A Study to Learn About Sickle Cell Disease In Adult Patients

NCT05407805 | Completed Results

• 2 other identifiers: C4071008NCT05407805
• Study Type: observational
• Target: 98
• Locations: 1 country
• Sites: 4

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 (6)

• Physician-reported Medical Utilization (MU) Vaso-occlusive Crisis (VOC) Rate

  Physician-reported MU VOC: defined as an acute episode of pain with no other cause other than a VOC event that required a medical facility visit or contact with a health care professional and treatment with oral or parenteral narcotics, or non-steroidal anti-inflammatory drugs. Acute chest syndrome, hepatic sequestration, splenic sequestration, and priapism (requiring a visit to a medical facility) were also considered MU VOC. Contact with healthcare professional included: called healthcare provider (or telemedicine visit) and received treatment, went to clinic and received treatment, went to emergency department and received treatment, admitted to the hospital and received treatment. VOC rate was derived as annualized rate. Annualized MU VOC rate = (Number of MU VOC events \* 365)/ (number of days in the observation period).

  Baseline up to Day 180

• VOC Day Rate

  A VOC was a complication of SCD characterized by vaso-occlusion presenting as recurrent pain episodes. VOC day was a self-report by the participant of experiencing a VOC during the past 24 hours. This was assessed through a dichotomous (Yes/No) item on the SCD ePRO system, "Did you have a pain crisis in the past 24 hours?" A response of "Yes" indicated a VOC day. VOC day rate was derived as annualized rate. VOC day rate = (Number of VOC days \* 365)/ (number of days in the observation period).

  Baseline up to Day 180

• Patient-reported VOC Event Rate

  A VOC was a complication of SCD characterized by vaso-occlusion presenting as recurrent pain episodes. Patient reported VOC Event is used to define a sequence of VOC days that could also include single intervening days with no pain crisis. The subsequent occurrence of two consecutive days with no pain crisis operationally defines the end of the respective VOC event. Rate were derived as annualized rate. Annualized VOC event rate = (Number of VOC events \* 365)/ (number of days in the observation period).

  Baseline up to Day 180

• Sickle Cell Disease (SCD) Electronic Patient Reported Outcome (ePRO) Daily Worst Pain Scores by VOC Status

  A VOC was a complication of SCD characterized by vaso-occlusion presenting as recurrent pain episodes. VOC day was defined as the day on which a SCD participant self-reports sickle pain crisis that was recorded in the SCD ePRO. A non-VOC day was defined as the day on which a SCD participant does not self-reports sickle pain crisis. Participants rated their pain by selecting the one number that best described their pain at its worst in the past 24 hours from 0-10, where 0= no pain and 10= as bad as you can imagine. Higher scores indicated worse pain. Data in this outcome measure was presented separately for VOC state and non-VOC state.

  Baseline up to Day 180

• Sickle Cell Disease Electronic Patient Reported Outcome Daily Worst Tiredness Scores by VOC Status

  A VOC was a complication of SCD characterized by vaso-occlusion presenting as recurrent pain episodes. VOC day was defined as the day on which a SCD participant self-reports sickle pain crisis that was recorded in the SCD ePRO. A non-VOC day was defined as the day on which a SCD participant does not self-reports sickle pain crisis. Participants rated their tiredness by selecting the one number that best described their tiredness at its worst in the past 24 hours from 0-10, where 0= no tiredness and 10= as bad as you can imagine. Higher scores indicated worse tiredness. Data in this outcome measure was presented separately for VOC state and non-VOC state.

  Baseline up to Day 180

• Sickle Cell Disease Electronic Patient Reported Outcome Daily Rating for Ability to Perform Usual Physical Activity (UPA) by VOC Status

  A VOC was a complication of SCD characterized by vaso-occlusion presenting as recurrent pain episodes. VOC day was defined as the day on which a SCD participant self-reports sickle pain crisis that was recorded in the SCD ePRO. A non-VOC day was defined as the day on which a SCD participant does not self-reports sickle pain crisis. A measure of participant's ability to perform their UPA (e.g. walking, climbing stairs, or household chores) during a VOC event was assessed on SCD ePRO recorded by participants using a scale ranging from 1-4 scale, where 1= able to perform with no difficulty and 4= unable to perform usual physical activities, where higher score indicated worse status. Data in this outcome measure was presented separately for VOC state and non-VOC state.

  Baseline up to Day 180

Secondary Outcomes (2)

• Percent Change in Physician-reported MU VOC Rate Per Unit of Change in VOC Day Rate

  Baseline up to Day 180

• Percent Change in Physician-reported MU VOC Rate Per Unit of Change in Patient-reported VOC Event Rate

  Baseline up to Day 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 (4)

Foundation for Sickle Cell Disease Research

Hollywood, Florida, 33024, 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

Results Point of Contact

• Title: Pfizer ClinicalTrials.gov Call Center
• Organization: Pfizer Inc.

ClinicalTrials.gov_Inquiries@pfizer.com

1-800-718-1021

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

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: June 10, 2026
• Results First Posted: June 10, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)