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

Hemophilia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05904210
• Other study ID #: F7TG2204
• Status: Completed
• Start date: November 7, 2023
• Est. completion date: May 31, 2024

Study information

• Verified date: June 2024
• Source: Laboratoire français de Fractionnement et de Biotechnologies
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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®.

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 information / eligibility

• Status: Completed
• Enrollment: 17
• Est. completion date: May 31, 2024
• Est. primary completion date: May 31, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

1. Patient received SEVENFACT® before study initiation.

2. 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

Related Conditions & MeSH terms

• Hemophilia

Intervention

Drug:
• SEVENFACT®
Coagulation Factor VIIa (Recombinant)

Locations

Country	Name	City	State
United States	Children's healthcare of Atlanta	Atlanta	Georgia
United States	University Hospitals Cleveland Medical Center	Cleveland	Ohio
United States	The Gulf States Hemophilia & Thrombophilia Center	Houston	Texas
United States	Luskin Orthopaedic Institute for Children	Los Angeles	California
United States	Valley Children's Hospital	Madera	California
United States	Hospital of the University of Pennsylvania	Philadelphia	Pennsylvania
United States	Mayo Clinic	Rochester	Minnesota
United States	SUNY Upstate Medical University, Pediatric Hematology/Oncology	Syracuse	New York

Sponsors (1)

Lead Sponsor	Collaborator
Laboratoire français de Fractionnement et de Biotechnologies

Country where clinical trial is conducted

United States, 

References & Publications (1)

Wang M, Lawrence JB, Quon DV, Ducore J, Simpson ML, Boggio LN, Mitchell IS, Yuan G, Alexander WA, Schved JF. PERSEPT 1: a phase 3 trial of activated eptacog beta for on-demand treatment of haemophilia inhibitor-related bleeding. Haemophilia. 2017 Nov;23(6):832-843. doi: 10.1111/hae.13301. Epub 2017 Aug 3. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	SEVENFACT® effectiveness	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.	For all events which took place from Jan 1st, 2021 up to site opening in 2023
Secondary	Patient profile analysis	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	For all events which took place from Jan 1st, 2021 up to site opening in 2023