Trial · NCT07895381
Registered outcomes20
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 20 of 20
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | 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. |
| Primary | 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. |
| Primary | 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. |
| Primary | 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. |
| Primary | 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. |
| Primary | 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. |
| Primary | 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). |
| Primary | 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). |
| Primary | 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. |
| Primary | 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). |
| Primary | 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). |
| Primary | 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. |
| Primary | 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. |
| Primary | 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. |
| Primary | 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. |
| Primary | 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. |
| Secondary | Number of patients receiving different dose of fitusiran and rate of dosing regimen changes since starting fitusiran: Cohort 1 & 2 | Over 12 months of treatment with fitusiran | |
| Secondary | Frequency of antithrombin (AT) and liver function test (LFT) performed: Cohort 1 & 2 | Over 12 months of treatment with fitusiran | |
| Secondary | Frequency and type of treatment access barriers among patients initiating fitusiran: Cohort 1 & 2 | Over 12 months of treatment with fitusiran | |
| Secondary | 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 | 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. |
Source ClinicalTrials.govtrials-2026-11-12-0.5.0
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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