Use of Hydroxyurea and Magnesium Pidolate for Treatment of Sickle Cell Disease

April 24, 2017 updated by: St. Jude Children's Research Hospital

Phase I Study of Combination Treatment With Hydroxyurea and Magnesium Pidolate in Patients With Sickle Cell Disease

The purpose of this study is to estimate the MTD of Mg pidolate in combination with HU in patients with sickle cell disease who have been on a therapeutic dose (15-30 mg/kg/day) of HU for at least 6 months.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

This is a Phase I clinical trial evaluating the combination of hydroxyurea and magnesium pidolate for patients with sickle cell disease with either hemoglobin SS disease or hemoglobin S beta thalassemia. Hydroxyurea and magnesium pidolate will be tested in pediatric and adolescent patients with sickle cell disease who already have been treated with hydroxyurea for a minimum of six months. Magnesium pidolate will be given in combination with hydroxyurea for six months. In successive small groups of patients, the dose of magnesium will be increased in order to eventually determine the maximum tolerated dose (MTD) and dose limiting toxicity (DLT) for magnesium when given in combination with hydroxyurea. The maximum tolerated dose is the highest drug dose that can be given safely to participants. The dose limiting toxicity is determined when drug side effects prevent an increase in dose.

Study Type

Interventional

Enrollment (Actual)

16

Phase

  • Phase 1

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
    • Tennessee
      • Memphis, Tennessee, United States, 38105
        • St. Jude Children's Research Hospital

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

3 years to 15 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  1. Age > 3 years and < 15 years at the time of study enrollment
  2. Diagnosis of Hb SS or Hb S beta thalassemia
  3. Hydroxyurea treatment for at least 6 months prior to study entry at dose of 15 - 30 mg/kg/day
  4. Compliance with taking HU treatment of at least 70 % for 6 months prior to study entry

Exclusion Criteria:

  1. Red blood cell transfusion within the last 3 months resulting in a level of Hb A of 10% or more
  2. Pregnancy or unwillingness to use effective birth control in sexually active subjects (females who state that they are sexually active)
  3. Renal dysfunction defined by a serum creatinine greater than 1.5 times the upper limit of normal for age
  4. Liver dysfunction defined by an ALT greater than twice the upper limit of normal for age
  5. Concomitant usage of an "antisickling" agent other than hydroxyurea
  6. Current use of Mg containing drugs
  7. Iron deficiency, defined by serum ferritin ≤ 10 ng/ml
  8. Concomitant chronic illness other than sickle cell anemia

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
Other: 1
Drug: Magnesium Pidolate, Hydroxyurea
Intervention Description: Mg pidolate in combination with HU in patients with sickle cell disease who have been on a therapeutic dose (15-30 mg/kg/day) of HU for at least 6 months. Mg pidolate will be given at an initial dose of 0.6 mEq/kg/day divided into 2 daily doses for the first cohort of patients. This dose will be escalated for the subsequent patient cohorts as defined by a classic Phase I design (according to toxicity).

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
To estimate the maximum tolerated dose of magnesium pidolate in combination with hydroxyurea in patients with sickle cell disease who have been on a therapeutic dose of hydroxyurea for at least six months.
Time Frame: Every 2 weeks for first 8 weeks; then every 4 weeks
Every 2 weeks for first 8 weeks; then every 4 weeks

Secondary Outcome Measures

Outcome Measure
Time Frame
To document the toxicity of the combination of hydroxyurea and magnesium pidolate.
Time Frame: Every 2 weeks for first 8 weeks; then every 4 weeks
Every 2 weeks for first 8 weeks; then every 4 weeks
To investigate the effect of the combination of hydroxyurea and magnesium on hematological parameters and red cell metabolism.
Time Frame: 3 months, 6 months, and 9 months
3 months, 6 months, and 9 months

Collaborators and Investigators

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

Sponsor

St. Jude Children's Research Hospital

Investigators

  • Principal Investigator: Winfred Wang, MD, St. Jude Children's Research Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

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

November 1, 2004

Primary Completion (Actual)

January 1, 2008

Study Completion (Actual)

January 1, 2008

Study Registration Dates

First Submitted

September 1, 2005

First Submitted That Met QC Criteria

September 1, 2005

First Posted (Estimate)

September 2, 2005

Study Record Updates

Last Update Posted (Actual)

April 26, 2017

Last Update Submitted That Met QC Criteria

April 24, 2017

Last Verified

February 1, 2010

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • HUMG1

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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