scholarly journals “Include me if you can”—reasons for low enrollment of pediatric patients in a psychopharmacological trial

Trials ◽  
2021 ◽  
Vol 22 (1) ◽  
Author(s):  
Larissa Niemeyer ◽  
Konstantin Mechler ◽  
Jan Buitelaar ◽  
Sarah Durston ◽  
Bram Gooskens ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Demographic Data ◽  
Autism Spectrum ◽  
Current Therapy ◽  
Future Research ◽  
Controlled Clinical Trial ◽  
Double Blind ◽  
Clinical Patient ◽  
Compulsive Disorder

Abstract Background Low recruitment in clinical trials is a common and costly problem which undermines medical research. This study aimed to investigate the challenges faced in recruiting children and adolescents with obsessive-compulsive disorder and autism spectrum disorder for a randomized, double-blind, placebo-controlled clinical trial and to analyze reasons for non-participation. The trial was part of the EU FP7 project TACTICS (Translational Adolescent and Childhood Therapeutic Interventions in Compulsive Syndromes). Methods Demographic data on pre-screening patients were collected systematically, including documented reasons for non-participation. Findings were grouped according to content, and descriptive statistical analyses of the data were performed. Results In total, n = 173 patients were pre-screened for potential participation in the clinical trial. Of these, only five (2.9%) were eventually enrolled. The main reasons for non-inclusion were as follows: failure to meet all inclusion criteria/meeting one or more of the exclusion criteria (n = 73; 42.2%), no interest in the trial or trials in general (n = 40; 23.1%), and not wanting changes to current therapy/medication (n = 14; 8.1%). Conclusions The findings from this study add valuable information to the existing knowledge on reasons for low clinical trial recruitment rates in pediatric psychiatric populations. Low enrollment and high exclusion rates raise the question of whether such selective study populations are representative of clinical patient cohorts. Consequently, the generalizability of the results of such trials may be limited. The present findings will be useful in the development of improved recruitment strategies and may guide future research in establishing the measurement of representativeness to ensure enhanced external validity in psychopharmacological clinical trials in pediatric populations. Trial registration EudraCT 2014-003080-38. Registered on 14 July 2014.

scholarly journals Immunogenicity and Safety of Inactivated Sabin-Strain Polio Vaccine “PoliovacSin”: Clinical Trials Phase I and II

Vaccines ◽  
2021 ◽  
Vol 9 (6) ◽  
pp. 565
Author(s):  
Anastasia Piniaeva ◽  
Georgy Ignatyev ◽  
Liubov Kozlovskaya ◽  
Yury Ivin ◽  
Anastasia Kovpak ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Phase I ◽  
Safety Profile ◽  
Neutralizing Antibody ◽  
Polio Eradication ◽  
Controlled Clinical Trial ◽  
Double Blind ◽  
Good Tolerability ◽  
Poliomyelitis Vaccine

Global polio eradication requires both safe and effective vaccines, and safe production processes. Sabin oral poliomyelitis vaccine (OPV) strains can evolve to virulent viruses and result in poliomyelitis outbreaks, and conventional inactivated poliomyelitis vaccine (Salk-IPV) production includes accumulation of large stocks of neurovirulent wild polioviruses. Therefore, IPV based on attenuated OPV strains seems a viable option. To increase the global supply of affordable inactivated vaccine in the still not-polio free world we developed an IPV made from the Sabin strains–PoliovacSin. Clinical trials included participants 18–60 years of age. A phase I single-center, randomized, double-blind placebo-controlled clinical trial included 60 participants, who received one dose of PoliovacSin or Placebo. A phase II multicenter, randomized, double-blind, comparative clinical trial included 200 participants, who received one dose of PoliovacSin or Imovax Polio. All vaccinations were well tolerated, and PoliovacSin had a comparable safety profile to the Placebo or the reference Imovax Polio preparations. A significant increase in neutralizing antibody levels to polioviruses types 1–3 (Sabin and wild) was observed in PoliovacSin and Imovax Polio vaccinated groups. Therefore, clinical trials confirmed good tolerability, low reactogenicity, and high safety profile of the PoliovacSin and its pronounced immunogenic properties. The preparation was approved for clinical trials involving infants.

scholarly journals Does synbiotic supplementation affect the quality of life in children with cystic fibrosis? A pilot randomized controlled clinical trial

2020 ◽  
Author(s):  
Nemat Bilan ◽  
Effat Marefat ◽  
Leila Nikniaz ◽  
Mahdieh Abbasalizad Farhangi ◽  
Zeinab Nikniaz
Keyword(s):  
Quality Of Life ◽  
Clinical Trial ◽  
Cystic Fibrosis ◽  
Demographic Data ◽  
Intervention Group ◽  
Controlled Clinical Trial ◽  
Double Blind ◽  
The Mean ◽  
Synbiotic Supplementation

Abstract Background: There is no clinical trial that assesses the effect synbiotic supplementation on HRQOL in CF children. Considering the importance of HRQOL as an essential primary outcome and determinant of therapeutic benefit in chronic diseases like cystic fibrosis, the present clinical trial aimed to determine the efficacy of synbiotic supplementation on HRQOL in children with CF.Methods: In the present double-blind randomized clinical trial, 40 CF children were randomly allocated to the two groups. The intervention group was supplemented with synbiotics supplements and the patients in the placebo group received maltodextrin for six months. Demographic data and information about antibiotic use were recorded using the questionnaire. The health-related quality of life was assessed using the Persian version of quality of life inventory questionnaires. Paired t-test and ANCOVA were used for statistical analysis. Results: Totally, 36 participants completed the trial. The mean score of HRQOL was 76.34±17.33. There were no significant differences between synbiotic and placebo groups regarding baseline demographic and quality of life characteristics. Compared with baseline values, the mean total score and subscores of quality of life did not change significantly after synbiotic and placebo supplementation (p>0.05). Moreover, the results of ANCOVA showed that there were no significant differences between the two groups regarding the post-trial value of HRQOL total score and subscores. Conclusion: According to results, six-month supplementation with synbiotic did not have a significant effect on the HRQOL in children with CF. However, further studies with larger sample sizes and using more disease-specific questionnaires are needed for a more precise conclusion. The protocol of the study was registered at Iranian registry clinical trials (Registration code: IRCT2017011732004N1; Registration date: 2017-02-14).

Pregabalin augmentation for resistant obsessive–compulsive disorder: a double-blind placebo-controlled clinical trial

CNS Spectrums ◽  
2019 ◽  
Vol 25 (4) ◽  
pp. 552-556
Author(s):  
Arash Mowla ◽  
Mehrnoosh Ghaedsharaf
Keyword(s):  
Clinical Trial ◽  
Placebo Group ◽  
Obsessive Compulsive Disorder ◽  
Controlled Clinical Trial ◽  
Obsessive Compulsive ◽  
Double Blind ◽  
Good Tolerability ◽  
Double Blind Placebo ◽  
Compulsive Disorder ◽  
Current Medication

AbstractBackground and objective.Glutamate dysfunction has been shown to be associated with pathophysiology of obsessive–compulsive disorder (OCD). Our objective is to survey the effects of pregabalin (a glutamate-modulating agent) as an augmenting treatment for resistant OCD.Patients and methods.In this 12-week double-blind placebo-controlled clinical trial, 56 patients with resistant OCD were randomly allocated to receive either pregabalin or placebo plus their current medication (sertraline). Yale–Brown Obsessive Compulsive Scale (Y-BOCS) was used to evaluate the outcomes. Adverse effects were also registered.Results.Of the 56 patients with resistant OCD who were randomly allocated in 2 groups of pregabalin (n = 28) and placebo group (n = 28), 42 patients (22 in pregabalin group and 20 in placebo group) completed the trial. Throughout the trial, the mean score decreased from 26.13± 7.03 to 8.81 ± 3.47 in the pregabalin group (p < 0) and from 26.85 ± 4.34 to 17.63 ± 4.22 in the placebo group (p < 0). At the end of trial, 16 (57.14%) patients in the pregabalin group and 2 (7.14%) patients in the placebo group showed more than 35% decline in YBOCS (p < .01). The pregabalin group showed good tolerability and safety.Conclusions.Our study revealed that pregabalin, as an augmenting medication, is more effective than placebo in the treatment of patients with resistant OCD.

scholarly journals Granisetron Augmentation of Sertraline in Obsessive-Compulsive Disorder: A Double-Blind Placebo-Controlled, Randomized Clinical Trial

2022 ◽  
Author(s):  
Ala Ghobadian ◽  
Saba Mokhtari ◽  
Behnam Shariati ◽  
Leila Kamalzadeh ◽  
Mohsen Shati ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Placebo Group ◽  
Obsessive Compulsive Disorder ◽  
Selective Serotonin Reuptake Inhibitors ◽  
Complete Response ◽  
Controlled Clinical Trial ◽  
P Value ◽  
Obsessive Compulsive ◽  
Double Blind ◽  
Compulsive Disorder

Abstract Background: Medications currently recommended for the treatment of Obsessive-Compulsive Disorder (OCD) usually relieve the severity of symptoms by as much as 20–30%, and satisfactory treatment is obtained in 40–60% of patients with OCD. Nevertheless, the remaining symptoms continue to impair the patients’ function. Therefore, it is necessary to investigate possible strategies to improve the mitigation of symptoms.In this study, the main objective was to examine and investigate the effectiveness of granisetron, which is a serotonin 5-hydroxytryptamine receptor type 3 (5-HT3) antagonist, as an adjunct therapy to selective serotonin reuptake inhibitors, for the purpose of ameliorating OCD symptoms. Methods: fifty-eight patients diagnosed with OCD, based on Diagnostic and Statistical Manual of Mental Disorders (DSM-5) criteria, who had a Yale-Brown obsessive-compulsive scale (Y-BOCS) score of more than 21 were recruited in a double-blinded, parallel-group, placebo-controlled, clinical trial of 10 weeks to receive either granisetron (1 mg twice daily) and sertraline (100 mg daily initially followed by 200 mg daily after week 4) or placebo and sertraline. The primary outcome was OCD symptoms measured by the Y-BOCS.Results: Y-BOCS total score significantly dropped in both groups (28.9 to 17.7 for granisetron and 27.5 to 19.3 for placebo group with a slightly greater drop for granisetron group), while the granisetron group experienced a significantly greater reduction in obsession scores (Greenhouse-Geisser F(2.32,97.57)=4.52,p-value=0.01). Moreover, in comparison with the placebo group, the proportion of the patients showing complete response was considerably higher among the granisetron group (P-value <0.01). No major adverse effects were observed in any of the groups. Conclusion: The results suggest that granisetron augmentation of sertraline may increase the rate of response in patients with moderate to severe non-refractory OCD. Further studies are suggested in this regard.Trial registration: The trial was registered at the Iranian Registry of Clinical Trials on 27/03/2019 (www.irct.ir; IRCT ID: IRCT20170123032145N3).

scholarly journals Does synbiotic supplementation affect the quality of life in children with cystic fibrosis? A pilot randomized controlled clinical trial

2020 ◽  
Author(s):  
Nemat Bilan ◽  
Effat Marefat ◽  
Leila Nikniaz ◽  
Mahdieh Abbasalizad Farhangi ◽  
Zeinab Nikniaz
Keyword(s):  
Quality Of Life ◽  
Clinical Trial ◽  
Cystic Fibrosis ◽  
Demographic Data ◽  
Intervention Group ◽  
Controlled Clinical Trial ◽  
Double Blind ◽  
The Mean ◽  
Synbiotic Supplementation

Abstract Background: There is no clinical trial that assesses the effect synbiotic supplementation on HRQOL in CF children. Considering the importance of HRQOL as an essential primary outcome and determinant of therapeutic benefit in chronic diseases like cystic fibrosis, the present clinical trial aimed to determine the efficacy of synbiotic supplementation on HRQOL in children with CF.Methods: In the present double-blind randomized clinical trial, 40 CF children were randomly allocated to the two groups. The intervention group was supplemented with synbiotics supplements and the patients in the placebo group received maltodextrin for six months. Demographic data and information about antibiotic use were recorded using the questionnaire. The health-related quality of life was assessed using the Persian version of quality of life inventory questionnaires. Paired t-test and ANCOVA were used for statistical analysis. Results: Totally, 36 participants completed the trial. The mean score of HRQOL was 76.34±17.33. There were no significant differences between synbiotic and placebo groups regarding baseline demographic and quality of life characteristics. Compared with baseline values, the mean total score and subscores of quality of life did not change significantly after synbiotic and placebo supplementation (p>0.05). Moreover, the results of ANCOVA showed that there were no significant differences between the two groups regarding the post-trial value of HRQOL total score and subscores. Conclusion: According to results, six-month supplementation with synbiotic did not have a significant effect on the HRQOL in children with CF. However, further studies with larger sample sizes and using more disease-specific questionnaires are needed for a more precise conclusion. The protocol of the study was registered at Iranian registry clinical trials (Registration code: IRCT2017011732004N1; Registration date: 2017-02-14).

scholarly journals Does synbiotic supplementation affect the quality of life in children with cystic fibrosis? A pilot randomized controlled clinical trial

2020 ◽  
Author(s):  
Nemat Bilan ◽  
Effat Marefat ◽  
Leila Nikniaz ◽  
Mahdieh Abbasalizad Farhangi ◽  
Zeinab Nikniaz
Keyword(s):  
Quality Of Life ◽  
Clinical Trial ◽  
Cystic Fibrosis ◽  
Demographic Data ◽  
Intervention Group ◽  
Controlled Clinical Trial ◽  
Double Blind ◽  
The Mean ◽  
Synbiotic Supplementation

Abstract Background: There is no clinical trial that assesses the effect synbiotic supplementation on HRQOL in CF children. Considering the importance of HRQOL as an essential primary outcome and determinant of therapeutic benefit in chronic diseases like cystic fibrosis, the present clinical trial aimed to determine the efficacy of synbiotic supplementation on HRQOL in children with CF.Methods: In the present double-blind randomized clinical trial, 40 CF children were randomly allocated to the two groups. The intervention group was supplemented with synbiotics supplements and the patients in the placebo group received maltodextrin for six months. Demographic data and information about antibiotic use were recorded using the questionnaire. The health-related quality of life was assessed using the Persian version of quality of life inventory questionnaires. Paired t-test and ANCOVA were used for statistical analysis. Results: Totally, 36 participants completed the trial. The mean score of HRQOL was 76.34±17.33. There were no significant differences between synbiotic and placebo groups regarding baseline demographic and quality of life characteristics. Compared with baseline values, the mean total score and subscores of quality of life did not change significantly after synbiotic and placebo supplementation (p>0.05). Moreover, the results of ANCOVA showed that there were no significant differences between the two groups regarding the post-trial value of HRQOL total score and subscores. Conclusion: According to results, six-month supplementation with synbiotic did not have a significant effect on the HEQOL in children with CF. However, further studies with larger sample sizes and using more disease-specific questionnaires are needed for a more precise conclusion. The protocol of the study was registered at Iranian registry clinical trials (Registration code: IRCT2017011732004N1; Registration date: 2017-02-14).

scholarly journals Does Synbiotic Supplementation Affect the Quality of Life in Children with Cystic Fibrosis? A Pilot Randomized Controlled Clinical Trial

2020 ◽  
Author(s):  
Nemat Bilan ◽  
Effat Marefat ◽  
Leila Nikniaz ◽  
Mahdieh Abbasalizad Farhangi ◽  
Zeinab Nikniaz
Keyword(s):  
Quality Of Life ◽  
Clinical Trial ◽  
Cystic Fibrosis ◽  
Demographic Data ◽  
Intervention Group ◽  
Controlled Clinical Trial ◽  
Double Blind ◽  
The Mean ◽  
Synbiotic Supplementation

Abstract Background There is no clinical trial that assesses the effect synbiotic supplementation on HRQOL in CF children. Considering the importance of HRQOL as an essential primary outcome and determinant of therapeutic benefit in chronic diseases like cystic fibrosis, the present clinical trial aimed to determine the efficacy of synbiotic supplementation on HRQOL in children with CF.Methods In the present double-blind randomized clinical trial, 40 CF children were randomly allocated to the two groups. The intervention group was supplemented with synbiotics supplements and the patients in the placebo group received maltodextrin for six months. Demographic data and information about antibiotic use were recorded using the questionnaire. The health-related quality of life was assessed using the Persian version of quality of life inventory questionnaires. Paired t-test and ANCOVA were used for statistical analysis.Results Totally, 36 participants completed the trial. The mean score of HRQOL was 76.34 ± 17.33. There were no significant differences between synbiotic and placebo groups regarding baseline demographic and quality of life characteristics. Compared with baseline values, the mean total score and subscores of quality of life did not change significantly after probiotic and placebo supplementation (p > 0.05). Moreover, the results of ANCOVA showed that there were no significant differences between the two groups regarding the post-trial value of HRQOL total score and subscores.Conclusion According to results, six-month supplementation with synbiotic did not have a significant effect on the HEQOL in children with CF. However, further studies with larger sample sizes and using more disease-specific questionnaires are needed for a more precise conclusion.

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