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

NCT05904210 | Completed FDA Drug

• 1 other identifier: F7TG2204
• Study Type: observational
• Target: 17
• Locations: 1 country
• Sites: 8

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®.

Conditions

Hemophilia

Keywords

inhibitors; eptacog beta; recombinant factor 7; real-world evidence

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Patient profile analysis

  For all events which took place from Jan 1st, 2021 up to site opening in 2023

Study Arms (1)

Patients treated by 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.

Drug: SEVENFACT®

Interventions

SEVENFACT® DRUG

Coagulation Factor VIIa (Recombinant)

Patients treated by SEVENFACT®

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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.

You may not qualify if:

• Not applicable

Sponsors & Collaborators

• Laboratoire français de Fractionnement et de Biotechnologies: lead

Study Sites (8)

Luskin Orthopaedic Institute for Children

Los Angeles, California, 90007, United States

Location

Valley Children's Hospital

Madera, California, 93636, United States

Location

Children's healthcare of Atlanta

Atlanta, Georgia, 30322, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

SUNY Upstate Medical University, Pediatric Hematology/Oncology

Syracuse, New York, 13210, United States

Location

University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44106, United States

Location

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

The Gulf States Hemophilia & Thrombophilia Center

Houston, Texas, 77030, United States

Location

Related 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.

  PMID: 28776894 BACKGROUND

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

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

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 16, 2023
• First Posted: June 15, 2023
• Study Start: November 7, 2023
• Primary Completion: May 9, 2024
• Study Completion: May 9, 2024
• Last Updated: June 25, 2024

Record last verified: 2024-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (8)