scholarly journals A Phase II Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy, Safety, and Tolerability of Arbaclofen Administered for the Treatment of Social Function in Children and Adolescents With Autism Spectrum Disorders: Study Protocol for AIMS-2-TRIALS-CT1

2021 ◽  
Vol 12 ◽  
Author(s):  
Mara Parellada ◽  
Antonia San José Cáceres ◽  
Melanie Palmer ◽  
Richard Delorme ◽  
Emily J. H. Jones ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Clinical Trial ◽  
Children And Adolescents ◽  
Phase Ii ◽  
Clinical Global Impression ◽  
Social Communication ◽  
Social Function ◽  
Autism Spectrum ◽  
Double Blind

Background: Autism Spectrum Disorder (ASD or autism) is characterized by difficulties in social communication and interaction, which negatively impact on individuals and their families' quality of life. Currently no pharmacological interventions have been shown to be effective for improving social communication in autism. Previous trials have indicated the potential of arbaclofen for improving social function among autistic children and adolescents with fluent speech. The AIMS2TRIALS-Clinical Trial 1 (AIMS-CT1) will examine whether arbaclofen is superior to placebo in improving social function and other secondary outcomes over 16 weeks, along with safety and tolerability profiles.Methods: AIMS-CT1 is an international, multi-site, double-blind, parallel group Phase II randomized clinical trial. It will include 130 males and females aged 5:0–17:11 years, with a diagnosis of ASD and fluent speech. Eligible participants will be randomized on a ratio of 1:1 for a 16-week treatment period. Medication will be titrated over 5 weeks. The primary outcome is the effect on social function from weeks 0 to 16 measured on the Socialization domain of the Vineland Adaptive Behavior Scales, 3rd editionTM. Secondary outcome measures include the CGI–S (Clinical Global Impression–Severity), CGI–I (Clinical Global Impression–Improvement), other areas of adaptive function, social communication and other autism symptoms, co-occurring behavior problems and health-related quality of life. Genetic and electrophysiological markers will be examined as potential stratifiers for treatment response. Exploratory novel digital technologies will also be used to measure change, examining simultaneously the validity of digital biomarkers in natural environments. The safety and tolerability of the drug will also be examined. Our protocol is very closely aligned with a parallel Canadian trial of 90 participants (ARBA Study, US NCT number: NCT03887676) to allow for secondary combined analyses. Outcomes will be compared using both an Intent-to-reat and Per Protocol approach.Discussion: The outcomes of this trial, combined with the parallel Canadian trial, will contribute to the evidence base for medications used to help social difficulties among young autistic individuals; demonstrate the capabilities of the AIMS-2-TRIALS network of academic centers to deliver clinical trials; and support future drug development.Clinical Trial Registration: EudraCT number: 2018-000942-21 and ClinicalTrials.gov registry number: NCT03682978. Currently under protocol v.7.2, dated 20.11.2020.

ISA247: Quality of Life Results from a Phase II, Randomized, Placebo-Controlled Study

2008 ◽  
Vol 12 (6) ◽  
pp. 268-275 ◽  
Author(s):  
Aditya K. Gupta ◽  
Richard G. Langley ◽  
Charles Lynde ◽  
Kirk Barber ◽  
Wayne Gulliver ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Phase Ii ◽  
Life Quality ◽  
Skin Condition ◽  
Controlled Study ◽  
Double Blind ◽  
Plaque Type ◽  
Psoriasis Disability Index ◽  
Stable Plaque

Background: Psoriasis is a chronic skin condition that can negatively affect a patient's quality of life (QoL), often hindering social functioning. ISA247, a novel psoriatic agent, has shown clinical efficacy in moderate to severe psoriasis sufferers, but its effect on QoL is currently not reported. Objective: The objective of this study was to assess the effect of ISA247 on the QoL in patients with stable, plaque-type psoriasis. Methods: A phase II, randomized, double-blind, placebo-controlled, parallel-group, multicenter study assessed the effects of ISA247 doses of 0.5 mg/kg/d ( n = 77) or 1.5 mg/kg/d ( n = 83) compared with placebo ( n = 41) for 12 weeks. QoL was assessed using the Dermatology Life Quality Index (DLQI) and Psoriasis Disability Index (PDI) scales. Results: ISA247 treatment (pooled groups) significantly improved QoL scores as assessed by both the DLQI and the PDI compared with those receiving placebo ( p < .05). Treatment with the higher dose of 1.5 mg/kg/d demonstrated a significantly greater response to many of the QoL scales compared with the 0.5 mg/kg/d group ( p < .05). Conclusions: ISA247 appears to improve the QoL while also providing effective treatment for chronic, moderate to severe, plaque-type psoriasis.

Oral Propolis, Nutritional Status and Quality of Life with Chemotherapy for Breast Cancer: A Randomized, Double-Blind Clinical Trial

2021 ◽  
pp. 1-9
Author(s):  
Seyed Hossein Davoodi ◽  
Vahid Yousefinejad ◽  
Bayazid Ghaderi ◽  
Mohammad Esmail Akbari ◽  
Shoaleh Darvishi ◽  
...  
Keyword(s):  
Breast Cancer ◽  
Quality Of Life ◽  
Clinical Trial ◽  
Nutritional Status ◽  
Double Blind ◽  
Double Blind Clinical Trial

scholarly journals A combination of mupirocin and acidic fibroblast growth factor for nipple fissure and nipple pain in breastfeeding women: protocol for a randomised, double-blind, controlled trial

BMJ Open ◽  
2019 ◽  
Vol 9 (3) ◽  
pp. e025526
Author(s):  
Xiaofang Lv ◽  
Rui Feng ◽  
Jingbo Zhai
Keyword(s):  
Quality Of Life ◽  
Clinical Trial ◽  
Growth Factor ◽  
Fibroblast Growth Factor ◽  
Treatment Period ◽  
Current Evidence ◽  
Fibroblast Growth ◽  
Acidic Fibroblast Growth Factor ◽  
Double Blind

IntroductionNipple fissure and nipple pain are common complaints among breastfeeding mothers. Studies found that mupirocin was effective in preventing and treating infections of damaged nipple and nipple pain. Acidic fibroblast growth factor (aFGF) plays an important role in wound healing. However, current evidence on the efficacy and safety of mupirocin plus aFGF for nipple fissure and nipple pain in breastfeeding women is inconclusive due to the lack of well-designed randomised controlled trials on this topic. The purpose of this study is to test the hypothesis that mupirocin plus aFGF is more effective than mupirocin alone for nipple fissure and nipple pain in breastfeeding women.Methods and analysisThis study is a randomised, double-blind, single-centre, parallel-group clinical trial. A total of 120 breastfeeding women with nipple fissure and nipple pain will be randomly assigned to either mupirocin plus aFGF group or mupirocin plus placebo group according to a computer-generated random allocation sequence. The treatment period lasts 14 days. The primary outcome is nipple pain intensity measured by the Visual Analogue Scale on day 14 during the treatment period. Secondary outcome measures include time to complete nipple pain relief, changes in the Nipple Trauma Score, time to complete healing of nipple trauma, quality of life measured by the Maternal Postpartum Quality of Life (MAPP-QOL) Questionnaire, the frequency of breast feeding, the rate of breastfeeding discontinuation, weight change in infants and adverse events.Ethics and disseminationThe study has gained approval from the Ethics Review Committee of Tianjin Central Hospital of Gynaecology Obstetrics on 22 January 2018 (approval no. 2018KY001). We plan to publish our research findings in a peer-reviewed academic journal and disseminate these findings in international conferences. This study has been registered with the Chinese Clinical Trial Registry.Trial registration numberChiCTR1800017248.

A phase II trial of risk-adapted treatment for muscle invasive bladder cancer after neoadjuvant accelerated MVAC.

2018 ◽  
Vol 36 (6_suppl) ◽  
pp. TPS537-TPS537 ◽  
Author(s):  
Daniel M. Geynisman ◽  
Philip Abbosh ◽  
Matthew R. Zibelman ◽  
Rebecca Feldman ◽  
David James McConkey ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Clinical Trial ◽  
Bladder Cancer ◽  
Active Surveillance ◽  
Phase Ii ◽  
Standard Of Care ◽  
Muscle Invasive Bladder Cancer ◽  
Muscle Invasive

TPS537 Background: Cisplatin-based neoadjuvant chemotherapy (NAC) followed by cystectomy (Cx) or chemoradiation (CRT) is the standard of care for urothelial carcinoma (UC) pts with muscle invasive bladder cancer (MIBC). Both Cx and CRT carry potential short and long-term toxicity and quality of life implications. Recent work has shown that mutations in DNA damage repair/response genes are predictive of pathologic downstaging after NAC at the time of Cx, with those pts achieving pT0 disease demonstrating excellent long-term survival (Van Allen et al. Cancer Discov. 2014; Plimack et al. Eur Urol. 2015; Liu et al. JAMA Oncol. 2016; Teo et al. CCR. 2017). Sparing pts Cx or CRT after NAC without compromising oncologic outcomes would improve quality of life and decrease morbidity. Methods: A phase II, parallel arm, multi-institutional clinical trial (NCT02710734) is being conducted to evaluate a risk-adapted approach to treatment of MIBC. Pts with cT2-T3N0M0 UC of the bladder, ECOG PS 0-1 and CrCl≥50 mL/min, undergo NAC with accelerated methotrexate, vinblastine, doxorubicin, and cisplatin. Simultaneously, the pre-NAC TURBT specimen is submitted for deep sequencing to identify variants in a panel of cancer-relevant genes (Caris Life Sciences, Phoenix, AZ). Those with an alteration in ATM, RB1, FANCC or ERCC2 and no clinical evidence of disease by restaging TUR and imaging post-NAC will begin a pre-defined active surveillance regimen that includes urinary cytological, cystoscopic, and radiographic evaluations. The remaining pts will undergo bladder-directed therapy at the discretion of the pt and clinician applying either intravesical therapy ( < cT2 post-NAC), CRT or Cx (≤cT2 post-NAC) or Cx (≥cT3 post-NAC). The primary objective is metastasis-free survival (MFS) at 2 years for all enrolled and evaluable pts. The trial has a non-inferiority design with a 14% margin between risk-adapted treatment (MFS = 78%) and standard-of-care (MFS = 64%) with a sample size of 70 pts, 82% power and a type I error of 0.045. Key secondary and translational objectives: assess the rate of UC recurrence in active surveillance pts; validate biomarkers of response to NAC; evaluate urinary biomarkers consistent with persistent UC. Clinical trial information: NCT02710734.

Clinical downstaging and quality of life after neoadjuvant chemotherapy in patients with locally advanced rectal cancer: Interim analysis from a phase II clinical trial.

2019 ◽  
Vol 37 (15_suppl) ◽  
pp. e15178-e15178
Author(s):  
Ahmed Abdalla ◽  
Amr M. Aref ◽  
Amer Alame ◽  
Danny Ma ◽  
Mohammed Barawi ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Clinical Trial ◽  
Rectal Cancer ◽  
Neoadjuvant Chemotherapy ◽  
Phase Ii ◽  
General Health ◽  
Locally Advanced ◽  
Advanced Rectal Cancer ◽  
Locally Advanced Rectal Cancer

e15178 Background: The role of neoadjuvant FOLFOX in achieving clinical downstaging and improvement in quality of life (QOL) in patients with locally advanced rectal cancer (LARC) remains to be established. We are conducting a phase II prospective clinical trial to evaluate the use of six cycles of FOLFOX as neoadjuvant chemotherapy in patients with T2-T3/N0-N+ rectal cancer. We now report tumor clinical downstaging and patient-reported QOL in our first patient cohort. Methods: Eleven Patients enrolled in our phase II prospective trial. Patients received three months of FOLFOX (infusional fluorouracil, leucovorin, and oxaliplatin) administered every two weeks. After three weeks of recovery, each patient was treated with conventional chemo-radiotherapy (5FU or capecitabine) All patients had an MRI and endorectal ultrasound at baseline and after completion of FOLFOX. A compilation of validated QOL questionnaires were also administered before and after FOLFOX. Results: A total of 11 patients completed the chemotherapy regimen. Based on pelvic MRI, complete clinical response (T0N0) was achieved by seven patients (64%), one patient (9%) was clinically downstaged but three (27%) didn’t have any changes following FOLFOX. Importantly, we found no disease progression during the FOLFOX course. QOL assessment after FOLFOX regimen showed trend towards improvement in general health, mobility, bladder control and psychological health. These changes in QOL were not statistically significant due to the small sample size. Patients self-grading of their general health before starting FOLFOX was 50% compared to 75% after. Of the five patients with pain at time of diagnosis, four reported complete pain relief while the fifth reported improvement from extreme to moderate pain. Three patients reported improvement in their anxiety/depression. In terms of bowel function, although there was trend towards improvement in the urgency subscale, other bowel functions subscales were unchanged. In general, scores for mobility, selfcare, and bladder function were slightly better after FOLFOX. Conclusions: This study suggests that adding only six cycles of neoadjuvant FOLFOX before CRT not only resulted in clinical downstaging of (LARC) but showed a trend toward improved QOL. This result provides some reassurance for oncologists that this approach does not diminish QOL with no risk of disease progression during the time of neoadjuvant chemotherapy. These findings need to be validated in a larger phase III trial.

Phase II clinical trial of nab-paclitaxel plus gemcitabine in elderly patients with previously untreated locally advanced or metastatic pancreatic adenocarcinoma: BIBABRAX study.

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. 4615-4615 ◽  
Author(s):  
Jaime Feliu Batlle ◽  
Monica Jorge Fernandez ◽  
Teresa Macarulla ◽  
Bartomeu Massuti ◽  
Ana Albero ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Clinical Trial ◽  
Elderly Patients ◽  
Phase Ii ◽  
Clinical Benefit ◽  
Performance Status ◽  
Locally Advanced ◽  
Standards Of Care ◽  
Ecog Ps

4615 Background: FOLFIRINOX and nab-paclitaxel plus gemcitabine (nab-P+G) are the standard of care in the first-line treatment of mPC patients (pt) with good performance status. However, no standards of care exist for elderly ( > 70 years) pt as they are usually excluded in clinical trials. This study aimed to evaluate whether the clinical benefit of nab-P+G could be extended to elderly pt with mPC. Methods: This was an open-label, single-arm, multicenter, phase II trial, to assess the efficacy and safety of Nab-P+G in elderly pt (≥ 70 years) with ECOG PS 0–1 and untreated unresectable locally advanced or metastatic PC. Pt received four-week cycles of intravenous (i.v.) nab-paclitaxel 125 mg/m2, followed by i.v. gemcitabine 1,000 mg/m2, on days 1, 8 and 15, until disease progression. Efficacy was evaluated according RECIST v 1.1 criteria and safety according NCI-CTCAE v 4.0 criteria. Results: Eighty pt were enrolled in the study. Median age was 74.6 years (range 70-87.9), 57.5% were men, 71% had ECOG PS 1 and 86% metastatic disease. 16.3% of patients had a history of prior tumor surgical resection, 12.5% received chemotherapy and 3.8% radiotherapy. Primary tumor was located in head (32.5%), tail (25.0%) and body (22.5%). Nab-P and G was reduced in 49% and 41% of pt respectively. 15 pt definitely interrupt study treatment due to toxicity: neurotoxicity (7), asthenia (5), neutropenia (1), leukocytosis (1) and hepatotoxicity (1). Time until definite deterioration (reduction ≥10 points as compared to baseline in EORTC-QLQ C30) was 1.6 months and deterioration-free rate at 3 months was 54.3%. Overall response rate was 13.8%, clinical benefit rate 67.5%, median PFS 7.2 months and median OS 9.2 months. The most common treatment-related adverse events were asthenia (60.0%), diarrhea (40.0%), neutropenia (33.8%), hair loss (28.8%), thrombocytopenia (26.3%), and nausea (23.8%). Only asthenia and neutropenia presented a relatively high incidence of grade 3 and 4 toxicities (21.3%). At least 1 SAE was reported in 55% of pt. Conclusions: BIBABRAX study confirms the clinical benefit of nab-P+G in an elderly population with mPC, in terms of survival, clinical response and tolerance, therefore it could be considered a treatment option for elderly patients. However, it was unable to demonstrate the preplanned benefit on the quality of life. Further research is needed on treatment strategies that could reduce deterioration of the quality of life in these pt. Clinical trial information: NCT02391662 .

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