A Study to Learn About Sickle Cell Disease In Adult Patients

Completed

• Conditions: Sickle Cell Disease
• Interventions: Other: Electronic Diary

• Registration Number: NCT05407805
• Lead Sponsor: Pfizer

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.

Detailed Description

Not available

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Study Start: 2022-02-10
• Study First Submitted: 2022-05-18
• Study First Submitted QC: 2022-06-02
• Study First Posted: 2022-06-07
• Primary Completion: 2024-06-24
• Study Completion: 2024-06-24
• Status Verified: 2024-07
• Last Update Submitted: 2024-07-12
• Last Update Posted: 2024-07-16

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 98

Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Control Group	Electronic Diary	SCD participants not on disease modifying treatment.
SCD Disease Modifying Treatment Group	Electronic Diary	SCD participants on a stable dose of a SCD disease modifying treatment regimen.

Primary Outcome Measures

Name	Time	Method
Average SCD ePRO daily worst tiredness score during VOC days	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	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	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	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	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	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	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	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	Day 1 to 180	Responsiveness of electronic patient reported outcomes between participants treated with SCD disease modifying treatment or no disease modifying treatment.

Secondary Outcome Measures

Name	Time	Method
Quantitative relationship between VOC Day rate and Physician-reported Medical Utilization VOC	Day 1 to 180	Quantitative measure of association between VOC Day rate and Physician-reported Medical Utilization VOC.
Quantitative relationship between VOC Day rate and Physician-reported Medical Utilization VOC rate across treatment groups	Day 1 to 180	Comparison of quantitative measures of treatment effect between VOC Day rate and Physician-reported Medical Utilization VOC rate.
Quantitative relationship between Patient-reported VOC Event rate and Physician-reported Medical Utilization VOC rate across treatment groups	Day 1 to 180	Comparison of quantitative measures of treatment effect between Patient-reported VOC Event rate and Physician-reported Medical Utilization VOC rate.
Quantitative relationship between Patient-reported VOC Event rate and Physician-reported Medical Utilization VOC rate	Day 1 to 180	Quantitative measure of association between Patient-reported VOC Event rate and Physician-reported Medical Utilization VOC rate.

Trial Locations

Locations (4)

Mid-Atlantic Permanente Medical Group Largo Medical Center; 🇺🇸 Upper Marlboro, Maryland, United States

Sanguine Biosciences, Inc.; 🇺🇸 Waltham, Massachusetts, United States

Cohen Children's Medical Center; 🇺🇸 New Hyde Park, New York, United States

Foundation for Sickle Cell Disease Research; 🇺🇸 Hollywood, Florida, United States