Retrospective Experience Assessing the Real-World Utilization and Effectiveness of SEVENFACT®
- Registration Number
- NCT05904210
- Lead Sponsor
- Laboratoire français de Fractionnement et de Biotechnologies
- Brief Summary
Retrospective Experience Assessing the Real-World Utilization and Effectiveness of SEVENFACT® \[coagulation factor VIIa (recombinant)-jncw\] in the USA.
The current study will provide a unique opportunity to describe the profile of the early patients selected for treatment with SEVENFACT® and to assess the real-world utilization (effectiveness, safety, tolerability, and modalities of treatment) of SEVENFACT®.
- Detailed Description
Parameters collected will include patient socio-demographics, medical history / comorbidities, bleeding disorder history, physical examination, vital signs, prior and concomitant bleeding disorder treatments, bleeding episode / surgery or invasive procedure / prophylaxis details, information on SEVENFACTâ treatment modalities, retrospective investigator rating of SEVENFACTâ effectiveness using the Clinical Global Impression Efficacy Index (CGI-E) and time for bleed control, Adverse Drug Experiences (ADEs) temporally associated with SEVENFACTâ administration, special situations that occurred during SEVENFACTâ administration, whether or not associated with ADE, laboratory testing for activation of the coagulation system or thrombosis, physical examination, and vital signs.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 17
- Patient received SEVENFACT® before study initiation.
- If collection of a written informed consent is required for an investigational site (see Section 5.1 General Informed Consent), patient has read, understood, and documented written informed consent/assent.
Not applicable
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Patients treated by SEVENFACT® SEVENFACT® Patients that initiated treatment with SEVENFACT® in real-world clinical care in the USA will be eligible. Data from eligible patients' medical charts, bleeding diaries, and medication logs will be extracted from the time of initiation of SEVENFACT® treatment and for each bleeding episode and surgery or invasive procedure requiring treatment with SEVENFACT® or for prophylaxis, up until the data collection at the investigational site.
- Primary Outcome Measures
Name Time Method SEVENFACT® effectiveness For all events which took place from Jan 1st, 2021 up to site opening in 2023 SEVENFACT® effectiveness will be evaluated based on the retrospective rating by the investigator using the Clinical Global Impression Efficacy Index (CGI-E) for each 'event' (bleeding episode, surgery or invasive procedure, or prophylaxis). Specifically, the proportion of patients receiving efficient treatment based on CGI-E will be estimated.
- Secondary Outcome Measures
Name Time Method Patient profile analysis For all events which took place from Jan 1st, 2021 up to site opening in 2023 The patient profile of patients treated with SEVENFACT® will be evaluated based on the patient's socio-demography, medical history, potential comorbidities, physical examination, current medications, and bleeding disorder history
Trial Locations
- Locations (8)
Valley Children's Hospital
🇺🇸Madera, California, United States
Luskin Orthopaedic Institute for Children
🇺🇸Los Angeles, California, United States
Children's healthcare of Atlanta
🇺🇸Atlanta, Georgia, United States
SUNY Upstate Medical University, Pediatric Hematology/Oncology
🇺🇸Syracuse, New York, United States
Hospital of the University of Pennsylvania
🇺🇸Philadelphia, Pennsylvania, United States
The Gulf States Hemophilia & Thrombophilia Center
🇺🇸Houston, Texas, United States
Mayo Clinic
🇺🇸Rochester, Minnesota, United States
University Hospitals Cleveland Medical Center
🇺🇸Cleveland, Ohio, United States