Study protocol: a multicenter, uncontrolled, open-label study of palivizumab in neonates, infants, and preschool children at high risk of severe respiratory syncytial virus infection

• Published in: BMC pediatrics Vol. 21; no. 1; pp. 106 - 9
• Main Authors: Mori, Masaaki, Watabe, Shinichi, Taguchi, Tomoaki, Hasegawa, Hisaya, Ishige, Mika, Tanuma, Naoyuki, Hirakawa, Akihiro, Koike, Ryuji, Kusuda, Satoshi
• Format: Journal Article
• Language: English
• Published: England BioMed Central Ltd 02.03.2021; BioMed Central; BMC
• Subjects: Airway stenosis; Antibodies, Monoclonal - therapeutic use; Antibodies, Monoclonal, Humanized - therapeutic use; Antiviral Agents - therapeutic use; Babies; Cardiovascular disease; Child; Child, Preschool; Children; Children & youth; Congenital diseases; Congenital esophageal atresia; Consent; Diseases; Dosage and administration; Down syndrome; Drugs; Efficacy; Esophagus; Gestational age; Hospitalization; Humans; Immunotherapy; Infant; Infant, Newborn; Infections; Inherited metabolic disease; Japan; Lung diseases; Metabolic disorders; Monoclonal antibodies; Multicenter Studies as Topic; Neonatal care; Neuromuscular disease; Neuromuscular diseases; Newborn babies; Palivizumab; Palivizumab - therapeutic use; Patient outcomes; Pediatric pharmacology; Pediatric research; Pediatrics; Pharmacokinetics; Premature birth; Preschool children; Prevention; Rare diseases; Regulatory approval; Respiratory syncytial virus; Respiratory syncytial virus infection; Respiratory Syncytial Virus Infections - prevention & control; Risk factors; Study Protocol; Ventilators; Japan; United States > US; Inherited metabolic disease; Palivizumab; Respiratory syncytial virus infection; Neuromuscular disease; Pulmonary hypoplasia; Efficacy; Pediatric patient; Airway stenosis; Congenital esophageal atresia
• ISSN: 1471-2431; 1471-2431
• DOI: 10.1186/s12887-021-02567-6

The prophylactic use of anti-respiratory syncytial virus (RSV) antibody (palivizumab) for severe RSV infection is not approved in Japan in specified groups of infants with neuromuscular diseases or other rare diseases associated with reduced ventilation competence or difficulty in expectoration, which increase the risk of exacerbation of severe RSV infection. The objective of this study is to investigate the efficacy, safety, and pharmacokinetics of palivizumab in pediatric patients with those rare diseases for which palivizumab is not indicated at present. This study is a multicenter, uncontrolled, open-label study planned to be carried out between July 1, 2019 and June 30, 2022 at 7 medical institutions in Japan. The study population will be recruited from among neonates, infants, or children aged 24 months or younger with a condition falling under any of the following 5 disease groups: pulmonary hypoplasia, airway stenosis, congenital esophageal atresia, inherited metabolic disease, or neuromuscular disease. The planned sample size is 18 subjects, including at least 3 subjects per disease group. Throughout the RSV season, at least 4 continuous doses of palivizumab will be administered intramuscularly at 15 mg/kg at intervals of 30 days. The efficacy and safety of palivizumab will be comprehensively evaluated based on the incidence of RSV-related hospitalization, and serum palivizumab concentration, serum anti-palivizumab antibody concentration, and the occurrence of adverse events/reactions after the start of palivizumab treatment. This study will evaluate the efficacy and safety of palivizumab in pediatric patients with rare diseases which place them at high risk of severe RSV infection, but which fall outside the current indications for palivizumab prophylaxis. The generated data will have implications for the regulatory approval of prophylactic palivizumab treatment in this patient group. This study has been prospectively registered in Japic Clinical Trials Information, which is managed and administered by the Japan Pharmaceutical Information Center (registration number: JapicCTI-194946 , registration date: September 10, 2019).