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Completed

NCT Number: NCT00143572

Use of Hydroxyurea and Magnesium Pidolate for Treatment of 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.

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Key information

Conditions

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

Age range

3 year–15 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

Exclusion criteria

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

Treatment and study plan

Magnesium Pidolate, Hydroxyurea

Drug

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).

Primary outcomes

  1. 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

Secondary outcomes

  1. 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

  2. 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

Sponsors and collaborators

Lead sponsor

St. Jude Children's Research Hospital

Other

Registry information

Official study title

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

Important dates

Study start
2004
Primary completion
2008
Study completion
2008
First posted
Sep 2, 2005
Registry last updated
Apr 26, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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