A LOW-INTERVENTIONAL LONGITUDINAL STUDY OF AN ELECTRONIC SICKLE CELL DISEASE PATIENT REPORTED OUTCOMES IN ADULT PARTICIPANTS AGED ≥18 YEARS OF AGE ON AND OFF HYDROXYUREA
Overview
- Phase
- Not Applicable
- Intervention
- Not specified
- Conditions
- Sickle Cell Disease
- Sponsor
- Pfizer
- Enrollment
- 98
- Locations
- 4
- Primary Endpoint
- Average SCD ePRO daily worst tiredness score during VOC days
- Status
- Completed
- Last Updated
- last year
Overview
Brief Summary
The purpose of this clinical trial is to evaluate the performance of the sickle cell disease (SCD) electronic diary in people with SCD who are on treatment that will change SCD and those not on such a treatment.
SCD is a type of condition when there are fewer red blood cells to carry oxygen around the body.
This disease can be passed on from parent to child and may cause pain, infections and damage to organs.
This study is seeking participants who:
- are confirmed with SCD
- are on a stable regimen of disease changing treatment or have not received any disease changing treatment before the start of the study and do not plan any changes in their treatment during the 6-month study observation period For 6 months, participants will be asked to complete a daily electronic diary to report on their experience in the past 24 hours with sickle cell pain crisis (if they got any treatment and what medications they took), worst pain, worst tiredness, and their ability to perform usual physical activities. We will compare the experiences of people who are taking SCD-modifying therapy to those that are not taking a SCD-modifying therapy.
Investigators
Eligibility Criteria
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Outcomes
Primary Outcomes
Average SCD ePRO daily worst tiredness score during VOC days
Time Frame: Day 1 to 180
Responsiveness of electronic patient reported outcomes between participants treated with SCD disease modifying treatment or no disease modifying treatment.
Average SCD ePRO daily worst tiredness score during non-VOC days
Time Frame: Day 1 to 180
Responsiveness of electronic patient reported outcomes between participants treated with SCD disease modifying treatment or no disease modifying treatment.
Physician-reported Medical Utilization vaso-occlusive crisis (VOC) rate
Time Frame: Day 1 to 180
Confirmation that the population is suitable for assessing responsiveness of electronic patient reported outcomes based upon a lower frequency rate of Physician reported Medical Utilization VOCs in the SCD disease modifying treatment group.
VOC Day rate
Time Frame: Day 1 to 180
Responsiveness of electronic patient reported outcomes between participants treated with SCD disease modifying treatment or no disease modifying treatment.
Patient-reported VOC Event rate
Time Frame: Day 1 to 180
Responsiveness of electronic patient reported outcomes between participants treated with SCD disease modifying treatment or no disease modifying treatment.
Average SCD ePRO daily worst pain score during VOC days
Time Frame: Day 1 to 180
Responsiveness of electronic patient reported outcomes between participants treated with SCD disease modifying treatment or no disease modifying treatment.
Average SCD ePRO daily worst pain score during non-VOC days
Time Frame: Day 1 to 180
Responsiveness of electronic patient reported outcomes between participants treated with SCD disease modifying treatment or no disease modifying treatment.
Average SCD ePRO daily rating for ability to perform usual physical activity during a non-VOC day
Time Frame: Day 1 to 180
Responsiveness of electronic patient reported outcomes between participants treated with SCD disease modifying treatment or no disease modifying treatment.
Average SCD ePRO daily rating for ability to perform usual physical activity during a VOC day
Time Frame: Day 1 to 180
Responsiveness of electronic patient reported outcomes between participants treated with SCD disease modifying treatment or no disease modifying treatment.
Secondary Outcomes
- Quantitative relationship between VOC Day rate and Physician-reported Medical Utilization VOC(Day 1 to 180)
- Quantitative relationship between VOC Day rate and Physician-reported Medical Utilization VOC rate across treatment groups(Day 1 to 180)
- Quantitative relationship between Patient-reported VOC Event rate and Physician-reported Medical Utilization VOC rate across treatment groups(Day 1 to 180)
- Quantitative relationship between Patient-reported VOC Event rate and Physician-reported Medical Utilization VOC rate(Day 1 to 180)