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Hydroxyurea and Transfusion (HAT)

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ClinicalTrials.gov Identifier: NCT03644953
Recruitment Status : Recruiting
First Posted : August 23, 2018
Last Update Posted : November 15, 2018
Sponsor:
Information provided by (Responsible Party):
Robert Nickel, Children's 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.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Drug: Hydroxyurea Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Intervention Model: Single Group Assignment
Intervention Model Description: Prospective, single arm, open label study of combination hydroxyurea and simple chronic transfusion.
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Hydroxyurea and Transfusion: Pilot Study of Combination Therapy for Patients With Sickle Cell Anemia
Actual Study Start Date : October 30, 2018
Estimated Primary Completion Date : August 2020
Estimated Study Completion Date : August 2020


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.




Primary Outcome Measures :
  1. Recruitment ratio [ Time Frame: 1 year ]
    number of participants who enroll on the study / total number of eligible subjects

  2. Retention ratio [ Time Frame: 1 year ]
    number participants who remain on study 1 year after HAT target dose / total number enrolled participants

  3. Hydroxyurea adherence ratio [ Time Frame: 1 year ]
    (hydroxyurea amount dispensed - amount returned) / prescribed amount between visits


Secondary Outcome Measures :
  1. 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

  2. Volume of red blood cells transfused per patient weight [ Time Frame: 1 year ]
    measure to evaluate the transfusion requirement of HAT



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Ages Eligible for Study:   2 Years to 24 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03644953


Contacts
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Contact: Robert S Nickel, MD, MSc 202-476-3122 ext 3122 RNickel@childrensnational.org
Contact: Jennifer Webb, MD 202-476-2856 JWebb@childrensnational.org

Locations
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United States, District of Columbia
Children's National Health System Recruiting
Washington, District of Columbia, United States, 20010
Contact: Robert S Nickel, MD, MSc         
Sponsors and Collaborators
Children's Research Institute

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Responsible Party: Robert Nickel, Principal Investigator, Children's Research Institute
ClinicalTrials.gov Identifier: NCT03644953     History of Changes
Other Study ID Numbers: Pro00010541
First Posted: August 23, 2018    Key Record Dates
Last Update Posted: November 15, 2018
Last Verified: November 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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
Keywords provided by Robert Nickel, Children's Research Institute:
Hydroxyurea
Transfusion
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Hydroxyurea
Antineoplastic Agents
Antisickling Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Nucleic Acid Synthesis Inhibitors