A Study to Learn About Sickle Cell Disease In Adult Patients

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

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 Overview

• Status: Completed
• Conditions: Sickle Cell Disease
• Intervention / Treatment: Other: Electronic Diary

• Study Type: Observational
• Enrollment (Actual): 98

Contacts and Locations

Study Locations

• United States
  • Florida
    • Hollywood, Florida, United States, 33024
      • Foundation for Sickle Cell Disease Research
  • Maryland
    • Largo, Maryland, United States, 20774
      • Mid-Atlantic Permanente Medical Group Largo Medical Center
    • Upper Marlboro, Maryland, United States, 20774
      • Mid-Atlantic Permanente Medical Group Largo Medical Center
  • Massachusetts
    • Waltham, Massachusetts, United States, 02451
      • Sanguine Biosciences, Inc.
  • New York
    • New Hyde Park, New York, United States, 11040
      • Cohen Children's Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Participants with a confirmed diagnosis of stable Sickle Cell Disease (SCD) (hemoglobin S inherited from both parents [HbS/S] or hemoglobin S inherited from one parent and hemoglobin beta thalassemia inherited from the other parent [HbS/beta-zero-thalassemia] genotype) who are either not on disease modifying treatment or on a stable dose of a SCD disease modifying treatment regimen.

Description

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.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Control Group; SCD participants not on disease modifying treatment.	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.; Other Names: SCD electronic patient reported outcome (ePRO)
SCD Disease Modifying Treatment Group; SCD participants on a stable dose of a SCD disease modifying treatment regimen.	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.; Other Names: SCD electronic patient reported outcome (ePRO)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
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
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
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
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
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
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
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
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
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
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
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

Collaborators and Investigators

• Sponsor: Pfizer
• Investigators: Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

Helpful Links

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

Study record dates

Study Major Dates

• Study Start (Actual): February 10, 2022
• Primary Completion (Actual): June 24, 2024
• Study Completion (Actual): June 24, 2024

Study Registration Dates

• First Submitted: May 18, 2022
• First Submitted That Met QC Criteria: June 2, 2022
• First Posted (Actual): June 7, 2022

Study Record Updates

• Last Update Posted (Actual): July 16, 2024
• Last Update Submitted That Met QC Criteria: July 12, 2024
• Last Verified: July 1, 2024

More Information

Terms related to this study

• Keywords: Vaso-occlusive crisis (VOC); Hemoglobin S (HbS)
• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Anemia, Sickle Cell
• Other Study ID Numbers: C4071008

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.