Status:
TERMINATED
A Low-Interventional Study of an Electronic Sickle Cell Disease Patient Reported Outcomes in Sickle Cell Participants
Lead Sponsor:
Sanguine Biosciences
Collaborating Sponsors:
Pfizer
Conditions:
Sickle Cell Disease
Sickle Cell Anemia
Eligibility:
All Genders
18-100 years
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...
Detailed Description
Pfizer is developing an ePRO that is completed on a daily basis using a diary to comprehensively self-report VOCs in participants with SCD and its impact on their lives.This is a prospective, low-inte...
Eligibility Criteria
Inclusion
- Inclusion Criteria - Control Group
- 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.
- Inclusion Criteria - HU Treatment Group
- 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.
- Exclusion Criteria:
- 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).
- Other Exclusions:
- Active use of illicit drug as determined by the investigator.
- A history or use of opioids will not be considered an exclusion if participant takes prescribed opioids for pain related to the underlying SCD.
- A history or use of cannabinoids is not exclusionary.
- History of alcohol abuse, dependence, or binge drinking within 6months of Screening as determined by the investigator.
- \- Binge drinking is defined as a pattern of 5 (male)and 4 (female) or more alcoholic drinks in about 2 hours. As a general rule, alcohol intake should not exceed 14 units per week (1unit = 8 ounces (240 mL) beer, 1 ounce (30mL) of 40% spirit or 3 ounces (90 mL) of wine).
- Unwilling or unable to comply with the criteria in the Lifestyle Considerations section (Section 5.3) of this protocol.
- Investigator site staff or Pfizer employees directly involved in the conduct of the study, site staff otherwise supervised by the investigator, and their respective family members.
- Lifestyle Considerations:
- Investigational drugs are not permitted during the study.
Exclusion
Key Trial Info
Start Date :
January 19 2022
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
April 10 2024
Estimated Enrollment :
52 Patients enrolled
Trial Details
Trial ID
NCT06503458
Start Date
January 19 2022
End Date
April 10 2024
Last Update
July 16 2024
Active Locations (1)
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1
Sanguine Biosciences
Woburn, Massachusetts, United States, 01801