Back to resultsHydroxyurea and Transfusion (HAT)
Primary Purpose
Sickle Cell Disease
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Hydroxyurea
Sponsored by
About this trial
This is an interventional treatment trial for Sickle Cell Disease focused on measuring Hydroxyurea, Transfusion
Eligibility Criteria
Inclusion Criteria:
- Diagnosis of SCA (Hb SS or Sβ0 thalassemia).
- On simple chronic transfusion therapy for stroke prevention (primary or secondary prevention) for ≥1 year with no plans to stop simple chronic transfusion in the next year.
Exclusion Criteria:
- Poor adherence to simple transfusion regimen as defined by having an HbS >45% at any time in the last year AND a transfusion interval >5 weeks.
- Treatment with hydroxyurea in the 12 months prior to study enrollment.
Abnormal initial laboratory values (temporary exclusions):
- Absolute neutrophil count <1.5 x 10^9/L
- Platelet count <100 x 10^9/L
- Serum creatinine more than twice upper limit for age
- Pregnancy or unwillingness to use a medically acceptable form of contraception if sexually active.
Sites / Locations
- Children's National Health System
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Hydroxyurea and Transfusion (HAT)
Arm Description
Combination hydroxyurea and simple chronic transfusion therapy
Outcomes
Primary Outcome Measures
Recruitment ratio
number of participants who enroll on the study / total number of eligible subjects
Retention ratio
number participants who remain on study 1 year after HAT target dose / total number enrolled participants
Hydroxyurea adherence ratio
(hydroxyurea amount dispensed - amount returned) / prescribed amount between visits
Secondary Outcome Measures
Proportion of subjects who develop an hemoglobin (Hb) S >45% AND an Hb >11.0 g/dL
incidence of above safety event will be monitored closely throughout the trial
Volume of red blood cells transfused per patient weight
measure to evaluate the transfusion requirement of HAT
Full Information
NCT ID
NCT03644953
First Posted
August 21, 2018
Last Updated
October 19, 2021
Sponsor
Children's National Research Institute
1. Study Identification
Unique Protocol Identification Number
NCT03644953
Brief Title
Hydroxyurea and Transfusion
Acronym
HAT
Official Title
Hydroxyurea and Transfusion: Pilot Study of Combination Therapy for Patients With Sickle Cell Anemia
Study Type
Interventional
2. Study Status
Record Verification Date
October 2021
Overall Recruitment Status
Completed
Study Start Date
October 30, 2018 (Actual)
Primary Completion Date
July 1, 2021 (Actual)
Study Completion Date
July 1, 2021 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Children's National Research Institute
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
This study will prospectively investigate the feasibility, safety, and transfusion requirements of adding hydroxyurea to simple chronic transfusions for patients with sickle cell anemia already on chronic transfusions.
Detailed Description
This is a single-arm, prospective study of hydroxyurea added to simple chronic transfusions, combination treatment termed hydroxyurea and transfusion (HAT). The primary objective of the study is to determine the feasibility of HAT for patients with sickle cell anemia (SCA) currently being treated only with simple transfusions for stroke prevention. Secondary objectives include: to evaluate the safety of HAT and to determine if HAT decreases transfusion requirements in this patient population. Exploratory objectives include: to evaluate with HAT changes in pre-transfusion laboratories and biomarkers of cerebrovascular disease progression, and to describe changes on brain imaging.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sickle Cell Disease
Keywords
Hydroxyurea, Transfusion
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Model Description
Prospective, single arm, open label study of combination hydroxyurea and simple chronic transfusion.
Masking
None (Open Label)
Allocation
N/A
Enrollment
14 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Hydroxyurea and Transfusion (HAT)
Arm Type
Experimental
Arm Description
Combination hydroxyurea and simple chronic transfusion therapy
Intervention Type
Drug
Intervention Name(s)
Hydroxyurea
Intervention Description
Participants will be started on hydroxyurea 20 ± 2.5 mg/kg/day in addition to continuing simple chronic transfusion every 4 weeks ± 1 week. Hydroxyurea will be dose escalated to achieve a HAT target dose (HAT-TD). Hydroxyurea will be increased by 5 mg/kg/day after 8 weeks of a dose if HAT-TD is not achieved (maximum daily dose 2,000 mg). After HAT-TD has been achieved, minor dose increases may occur if subject grows a significant amount to maintain the same mg/kg dose.
Primary Outcome Measure Information:
Title
Recruitment ratio
Description
number of participants who enroll on the study / total number of eligible subjects
Time Frame
1 year
Title
Retention ratio
Description
number participants who remain on study 1 year after HAT target dose / total number enrolled participants
Time Frame
1 year
Title
Hydroxyurea adherence ratio
Description
(hydroxyurea amount dispensed - amount returned) / prescribed amount between visits
Time Frame
1 year
Secondary Outcome Measure Information:
Title
Proportion of subjects who develop an hemoglobin (Hb) S >45% AND an Hb >11.0 g/dL
Description
incidence of above safety event will be monitored closely throughout the trial
Time Frame
1 year
Title
Volume of red blood cells transfused per patient weight
Description
measure to evaluate the transfusion requirement of HAT
Time Frame
1 year
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
24 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Diagnosis of SCA (Hb SS or Sβ0 thalassemia).
On simple chronic transfusion therapy for stroke prevention (primary or secondary prevention) for ≥1 year with no plans to stop simple chronic transfusion in the next year.
Exclusion Criteria:
Poor adherence to simple transfusion regimen as defined by having an HbS >45% at any time in the last year AND a transfusion interval >5 weeks.
Treatment with hydroxyurea in the 12 months prior to study enrollment.
Abnormal initial laboratory values (temporary exclusions):
Absolute neutrophil count <1.5 x 10^9/L
Platelet count <100 x 10^9/L
Serum creatinine more than twice upper limit for age
Pregnancy or unwillingness to use a medically acceptable form of contraception if sexually active.
Facility Information:
Facility Name
Children's National Health System
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20010
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Hydroxyurea and Transfusion
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