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Clinical Trials in the USA / NCT07895381
Starting soon Observational

A Study to Describe Patient REal-world FitusiRan ExpeRience

NCT07895381 · tracked via the Priya Life Science USA tracker
Sponsor
Sanofi
Phase
Observational
Started
2026-10-07
Last updated
2026-10-26

Condition(s) studied

Hemophilia A or B

Study 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. 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.

Eligibility

Sex
ALL
Min age
12 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria: * 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 * 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 Exclusion Criteria: * 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.

Primary outcome measure(s)

  • Patient-reported satisfaction with fitusiran measured by patient treatment preference questionnaire: Cohort 1 & 2 — Over 12 months of treatment with fitusiran
    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.
  • Patient-reported satisfaction with previous treatment measured by bespoke qualitative patient survey and interview: Cohorts 1 & 2 — Over 12 months of treatment with fitusiran
    The Bespoke survey covering patient satisfaction and transition to, or changes made while taking Fitusiran.
  • Patient-reported adherence to fitusiran measured by patient treatment preference questionnaire: Cohort 1 — Over 12 months of treatment with fitusiran
    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.
  • Patient-reported reasons for switching to or starting and staying on fitusiran assessed through bespoke qualitative patient survey and interview: Cohort 1 & 2 — Over 12 months of treatment with fitusiran
    The Bespoke survey covering patient satisfaction and transition to, or changes made while taking Fitusiran.
  • Patient-reported disadvantages of using fitusiran assessed by bespoke qualitative patient survey and interview: Cohort 1 & 2 — Over 12 months of treatment with fitusiran
    The Bespoke survey covering patient satisfaction and transition to, or changes made while taking Fitusiran.
  • Patient-reported willingness to continue fitusiran assessed through patient treatment preference questionnaire: Cohort 1 & 2 — Over 12 months of treatment with fitusiran
    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.
  • Change from baseline in patient-reported physical activity since starting fitusiran using PROBE questionnaire, EQ-5D-5L, EQ-5D-Y-3L: Cohort 1 — Over 12 months of treatment with fitusiran
    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).
  • Change from baseline in patient-reported joint health since starting fitusiran using PROBE questionnaire, EQ-5D-5L, EQ-5D-Y-3L: Cohort 1 — Over 12 months of treatment with fitusiran
    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).
  • Changes in patient reported outcomes, burdens and experiences (PROBE): Cohort 1 — Over 12 months of treatment with fitusiran
    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.
  • Changes in EQ-5D-5L (for adult participants): Cohort 1 & 2 — Over 12 months of treatment with fitusiran
    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).
  • Changes in EQ-5D-Y-3L (for adolescent participants): Cohort 1 & 2 — Over 12 months of treatment with fitusiran
    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).
  • 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 — Over 12 months of treatment with fitusiran
    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.
  • Changes in patient-reported hemophilia disease and treatment-related pain since starting fitusiran measured by PROMIS v2.0 - pain intensity 3a: Cohort 1 — Over 12 months of treatment with fitusiran
    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.
  • 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 — Over 12 months of treatment with fitusiran
    The Bespoke survey covering patient satisfaction and transition to, or changes made while taking Fitusiran.
  • Number of patient-reported antithrombin (AT) test and associated impacts measured by the bespoke qualitative patient survey and interview: Cohort 1 & 2 — Over 12 months of treatment with fitusiran
    The Bespoke survey covering patient satisfaction and transition to, or changes made while taking Fitusiran.
  • Number of patient-reported liver function test (LFT) and associated impacts measured by the bespoke qualitative patient survey and interview: Cohort 1 & 2 — Over 12 months of treatment with fitusiran
    The Bespoke survey covering patient satisfaction and transition to, or changes made while taking Fitusiran.

Trial sites (1)

FacilityCityRegionStatus
Concentrics Center for Research Indianapolis Indiana

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07895381 on ClinicalTrials.gov ↗ ← All trials in the USA