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

This study has been completed.
Sponsor:
Information provided by:
St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier:
NCT00143572
First received: September 1, 2005
Last updated: February 23, 2010
Last verified: February 2010
  Purpose

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.


Condition Intervention Phase
Anemia, Sickle Cell
Drug: Magnesium Pidolate, Hydroxyurea
Phase 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Phase I Study of Combination Treatment With Hydroxyurea and Magnesium Pidolate in Patients With Sickle Cell Disease

Resource links provided by NLM:


Further study details as provided by St. Jude Children's Research Hospital:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • 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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]

Enrollment: 16
Study Start Date: November 2004
Study Completion Date: January 2008
Primary Completion Date: January 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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).

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.

  Eligibility

Ages Eligible for Study:   3 Years to 15 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00143572

Locations
United States, Tennessee
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Sponsors and Collaborators
St. Jude Children's Research Hospital
Investigators
Principal Investigator: Winfred Wang, MD St. Jude Children's Research Hospital
  More Information

Additional Information:
No publications provided

Responsible Party: Winfred Wang,MD / Principal Investigator, St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier: NCT00143572     History of Changes
Other Study ID Numbers: HUMG1
Study First Received: September 1, 2005
Last Updated: February 23, 2010
Health Authority: United States: Food and Drug Administration

Keywords provided by St. Jude Children's Research Hospital:
Hematologic Diseases
Anemia, Sickle Cell

Additional relevant MeSH terms:
Anemia
Anemia, Sickle Cell
Hematologic Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hemoglobinopathies
Genetic Diseases, Inborn
Hydroxyurea
Antineoplastic Agents
Therapeutic Uses
Pharmacologic Actions
Antisickling Agents
Hematologic Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Nucleic Acid Synthesis Inhibitors

ClinicalTrials.gov processed this record on August 28, 2014