Hydroxyurea and Transfusion

Phase 2

Completed

• Conditions: Sickle Cell Disease
• Interventions: Drug: Hydroxyurea

• Registration Number: NCT03644953
• Lead Sponsor: Children's National Research Institute

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.

Study Timeline

• Study First Submitted: 2018-08-21
• Study First Submitted QC: 2018-08-22
• Study First Posted: 2018-08-23
• Study Start: 2018-10-30
• Primary Completion: 2021-07-01
• Study Completion: 2021-07-01
• Status Verified: 2021-10
• Last Update Submitted: 2021-10-19
• Last Update Posted: 2021-10-21

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 14

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Hydroxyurea and Transfusion (HAT)	Hydroxyurea	Combination hydroxyurea and simple chronic transfusion therapy

Primary Outcome Measures

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

Secondary Outcome Measures

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

Trial Locations

Locations (1)

Children's National Health System; 🇺🇸 Washington, District of Columbia, United States