Hydroxyurea and Transfusion

NCT03644953 | Completed Phase 2 DMC FDA Drug

• 1 other identifier: Pro00010541
• Study Type: interventional
• Target: 14
• Locations: 1 country
• Sites: 1

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.

Conditions

Sickle Cell Disease

Keywords

Hydroxyurea; Transfusion

Outcome Measures

Primary Outcomes (3)

• Recruitment ratio

  number of participants who enroll on the study / total number of eligible subjects

  1 year

• Retention ratio

  number participants who remain on study 1 year after HAT target dose / total number enrolled participants

  1 year

• Hydroxyurea adherence ratio

  (hydroxyurea amount dispensed - amount returned) / prescribed amount between visits

  1 year

Secondary Outcomes (2)

• Proportion of subjects who develop an hemoglobin (Hb) S >45% AND an Hb >11.0 g/dL

  1 year

• Volume of red blood cells transfused per patient weight

  1 year

Study Arms (1)

Hydroxyurea and Transfusion (HAT)

EXPERIMENTAL

Combination hydroxyurea and simple chronic transfusion therapy

Drug: Hydroxyurea

Interventions

Hydroxyurea 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.

Hydroxyurea and Transfusion (HAT)

Eligibility Criteria

• Age: 2 Years - 24 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Children's National Research Institute: lead

Study Sites (1)

Children's National Health System

Washington D.C., District of Columbia, 20010, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

Hydroxyurea

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Urea; Amides; Organic Chemicals

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Model Details: Prospective, single arm, open label study of combination hydroxyurea and simple chronic transfusion.

Study Record Dates

• First Submitted: August 21, 2018
• First Posted: August 23, 2018
• Study Start: October 30, 2018
• Primary Completion: July 1, 2021
• Study Completion: July 1, 2021
• Last Updated: October 21, 2021

Record last verified: 2021-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)