A Study to Learn About Sickle Cell Disease In Adult Patients (NCT05407805) | Clinical Trial Compass
CompletedNot Applicable
A Study to Learn About Sickle Cell Disease In Adult Patients
United States98 participantsStarted 2022-02-10
Plain-language 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.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria (All Groups):
\- Confirmed diagnosis of stable SCD (HbS/S or HbS/beta-zero-thalassemia).
Additional Inclusion Criteria (No Disease Modifying Treatment Control Group):
* Have experienced ≥1 episode(s) of medical utilization (MU) VOC within 12 months prior to Screening.
* Data available for number of MU VOC(s) during the 12-month interval prior to Screening and a value for %fetal hemoglobin (HbF) collected subsequent to 1 year of age in the absence of recent transfusion.
Additional Inclusion Criteria (SCD Disease Modifying Treatment Group):
* Have experienced ≥1 episode(s) of MU VOC within 12 months prior to initiation of HU and/or crizanlizumab (whichever was initiated earlier).
* Must be on a stable dose of their SCD treatment regimen ≥8 weeks prior to Day 1 with the intent of remaining on the same dose throughout the study, unless adjustments are medically necessary due to bone marrow suppression, in accordance with published guidelines and/or product specific guidance (eg, package label). Accepted SCD disease modifying treatment regimens include:
* HU alone and/or in combination with crizanlizumab, L-glutamine and/or voxelotor; or
* Crizanlizumab alone and/or in combination with HU, L-glutamine and/or voxelotor.
* Data available for number of MU VOC(s) during the 12-month interval prior to initiation of any SCD disease modifying treatment, as described above, and a value for %HbF collected subsequent to 1 year of age, prior to initiation of any …
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Physician-reported Medical Utilization (MU) Vaso-occlusive Crisis (VOC) Rate
Timeframe: Baseline up to Day 180
2
VOC Day Rate
Timeframe: Baseline up to Day 180
3
Patient-reported VOC Event Rate
Timeframe: Baseline up to Day 180
4
Sickle Cell Disease (SCD) Electronic Patient Reported Outcome (ePRO) Daily Worst Pain Scores by VOC Status
Timeframe: Baseline up to Day 180
5
Sickle Cell Disease Electronic Patient Reported Outcome Daily Worst Tiredness Scores by VOC Status
Timeframe: Baseline up to Day 180
6
Sickle Cell Disease Electronic Patient Reported Outcome Daily Rating for Ability to Perform Usual Physical Activity (UPA) by VOC Status