A Study to Learn About Sickle Cell Disease In Adult Patients

Sickle Cell Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05407805
• Other study ID #: C4071008
• Status: Recruiting
• Start date: February 10, 2022
• Est. completion date: June 22, 2024

Study information

• Verified date: December 2023
• Source: Pfizer
• Contact: Pfizer CT.gov Call Center
• Phone: 1-800-718-1021
• Email: ClinicalTrials.gov_Inquiries[@]pfizer.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 150
• Est. completion date: June 22, 2024
• Est. primary completion date: June 22, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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 HU treatment, and in the absence of recent transfusion.

Exclusion Criteria (All Groups):

• 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.

Additional Exclusion Criteria (No Disease Modifying Treatment Control Group):

• 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.

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

Intervention

Other:
• 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.

Locations

Country	Name	City	State
United States	Foundation for Sickle Cell Disease Research	Hollywood	Florida
United States	Mid-Atlantic Permanente Medical Group Largo Medical Center	Largo	Maryland
United States	Cohen Children's Medical Center	New Hyde Park	New York
United States	Mid-Atlantic Permanente Medical Group Largo Medical Center	Upper Marlboro	Maryland
United States	Sanguine Biosciences, Inc.	Waltham	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Physician-reported Medical Utilization vaso-occlusive crisis (VOC) rate	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.	Day 1 to 180
Primary	VOC Day rate	Responsiveness of electronic patient reported outcomes between participants treated with SCD disease modifying treatment or no disease modifying treatment.	Day 1 to 180
Primary	Patient-reported VOC Event rate	Responsiveness of electronic patient reported outcomes between participants treated with SCD disease modifying treatment or no disease modifying treatment.	Day 1 to 180
Primary	Average SCD ePRO daily worst pain score during VOC days	Responsiveness of electronic patient reported outcomes between participants treated with SCD disease modifying treatment or no disease modifying treatment.	Day 1 to 180
Primary	Average SCD ePRO daily worst pain score during non-VOC days	Responsiveness of electronic patient reported outcomes between participants treated with SCD disease modifying treatment or no disease modifying treatment.	Day 1 to 180
Primary	Average SCD ePRO daily worst tiredness score during VOC days	Responsiveness of electronic patient reported outcomes between participants treated with SCD disease modifying treatment or no disease modifying treatment.	Day 1 to 180
Primary	Average SCD ePRO daily worst tiredness score during non-VOC days	Responsiveness of electronic patient reported outcomes between participants treated with SCD disease modifying treatment or no disease modifying treatment.	Day 1 to 180
Primary	Average SCD ePRO daily rating for ability to perform usual physical activity during a VOC day	Responsiveness of electronic patient reported outcomes between participants treated with SCD disease modifying treatment or no disease modifying treatment.	Day 1 to 180
Primary	Average SCD ePRO daily rating for ability to perform usual physical activity during a non-VOC day	Responsiveness of electronic patient reported outcomes between participants treated with SCD disease modifying treatment or no disease modifying treatment.	Day 1 to 180
Secondary	Quantitative relationship between VOC Day rate and Physician-reported Medical Utilization VOC	Quantitative measure of association between VOC Day rate and Physician-reported Medical Utilization VOC.	Day 1 to 180
Secondary	Quantitative relationship between VOC Day rate and Physician-reported Medical Utilization VOC rate across treatment groups	Comparison of quantitative measures of treatment effect between VOC Day rate and Physician-reported Medical Utilization VOC rate.	Day 1 to 180
Secondary	Quantitative relationship between Patient-reported VOC Event rate and Physician-reported Medical Utilization VOC rate	Quantitative measure of association between Patient-reported VOC Event rate and Physician-reported Medical Utilization VOC rate.	Day 1 to 180
Secondary	Quantitative relationship between Patient-reported VOC Event rate and Physician-reported Medical Utilization VOC rate across treatment groups	Comparison of quantitative measures of treatment effect between Patient-reported VOC Event rate and Physician-reported Medical Utilization VOC rate.	Day 1 to 180

External Links

•   To obtain contact information for a study center near you, click here.