Masaaki Mori1, Shinichi Watabe2, Tomoaki Taguchi3,4, Hisaya Hasegawa5, Mika Ishige6, Naoyuki Tanuma7, Akihiro Hirakawa8, Ryuji Koike8, Satoshi Kusuda9. 1. Department of Pediatrics, Tokyo Medical and Dental University Medical Hospital, 1-5-45 Yushima, Bunkyo-ku, Tokyo, 113-8519, Japan. mori.phv@tmd.ac.jp. 2. Department of Pediatrics, Kurashiki Central Hospital, 1-1-1 Miwa, Kurashiki, Okayama, 710-8602, Japan. 3. Department of Pediatric Surgery, Kyushu University Hospital, 3-1-1 Maidashi, Higashi-ku, Fukuoka, 812-8582, Japan. 4. present affiliation: Fukuoka College of Health Science, 2-15-1 Tamura, Sawara-ku, Fukuoka, 814-0193, Japan. 5. Division of Neonatal Intensive Care, Tokyo Women's Medical University Medical Center East, 2-1-10 Nishiogu, Arakawa-ku, Tokyo, 116-8567, Japan. 6. Department of Pediatrics and Child Health, Nihon University School of Medicine, 1-6 Kanda Surugadai, Chiyoda-ku, Tokyo, 101-8309, Japan. 7. Department of Pediatrics, Tokyo Metropolitan Fuchu Medical Center for the Disabled, 2-9-2 Musashidai, Fuchu, Tokyo, 183-8553, Japan. 8. Clinical Research Center, Tokyo Medical and Dental University Medical Hospital, 1-5-45 Yushima, Bunkyo-ku, Tokyo, 113-8519, Japan. 9. Department of Pediatrics, Faculty of Medicine, Kyorin University, 6-20-2 Shinkawa, Mitaka, Tokyo, 181-8611, Japan.
Abstract
BACKGROUND: 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. METHODS/ DESIGN: 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. DISCUSSION: 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. TRIAL REGISTRATION: 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).
BACKGROUND: 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. METHODS/ DESIGN: 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. DISCUSSION: 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. TRIAL REGISTRATION: 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).
Authors: S Johnson; C Oliver; G A Prince; V G Hemming; D S Pfarr; S C Wang; M Dormitzer; J O'Grady; S Koenig; J K Tamura; R Woods; G Bansal; D Couchenour; E Tsao; W C Hall; J F Young Journal: J Infect Dis Date: 1997-11 Impact factor: 5.226
Authors: Anja Wilkesmann; Roland A Ammann; Oliver Schildgen; Anna M Eis-Hübinger; Andreas Müller; Jürgen Seidenberg; Volker Stephan; Christian Rieger; Egbert Herting; Thorsten Wygold; Friedeman Hornschuh; Jessie R Groothuis; Arne Simon Journal: Pediatr Infect Dis J Date: 2007-06 Impact factor: 2.129