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

NCT06503458 | Terminated

• 1 other identifier: C4071008
• Study Type: observational
• Target: 52
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to learn more about painful crisis in people with Sickle Cell Disease. For this reason, Pfizer is conducting a study to understand how people with Sickle Cell Disease feel on a daily basis, treat these painful episodes including if they treat themselves at home or go to a doctor's office/emergency room, as well as the types of medications that are taken during these episodes.

Conditions

Sickle Cell Disease; Sickle Cell Anemia

Keywords

Sickle Cell Disease; Sickle Cell Anemia; SCD

Outcome Measures

Primary Outcomes (2)

• Primary Objective #1:

  To assess whether the effect of HU on frequency of physician-reported MU VOC is observed in the study population, the annualized rate reduction in those on HU as compared to matched participants in control group will be evaluated. The null hypothesis is that the annualized physician-reported MU VOC rate in HU treatment group is not different from one in control group.

  6 months

• Primary Objective #2

  To assess whether the effect of HU on frequency of patient-reported crisis rates (VOC Day rate and Patient-reported VOC Event rate) calculated using SCD ePRO is observed in the study population, the annualized rates reduction in HU treatment group as compared to matched participants in control group will be evaluated. The corresponding null hypotheses are that the annualized patient-reported crisis rates in HU treatment group are not different from ones in control group.

  6 months

Secondary Outcomes (1)

• Association Between Patient-Reported Crisis Rates and Physician-Reported MU VOC rate

  6 months

Study Arms (2)

Control Group

1. Have experienced ≥1 episode(s) of MU VOC requiring documented unplanned medical intervention (eg, clinic, physician's office, emergency room or admission) requiring opioid or IV non-steroidal anti-inflammatory drug treatment within 12 months prior to Screening. 2. Participant has no history of past treatment with HU unless the reason for discontinuation was pregnancy (or pregnancy of a partner). 3. Data available for number of MU VOC(s) during the 12-month interval prior to Screening and a value for %HbF collected subsequent to 1 year of age in the absence of recent transfusion27(see Inclusion Criteria #2 - All Groups)

HU Treatment Group

1. Have experienced ≥1 episode(s) of MU VOC requiring documented unplanned medical intervention (eg, clinic, physician's office, emergency room or admission) requiring opioid or IV non-steroidal anti-inflammatory drug treatment within12 months prior to initiation of HU. 2. Must be on a stable dose of HU ≥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. 3. Data available for number of MU VOC(s) during the 12-month interval prior to initiation of HU treatment and a value for %HbF collected subsequent to 1 year of age, prior to initiation of HU treatment and in the absence of recent transfusion27(see Inclusion Criteria #2 - All Groups).

Eligibility Criteria

• Age: 18 Years - 100 Years
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Approximately 200 participants with SCD (HbS/S or HbS/β-thal) will be enrolled into two concurrentgroups, those who are either not on disease modifying treatment (control group\[Group 1\], approximately 150 participants) or on a stable dose of HU(HU treatment group\[Group 2\], approximately 50 participants), will be enrolled in this study.

You may qualify if:

• Have experienced ≥1 episode(s) of MU VOC requiring documented unplanned medical intervention (eg, clinic, physician's office, emergency room or admission) requiring opioid or IV non-steroidal anti-inflammatory drug treatment within 12 months prior to Screening.
• Participant has no history of past treatment with HU unless the reason for discontinuation was pregnancy (or pregnancy of a partner).
• Data available for number of MU VOC(s) during the 12-month interval prior to Screening and a value for %HbF collected subsequent to 1 year of age in the absence of recent transfusion.
• Have experienced ≥1 episode(s) of MU VOC requiring documented unplanned medical intervention (eg, clinic, physician's office, emergency room or admission) requiring opioid or IV non-steroidal anti-inflammatory drug treatment within12 months prior to initiation of HU.
• Must be on a stable dose of HU ≥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.
• Data available for number of MU VOC(s) during the 12-month interval prior to initiation of HU treatment and a value for %HbF collected subsequent to 1 year of age, prior to initiation of HU treatment and in the absence of recent transfusion.

You may not qualify if:

• Medical Conditions:
• Evidence or history of 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.
• Participants with any of the following acute or chronic infections or infection history as self-reported and/or assessed from medical records:
• Fever ≤7 days of Screening;
• Any infection requiring treatment with anti-infective drug(s) ≤2 weeks ofScreening;
• COVID-19 infection unless 10 days have elapsed since symptoms first appeared, participant is without symptoms for ≥24 hours and is not experiencing post-COVID-19 symptoms.
• Marked bone marrow suppression as evidenced by any of the following as per medical record: severe anemia, neutropenia (ANC \<1500 mm3WBC), thrombocytopenia (platelet count \<100,000 mm3) ≤8 weeks of Day 1.
• Major surgery \<3 months prior to Day 1 as self-reported and/or assessed from medical records or planned significant medical procedures during the study.
• Females who are pregnant or plan to become pregnant during the study.
• Other medical or psychiatric condition including cognitive impairment that prevents accurate reporting of pain and/or assessment of SCD symptoms, recent (within the past year) or active suicidal ideation/behavior, or laboratory abnormality that may increase the risk of study participation or, in the investigator's judgment, make the participant inappropriate for the study.
• Prior/Concomitant Therapy:
• History of hematopoietic stem cell transplant or treatment with gene therapy as assessed from medical records.
• History of simple transfusion ≤4 weeks of Day 1 as assessed from medical records.Prior/Concurrent Clinical Study Experience:
• Previous administration with an investigational drug within 30days (or as determined by the local requirement) or 5half-lives preceding Day 1 (whichever is longer).
• Active use of illicit drug as determined by the investigator.

Sponsors & Collaborators

• Sanguine Biosciences: lead
• Pfizer: collaborator

Study Sites (1)

Sanguine Biosciences

Woburn, Massachusetts, 01801, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell

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 Design

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

Study Record Dates

• First Submitted: February 8, 2022
• First Posted: July 16, 2024
• Study Start: January 19, 2022
• Primary Completion: December 23, 2023
• Study Completion: April 10, 2024
• Last Updated: July 16, 2024

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)