Retrospective Experience Assessing the Real-World Utilization and Effectiveness of SEVENFACT®

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®.

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Key information

Conditions

Hemophilia Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemophilia A Hemorrhagic Disorders

Sex eligibility

All sexes

Study type

Observational

Primary location

Luskin Orthopaedic Institute for Children, Los Angeles, California, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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.

Exclusion criteria

Not applicable

Treatment and study plan

SEVENFACT®

Drug

Coagulation Factor VIIa (Recombinant)

Primary outcomes

SEVENFACT® effectiveness

Time frame: 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 outcomes

Patient profile analysis

Time frame: 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

Sponsors and collaborators

Lead sponsor

Laboratoire français de Fractionnement et de Biotechnologies

Industry

Registry information

Official study title

Retrospective Experience Assessing the Real-World Utilization and Effectiveness of SEVENFACT® [Coagulation Factor VIIa (Recombinant) - Jncw] in the USA

Important dates

Study start: 2023
Primary completion: 2024
Study completion: 2024
First posted: Jun 15, 2023
Registry last updated: Jun 25, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed