Clinical trial NCT05904210: Retrospective Experience Assessing the Real-World Utilization and Effectiveness of SEVENFACT®
Official title: Retrospective Experience Assessing the Real-World Utilization and Effectiveness of SEVENFACT® [Coagulation Factor VIIa (Recombinant) - Jncw] in the USA
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemophilia
Keywords: inhibitors
eptacog beta
recombinant factor 7
real-world evidence
Lead sponsor: Laboratoire français de Fractionnement et de Biotechnologies
Sponsor class: INDUSTRY
First posted: 2023-06-15
Enrollment: 17
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Full Analysis Set (FAS) defined as all patients that received at least one dose of SEVENFACT®. Missing data will not be imputed, and all analyses will be based on observed data only.

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.

Eligibility criteria: 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

Primary outcomes:
measure: SEVENFACT® effectiveness
timeFrame: For all events which took place from Jan 1st, 2021 up to site opening in 2023
Secondary outcomes:
measure: Patient profile analysis
timeFrame: For all events which took place from Jan 1st, 2021 up to site opening in 2023
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Luskin Orthopaedic Institute for Children
state: California
city: Madera
country: United States
facility: Valley Children's Hospital
state: California
city: Atlanta
country: United States
facility: Children's healthcare of Atlanta
state: Georgia
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: Syracuse
country: United States
facility: SUNY Upstate Medical University, Pediatric Hematology/Oncology
state: New York
city: Cleveland
country: United States
facility: University Hospitals Cleveland Medical Center
state: Ohio
city: Philadelphia
country: United States
facility: Hospital of the University of Pennsylvania
state: Pennsylvania
city: Houston
country: United States
facility: The Gulf States Hemophilia & Thrombophilia Center
state: Texas
Study officials:
References:
citation: 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.