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Hemophilia A or BPREFERNCT07895381

A Study to Describe Patient REal-world FitusiRan ExpeRience

An observational study of Hemophilia A or B, run by Sanofi at 1 site in United States.

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Sites and contacts
Official record
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Trial summary

This is a real-world, mixed-methods observational study conducted among adolescent (ages 12-17 years) and adult participants diagnosed with hemophilia A or B, with or without Factor VIII (FVIII) or Factor IX (FIX) inhibitors, who are treated with fitusiran. Read more about this study

Who is it for?
Ages 12 years and older
Conditions the study is for
Hemophilia A or B
How many people
100 participants
Where
1 siteUnited States
Study dates
7 Oct 2026to 2 Mar 2029
Phase
Observational
Are there results?
Not yet: the study is under way

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 12 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Study population: This study aims to enroll up to an estimated 100 participants across cohorts.

Who may be able to join

  • Participant is English speaking
  • Participant is ≥ 12 years of age at the time of consent
  • -- Signed written informed consent by participants who are legally adults (i.e., have reached the age of majority as defined by local law) or legally authorized representative (e.g., parent) and assent (for participants under the age of majority by local law)
  • Participant has a diagnosis of hemophilia A or B
  • Participant is using fitusiran (QFITLIA) for:
  • Prospective cohort: < 6 months at the time of consent
Show all 12Show fewer
  • Cross-sectional cohort: 9-15 months at the time of consent
  • Participant has access to an internet-connected computer or mobile device
  • Participant is willing and able to participate in:
  • Prospective cohort: Up to three 30-minute online surveys and two 60 minute virtual interviews over an approximately 12-month period
  • Cross-sectional cohort: A 30-minute online survey and 60-minute virtual interview
  • Participant resides in the US

Who cannot join

  • Prospective cohort: Participated in a clinical trial for fitusiran
  • Cross-sectional cohort: Participated in the prospective cohort
  • The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Trial Transparency email recommended (Toll free for US & Canada) contact

800-633-1610 ext. option 6

Contact-US@sanofi.com

United States1 site
  • Concentrics Center for Research

    Indianapolis, Indiana, 46240

Other trials in United States

About this study

Official title: Patient Experience With Fitusiran Prophylaxis in Adolescents and Adults With Hemophilia A or B With or Without Factor VIII or Factor IX Inhibitors in a Real-world Setting

This is a real-world, mixed-methods observational study conducted among adolescent (ages 12-17 years) and adult participants diagnosed with hemophilia A or B, with or without Factor VIII (FVIII) or Factor IX (FIX) inhibitors, who are treated with fitusiran. The study includes a prospective longitudinal cohort (Cohort 1) and a cross-sectional cohort (Cohort 2).

Data collection includes patient-reported outcomes via online surveys, qualitative interviews conducted via teleconference, and electronic medical record (EMR) review to describe real-world patient experiences, treatment satisfaction, adherence, and clinical management of fitusiran.

Medical subject headings
Hemophilia A

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Study groups in NCT07895381
Study groupRoleWhat they receive
Cohort 1Participants who have started fitusiran treatment within 0 to 5 months before baseline.
Cohort 2Participants who have started fitusiran treatment within 9 to 15 months before baseline.

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Patient-reported satisfaction with fitusiran measured by patient treatment preference questionnaire: Cohort 1 & 2

    The Patient Treatment Preference Questionnaire is a five-item instrument designed to evaluate treatment preferences. Items assess satisfaction with treatment, preferred treatment options, and willingness to continue treatment.

    Measured: Over 12 months of treatment with fitusiran

  2. Patient-reported satisfaction with previous treatment measured by bespoke qualitative patient survey and interview: Cohorts 1 & 2

    The Bespoke survey covering patient satisfaction and transition to, or changes made while taking Fitusiran.

    Measured: Over 12 months of treatment with fitusiran

  3. Patient-reported adherence to fitusiran measured by patient treatment preference questionnaire: Cohort 1

    The Patient Treatment Preference Questionnaire is a five-item instrument designed to evaluate treatment preferences. Items assess satisfaction with treatment, preferred treatment options, and willingness to continue treatment.

    Measured: Over 12 months of treatment with fitusiran

  4. Patient-reported reasons for switching to or starting and staying on fitusiran assessed through bespoke qualitative patient survey and interview: Cohort 1 & 2

    The Bespoke survey covering patient satisfaction and transition to, or changes made while taking Fitusiran.

    Measured: Over 12 months of treatment with fitusiran

  5. Patient-reported disadvantages of using fitusiran assessed by bespoke qualitative patient survey and interview: Cohort 1 & 2

    The Bespoke survey covering patient satisfaction and transition to, or changes made while taking Fitusiran.

    Measured: Over 12 months of treatment with fitusiran

  6. Patient-reported willingness to continue fitusiran assessed through patient treatment preference questionnaire: Cohort 1 & 2

    The Patient Treatment Preference Questionnaire is a five-item instrument designed to evaluate treatment preferences. Items assess satisfaction with treatment, preferred treatment options, and willingness to continue treatment.

    Measured: Over 12 months of treatment with fitusiran

  7. Change from baseline in patient-reported physical activity since starting fitusiran using PROBE questionnaire, EQ-5D-5L, EQ-5D-Y-3L: Cohort 1

    The Patient-Reported Outcomes Burdens and Experiences (PROBE) questionnaire is designed to assess the health status, treatment experiences, and QoL of individuals with hemophilia. Total PROBE score ranges from 0 to 1; a lower score means higher burden.

    The EQ-5D-5L is an adult measure of general health-related quality of life and health status, composed of five items and a visual analogue scale (VAS). VAS score ranges from 0 (worst imaginable health) to 100 (best imaginable health).

    The EQ-5D-Y-3L assesses the same health domains as the EQ-5D-5L but uses simplified, child-friendly language. VAS score ranges from 0 (worst imaginable health) to 100 (best imaginable health).

    Measured: Over 12 months of treatment with fitusiran

  8. Change from baseline in patient-reported joint health since starting fitusiran using PROBE questionnaire, EQ-5D-5L, EQ-5D-Y-3L: Cohort 1

    The Patient-Reported Outcomes Burdens and Experiences (PROBE) questionnaire is designed to assess the health status, treatment experiences, and QoL of individuals with hemophilia. Total PROBE score ranges from 0 to 1; a lower score means higher burden.

    The EQ-5D-5L is an adult measure of general health-related quality of life and health status, composed of five items and a visual analogue scale (VAS). VAS score ranges from 0 (worst imaginable health) to 100 (best imaginable health).

    The EQ-5D-Y-3L assesses the same health domains as the EQ-5D-5L but uses simplified, child-friendly language. VAS score ranges from 0 (worst imaginable health) to 100 (best imaginable health).

    Measured: Over 12 months of treatment with fitusiran

  9. Changes in patient reported outcomes, burdens and experiences (PROBE): Cohort 1

    The Patient-Reported Outcomes Burdens and Experiences (PROBE) questionnaire assesses health status, treatment experiences, and quality of life of individuals with hemophilia. Total PROBE score ranges from 0 to 1; a lower score means higher burden.

    Measured: Over 12 months of treatment with fitusiran

  10. Changes in EQ-5D-5L (for adult participants): Cohort 1 & 2

    The EQ-5D-5L is an adult measure of general health-related quality of life and health status, composed of five items and a visual analogue scale (VAS). VAS score ranges from 0 (worst imaginable health) to 100 (best imaginable health).

    Measured: Over 12 months of treatment with fitusiran

  11. Changes in EQ-5D-Y-3L (for adolescent participants): Cohort 1 & 2

    The EQ-5D-Y-3L is a tool to measure general health-related quality of life and health status in child-friendly language. It is composed of five items and a visual analogue scale (VAS). VAS score ranges from 0 (worst imaginable health) to 100 (best imaginable health).

    Measured: Over 12 months of treatment with fitusiran

  12. Patient-reported change in work or school attendance and productivity since starting fitusiran via the Work Productivity and Activity Impairment Questionnaire plus Classroom Impairment Questionnaire adapted for hemophilia patients (WPAI+CIQ:HS): Cohort 1

    The Work Productivity and Activity Impairment Questionnaire plus Classroom Impairment Questions for Hemophilia Specific (WPAI+CIQ:HS). Score ranged from 0-10; where 0 indicates less productivity and 10 indicates greater impairment.

    Measured: Over 12 months of treatment with fitusiran

  13. Changes in patient-reported hemophilia disease and treatment-related pain since starting fitusiran measured by PROMIS v2.0 - pain intensity 3a: Cohort 1

    The Patient-Reported Outcomes Measurement Information System (PROMIS) v2.0 Pain Intensity 3a short form is a patient-reported measure of hemophilia disease and treatment-related pain intensity. Score range from 3 to 15, the higher the score, the greater the pain.

    Measured: Over 12 months of treatment with fitusiran

  14. Changes in patient-reported mental health status, such as stress and anxiety, since starting fitusiran, measured by the bespoke qualitative patient survey and interview: Cohorts 1 & 2

    The Bespoke survey covering patient satisfaction and transition to, or changes made while taking Fitusiran.

    Measured: Over 12 months of treatment with fitusiran

  15. Number of patient-reported antithrombin (AT) test and associated impacts measured by the bespoke qualitative patient survey and interview: Cohort 1 & 2

    The Bespoke survey covering patient satisfaction and transition to, or changes made while taking Fitusiran.

    Measured: Over 12 months of treatment with fitusiran

  16. Number of patient-reported liver function test (LFT) and associated impacts measured by the bespoke qualitative patient survey and interview: Cohort 1 & 2

    The Bespoke survey covering patient satisfaction and transition to, or changes made while taking Fitusiran.

    Measured: Over 12 months of treatment with fitusiran

Secondary outcomes (4)
  1. Number of patients receiving different dose of fitusiran and rate of dosing regimen changes since starting fitusiran: Cohort 1 & 2

    Measured: Over 12 months of treatment with fitusiran

  2. Frequency of antithrombin (AT) and liver function test (LFT) performed: Cohort 1 & 2

    Measured: Over 12 months of treatment with fitusiran

  3. Frequency and type of treatment access barriers among patients initiating fitusiran: Cohort 1 & 2

    Measured: Over 12 months of treatment with fitusiran

  4. Patient-reported treatment satisfaction and compliance related to dose changes and longer dose adjustment period using patient treatment preference questionnaire and qualitative patient interview: Cohort 1 & 2

    The Patient Treatment Preference Questionnaire is a five-item instrument designed to evaluate treatment preferences. Items assess satisfaction with treatment, preferred treatment options, and willingness to continue treatment.

    Measured: Over 12 months of treatment with fitusiran

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted26 Oct 2026
  2. Study start7 Oct 2026 (estimated)
  3. Primary completion2 Mar 2029 (estimated)
  4. Study completion2 Mar 2029 (estimated)

Resources

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
Prospective
Enrolment
100 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07895381
Sponsor's protocol ID
OBS22863
Other IDs
U1111-1338-4278
Acronym
PREFER
Status last verified
Oct 2026
First submitted
6 Oct 2026
Last updated
26 Oct 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Questions about this trial

What is this clinical trial studying?

An observational study of Hemophilia A or B, run by Sanofi at 1 site 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 12 inclusion and 3 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: opening soon, as of the sponsor's last update on 26 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site 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?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Trial Transparency email recommended (Toll free for US & Canada) (800-633-1610 ext. option 6, Contact-US@sanofi.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Patient-reported satisfaction with fitusiran measured by patient treatment preference questionnaire: Cohort 1 & 2 (time frame: Over 12 months of treatment with fitusiran). The study lists 16 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?
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 26 Oct 2026.

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