Sickle Cell DiseaseNCT02868138
Study of Hydroxyurea to Treat Sickle Cell Disease
An observational study of Sickle Cell Disease, run by Mersin University at 1 site in Turkey (Türkiye).
Trial summary
The aim of this single-center observational study was to evaluate quality of life, clinical effectiveness, and satisfaction in pediatric and young adult patients with sickle cell disease receiving hydroxyurea. Read more about this study
- Who is it for?
- Ages 7 to 22 years
- Conditions the study is for
- Sickle Cell Disease
- How many people
- 50 participants
- Where
- 1 siteTurkey (Türkiye)
- Study dates
- May 2016to Jul 2016
- 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 7 to 22 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Pediatric (7-17 years) and young adult (18-22 years) patients with sickle cell disease who received hydroxyurea
Who may be able to join
- Patients willing to participate in the study
- Pediatric and young adult patients
- Patients diagnosed with HbSS or HbSβ0 sickle cell disease
- Patients receiving hydroxyurea for at least a year.
Who cannot join
- Patients not meeting the inclusion criteria
- Patients not willing to participate in the study
- Patients diagnosed with other types of anemia except HbSS or HbSβ0 sickle cell disease
- Patients not receiving hydroxyurea for at least a year
- Patients having other conditions such as physical and/or mental difficulties which may affect their quality of life
- Patients having any contrindication against hydroxyurea
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Evaluation of Clinical Effectiveness, Quality of Life, and Compliance in Patients With Sickle Cell Disease Receiving Hydroxyurea
The aim of this single-center observational study was to evaluate quality of life, clinical effectiveness, and satisfaction in pediatric and young adult patients with sickle cell disease receiving hydroxyurea.
Read the detailed description
In this study, 34 pediatric (HbSS: n= 5; HbSβ0: n= 29) and 16 (HbSS: n=5; HbSβ0: n= 11) young adult adult patients with sickle cell disease receiving hydroxyurea for at least a year were participated. Upon receipt of Informed Consent Form, Case Report Form, Demographic Data Collection Form, Child Health Questionnaire-Parent Form, Life Quality Survey Short Form-36, and Hydroxyurea Therapy Satisfaction Survey were used to obtain data for effectiveness of hydroxyurea therapy and parameters that may affect compliance to treatment and life quality of the participants.
Regarding the normal ranges, ferritin, hemoglobin A, A2, F, and S, platelet, mean corpuscular volume, erythrocyte distribution width, basophil percentage, monocyte, monocyte percentage, total bilirubin, direct bilirubin, and C-reactive protein values were higher while hemoglobin, hematocrit, and erythrocyte values were lower in these patients. Our findings regarding quality of life and satisfaction with hydroxyurea therapy indicated that the patients with sickle cell disease had lower scores.
Demographic, clinical, and therapeutic variables as well as comorbid diseases and concomitant drug use when considered together, these findings suggest that the health quality and compliance of the pediatric and young adult patients to therapy might be low due to not sufficiently effective hydroxyurea therapy in addition to comorbidities, concomitant drug use, and side effects.
- Conditions
- Sickle Cell Disease
- Keywords
- Sickle cell disease, Hydroxyurea, Compliance
- Medical subject headings
- Anemia, Sickle Cell, Patient Compliance
What is being measured
The primary outcome is the main question the study is designed to answer.
Effectiveness and acceptance of hydroxyurea therapy in patients
Measured: Up to 12 weeks
Secondary outcomes (6)
Demographic characteristics of patients using Demographic Data Collection Form
Measured: Up to 12 weeks
Clinical characteristics of patients using Case Report Form
Measured: Up to 12 weeks
Health status of pediatric patients using Child Health Questionnaire-Parent Form
Measured: Up to 12 weeks
Quality of life of young adult patients using Life Quality Survey Short Form-36
Measured: Up to 12 weeks
Effectiveness of hydroxyurea therapy in patients using Case Report Form
Measured: Up to 12 weeks
Acceptance of hydroxyurea therapy in patients using Hydroxyurea Satisfaction Survey
Measured: Up to 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Aug 2016 (estimated)
- Study startMay 2016
- Primary completionJul 2016
- Study completionJul 2016
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.
- More trials run by Mersin UniversityThe sponsor's other registered studies.
- 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
- 50 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mersin University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Bahar TUNCTAN, Mersin University
- Investigators
- BAHAR TUNCTAN, Ph.D., Study chair, MERSIN UNIVERSITY FACULTY OF PHARMACY DEPARTMENT OF PHARMACOLOGY
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02868138
- Sponsor's protocol ID
- MEUKAEK-2016/27 - 16-AKD-19
- Status last verified
- Aug 2016
- First submitted
- 8 Aug 2016
- Last updated
- 16 Aug 2016
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 16 Aug 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of Clinical Effectiveness, Quality of Life, and Compliance in Patients With Sickle Cell Disease Receiving Hydroxyurea
- ClinicalTrials.gov ID
- NCT02868138
- Acronym / sponsor ID
- MEUKAEK-2016/27 - 16-AKD-19
- Conditions
- Sickle Cell Disease
- Keywords
- Sickle cell disease, Hydroxyurea, Compliance
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 50 participants (actual)
- Lead sponsor
- Mersin UniversityUniversity, hospital or other
- Study start
- May 2016
- Primary completion
- Jul 2016 (actual)
- Study completion
- Jul 2016 (actual)
- First posted
- 16 Aug 2016 (estimated)
- 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 aim of this single-center observational study was to evaluate quality of life, clinical effectiveness, and satisfaction in pediatric and young adult patients with sickle cell disease receiving hydroxyurea.
Detailed description
In this study, 34 pediatric (HbSS: n= 5; HbSβ0: n= 29) and 16 (HbSS: n=5; HbSβ0: n= 11) young adult adult patients with sickle cell disease receiving hydroxyurea for at least a year were participated. Upon receipt of Informed Consent Form, Case Report Form, Demographic Data Collection Form, Child Health Questionnaire-Parent Form, Life Quality Survey Short Form-36, and Hydroxyurea Therapy Satisfaction Survey were used to obtain data for effectiveness of hydroxyurea therapy and parameters that may affect compliance to treatment and life quality of the participants.
Regarding the normal ranges, ferritin, hemoglobin A, A2, F, and S, platelet, mean corpuscular volume, erythrocyte distribution width, basophil percentage, monocyte, monocyte percentage, total bilirubin, direct bilirubin, and C-reactive protein values were higher while hemoglobin, hematocrit, and erythrocyte values were lower in these patients. Our findings regarding quality of life and satisfaction with hydroxyurea therapy indicated that the patients with sickle cell disease had lower scores.
Demographic, clinical, and therapeutic variables as well as comorbid diseases and concomitant drug use when considered together, these findings suggest that the health quality and compliance of the pediatric and young adult patients to therapy might be low due to not sufficiently effective hydroxyurea therapy in addition to comorbidities, concomitant drug use, and side effects.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 7 to 22 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Pediatric (7-17 years) and young adult (18-22 years) patients with sickle cell disease who received hydroxyurea
Key inclusion criteria (4 listed)
- Patients willing to participate in the study
- Pediatric and young adult patients
- Patients diagnosed with HbSS or HbSβ0 sickle cell disease
- Patients receiving hydroxyurea for at least a year.
Key exclusion criteria (6 listed)
- Patients not meeting the inclusion criteria
- Patients not willing to participate in the study
- Patients diagnosed with other types of anemia except HbSS or HbSβ0 sickle cell disease
- Patients not receiving hydroxyurea for at least a year
- Patients having other conditions such as physical and/or mental difficulties which may affect their quality of life
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
Effectiveness and acceptance of hydroxyurea therapy in patients
Time frame: Up to 12 weeks
6 secondary outcomes
Demographic characteristics of patients using Demographic Data Collection Form
Time frame: Up to 12 weeks
Clinical characteristics of patients using Case Report Form
Time frame: Up to 12 weeks
Health status of pediatric patients using Child Health Questionnaire-Parent Form
Time frame: Up to 12 weeks
Quality of life of young adult patients using Life Quality Survey Short Form-36
Time frame: Up to 12 weeks
Effectiveness of hydroxyurea therapy in patients using Case Report Form
Time frame: Up to 12 weeks
Acceptance of hydroxyurea therapy in patients using Hydroxyurea Satisfaction Survey
Time frame: Up to 12 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Aug 2016.
- Individual participant dataSharing planned: Yes
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.
BAHAR TUNCTAN, Ph.D. study chair
MERSIN UNIVERSITY FACULTY OF PHARMACY DEPARTMENT OF PHARMACOLOGY
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, run by Mersin University at 1 site in Turkey (Türkiye).
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 4 inclusion and 6 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: finished, as of the sponsor's last update on 16 Aug 2016. 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 Turkey (Türkiye). 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Effectiveness and acceptance of hydroxyurea therapy in patients (time frame: Up to 12 weeks). 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 Aug 2016.