Perspectives in Clinical Research最新文献

Informed consent is a foundation of the ethical conduct of research involving human participants. Based on the ethical principle of respect for persons, the goal of informed consent is to ensure that participants are aware of the risks and potential benefits and make a voluntary decision about participating in clinical trial research. The extraordinary scientific advances happening globally, have demonstrated the potential of regenerative therapies in transforming the health of the nation by providing a therapeutic option for diseases that were previously considered incurable. These therapies, which include cells and gene therapy (GT) labeled as Advanced Therapeutic Medicinal Products globally, have complex mechanisms of action. Owing to their highly personalized and intricate nature of these therapies, developing the latter often presents unique challenges above and beyond those encountered for small molecule drugs. We recently looked through some cell and GT clinical trials and realized the lacunae in the informed consent form (ICF) provided by the investigators. Especially in a country like India, where the general understanding and perception of patients is limited regarding clinical trials, it is felt that any lapses in the consent process may jeopardize the informed decision-making and safety of the participants and tarnish the reputation of India globally. The present article highlights the need for appropriate patient and public education on the various aspects of cell and gene therapies and aims to address all the elements of ICF in light of the challenges associated with these innovative therapies.

Validity and reliability refer to the accuracy and consistency of a research tool. In the previous article in this series, we examined the development of a research questionnaire. In this article, we discuss the methods of determining the validity and reliability of a research questionnaire.

Context: Type 1 diabetes mellitus (T1DM) is a chronic disease, mainly observed in children or youth, with a significantly increased incidence in young children. Structured therapeutic patient education (TPE) is a must to help them manage their disease effectively and lead a healthy lifestyle.

Aims: This study aimed to assess the effects of a structured TPE program on glycemic markers and quality of life (QOL) of T1DM children and adolescents in Fez city, Morocco.

Settings and design: It is a quasi-experimental study.

Materials and methods: One hundred T1DM children and adolescents, aged 8-18, participated in a TPE intervention at the pediatric department in a hospital center in Fez, Morocco. Glycemic markers were measured and QOL was assessed by a validated questionnaire.

Statistical analysis used: Parametric and nonparametric tests were used and statistical significance determined by P < 0.05.

Results: At 3 months' follow-up, both global and dimensional QOL mean scores improved significantly (P ≤ 0.0001), whereas glycosylated hemoglobin (HbA1c) decreased (10.28% vs. 10.62%), tough with no statistical significance (P = 0.160). Furthermore, a significant improvement was observed in the maximum preprandial (2, 11 g/L [1.51-2.58] vs. 2, 37 g/L [1.81-3.21], P = 0.001) and postprandial blood glucose levels (2, 50 g/L [1.90-3.27] vs. 2, 95 g/L [2.07-3.99], P = 0.001) after 3 months; with no significant change in their minimum.

Conclusion: Although this TPE intervention was more effective in improving patients' QOL than their HbA1c, it is worth striving to implement regular TPE programs for T1DM pediatric patients and adjust them to achieve a better patients' glycemic markers levels.