Sickle Cell DiseaseNCT06503458
A Low-Interventional Study of an Electronic Sickle Cell Disease Patient Reported Outcomes in Sickle Cell Participants
An observational study of Sickle Cell Disease and Sickle Cell Anemia, run by Sanguine Biosciences at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Sickle Cell Disease, Sickle Cell Anemia
- Type of treatment
- Injection or infusion
- How many people
- 52 participants
- Where
- 1 siteUnited States
- Study dates
- 19 Jan 2022to 10 Apr 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
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.
Who may be able to join
- 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.
Who cannot join
- 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.
Show all 24Show fewer
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Sickle Cell Disease Patient Reported Outcomes in Adult Participants Aged ≥18 Years of Age On and Off Hydroxyurea
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.
Read the 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-intervention study to evaluate the responsiveness of the SCD ePRO in a therapeutic setting. Efficacy of drugs intended to reduce the frequency of VOC has historically been assessed based on frequency of VOC with an operational definition for VOC that requires MU, an endpoint approach that is limited in its utility for assessing benefit of therapeutic interventions across the totality of the disease experience. Thus, despite current available treatments, many patients with SCD still experience VOCs and there remains a significant unmet medical need and an opportunity to improve on existing endpoints. While previous studies have been conducted to consider this endpoint, they have not been conducted in a therapeutic setting and therefore unable to assess the responsiveness of theSCD ePRO.1,2This study aims to assess the responsiveness of the patient-reported endpoints in participants with SCD who are not on a disease modifying therapy versus those who are on the disease modifying treatment HU.
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia
- Keywords
- Sickle Cell Disease, Sickle Cell Anemia, SCD
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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). |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 6 months
Secondary outcomes (1)
Association Between Patient-Reported Crisis Rates and Physician-Reported MU VOC rate
Patient-reported crisis rates are determined by VOC Day rate and patient-reported VOC Event rate and is calculated using the SCD ePRO. These will be compared to physician-reported MU VOC rate to assess if there is an association between them. The assessment will be done in the matched sample, as well as in those in HU treatment group and in matched control group participants. The corresponding null hypotheses are that the annualized patient-reported crisis rates are not associated with physician-reported MU VOC.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jul 2024
- Study start19 Jan 2022
- Primary completion23 Dec 2023
- Study completion10 Apr 2024
Why it stopped: Low enrollment, cohorts were not able to be filled
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 52 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sanguine Biosciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Pfizer
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06503458
- Sponsor's protocol ID
- C4071008
- Status last verified
- Jul 2024
- First submitted
- 8 Feb 2022
- Last updated
- 16 Jul 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Sickle Cell Disease Patient Reported Outcomes in Adult Participants Aged ≥18 Years of Age On and Off Hydroxyurea
- ClinicalTrials.gov ID
- NCT06503458
- Acronym / sponsor ID
- C4071008
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia
- Keywords
- Sickle Cell Disease, Sickle Cell Anemia, SCD
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Low enrollment, cohorts were not able to be filled
- Enrolment
- 52 participants (actual)
- Lead sponsor
- Sanguine BiosciencesIndustry (pharma, biotech, devices)
- Collaborators
- Pfizer
- Study start
- 19 Jan 2022 (actual)
- Primary completion
- 23 Dec 2023 (actual)
- Study completion
- 10 Apr 2024 (actual)
- First posted
- 16 Jul 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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-intervention study to evaluate the responsiveness of the SCD ePRO in a therapeutic setting. Efficacy of drugs intended to reduce the frequency of VOC has historically been assessed based on frequency of VOC with an operational definition for VOC that requires MU, an endpoint approach that is limited in its utility for assessing benefit of therapeutic interventions across the totality of the disease experience. Thus, despite current available treatments, many patients with SCD still experience VOCs and there remains a significant unmet medical need and an opportunity to improve on existing endpoints. While previous studies have been conducted to consider this endpoint, they have not been conducted in a therapeutic setting and therefore unable to assess the responsiveness of theSCD ePRO.1,2This study aims to assess the responsiveness of the patient-reported endpoints in participants with SCD who are not on a disease modifying therapy versus those who are on the disease modifying treatment HU.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 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). |
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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.
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (24 listed)
- 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:
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Primary Objective #1:
Time frame: 6 months
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.
Primary Objective #2
Time frame: 6 months
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.
1 secondary outcome
Association Between Patient-Reported Crisis Rates and Physician-Reported MU VOC rate
Time frame: 6 months
Results and safety
The study was stopped early (reason given: Low enrollment, cohorts were not able to be filled). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jul 2024.
- Individual participant dataSharing planned: No
Related trials for Sickle Cell Disease
Active, not recruitingPhase 1/2
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat
Sickle Cell Disease, Hemolytic Anemia
RecruitingObservational study
HPC Offered for PRESERVE Expansion
Bone Marrow Transplant (BMT)
RecruitingPhase 1
Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
Sickle Cell Disorders, Sickle Cell Disease
RecruitingPhase 4
Clinical and Biomarker Effects of Depot Medroxyprogesterone Acetate in Females With Sickle Cell Disease
Sickle Cell Disease (SCD), Vaso-Occlusive Pain Episode in Sickle Cell Disease
Not yet recruitingPhase 1
Buprenorphine for the Management of Pain in Children With Sickle Cell Disease
Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Sickle Cell Disease and Sickle Cell Anemia, run by Sanguine Biosciences at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 24 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 16 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: Low enrollment, cohorts were not able to be filled). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Primary Objective #1: (time frame: 6 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jul 2024.