Sickle Cell AnemiaPhase 2NCT03020615
Hydroxyurea Management in Kids: Intensive Versus Stable Dosage Strategies
A Phase 2 study testing Hydroxyurea in people with Sickle Cell Anemia, run by St. Jude Children's Research Hospital at 4 sites in United States.
Trial summary
This is a pilot study, single-blind, randomized, multicenter, therapeutic clinical trial designed to evaluate the feasibility of enrolling infants and toddlers (9 months to 36 months) with sickle cell anemia (SCA; HbSS or HbSβ^0thalassemia), regardless of disease severity, to a therapeutic trial. Read more about this study
- Who is it for?
- Ages 9 to 36 months
- Conditions the study is for
- Sickle Cell Anemia
- What is being tested?
- Hydroxyurea
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 58 participants
- Where
- 4 sitesUnited States
- Study dates
- 12 May 2017to 8 Jun 2020
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 9 to 36 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Children with HbSS or sickle hemoglobin (HbS)/β^0thalassemia
- ≥9 to ≤ 36 months of age at study initiation
- Enrollment will occur irrespective of clinical severity
Who cannot join
- Permanent:
- Receiving chronic red blood cell transfusion therapy.
- Condition or chronic illness, which in the opinion of the PI makes participation unsafe.
- Transient (participants may be re-evaluated after ≥14 days):
- Recent (<30 days) participation in another clinical intervention trial utilizing an investigational new drug/investigational device exemption (IND/IDE) agent.
- Erythrocyte transfusion in the past 2 months.
Show all 13Show fewer
- Laboratory Assessments:
- Hemoglobin <6.0 g/dL
- Absolute reticulocyte count <80 * 10^3/µL if hemoglobin <9.0 mg/dL
- Absolute neutrophil count <1.5 * 10^3/µL
- Platelet count <100 * 10^3/µL
- Serum creatinine > twice the upper limit of normal for age
- Alanine aminotransferase (ALT) > twice the upper limit of normal
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Emory University/Children's Health Care of Atlanta
Atlanta, Georgia, 30322
University of Texas Southwestern Medical Center at Dallas
Dallas, Texas, 75390-9063
University of Mississippi Medical Center
Jackson, Mississippi, 39216
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
About this study
This is a pilot study, single-blind, randomized, multicenter, therapeutic clinical trial designed to evaluate the feasibility of enrolling infants and toddlers (9 months to 36 months) with sickle cell anemia (SCA; HbSS or HbSβ^0thalassemia), regardless of disease severity, to a therapeutic trial. A prior clinical trial at St. Jude Children's Research Hospital (SJCRH) (BABYHUG, NCT01783990) demonstrated that a fixed dose (20 mg/kg/day) of hydroxyurea was safe and effective in decreasing SCA-related complications in very young children (9-18 months), and largely due to these findings, hydroxyurea is recommended to be offered to all children (≥9 months old) with SCA, independent of disease severity. Nevertheless, children in the treatment arm of BABYHUG continued to experience vaso-occlusive symptoms and to incur organ damage. In clinical trials of older children with SCA, intensification of hydroxyurea to a maximum tolerated dosage (MTD), defined by mild to moderate myelosuppression, may be associated with improved laboratory parameters compared to fixed lower-dosing, but the clinical benefits gained from dose intensification have not been described. Therefore, in this trial, children in the standard treatment arm will receive a fixed dose of hydroxyurea (20 mg/kg/day), and participants in the experimental arm will receive hydroxyurea intensified to MTD, defined by a goal absolute neutrophil count (ANC) of 1500-3000 cells/µL. This trial aims to establish a multicenter infrastructure that will identify, enroll and randomize very young children (9-36 months) to receive fixed dose versus intensified-dose hydroxyurea in a single blinded manner, and to obtain prospective pilot data comparing the clinical and laboratory outcomes between the treatment arms to facilitate design of a definitive phase III trial.
Read the detailed description
All participants will initially receive hydroxyurea at a dose of ~20 mg/kg/day in an open label fashion for eight weeks (± 2 weeks) prior to randomization. Participants will receive monthly medical evaluations (every 4 ± 2 weeks) where they will have height and weight measurements, medical history, physical examination, and medication adherence assessments. During these monthly visits complete blood counts with absolute reticulocyte count will be monitored. Hemoglobin electrophoresis, complete serum chemistries, urinalysis, lactate dehydrogenase and quality of life measurements will be obtained every 20 (±2) weeks. Transcranial Doppler (TCD) ultrasound velocities will be obtained at study entry (in participants ≥2 years of age) and study exit. Participants randomized to receive hydroxyurea at MTD will have their dose increased by 5 mg/kg/day every 8 weeks, in the absence of toxicity, until a goal ANC of 1500-3000 cells/µL is achieved, up to a maximum of 35 mg/kg/day.
Both groups will receive their assigned treatment for 48 weeks (± 3 weeks). Participants will be in the study for a total of 56 weeks (± 3 weeks) and have 14 clinic visits to the St. Jude outpatient Hematology Clinic during that time. After the 56 weeks, participants will be followed for an additional 30 days for side effects and will then be taken off study.
- Conditions
- Sickle Cell Anemia
- Being tested
- Hydroxyurea
- Keywords
- Sickle cell, Hydroxyurea, Infants
- 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.
HydroxyureaDrug
Also called HU
Given orally once daily.
| Study group | Role | What they receive |
|---|---|---|
| Stable Dosing | Active comparator | In the first 8 weeks (± 2 weeks) of this study, participants will receive standard treatment [a fixed dose of 20 (± 2.5) mg/kg/day of hydroxyurea]. After 8 weeks (± 2 weeks) of standard treatment, participants will be randomized (like flipping a coin) to one of two treatment groups. Group 1 (Stable Dosing) continues standard treatment. Receives: Drug: Hydroxyurea |
| Intensive Dosing | Experimental | In the first 8 weeks (± 2 weeks) of this study, participants will receive standard treatment [a fixed dose of 20 (± 2.5) mg/kg/day of hydroxyurea]. After 8 weeks (± 2 weeks) of standard treatment, participants will be randomized (like flipping a coin) to one of two treatment groups. Group 2 (Intensive Dosing) will have their HU dose increased by 5 mg/kg/day every 8 weeks up to a maximum of 35 mg/kg/day. Receives: Drug: Hydroxyurea |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Patients Enrolled.
A count of the number of patients enrolled will be provided.
Measured: at baseline
Number of Patients Randomized
A count of the number of patients randomized will be provided.
Measured: Eight weeks (± 2 weeks) after study enrollment
Number of Randomized Patients With ≥80% Chronic Medication Compliance
Chronic medication compliance is defined based on medication possession ratio (MPR), a measure of the percentage of time that a patient has access to medication. Each participant's MPR is calculated as [(days medication in family's possession/days prescribed medication) * 100].
Measured: At completion of therapy, up to 56 weeks after study enrollment
Number of Patients Who Have the % Fetal Hemoglobin (%HbF) Collected at Baseline and at Study Exit
The number of patients who have successfully provided %HbF at baseline and study exit will be provided.
Measured: At baseline and at completion of the protocol, up to 56 weeks after study enrollment
Secondary outcomes (35)
Frequency by Reason Given for Refusal for Study Participation
Descriptive statistics of count and frequency will be provided for participants who were approached but refused to be enrolled on the study.
Measured: Once, at enrollment
Number of Patients With Hospitalizations by Arm
The number of patients with hospitalizations will be provided by arm. This analysis approach is different than what was written in the protocol due to small number of participants with hospitalizations and small number of hospitalization events.
Measured: From baseline through completion of therapy, up to 56 weeks
Cumulative Number of Hospitalizations by Arms
The total number of hospitalization events will be provided by arms. This analysis approach is different than what was written in the protocol due to small number of participants with hospitalizations and small number of hospitalization events.
Measured: From baseline through completion of therapy, up to 56 weeks
Mean Change in Hemoglobin (g/dL)
Descriptive statistics of the change between baseline and completion of the study will be provided and will be compared between two treatment arms using two sample t-test or exact Wilcoxon Rank Sum test depending on the normality of the data tested by the Shapiro-Wilk test.
Measured: From baseline at study entry to completion of therapy, up to 56 weeks
Median Change in Hemoglobin (g/dL)
Descriptive statistics of the change between baseline and completion of the study will be provided.
Measured: From baseline at study entry to completion of therapy, up to 56 weeks
Mean Change in Fetal Hemoglobin (%)
Descriptive statistics of the change between baseline and completion of the study will be provided.
Measured: From baseline at study entry to completion of therapy, up to 56 weeks
Median Change in Fetal Hemoglobin (%)
Descriptive statistics of the change between baseline and completion of the study will be provided and will be compared between two treatment arms using two sample t-test or exact Wilcoxon Rank Sum test depending on the normality of the data tested by the Shapiro-Wilk test.
Measured: From baseline at study entry to completion of therapy, up to 56 weeks
Mean Change in Mean Corpuscular Volume (fL)
Descriptive statistics of the change between baseline and completion of the study will be provided.
Measured: From baseline at study entry to completion of therapy, up to 56 weeks
Median Change in Mean Corpuscular Volume (fL)
Descriptive statistics of the change between baseline and completion of the study will be provided and will be compared between two treatment arms using two sample t-test or exact Wilcoxon Rank Sum test depending on the normality of the data tested by the Shapiro-Wilk test.
Measured: From baseline at study entry to completion of therapy, up to 56 weeks
Mean Change in Absolute Reticulocyte Count (*10^3 Reticulocytes/µL)
Descriptive statistics of the change between baseline and completion of the study will be provided and will be compared between two treatment arms using two sample t-test or exact Wilcoxon Rank Sum test depending on the normality of the data tested by the Shapiro-Wilk test.
Measured: From baseline at study entry to completion of therapy, up to 56 weeks
Median Change in Absolute Reticulocyte Count (*10^3 Reticulocytes/µL)
Descriptive statistics of the change between baseline and completion of the study will be provided.
Measured: From baseline at study entry to completion of therapy, up to 56 weeks
Mean Change in White Blood Cell Count (*10^3 White Blood Cells/µL)
Descriptive statistics of the change between baseline and completion of the study will be provided.
Measured: From baseline at study entry to completion of therapy, up to 56 weeks
Median Change in White Blood Cell Count (*10^3 White Blood Cells/µL)
Descriptive statistics of the change between baseline and completion of the study will be provided and will be compared between two treatment arms using two sample t-test or exact Wilcoxon Rank Sum test depending on the normality of the data tested by the Shapiro-Wilk test.
Measured: From baseline at study entry to completion of therapy, up to 56 weeks
Mean Change in Absolute Neutrophil Count (*10^3 Neutrophils/µL)
Descriptive statistics of the change between baseline and completion of the study will be provided.
Measured: From baseline at study entry to completion of therapy, up to 56 weeks
Median Change in Absolute Neutrophil Count (*10^3 Neutrophils/µL)
Descriptive statistics of the change between baseline and completion of the study will be provided and will be compared between two treatment arms using two sample t-test or exact Wilcoxon Rank Sum test depending on the normality of the data tested by the Shapiro-Wilk test.
Measured: From baseline at study entry to completion of therapy, up to 56 weeks
Mean Change in Platelet Count (*10^3 Platelets/µL)
Descriptive statistics of the change between baseline and completion of the study will be provided.
Measured: From baseline at study entry to completion of therapy, up to 56 weeks
Median Change in Platelet Count (*10^3 Platelets/µL)
Descriptive statistics of the change between baseline and completion of the study will be provided and will be compared between two treatment arms using two sample t-test or exact Wilcoxon Rank Sum test depending on the normality of the data tested by the Shapiro-Wilk test.
Measured: From baseline at study entry to completion of therapy, up to 56 weeks
Mean Change in Bilirubin (mg/dL)
Descriptive statistics of the change between baseline and completion of the study will be provided.
Measured: From baseline at study entry to completion of therapy, up to 56 weeks
Median Change in Bilirubin (mg/dL)
Descriptive statistics of the change between baseline and completion of the study will be provided and will be compared between two treatment arms using two sample t-test or exact Wilcoxon Rank Sum test depending on the normality of the data tested by the Shapiro-Wilk test.
Measured: From baseline at study entry to completion of therapy, up to 56 weeks
Mean Change in Lactate Dehydrogenase (Units/L)
Descriptive statistics of the change between baseline and completion of the study will be provided and will be compared between two treatment arms using two sample t-test or exact Wilcoxon Rank Sum test depending on the normality of the data tested by the Shapiro-Wilk test.
Measured: From baseline at study entry to completion of therapy, up to 56 weeks
Median Change in Lactate Dehydrogenase (Units/L)
Descriptive statistics of the change between baseline and completion of the study will be provided.
Measured: From baseline at study entry to completion of therapy, up to 56 weeks
Number of Participants Who do Not Have Normal Transcranial Doppler (TCD) Ultrasound Velocities
Normal TCD velocities will be defined as TCD velocities <170 cm/s.
Measured: From baseline at study entry to completion of therapy, up to 56 weeks
Number of Participants Who Undergo Surgery
Any operative procedure will be included.
Measured: From start of therapy through completion of therapy, up to 56 weeks
Number of Participants Who Undergo Transfusion
Transfusion will be defined as the provision of red blood cells to correct anemia.
Measured: From start of therapy through completion of therapy, up to 56 weeks
Number of Patients With Toxicities Related to Hydroxyurea Dosing
Number of patients with toxicities to include: neutropenia (ANC <1000*/µL), reticulocytopenia (ARC <80*10^3/µL and concomitant anemia (hemoglobin <6 g/dL), and thrombocytopenia (platelets <100*10^3/µL).
Measured: From start of therapy through completion of therapy, up to 56 weeks
Number of Toxicities Related to Hydroxyurea Dosing
Number of toxicities will be reported to include: neutropenia (ANC <1000*/µL), reticulocytopenia (ARC <80*10^3/µL and concomitant anemia (hemoglobin <6 g/dL), and thrombocytopenia (platelets <100*10^3/µL).
Measured: From start of therapy through completion of therapy, up to 56 weeks
Change in Pain and Hurt Score
Change in PedsQL 4.0 score will be reported. Scores are based on a 100 point scale, 0-100 with higher scores indicating a better quality of life. We are taking the exit visit score and subtracting the baseline score.
Measured: From baseline at study entry to completion of therapy, up to 56 weeks
Change in Pain Impact Score
Change in PedsQL 4.0 score will be reported. Scores are based on a 100 point scale, 0-100 with higher scores indicating a better quality of life. We are taking the exit visit score and subtracting the baseline score.
Measured: From baseline at study entry to completion of therapy, up to 56 weeks
Change in Pain Management Score
Change in PedsQL 4.0 score will be reported. Scores are based on a 100 point scale, 0-100 with higher scores indicating a better quality of life. We are taking the exit visit score and subtracting the baseline score.
Measured: From baseline at study entry to completion of therapy, up to 56 weeks
Change in Worry I Score
Change in PedsQL 4.0 score will be reported. Scores are based on a 100 point scale, 0-100 with higher scores indicating a better quality of life. We are taking the exit visit score and subtracting the baseline score.
Measured: From baseline at study entry to completion of therapy, up to 56 weeks
Change in Worry II Score
Change in PedsQL 4.0 score will be reported. Scores are based on a 100 point scale, 0-100 with higher scores indicating a better quality of life. We are taking the exit visit score and subtracting the baseline score.
Measured: From baseline at study entry to completion of therapy, up to 56 weeks
Change in Emotions Score
Change in PedsQL 4.0 score will be reported. Scores are based on a 100 point scale, 0-100 with higher scores indicating a better quality of life. We are taking the exit visit score and subtracting the baseline score.
Measured: From baseline at study entry to completion of therapy, up to 56 weeks
Change in Treatment Score
Change in PedsQL 4.0 score will be reported. Scores are based on a 100 point scale, 0-100 with higher scores indicating a better quality of life. We are taking the exit visit score and subtracting the baseline score.
Measured: From baseline at study entry to completion of therapy, up to 56 weeks
Change in Communication I Score
Change in PedsQL 4.0 score will be reported. Scores are based on a 100 point scale, 0-100 with higher scores indicating a better quality of life. We are taking the exit visit score and subtracting the baseline score.
Measured: From baseline at study entry to completion of therapy, up to 56 weeks
Change in Communication II Score
Change in PedsQL 4.0 score will be reported. Scores are based on a 100 point scale, 0-100 with higher scores indicating a better quality of life. We are taking the exit visit score and subtracting the baseline score.
Measured: From baseline at study entry to completion of therapy, up to 56 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jan 2017 (estimated)
- Study start12 May 2017
- Primary completion8 Jun 2020
- Study completion8 Jun 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Sickle Cell AnemiaStudies of the same condition, open and under way.
- More trials run by St. Jude Children's Research HospitalThe 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Investigator
- Enrolment
- 58 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Sponsor
- Investigators
- Jeremie Estepp, MD, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03020615
- Sponsor's protocol ID
- HUGKISS
- Other IDs
- R34HL127162
- Status last verified
- May 2021
- First submitted
- 11 Jan 2017
- Last updated
- 25 Jun 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Hydroxyurea Management in Kids: Intensive Versus Stable Dosage Strategies
- ClinicalTrials.gov ID
- NCT03020615
- Acronym / sponsor ID
- HUGKISS
- Conditions
- Sickle Cell Anemia
- Keywords
- Sickle cell, Hydroxyurea, Infants
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 58 participants (actual)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- 12 May 2017 (actual)
- Primary completion
- 8 Jun 2020 (actual)
- Study completion
- 8 Jun 2020 (actual)
- First posted
- 13 Jan 2017 (estimated)
- Results
- Posted 25 Jun 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a pilot study, single-blind, randomized, multicenter, therapeutic clinical trial designed to evaluate the feasibility of enrolling infants and toddlers (9 months to 36 months) with sickle cell anemia (SCA; HbSS or HbSβ^0thalassemia), regardless of disease severity, to a therapeutic trial. A prior clinical trial at St. Jude Children's Research Hospital (SJCRH) (BABYHUG, NCT01783990) demonstrated that a fixed dose (20 mg/kg/day) of hydroxyurea was safe and effective in decreasing SCA-related complications in very young children (9-18 months), and largely due to these findings, hydroxyurea is recommended to be offered to all children (≥9 months old) with SCA, independent of disease severity. Nevertheless, children in the treatment arm of BABYHUG continued to experience vaso-occlusive symptoms and to incur organ damage. In clinical trials of older children with SCA, intensification of hydroxyurea to a maximum tolerated dosage (MTD), defined by mild to moderate myelosuppression, may be associated with improved laboratory parameters compared to fixed lower-dosing, but the clinical benefits gained from dose intensification have not been described. Therefore, in this trial, children in the standard treatment arm will receive a fixed dose of hydroxyurea (20 mg/kg/day), and participants in the experimental arm will receive hydroxyurea intensified to MTD, defined by a goal absolute neutrophil count (ANC) of 1500-3000 cells/µL. This trial aims to establish a multicenter infrastructure that will identify, enroll and randomize very young children (9-36 months) to receive fixed dose versus intensified-dose hydroxyurea in a single blinded manner, and to obtain prospective pilot data comparing the clinical and laboratory outcomes between the treatment arms to facilitate design of a definitive phase III trial.
Detailed description
All participants will initially receive hydroxyurea at a dose of ~20 mg/kg/day in an open label fashion for eight weeks (± 2 weeks) prior to randomization. Participants will receive monthly medical evaluations (every 4 ± 2 weeks) where they will have height and weight measurements, medical history, physical examination, and medication adherence assessments. During these monthly visits complete blood counts with absolute reticulocyte count will be monitored. Hemoglobin electrophoresis, complete serum chemistries, urinalysis, lactate dehydrogenase and quality of life measurements will be obtained every 20 (±2) weeks. Transcranial Doppler (TCD) ultrasound velocities will be obtained at study entry (in participants ≥2 years of age) and study exit. Participants randomized to receive hydroxyurea at MTD will have their dose increased by 5 mg/kg/day every 8 weeks, in the absence of toxicity, until a goal ANC of 1500-3000 cells/µL is achieved, up to a maximum of 35 mg/kg/day.
Both groups will receive their assigned treatment for 48 weeks (± 3 weeks). Participants will be in the study for a total of 56 weeks (± 3 weeks) and have 14 clinic visits to the St. Jude outpatient Hematology Clinic during that time. After the 56 weeks, participants will be followed for an additional 30 days for side effects and will then be taken off study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Stable Dosing | Active comparator | Drug: HydroxyureaIn the first 8 weeks (± 2 weeks) of this study, participants will receive standard treatment [a fixed dose of 20 (± 2.5) mg/kg/day of hydroxyurea]. After 8 weeks (± 2 weeks) of standard treatment, participants will be randomized (like flipping a coin) to one of two treatment groups. Group 1 (Stable Dosing) continues standard treatment. |
| Intensive Dosing | Experimental | Drug: HydroxyureaIn the first 8 weeks (± 2 weeks) of this study, participants will receive standard treatment [a fixed dose of 20 (± 2.5) mg/kg/day of hydroxyurea]. After 8 weeks (± 2 weeks) of standard treatment, participants will be randomized (like flipping a coin) to one of two treatment groups. Group 2 (Intensive Dosing) will have their HU dose increased by 5 mg/kg/day every 8 weeks up to a maximum of 35 mg/kg/day. |
Who may qualify
- Ages
- 9 to 36 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Children with HbSS or sickle hemoglobin (HbS)/β^0thalassemia
- ≥9 to ≤ 36 months of age at study initiation
- Enrollment will occur irrespective of clinical severity
Key exclusion criteria (13 listed)
- Receiving chronic red blood cell transfusion therapy.
- Condition or chronic illness, which in the opinion of the PI makes participation unsafe.
- Recent (<30 days) participation in another clinical intervention trial utilizing an investigational new drug/investigational device exemption (IND/IDE) agent.
- Erythrocyte transfusion in the past 2 months.
- Laboratory Assessments:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| HydroxyureaAlso: HUGiven orally once daily. | Drug | Intensive Dosing, Stable Dosing |
Primary outcome
Number of Patients Enrolled.
Time frame: at baseline
A count of the number of patients enrolled will be provided.
Number of Patients Randomized
Time frame: Eight weeks (± 2 weeks) after study enrollment
A count of the number of patients randomized will be provided.
Number of Randomized Patients With ≥80% Chronic Medication Compliance
Time frame: At completion of therapy, up to 56 weeks after study enrollment
Chronic medication compliance is defined based on medication possession ratio (MPR), a measure of the percentage of time that a patient has access to medication. Each participant's MPR is calculated as [(days medication in family's possession/days prescribed medication) * 100].
Number of Patients Who Have the % Fetal Hemoglobin (%HbF) Collected at Baseline and at Study Exit
Time frame: At baseline and at completion of the protocol, up to 56 weeks after study enrollment
The number of patients who have successfully provided %HbF at baseline and study exit will be provided.
35 secondary outcomes
Frequency by Reason Given for Refusal for Study Participation
Time frame: Once, at enrollment
Number of Patients With Hospitalizations by Arm
Time frame: From baseline through completion of therapy, up to 56 weeks
Cumulative Number of Hospitalizations by Arms
Time frame: From baseline through completion of therapy, up to 56 weeks
Mean Change in Hemoglobin (g/dL)
Time frame: From baseline at study entry to completion of therapy, up to 56 weeks
Median Change in Hemoglobin (g/dL)
Time frame: From baseline at study entry to completion of therapy, up to 56 weeks
Mean Change in Fetal Hemoglobin (%)
Time frame: From baseline at study entry to completion of therapy, up to 56 weeks
Median Change in Fetal Hemoglobin (%)
Time frame: From baseline at study entry to completion of therapy, up to 56 weeks
Mean Change in Mean Corpuscular Volume (fL)
Time frame: From baseline at study entry to completion of therapy, up to 56 weeks
Median Change in Mean Corpuscular Volume (fL)
Time frame: From baseline at study entry to completion of therapy, up to 56 weeks
Mean Change in Absolute Reticulocyte Count (*10^3 Reticulocytes/µL)
Time frame: From baseline at study entry to completion of therapy, up to 56 weeks
Median Change in Absolute Reticulocyte Count (*10^3 Reticulocytes/µL)
Time frame: From baseline at study entry to completion of therapy, up to 56 weeks
Mean Change in White Blood Cell Count (*10^3 White Blood Cells/µL)
Time frame: From baseline at study entry to completion of therapy, up to 56 weeks
Median Change in White Blood Cell Count (*10^3 White Blood Cells/µL)
Time frame: From baseline at study entry to completion of therapy, up to 56 weeks
Mean Change in Absolute Neutrophil Count (*10^3 Neutrophils/µL)
Time frame: From baseline at study entry to completion of therapy, up to 56 weeks
Median Change in Absolute Neutrophil Count (*10^3 Neutrophils/µL)
Time frame: From baseline at study entry to completion of therapy, up to 56 weeks
Mean Change in Platelet Count (*10^3 Platelets/µL)
Time frame: From baseline at study entry to completion of therapy, up to 56 weeks
Median Change in Platelet Count (*10^3 Platelets/µL)
Time frame: From baseline at study entry to completion of therapy, up to 56 weeks
Mean Change in Bilirubin (mg/dL)
Time frame: From baseline at study entry to completion of therapy, up to 56 weeks
Median Change in Bilirubin (mg/dL)
Time frame: From baseline at study entry to completion of therapy, up to 56 weeks
Mean Change in Lactate Dehydrogenase (Units/L)
Time frame: From baseline at study entry to completion of therapy, up to 56 weeks
Median Change in Lactate Dehydrogenase (Units/L)
Time frame: From baseline at study entry to completion of therapy, up to 56 weeks
Number of Participants Who do Not Have Normal Transcranial Doppler (TCD) Ultrasound Velocities
Time frame: From baseline at study entry to completion of therapy, up to 56 weeks
Number of Participants Who Undergo Surgery
Time frame: From start of therapy through completion of therapy, up to 56 weeks
Number of Participants Who Undergo Transfusion
Time frame: From start of therapy through completion of therapy, up to 56 weeks
Number of Patients With Toxicities Related to Hydroxyurea Dosing
Time frame: From start of therapy through completion of therapy, up to 56 weeks
Number of Toxicities Related to Hydroxyurea Dosing
Time frame: From start of therapy through completion of therapy, up to 56 weeks
Change in Pain and Hurt Score
Time frame: From baseline at study entry to completion of therapy, up to 56 weeks
Change in Pain Impact Score
Time frame: From baseline at study entry to completion of therapy, up to 56 weeks
Change in Pain Management Score
Time frame: From baseline at study entry to completion of therapy, up to 56 weeks
Change in Worry I Score
Time frame: From baseline at study entry to completion of therapy, up to 56 weeks
Change in Worry II Score
Time frame: From baseline at study entry to completion of therapy, up to 56 weeks
Change in Emotions Score
Time frame: From baseline at study entry to completion of therapy, up to 56 weeks
Change in Treatment Score
Time frame: From baseline at study entry to completion of therapy, up to 56 weeks
Change in Communication I Score
Time frame: From baseline at study entry to completion of therapy, up to 56 weeks
Change in Communication II Score
Time frame: From baseline at study entry to completion of therapy, up to 56 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jun 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- St. Jude Children's Research HospitalLink provided by the sponsor.
- Clinical Trials Open at St. JudeLink provided by the sponsor.
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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.
Jeremie Estepp, MD principal investigator
St. Jude Children's Research Hospital
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Hydroxyurea in people with Sickle Cell Anemia, run by St. Jude Children's Research Hospital at 4 sites 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 3 inclusion and 13 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 25 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 25 Jun 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Patients Enrolled. (time frame: at baseline). The study lists 4 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 25 Jun 2021.