At a glance
ClinicalIndex Comparison Record- ✓Confirmed diagnosis of stable sickle cell disease (HbS/S or HbS/beta-zero-thalassemia)
- ✓Control group: ≥1 medically-utilized VOC episode within 12 months prior to screening
- ✓Disease-modifying treatment group: ≥1 medically-utilized VOC episode within 12 months prior to HU and/or crizanlizumab initiation
- ✓Disease-modifying treatment group: stable dose ≥8 weeks prior to Day 1, intending to remain on same dose unless medically necessary adjustments needed
- ✕Clinically significant hematological (non-SCD), renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, psychiatric, or neurological disease
- ✕Marked bone marrow suppression: ANC <1000/mm³, WBC <1000/mm³, or platelets <100,000/mm³ within ≤8 weeks of Day 1
- ✕Prior hematopoietic stem cell transplant or gene therapy
- ✕Simple transfusion within ≤4 weeks prior to Day 1
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
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
In Brief
An observational study evaluating Electronic Diary for Sickle Cell Disease. Completed, enrolled 98 participants across 4 sites.
Detailed 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.
Study Details
Timeline
Arms & Interventions
SCD participants not on disease modifying treatment.
SCD participants on a stable dose of a SCD disease modifying treatment regimen.
Interventions
Participants will be asked to complete a daily electronic patient reported outcome diary entry to report on their experience in the past 24 hours.