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Issues, challenges, and the way forward in conducting clinical trials among neonates: investigators' perspective
Sindhu Sivanandan1, Kajal Jain2, Nishad Plakkal1
1Jawaharlal Institute of Postgraduate Medical Education and Research (JIPMER), Puducherry, India.
Insights
Conducting clinical trials in neonates is crucial. This study details challenges and tools from a trial of goat lung surfactant extract (GLSE) for respiratory distress syndrome (RDS) in preterm infants.
Area of Science:
- Neonatology
- Pharmacology
- Clinical Research
Background:
- Limited drug trials in neonates lead to off-label medication use.
- Neonates face risks from unlicensed drugs, including toxicity and ineffective treatment.
- Ethical and operational hurdles should not prevent essential neonatal drug trials.
Purpose of the Study:
- To share challenges encountered during a combined phase-2 and phase-3 trial.
- To inform stakeholders about conducting neonatal drug trials.
- To foster discussion and provide adaptable tools for future research.
Main Methods:
- Combined phase-2 and phase-3 clinical trial design.
- Investigation of an indigenous goat lung surfactant extract (GLSE).
- Focus on treatment of respiratory distress syndrome (RDS) in preterm neonates.
Main Results:
- Identification of specific challenges in neonatal clinical trials.
- Development of practical tools and methodologies for trial implementation.
- Successful adaptation of trial protocols for a specific neonatal population.
Conclusions:
- Neonatal clinical trials are feasible despite ethical and operational complexities.
- Sharing trial experiences and developed tools can facilitate future research.
- Addressing challenges in neonatal drug development is vital for improving infant health outcomes.
Abstract:
Clinical trials are essential to test the safety and efficacy of new treatments in any population. The paucity of drug trials especially in the neonatal population has led to the widespread use of unlicensed or off-label medications, exposing them to the risks of drug toxicity and ineffective treatment. Ethical and operational challenges are no longer considered valid excuses for not conducting drug trials in neonates. We recently participated in a combined phase-2 and phase-3 trial investigating a new indigenous goat lung surfactant extract (GLSE) for the treatment of respiratory distress syndrome (RDS) in preterm neonates. In this article, we share pertinent challenges faced by us during the trial to better inform and foster-positive discussion among drug developers, administrators, regulatory authorities, patient advocacy groups, and researchers. Also, we provide many tools developed for the GLSE trial that can be modified and used by prospective trialists.