A Study to Learn About Sickle Cell Disease In Adult Patients

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Total Enrollment

98 participants

Study Classification

OBSERVATIONAL

Study Start Date

2022-02-10

Study Completion Date

2024-06-24

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

A Low-Interventional Study of an Electronic Sickle Cell Disease Patient Reported Outcomes in Sickle Cell Participants

NCT06503458

Sickle Cell Disease Sickle Cell Anemia

TERMINATED

Assessment of Algorithm-Based Hydroxyurea Dosing on Fetal Hemoglobin Response, Acute Complications, and Organ Function in People With Sickle Cell Disease

NCT02225132

Sickle Cell Disease

COMPLETED PHASE1/PHASE2

Hydroxyurea for the Treatment of Patients With Sickle Cell Anemia

NCT00001197

Sickle Cell Anemia

COMPLETED PHASE2

Risk Clinical Stratification of Sickle Cell Disease in Nigeria, Assessment of Efficacy/Safety of Hydroxyurea Treatment

NCT02149537

Sickle Cell Disease Sickle Cell Anemia

COMPLETED PHASE4

Hydroxyurea and Erythropoietin to Treat Sickle Cell Anemia

NCT00270478

Sickle Cell Disease Chronic Kidney Disease Pulmonary Hypertension

COMPLETED PHASE1

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Sickle Cell Disease

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Control Group

SCD participants not on disease modifying treatment.

Electronic Diary

Intervention Type OTHER

Participants will be asked to complete a daily electronic patient reported outcome diary entry to report on their experience in the past 24 hours.

SCD Disease Modifying Treatment Group

SCD participants on a stable dose of a SCD disease modifying treatment regimen.

Electronic Diary

Intervention Type OTHER

Participants will be asked to complete a daily electronic patient reported outcome diary entry to report on their experience in the past 24 hours.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Electronic Diary

Participants will be asked to complete a daily electronic patient reported outcome diary entry to report on their experience in the past 24 hours.

Intervention Type OTHER

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

SCD electronic patient reported outcome (ePRO)

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

\- Confirmed diagnosis of stable SCD (HbS/S or HbS/beta-zero-thalassemia).

* 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.

* 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 HU treatment, and in the absence of recent transfusion.

Exclusion Criteria

* Evidence or history of ongoing (condition or sequelae) clinically significant hematological (non-SCD), renal, endocrine, pulmonary, gastrointestinal, cardiovascular (including overt stroke but excluding silent cerebral infarct), hepatic (excluding cholelithiasis), psychiatric or neurological disease as assessed from medical records.
* Marked ongoing bone marrow suppression as evidenced by any of the following as per medical record: severe anemia, absolute neutrophil count (ANC) \<1000 mm3 white blood cell (WBC), thrombocytopenia (platelet count \<100,000 mm3) within ≤8 weeks prior to Day 1 enrollment.
* History of hematopoietic stem cell transplant or treatment with gene therapy as assessed from medical records.
* History of simple transfusion within ≤4 weeks prior to Day 1 enrollment as assessed from medical records or participant self-report.
* History of chronic transfusion/exchange transfusion within ≤12 weeks prior to Day 1 enrollment as assessed from medical records or participant self-report and/or plan to initiate such treatment during the 6-month observation period.

* Participant received HU and/or crizanlizumab at any time within ≤18 months of Day 1 enrollment and treatment(s) was discontinued due to lack of efficacy (no reduction in the frequency of VOCs, documented or perceived) and/or plan to initiate said treatment(s) during the 6-month observation period.
* Participant received voxelotor or L-glutamine within ≤4 weeks of Day 1 enrollment and/or plan to initiate said treatment(s) during the 6-month observation period.

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No