Ignite Creation Date:
2025-12-25 @ 12:21 AM
Ignite Modification Date:
2026-01-05 @ 5:57 PM
Study NCT ID:
NCT06503458
Status:
TERMINATED
Last Update Posted:
2024-07-16
First Post:
2022-02-08
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Low-Interventional Study of an Electronic Sickle Cell Disease Patient Reported Outcomes in Sickle Cell Participants
Sponsor:
Sanguine Biosciences
Organization:
Study Overview
Official Title:
Sickle Cell Disease Patient Reported Outcomes in Adult Participants Aged ≥18 Years of Age On and Off Hydroxyurea
Status:
TERMINATED
Status Verified Date:
2024-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Low enrollment, cohorts were not able to be filled
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
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.
Detailed Description:
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 Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: