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

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®.

Study Overview

• Status: Recruiting
• Conditions: Hemophilia
• Intervention / Treatment: Drug: 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.

• Study Type: Observational
• Enrollment (Estimated): 20

Contacts and Locations

• Study Contact: Name: Pascaline BELLEVILLE; Phone Number: +33169827217; Email: bellevillep@lfb.fr

Study Locations

• United States
  • California
    • Los Angeles, California, United States, 90007
      • Recruiting
      • Luskin Orthopaedic Institute for Children
      • Contact: Doris Quon, MD
    • Madera, California, United States, 93636
      • Recruiting
      • Valley Children's Hospital
      • Contact: Vinod Balasa, MD
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Recruiting
      • Children's Healthcare of Atlanta
      • Contact: Michael White, MD
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Recruiting
      • Mayo Clinic
      • Contact: Ahmad Al-Huniti, MD
  • New York
    • Syracuse, New York, United States, 13210
      • Recruiting
      • SUNY Upstate Medical University, Pediatric Hematology/Oncology
      • Contact: Andrea Dvorak, MD
  • Ohio
    • Cleveland, Ohio, United States, 44106
      • Recruiting
      • University Hospitals Cleveland Medical Center
      • Contact: Sanjay Ahuja, MD
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Not yet recruiting
      • Hospital of the University of Pennsylvania
      • Contact: Adam Cuker, MD
  • Texas
    • Houston, Texas, United States, 77030
      • Recruiting
      • The Gulf States Hemophilia & Thrombophilia Center
      • Contact: Miguel ESCOBAR, MD
  • Washington
    • Northwest, Washington, United States, 20007
      • Not yet recruiting
      • MedStar Georgetown University Hospital
      • Contact: Craig Kessler, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• 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.

Description

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

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

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®; Coagulation Factor VIIa (Recombinant)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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

Collaborators and Investigators

• Sponsor: Laboratoire français de Fractionnement et de Biotechnologies

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start (Actual): November 7, 2023
• Primary Completion (Estimated): June 1, 2024
• Study Completion (Estimated): June 1, 2024

Study Registration Dates

• First Submitted: May 16, 2023
• First Submitted That Met QC Criteria: June 6, 2023
• First Posted (Actual): June 15, 2023

Study Record Updates

• Last Update Posted (Estimated): May 3, 2024
• Last Update Submitted That Met QC Criteria: May 2, 2024
• Last Verified: May 1, 2024

More Information

Terms related to this study

• Keywords: real-world evidence; inhibitors; eptacog beta; recombinant factor 7
• Other Study ID Numbers: F7TG2204

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No