scholarly journals A nonrandomized phase 2 trial of oral thymic peptides in hospitalized patients with Covid-19

2021 ◽  
Author(s):  
Héctor M. Ramos-Zaldívar ◽  
Karla G. Reyes-Perdomo ◽  
Nelson A. Espinoza-Moreno ◽  
Ernesto Tomás Dox-Cruz ◽  
Thania Camila Aguirre Urbina ◽  
...  
Keyword(s):  
Side Effects ◽  
Adverse Events ◽  
Standard Care ◽  
Registry Data ◽  
Control Group ◽  
Phase 2 ◽  
Safety And Efficacy ◽  
Link Type ◽  
Phase 2 Trial ◽  
Thymic Peptides

AbstractBackgroundCoronavirus disease 2019 (Covid-19) active cases continue to demand the development of safe and effective treatments. This is the first clinical trial to evaluate the safety and efficacy of oral thymic peptides.MethodsWe conducted a nonrandomized phase 2 trial with a historic control group to evaluate the safety and efficacy of a daily 250-mg oral dose of thymic peptides in the treatment of hospitalized Covid-19 patients. Comparison based on standard care from registry data was performed after propensity score matching. The primary outcomes were survival, time to recovery and the number of participants with treatment-related adverse events or side effects by day 20.ResultsA total of 44 patients were analyzed in this study, 22 in the thymic peptides group and 22 in the standard care group. There were no deaths in the intervention group, compared to 24% mortality in standard care by day 20 (log-rank P=0.02). The Kaplan-Meier analysis showed a significantly shorter time to recovery by day 20 in the thymic peptides group as compared with standard care (median, 6 days vs. 12 days; hazard ratio for recovery, 2.75 [95% confidence interval, 1.34 to 5.62]; log-rank P=0.002). No side effects or adverse events were reported.ConclusionIn patients hospitalized with Covid-19, the use of thymic peptides reported no side effects, adverse events, or deaths by day 20. When compared with registry data, a significantly shorter time to recovery and mortality reduction was measured. The Catholic University of Honduras Medical Research Group (GIMUNICAH) is working on a more extensive phase 3 trial.Trial registrationClinicalTrials.govNCT04771013. February 25, 2021.

scholarly journals 80 Distraction in the ED using Virtual reality for Intravenous Needs in Children to Improve comfort -the DEVINCI project: A Pilot RCT

2020 ◽  
Vol 25 (Supplement_2) ◽  
pp. e33-e33
Author(s):  
Evelyne D Trottier ◽  
Esli Osmanlliu ◽  
Benoit Bailey ◽  
Maryse Lagacé ◽  
Marisol Sanchez ◽  
...  
Keyword(s):  
Virtual Reality ◽  
Side Effects ◽  
Adverse Events ◽  
Standard Care ◽  
Healthcare Providers ◽  
Intervention Group ◽  
Procedural Pain ◽  
Control Group ◽  
Memory Of Pain ◽  
Pilot Rct

Abstract Background Venipuncture is a frequent cause of pain and distress in the pediatric emergency department (ED). Healthcare professionals must optimize patient comfort during such procedures, given the associated short- and long-term adverse events. Distraction, which can improve patient experience, remains the most studied psychological intervention. Virtual reality (VR) is a method of immersive distraction that can contribute to the multi-modal management of procedural pain and distress. Its use in the healthcare setting has become more accessible due to the development of more portable and affordable systems. Objectives The primary outcome of this study was to determine the feasibility and acceptability of a VR distraction device in the pediatric ED. The secondary outcomes were to examine the preliminary effects of the addition of VR to standard practice on children’s pain, distress, and memory of pain associated with venipunctures in the pediatric ED. Design/Methods This pilot RCT was performed at a tertiary pediatric centre. Children 7-17 years old requiring a venipuncture in the ED were recruited. Participants were randomized to either a control group (standard care) or intervention group (standard care+VR). A priori feasibility and acceptability criteria were established following expert consensus (nurses/physicians working in the ED): - 80% of families approached for recruitment consent to participate; - 80% of recruited patients finish the game as planned (intervention group); - Mean >7 on the 0-10 satisfaction scale (patients/parents/healthcare providers); - No serious adverse events. The principal clinical outcome was the mean level of procedural pain as measured by the verbal numerical rating scale (VNRS). Auto-evaluation of procedural-related distress was performed using the Child Fear Scale (CFS). The level of distress related to the procedure was also evaluated by proxy using the Procedure Behavior Check List (PBCL). Memory of pain was measured at 24h using the VNRS. Side effects were documented. Results A total of 63 patients were recruited between December 2018 and April 2019 (one was excluded as the venipuncture was later cancelled). The results showed feasibility and acceptability of VR in the pediatric ED with: -79% recruitment rate of eligible families -90% rate of VR game completion as per protocol (reason for not completing the game: prior headache, wanting to see, VR not working well) and -overall high mean satisfaction levels. In addition, there was a significantly higher level of satisfaction among healthcare providers in the intervention group, and 93% of those were willing to use this technology again for the same procedure. Regarding clinical outcomes, there was no significant difference between groups in self-reported procedural pain, but evaluation of distress by proxy (10/40 vs 13.2/40, p = 0.007) and memory of pain at 24 hours (2.4 vs 4.2, p = 0.027) were significantly lower in the intervention group. Venipuncture was successful on first attempt in 23/31 patients (74%) in the VR group and 15/30 (50%) patients in the control group (p = 0.039). Five of the 31 patients (16%) in the VR group reported side effects, which were mild and self-resolving. Conclusion The addition of VR to standard of care for pain and distress management related to venipunctures in the pediatric ED is a feasible and acceptable intervention. Side effects related to VR were few and self-resolving. Both experimental groups were similar with respect to self-reported procedural pain. A lower level of procedural distress, lower memory of pain at 24 hours, and higher venipuncture first attempt success rate were observed in the VR group.

scholarly journals Safety and efficacy of autologous non-hematopoietic enriched stem cell nebulization in COVID-19 patients: a randomized clinical trial, Abu Dhabi 2020

2021 ◽  
Vol 6 (1) ◽  
Author(s):  
Yendry Ventura-Carmenate ◽  
Fatima Mohammed Alkaabi ◽  
Yandy Marx Castillo-Aleman ◽  
Carlos Agustin Villegas-Valverde ◽  
Yasmine Maher Ahmed ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Stem Cell ◽  
Adverse Events ◽  
Standard Care ◽  
Abu Dhabi ◽  
Ordinal Scale ◽  
Control Group ◽  
Safety And Efficacy ◽  
Point Reduction ◽  
The Impact

Abstract Background The novel SARS-CoV-2 has caused the coronavirus disease 2019 (COVID-19) pandemic. Currently, with insufficient worldwide vaccination rates, identifying treatment solutions to reduce the impact of the virus is urgently needed. Method An adaptive, multicentric, open-label, and randomized controlled phase I/II clinical trial entitled the “SENTAD-COVID Study” was conducted by the Abu Dhabi Stem Cells Center under exceptional conditional approval by the Emirates Institutional Review Board (IRB) for COVID-19 Research Committee from April 4th to July 31st, 2020, using an autologous peripheral blood non-hematopoietic enriched stem cell cocktail (PB-NHESC-C) administered by compressor (jet) nebulization as a complement to standard care therapy. The primary endpoints include safety and efficacy assessments, adverse events, the mortality rate within 28 days, and the time to clinical improvement as measured by a 2-point reduction on a seven-category ordinal scale or discharge from the hospital whichever occurred first. Results The study included a total of 139 randomized COVID-19 patients, with 69 in the experimental group and 70 in the control group (standard care). Overall survival was 94.20% for the cocktail-treated group vs. 90.27% for the control group. Adverse events were reported in 50 (72.46%) patients receiving PB-NHESC-C and 51 (72.85%) in the control group (p = 0.9590), with signs and symptoms commonly found in COVID-19. After the first 9 days of the intervention, 67.3% of cocktail-treated patients recovered and were released from hospitals compared to 53.1% (RR = 0.84; 95% CI, 0.56–1.28) in the control group. Improvement, i.e., at least a 2-point reduction in the severity scale, was more frequently observed in cocktail-treated patients (42.0%) than in controls (17.0%) (RR = 0.69; 95% CI, 0.56–0.88). Conclusions Cocktail treatment improved clinical outcomes without increasing adverse events. Thus, the nebulization of PB-NHESC-C was safe and effective for treatment in most of these patients. Trial registration ClinicalTrials.gov. NCT04473170. It was retrospectively registered on July 16th, 2020.

scholarly journals Safety and Efficacy of Autologous Non-Hematopoietic Enriched Stem Cell Nebulization in Covid-19 Patients. A Randomized Clinical Trial, Abu Dhabi 2020.

2021 ◽  
Author(s):  
Yendry Ventura Carmenate ◽  
Fatima Mohammed Alkaabi ◽  
Yandy Marx Castillo Aleman ◽  
Carlos Agustin Villegas Valverde ◽  
Yasmine Maher Ahmed ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Stem Cell ◽  
Adverse Events ◽  
Standard Care ◽  
Abu Dhabi ◽  
Ordinal Scale ◽  
Control Group ◽  
Safety And Efficacy ◽  
Point Reduction ◽  
The Impact

Abstract Background: The novel SARS-Cov-2 has caused the COVID-19 pandemic. Currently, with insufficient worldwide vaccination rates, the identification of treatment solutions to reduce the impact of the virus is urgently needed. Method: An adaptive, multicentric, open-label, and randomized controlled phase I/II clinical trial entitled the “SENTAD-COVID Study” was conducted by the Abu Dhabi Stem Cells Center under conditional exceptional approval by the Emirates Institutional Review Board (IRB) for COVID-19 Research Committee from April 4 to July 31, 2020, using an autologous peripheral blood nonhematopoietic enriched stem cell cocktail (PB-NHESC-C) administered by compressor (jet) nebulization as a complement to standard care therapy. The primary endpoints include safety and efficacy assessments, adverse events, the mortality rate within 28 days, and the time to clinical improvement as measured by a 2-point reduction on a seven-category ordinal scale or discharge from the hospital, whichever occurred first. Results: The study included a total of 139 randomized COVID-19 patients with 69 in the experimental group and 70 in the control group (standard care). Overall survival was 94.20% for the cocktail-treated group vs. 90.27% for the control group. Adverse events occurred in 43 (62.32%) patients receiving PB-NHESC-C vs. 44 (62.86%) in the control group, and most adverse events were related to the disease. After the first nine days of the intervention, 67.3% of cocktail-treated patients recovered and were released from hospitals compared to 53.1% (RR=0.84; 95% CI, 0.56-1.28) in the control group. Improvement, i.e., at least a 2-point reduction in the severity scale, was more frequently observed in cocktail-treated patients (42.0%) than in controls (17.0%) (RR=0.69; 95% CI, 0.56-0.88). Conclusions: Cocktail treatment improved clinical outcomes without increasing adverse events. Thus, the nebulization of PB-NHESC-C was safe and effective for treatment in most of these patients. Trial registration: ClinicalTrials.gov. NCT04473170. Registered 16 July 2020. Retrospectively registered. https://clinicaltrials.gov/ct2/show/NCT04473170.

scholarly journals A Randomized, Evaluator-Blind, Phase 2 Study Comparing the Safety and Efficacy of Omadacycline to Those of Linezolid for Treatment of Complicated Skin and Skin Structure Infections

2012 ◽  
Vol 56 (11) ◽  
pp. 5650-5654 ◽  
Author(s):  
Gary J. Noel ◽  
Michael P. Draper ◽  
Howard Hait ◽  
S. Ken Tanaka ◽  
Robert D. Arbeit
Keyword(s):  
Adverse Events ◽  
Clinical Success ◽  
Phase 2 ◽  
Success Rates ◽  
Safety And Efficacy ◽  
Content Type ◽  
Skin Structure ◽  
Phase 2 Trial ◽  
Complicated Skin ◽  
Intent To Treat

ABSTRACTA randomized, investigator-blind, multicenter phase 2 trial involving patients with complicated skin and skin structure infections (cSSSI) compared the safety and efficacy of omadacycline, a broad-spectrum agent with activity against methicillin-resistantStaphylococcus aureus(MRSA), to those of linezolid (with or without aztreonam). Patients were randomized 1:1 to omadacycline (100 mg intravenously [i.v.] once a day [QD] with an option to transition to 200 mg orally QD) or linezolid (600 mg i.v. twice daily [BID] with an option to transition to 600 mg orally BID) at 11 U.S. sites. Patients suspected or documented to have infections caused by Gram-negative bacteria were given aztreonam (2 g i.v. every 12 h [q12h]) if randomized to linezolid or matching placebo infusions if randomized to omadacycline. Adverse events were reported in 46 (41.4%) omadacycline-treated and 55 (50.9%) linezolid-treated patients. Adverse events related to treatment were assessed by investigators in 24 (21.6%) omadacycline-treated and 33 (30.6%) linezolid-treated patients. The gastrointestinal tract was most commonly involved, with adverse events reported in 21 (18.9%) patients exposed to omadacycline and 20 (18.5%) exposed to linezolid. Rates of successful clinical response in the intent-to-treat (ITT) and clinical evaluable (CE) populations favored omadacycline (ITT, 88.3% versus 75.9%; 95% confidence interval [CI], 1.9 to 22.9; CE, 98.0% versus 93.2%; 95% CI, −1.7 to 11.3). For microbiologically evaluable (ME) patients withS. aureusinfections, the clinical success rates were 97.2% (70/72) in omadacycline-treated and 92.7% (51/55) in linezolid-treated patients. This phase 2 experience supports conclusions that omadacycline is well tolerated in cSSSI patients and that this aminomethylcycline has potential to be an effective treatment for serious skin infections.

scholarly journals A phase 1 and randomized controlled phase 2 trial of the safety and efficacy of the combination of gemcitabine and docetaxel with ontuxizumab (MORAb‐004) in metastatic soft‐tissue sarcomas

Cancer ◽  
2019 ◽  
Vol 125 (14) ◽  
pp. 2445-2454 ◽  
Author(s):  
Robin L. Jones ◽  
Sant P. Chawla ◽  
Steven Attia ◽  
Patrick Schöffski ◽  
Hans Gelderblom ◽  
...  
Keyword(s):  
Soft Tissue ◽  
Phase 1 ◽  
Soft Tissue Sarcomas ◽  
Phase 2 ◽  
Safety And Efficacy ◽  
Randomized Controlled ◽  
Phase 2 Trial

scholarly journals Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial

2021 ◽  
pp. 1-13
Author(s):  
Craig M. McDonald ◽  
Perry B. Shieh ◽  
Hoda Z. Abdel-Hamid ◽  
Anne M. Connolly ◽  
Emma Ciafaloni ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Natural History ◽  
Adverse Events ◽  
Exon Skipping ◽  
Control Group ◽  
Phase 2 ◽  
Open Label ◽  
M Phase ◽  
Positive Treatment

Background Eteplirsen received accelerated FDA approval for treatment of Duchenne muscular dystrophy (DMD) with mutations amenable to exon 51 skipping, based on demonstrated dystrophin production. Objective To report results from PROMOVI, a phase 3, multicenter, open-label study evaluating efficacy and safety of eteplirsen in a larger cohort. Methods Ambulatory patients aged 7–16 years, with confirmed mutations amenable to exon 51 skipping, received eteplirsen 30 mg/kg/week intravenously for 96 weeks. An untreated cohort with DMD not amenable to exon 51 skipping was also enrolled. Results 78/79 eteplirsen-treated patients completed 96 weeks of treatment. 15/30 untreated patients completed the study; this cohort was considered an inappropriate control group because of genotype-driven differences in clinical trajectory. At Week 96, eteplirsen-treated patients showed increased exon skipping (18.7-fold) and dystrophin protein (7-fold) versus baseline. Post-hoc comparisons with patients from eteplirsen phase 2 studies (4658-201/202) and mutation-matched external natural history controls confirmed previous results, suggesting clinically notable attenuation of decline on the 6-minute walk test over 96 weeks (PROMOVI: –68.9 m; phase 2 studies: –67.3 m; external controls: –133.8 m) and significant attenuation of percent predicted forced vital capacity annual decline (PROMOVI: –3.3%, phase 2 studies: –2.2%, external controls: –6.0%; p <  0.001). Adverse events were generally mild to moderate and unrelated to eteplirsen. Most frequent treatment-related adverse events were headache and vomiting; none led to treatment discontinuation. Conclusions This large, multicenter study contributes to the growing body of evidence for eteplirsen, confirming a positive treatment effect, favorable safety profile, and slowing of disease progression versus natural history.

Repurposing Domperidone in Secondary Progressive MS

Neurology ◽  
2021 ◽  
pp. 10.1212/WNL.0000000000011863
Author(s):  
Marcus W. Koch ◽  
Kayla Sage ◽  
Sharanjit Kaur ◽  
Janet Kim ◽  
Graziela Cerchiaro ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Adverse Events ◽  
Progressive Multiple Sclerosis ◽  
Secondary Progressive Multiple Sclerosis ◽  
Phase 2 ◽  
Disability Progression ◽  
Two Stage ◽  
Serious Adverse Events ◽  
Secondary Progressive ◽  
Link Type

ObjectiveTo assess whether treatment with the generic drug domperidone can reduce the progression of disability in secondary progressive multiple sclerosis (SPMS), we conducted a phase 2 futility trial following the Simon two-stage design.MethodsWe enrolled patients in an open-label, Simon two-stage, single-center, phase 2, single-arm futility trial at the Calgary MS Clinic if they met the following criteria: age 18–60 years, SPMS, screening EDSS score of 4.0–6.5 and screening T25FW of 9 seconds or more. Patients received domperidone 10 mg QID for one year. The primary outcome was worsening of disability, defined as worsening of the T25FW performance by 20% or more at 12 months compared to at baseline. This trial is registered with ClinicalTrials.gov, number NCT02308137.ResultsBetween February 13, 2015 and January 3, 2020, 110 patients were screened, 81 received treatment, 64 completed follow-up, of whom 62 were analysed. The study did not meet its primary endpoint: 22 of 62 (35%) patients experienced significant worsening of disability, which is close to the expected proportion of 40%, and above the pre-defined futility threshold. Patients with higher prolactin levels during the study had a significantly lower risk of disability progression, which may warrant further investigation. Domperidone treatment was reasonably well tolerated, but adverse events occurred in 84% and serious adverse events in 15% of patients.ConclusionsDomperidone treatment could not reject futility in reducing disability progression in SPMS. The Simon two-stage trial model may be a useful model for phase 2 studies in progressive MS.Classification of evidenceThis study provides Class III evidence that in individuals with secondary progressive multiple sclerosis participating in a futility trial, domperidone treatment could not reject futility in reducing disability progression at 12 months.

Adaptive, Dose-finding Phase 2 Trial Evaluating the Safety and Efficacy of ABT-089 in Mild to Moderate Alzheimer Disease

2015 ◽  
Vol 29 (3) ◽  
pp. 192-199 ◽  
Author(s):  
Robert A. Lenz ◽  
Yili L. Pritchett ◽  
Scott M. Berry ◽  
Daniel A. Llano ◽  
Shu Han ◽  
...  
Keyword(s):  
Alzheimer Disease ◽  
Dose Finding ◽  
Phase 2 ◽  
Safety And Efficacy ◽  
Phase 2 Trial
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