All preplanned sensitivity analyses, including the intent-to-treat population, statistically favored pazopanib. Less fatigue and better overall quality of life were the main reasons for preferring pazopanib, with less diarrhea being the most cited reason for preferring sunitinib. Physicians also preferred pazopanib (61%) over sunitinib (22%); 17% expressed no preference. Adverse events were consistent with each drug's known profile. Pazopanib was superior to sunitinib in HRQoL measures evaluating fatigue, hand/foot soreness, and mouth/throat soreness. Conclusion This innovative cross-over trial demonstrated a significant patient preference for pazopanib over sunitinib, with HRQoL and safety as key influencing factors.

scholarly journals Effect of Transcutaneous Electrical Nerve Stimulation on Pain, Function, and Quality of Life in Fibromyalgia: A Double-Blind Randomized Clinical Trial

2015 ◽  
Vol 95 (1) ◽  
pp. 129-140 ◽  
Author(s):  
Brian Noehren ◽  
Dana L. Dailey ◽  
Barbara A. Rakel ◽  
Carol G.T. Vance ◽  
Miriam B. Zimmerman ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Clinical Trial ◽  
Nerve Stimulation ◽  
Transcutaneous Electrical Nerve Stimulation ◽  
Treatment Modalities ◽  
Double Blind ◽  
Electrical Nerve Stimulation ◽  
Double Blind Placebo ◽  
Patient Reported

BackgroundFibromyalgia is a common chronic pain condition that has a significant impact on quality of life and often leads to disability. To date, there have been few well-controlled trials assessing the utility of nonpharmacological treatment modalities such as transcutaneous electrical nerve stimulation (TENS) in the management of pain and improvement in function in individuals with fibromyalgia.ObjectivesThe purpose of this study will be to complete a long-term, multicenter study to assess the effects of TENS in women with fibromyalgia.DesignThis will be a phase II randomized, double-blind, placebo-controlled, multicenter clinical trial.ParticipantsThree hundred forty-three participants with fibromyalgia will be recruited for this study.InterventionParticipants will be randomly assigned to 1 of 3 groups: the intervention (TENS), placebo, or no treatment. After completing the randomized period, all participants will receive the intervention for 1 month. The participants will be asked to use TENS at the highest tolerable level for at least 2 hours daily during physical activity.MeasurementsThe primary outcome will be pain with movement, with secondary outcomes assessing functional abilities, patient-reported outcomes, and quantitative sensory testing.LimitationsBecause having participants refrain from their typical medications is not practical, their usage and any change in medication use will be recorded.ConclusionsThe results of this study will provide some of the first evidence from a large-scale, double-blind, placebo-controlled trial on the effectiveness of TENS on pain control and quality-of-life changes in patients with fibromyalgia.

Patient-reported diarrhea impact on physical functioning and quality of life in clinical trial data submitted to the U.S. Food and Drug Administration.

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. e19105-e19105
Author(s):  
Ting-Yu Chen ◽  
Bellinda King-Kallimanis ◽  
Lyna Merzoug ◽  
Mallorie Fiero ◽  
Jennifer J Gao ◽  
...  
Keyword(s):  
Breast Cancer ◽  
Quality Of Life ◽  
Clinical Trial Data ◽  
Life Questionnaire ◽  
Double Blind ◽  
Patient Reported ◽  
Related Quality ◽  
Cancer Trials ◽  
And Function

e19105 Background: Patient-reported outcomes can provide symptom and function data that complement standard oncology endpoints. Frequently, trials will conclude there was no clinically meaningful detriment to health-related quality of life (HRQL) or function, even when notable toxicity is observed. It is possible that mean change from baseline analyses obscures meaningful change in subgroups experiencing symptomatic toxicity. In this study, we explore how patients’ response to a diarrhea item related to physical function (PF) and HRQL in trials submitted to US FDA. Methods: We analyzed 3 randomized, double-blind breast cancer trials (early to late line metastatic) where diarrhea was a more common AE-symptom in the treatment arm, but there was not a large detriment in the mean change from baseline for HRQL and PF. Trials included the EORTC Quality of Life Questionnaire (QLQ-C30), which captures patient-reported HRQL, symptoms, and functioning. Higher scores (range 0-100) indicate better functioning and HRQL. Symptoms were measured with a 4-point scale; not at all to very much. Descriptive statistics were used to analyze diarrhea, PF, and HRQL over time. Results: Patients reporting very much diarrhea at month 3 had worse PF and HRQL compared to patients reporting no diarrhea . The range of difference between patients who reported very much diarrhea and those with none was 8-18 points for PF across trials. For HRQL scores, the range was 13–17 points worse. This trend was also seen in the control arm and at other times. Conclusions: In this set of breast cancer trials with differences in diarrhea by arm, reporting “no meaningful difference in PF or HRQL between the arms” is insufficient and potentially misleading. A more informative interpretation is that an exploratory analysis of HRQL and PF did not show in the investigational arm; there was a greater proportion of patients reporting diarrhea on the treatment arm; and patients reporting more frequent diarrhea reported lower HRQL and PF compared to patients with no diarrhea, regardless of arm. [Table: see text]

Comparison of Flavoxate Hydrochloride in Daily Dosages of 600 versus 1200 mg for the Treatment of Urgency and Urge Incontinence

1988 ◽  
Vol 16 (3) ◽  
pp. 244-248 ◽  
Author(s):  
R. Milani ◽  
S. Scalambrino ◽  
S. Carrera ◽  
P. Pezzoli ◽  
R. Ruffmann
Keyword(s):  
Quality Of Life ◽  
Side Effect ◽  
Urge Incontinence ◽  
Double Blind ◽  
Free Treatment ◽  
Parallel Group ◽  
Flavoxate Hydrochloride ◽  
Parallel Group Trial ◽  
Group Trial

Flavoxate hydrochloride at a daily dosage of 600 mg was compared to a daily dosage of 1200 mg for the treatment of unstable bladder. Twenty-seven patients were treated for 4 weeks in a double-blind, randomized, parallel-group trial. Clinically, both schedules were equally successful. In urodynamic terms, however, particularly with respect to uninhibited detrusor contractions, 1200 mg/day was significantly superior to 600 mg/day. Tolerability was excellent for both regimens. The side-effect free treatment of urgency and urge incontinence is of paramount importance for a patient's quality of life.

scholarly journals A randomised, placebo-controlled study of RIPK1 inhibitor GSK2982772 in patients with active ulcerative colitis

2021 ◽  
Vol 8 (1) ◽  
pp. e000680
Author(s):  
Kathy Weisel ◽  
Nicola Scott ◽  
Scott Berger ◽  
Susanne Wang ◽  
Kurt Brown ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Ulcerative Colitis ◽  
Disease Activity ◽  
Controlled Study ◽  
Experimental Medicine ◽  
Open Label ◽  
Double Blind ◽  
Treatment Groups ◽  
To Receive

ObjectiveTumour necrosis factor signalling via the receptor-interacting protein kinase 1 (RIPK1) pathway regulates colonic inflammation suggesting that RIPK1 inhibition may be a potential therapeutic target in ulcerative colitis (UC). This phase IIa, randomised, double-blind experimental medicine study investigated the safety, pharmacokinetics (PK), pharmacodynamics (PD) and preliminary efficacy of the RIPK1 inhibitor GSK2982772 in patients with active UC.DesignIn part A, prior to a protocol amendment, one patient was randomised to receive GSK2982772 60 mg twice daily for 42 days. After the amendment, patients were randomised 2:1 to receive GSK2982772 60 mg or placebo three times daily for 42 days. In part B, all patients switched to open-label GSK2982772 60 mg three times daily for 42 days. Safety, PK, PD biomarkers, histological disease activity, clinical efficacy and quality of life were assessed at days 43 and 85.ResultsThirty-six patients were randomised (n=12, placebo/open-label GSK2982772; n=24, GSK2982772/open-label GSK2982772). Most adverse events were mild, with headache reported the most frequently across groups (placebo/open-label GSK2982772, n=2 (17%); GSK2982772/open-label GSK2982772, n=8 (33%)). GSK2982772 was well distributed into colonic tissue, with generally higher concentrations in colonic biopsy samples versus plasma. No apparent differences between treatment groups were observed for PD, histological disease activity, clinical disease activity or quality-of-life measures. At screening, all patients had Mayo endoscopic scores of 2 or 3. At day 43, no patients in the placebo/open-label GSK2982772 group achieved Mayo endoscopic scores of 0 or 1 vs 3/24 (13%) for GSK2982772/open-label GSK2982772. At day 85, 1/9 (11%) achieved scores of 0 or one for placebo/open-label GSK2982772 vs 3/22 (14%) for GSK2982772/open-label GSK2982772.ConclusionGSK2982772 was generally well tolerated, with no treatment-related safety concerns identified. However, no significant differences in efficacy were observed between treatment groups, suggesting that GSK2982772 as monotherapy is not a promising treatment for patients with active UC.Trial registration numberNCT02903966.

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