scholarly journals Phase II, Randomized, Open-Label Study of Pegfilgrastim-Supported VDC/IE Chemotherapy in Pediatric Sarcoma Patients

2010 ◽  
Vol 28 (8) ◽  
pp. 1329-1336 ◽  
Author(s):  
Sheri L. Spunt ◽  
Helen Irving ◽  
Jami Frost ◽  
Leonard Sender ◽  
Matthew Guo ◽  
...  
Keyword(s):  
Pediatric Patients ◽  
Age Groups ◽  
Efficacy And Safety ◽  
Older Children ◽  
Open Label ◽  
Myelosuppressive Chemotherapy ◽  
Open Label Study ◽  
Label Study ◽  
Pediatric Sarcoma ◽  
To Receive

Purpose This multicenter, randomized, open-label study evaluated the efficacy, safety, and pharmacokinetics of a single subcutaneous pegfilgrastim injection with daily subcutaneous filgrastim administration in pediatric patients receiving myelosuppressive chemotherapy for sarcoma. Patients and Methods Forty-four patients with previously untreated, biopsy-proven sarcoma stratified into three age groups (0-5, 6-11, and 12-21 years) were randomly assigned in a 6:1 randomization ratio to receive a single pegfilgrastim dose of 100 μg/kg (n = 38) or daily filgrastim doses of 5 μg/kg (n = 6) after chemotherapy (cycles 1 and 3: vincristine-doxorubicin-cyclophosphamide; cycles 2 and 4: ifosfamide-etoposide). The duration of grade 4 neutropenia, time to neutrophil recovery, incidence of febrile neutropenia, and adverse events were recorded. Results Pegfilgrastim and filgrastim were similar for all efficacy and safety end points, and their pharmacokinetic profiles were consistent with those in adults. Younger children experienced more protracted neutropenia and had higher median pegfilgrastim exposure than older children. Conclusion A single dose of pegfilgrastim at 100 μg/kg administered once per chemotherapy cycle is comparable to daily injections of filgrastim at 5 μg/kg for pediatric sarcoma patients receiving myelosuppressive chemotherapy.

scholarly journals 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

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Masaaki Mori ◽  
Shinichi Watabe ◽  
Tomoaki Taguchi ◽  
Hisaya Hasegawa ◽  
Mika Ishige ◽  
...  
Keyword(s):  
Respiratory Syncytial Virus ◽  
High Risk ◽  
Rare Diseases ◽  
Pediatric Patients ◽  
Efficacy And Safety ◽  
Open Label ◽  
Open Label Study ◽  
Label Study ◽  
Rsv Infection ◽  
Syncytial Virus

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).

scholarly journals Efficacy and Safety of Calcipotriol/Betamethasone Dipropionate Ointment for the Treatment of Trachyonychia: An Open-Label Study

2015 ◽  
Vol 27 (4) ◽  
pp. 371 ◽  
Author(s):  
Jung-Min Park ◽  
Hyun-Ho Cho ◽  
Won-Jeong Kim ◽  
Je-Ho Mun ◽  
Margaret Song ◽  
...  
Keyword(s):  
Betamethasone Dipropionate ◽  
Efficacy And Safety ◽  
Open Label ◽  
Open Label Study ◽  
Label Study ◽  
Calcipotriol Betamethasone

scholarly journals Efficacy and Safety of Thirst-Quenching Lozenges for Xerostomia in Patients Undergoing Hemodialysis: A Prospective, Single-Arm, Open-Label Study

2020 ◽  
Vol 6 (2) ◽  
Author(s):  
Sahay Manisha ◽  
Almeida Alan ◽  
Naqvi Syed Mujtaba Hussain ◽  
Venugopal Hariharan ◽  
Kale Ravindra Machhindra ◽  
...  
Keyword(s):  
Efficacy And Safety ◽  
Open Label ◽  
Open Label Study ◽  
Label Study
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