A Study to Learn About Sickle Cell Disease In Adult Patients

NCT05407805 | Completed

• 1 other identifier: C4071008
• Study Type: observational
• Target: 98
• Locations: 1 country
• Sites: 5

Brief 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.

Conditions

Sickle Cell Disease

Keywords

Vaso-occlusive crisis (VOC); Hemoglobin S (HbS)

Outcome Measures

Primary Outcomes (9)

• 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 Outcomes (4)

• Quantitative relationship 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

  Day 1 to 180

• Quantitative relationship 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

  Day 1 to 180

Study Arms (2)

Control Group

SCD participants not on disease modifying treatment.

Other: Electronic Diary

SCD Disease Modifying Treatment Group

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

Other: Electronic Diary

Interventions

Electronic Diary 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.

Also known as: SCD electronic patient reported outcome (ePRO)

Control Group; SCD Disease Modifying Treatment Group

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• \- 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Pfizer: lead

Study Sites (5)

Foundation for Sickle Cell Disease Research

Hollywood, Florida, 33024, United States

Location

Mid-Atlantic Permanente Medical Group Largo Medical Center

Largo, Maryland, 20774, United States

Location

Mid-Atlantic Permanente Medical Group Largo Medical Center

Upper Marlboro, Maryland, 20774, United States

Location

Sanguine Biosciences, Inc.

Waltham, Massachusetts, 02451, United States

Location

Cohen Children's Medical Center

New Hyde Park, New York, 11040, United States

Location

Related Links

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

MeSH Terms

Conditions

Anemia, Sickle Cell; Vaso-Occlusive Crises; Sickle Cell Trait

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 18, 2022
• First Posted: June 7, 2022
• Study Start: February 10, 2022
• Primary Completion: June 24, 2024
• Study Completion: June 24, 2024
• Last Updated: July 16, 2024

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (5)