The purpose of this study is to learn more about painful crisis in people with Sickle Cell Disease. For this reason, Pfizer is conducting a study to understand how people with Sickle Cell Disease feel on a daily basis, treat these painful episodes including if they treat themselves at home or go to a doctor's office/emergency room, as well as the types of medications that are taken during these episodes.
Pfizer is developing an ePRO that is completed on a daily basis using a diary to comprehensively self-report VOCs in participants with SCD and its impact on their lives.This is a prospective, low-intervention study to evaluate the responsiveness of the SCD ePRO in a therapeutic setting. Efficacy of drugs intended to reduce the frequency of VOC has historically been assessed based on frequency of VOC with an operational definition for VOC that requires MU, an endpoint approach that is limited in its utility for assessing benefit of therapeutic interventions across the totality of the disease experience. Thus, despite current available treatments, many patients with SCD still experience VOCs and there remains a significant unmet medical need and an opportunity to improve on existing endpoints. While previous studies have been conducted to consider this endpoint, they have not been conducted in a therapeutic setting and therefore unable to assess the responsiveness of theSCD ePRO.1,2This study aims to assess the responsiveness of the patient-reported endpoints in participants with SCD who are not on a disease modifying therapy versus those who are on the disease modifying treatment HU.
Study Type
OBSERVATIONAL
Enrollment
52
Sanguine Biosciences
Woburn, Massachusetts, United States
Primary Objective #1:
To assess whether the effect of HU on frequency of physician-reported MU VOC is observed in the study population, the annualized rate reduction in those on HU as compared to matched participants in control group will be evaluated. The null hypothesis is that the annualized physician-reported MU VOC rate in HU treatment group is not different from one in control group.
Time frame: 6 months
Primary Objective #2
To assess whether the effect of HU on frequency of patient-reported crisis rates (VOC Day rate and Patient-reported VOC Event rate) calculated using SCD ePRO is observed in the study population, the annualized rates reduction in HU treatment group as compared to matched participants in control group will be evaluated. The corresponding null hypotheses are that the annualized patient-reported crisis rates in HU treatment group are not different from ones in control group.
Time frame: 6 months
Association Between Patient-Reported Crisis Rates and Physician-Reported MU VOC rate
Patient-reported crisis rates are determined by VOC Day rate and patient-reported VOC Event rate and is calculated using the SCD ePRO. These will be compared to physician-reported MU VOC rate to assess if there is an association between them. The assessment will be done in the matched sample, as well as in those in HU treatment group and in matched control group participants. The corresponding null hypotheses are that the annualized patient-reported crisis rates are not associated with physician-reported MU VOC.
Time frame: 6 months
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