Hydroxyurea and Transfusion (HAT)

October 19, 2021 updated by: Robert Nickel, Children's National Research Institute

Hydroxyurea and Transfusion: Pilot Study of Combination Therapy for Patients With Sickle Cell Anemia

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

14

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • District of Columbia
      • Washington, District of Columbia, United States, 20010
        • Children's National Health System

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 24 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  1. Diagnosis of SCA (Hb SS or Sβ0 thalassemia).
  2. 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:

  1. 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.
  2. Treatment with hydroxyurea in the 12 months prior to study enrollment.

  3. Abnormal initial laboratory values (temporary exclusions):

    1. Absolute neutrophil count <1.5 x 10^9/L
    2. Platelet count <100 x 10^9/L
    3. Serum creatinine more than twice upper limit for age
  4. Pregnancy or unwillingness to use a medically acceptable form of contraception if sexually active.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Hydroxyurea and Transfusion (HAT)
Combination hydroxyurea and simple chronic transfusion therapy
Drug: Hydroxyurea
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Recruitment ratio
Time Frame: 1 year
number of participants who enroll on the study / total number of eligible subjects
1 year
Retention ratio
Time Frame: 1 year
number participants who remain on study 1 year after HAT target dose / total number enrolled participants
1 year
Hydroxyurea adherence ratio
Time Frame: 1 year
(hydroxyurea amount dispensed - amount returned) / prescribed amount between visits
1 year

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Proportion of subjects who develop an hemoglobin (Hb) S >45% AND an Hb >11.0 g/dL
Time Frame: 1 year
incidence of above safety event will be monitored closely throughout the trial
1 year
Volume of red blood cells transfused per patient weight
Time Frame: 1 year
measure to evaluate the transfusion requirement of HAT
1 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Children's National Research Institute

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 30, 2018

Primary Completion (Actual)

July 1, 2021

Study Completion (Actual)

July 1, 2021

Study Registration Dates

First Submitted

August 21, 2018

First Submitted That Met QC Criteria

August 22, 2018

First Posted (Actual)

August 23, 2018

Study Record Updates

Last Update Posted (Actual)

October 21, 2021

Last Update Submitted That Met QC Criteria

October 19, 2021

Last Verified

October 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • Pro00010541

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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