Hydroxyurea and Transfusion

NCT ID: NCT03644953

Last Updated: 2021-10-21

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 14 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-10-30
• Study Completion Date: 2021-07-01

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.

Conditions

Sickle Cell Disease

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Hydroxyurea and Transfusion (HAT)

Combination hydroxyurea and simple chronic transfusion therapy

Group Type: EXPERIMENTAL

Hydroxyurea

Intervention Type: DRUG

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.

Interventions

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.

Intervention Type: DRUG

Eligibility Criteria

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.

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 24 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Children's National Research Institute

OTHER

Sponsor Role: lead

Responsible Party

Robert Nickel

Principal Investigator

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

Children's National Health System

Washington D.C., District of Columbia, United States

Countries

United States

Other Identifiers

Pro00010541

Identifier Type: -

Identifier Source: org_study_id