Assessment of Algorithm-Based Hydroxyurea Dosing on Fetal Hemoglobin Response, Acute Complications, and Organ Function in People With Sickle Cell Disease
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|ClinicalTrials.gov Identifier: NCT02225132|
Recruitment Status : Completed
First Posted : August 26, 2014
Last Update Posted : August 9, 2018
- Sickle cell disease (SCD) is a blood disease. The drug hydroxyurea (HU) is approved to prevent pain crises in people with SCD. Researchers want to see how higher doses of HU affect the blood. This will help them learn about the right dosage of HU to give to people with SCD.
- To improve hydroxyurea dosing in people with SCD.
- People age 15 or older with homozygous SCD (HbSS).
- Participants will be screened with medical history, physical exam, medication review, and blood and urine tests.
- Participants will be in the study for about 15 months.
- First 3 months: monthly study visits with blood and urine tests.
- After 3 months: participants will take HU as a capsule by mouth. If you are already taking HU, your dose will be increased.
- Within a month of starting or increasing HU: participants will keep a daily pain diary for 2 weeks. They will have an echocardiogram (ultrasound) of the heart, a 6-minute walk test. They will complete a quality-of-life questionnaire.
- Participants will visit every month until they reach their highest tolerated dose of HU. They may need to come as often as every week sometimes to closely monitor their blood counts. Then they will alternate a phone call one month and a visit the next. At the visits, participants will bring their pill bottle, answer questions about side effects, and have blood tests.
- Every 2 months, participants will have a medical history, physical exam, and blood tests.
- Every 4 months, participants will have blood and urine tests. They will also complete another 2-week pain diary and quality-of-life questionnaire.
- About 12 months after starting or increasing HU, participants will have blood tests, an echocardiogram, and a 6-minute walk test.
|Condition or disease||Intervention/treatment||Phase|
|Sickle Cell Disease||Drug: Hydroxyurea||Early Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||15 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Assessment of Computerized Algorithm-Based Hydroxyurea Dosing on Fetal Hemoglobin Response, Acute Complications, and Organ Function in Subjects With Sickle Cell Disease|
|Study Start Date :||August 23, 2014|
|Actual Primary Completion Date :||May 24, 2018|
|Actual Study Completion Date :||May 24, 2018|
- Maximal HbF level achieved after initiating/continuing HU as compared to an average of the 3 HbF levels obtained during the run-in period. [ Time Frame: 15 months ]
- Number of hospitalizations for pain crises and acute chest syndrome as compared to the 1 year prior to HU initiation or dose increase. [ Time Frame: 12 months ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02225132
|United States, Maryland|
|National Institutes of Health Clinical Center, 9000 Rockville Pike|
|Bethesda, Maryland, United States, 20892|
|Principal Investigator:||Courtney D Fitzhugh, M.D.||National Heart, Lung, and Blood Institute (NHLBI)|