HemophiliaNCT05904210
Retrospective Experience Assessing the Real-World Utilization and Effectiveness of SEVENFACT®
An observational study of Hemophilia, run by Laboratoire français de Fractionnement et de Biotechnologies at 8 sites in United States.
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… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hemophilia
- What is being tested?
- SEVENFACT®
- How many people
- 17 participants
- Where
- 8 sitesUnited States
- Study dates
- 7 Nov 2023to 9 May 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- 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.
Who cannot join
- Not applicable
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
United States8 sites
Children's healthcare of Atlanta
Atlanta, Georgia, 30322
University Hospitals Cleveland Medical Center
Cleveland, Ohio, 44106
The Gulf States Hemophilia & Thrombophilia Center
Houston, Texas, 77030
Luskin Orthopaedic Institute for Children
Los Angeles, California, 90007
Valley Children's Hospital
Madera, California, 93636
Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Mayo Clinic
Rochester, Minnesota, 55905
SUNY Upstate Medical University, Pediatric Hematology/Oncology
Syracuse, New York, 13210
About this study
Official title: 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®.
Read the 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.
- Conditions
- Hemophilia
- Being tested
- SEVENFACT®
- Keywords
- inhibitors, eptacog beta, recombinant factor 7, real-world evidence
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SEVENFACT®Drug
Coagulation Factor VIIa (Recombinant)
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: SEVENFACT® |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: For all events which took place from Jan 1st, 2021 up to site opening in 2023
Secondary outcomes (1)
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
Measured: For all events which took place from Jan 1st, 2021 up to site opening in 2023
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jun 2023
- Study start7 Nov 2023
- Primary completion9 May 2024
- Study completion9 May 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for HemophiliaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- Laboratoire français de Fractionnement et de Biotechnologies
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05904210
- Sponsor's protocol ID
- F7TG2204
- Status last verified
- Jun 2024
- First submitted
- 16 May 2023
- Last updated
- 25 Jun 2024
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
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.
PubMed 28776894
Study at a glance
From the registry record, last updated by the sponsor on 25 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Retrospective Experience Assessing the Real-World Utilization and Effectiveness of SEVENFACT® [Coagulation Factor VIIa (Recombinant) - Jncw] in the USA
- ClinicalTrials.gov ID
- NCT05904210
- Acronym / sponsor ID
- F7TG2204
- Conditions
- Hemophilia
- Keywords
- inhibitors, eptacog beta, recombinant factor 7, real-world evidence
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Laboratoire français de Fractionnement et de BiotechnologiesIndustry (pharma, biotech, devices)
- Study start
- 7 Nov 2023 (actual)
- Primary completion
- 9 May 2024 (actual)
- Study completion
- 9 May 2024 (actual)
- First posted
- 15 Jun 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients treated by SEVENFACT® | – | Drug: 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. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (2 listed)
- 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.
Key exclusion criteria (1 listed)
- Not applicable
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SEVENFACT®Coagulation Factor VIIa (Recombinant) | Drug | Patients treated by SEVENFACT® |
Primary outcome
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.
1 secondary outcome
Patient profile analysis
Time frame: For all events which took place from Jan 1st, 2021 up to site opening in 2023
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jun 2024.
- Background: PubMed 28776894Wang 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia, run by Laboratoire français de Fractionnement et de Biotechnologies at 8 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 Jun 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
SEVENFACT® effectiveness (time frame: For all events which took place from Jan 1st, 2021 up to site opening in 2023). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05904210, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Jun 2024.