Back to resultsUse of Hydroxyurea and Magnesium Pidolate for Treatment of Sickle Cell Disease
Primary Purpose
Anemia, Sickle Cell
Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Magnesium Pidolate, Hydroxyurea
Sponsored by
About this trial
This is an interventional treatment trial for Anemia, Sickle Cell focused on measuring Hematologic Diseases, Anemia, Sickle Cell
Eligibility Criteria
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
Sites / Locations
- St. Jude Children's Research Hospital
Arms of the Study
Arm 1
Arm Type
Other
Arm Label
1
Arm Description
Outcomes
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.
Secondary Outcome Measures
To document the toxicity of the combination of hydroxyurea and magnesium pidolate.
To investigate the effect of the combination of hydroxyurea and magnesium on hematological parameters and red cell metabolism.
Full Information
NCT ID
NCT00143572
First Posted
September 1, 2005
Last Updated
April 24, 2017
Sponsor
St. Jude Children's Research Hospital
1. Study Identification
Unique Protocol Identification Number
NCT00143572
Brief Title
Use of Hydroxyurea and Magnesium Pidolate for Treatment of Sickle Cell Disease
Official Title
Phase I Study of Combination Treatment With Hydroxyurea and Magnesium Pidolate in Patients With Sickle Cell Disease
Study Type
Interventional
2. Study Status
Record Verification Date
February 2010
Overall Recruitment Status
Completed
Study Start Date
November 2004 (undefined)
Primary Completion Date
January 2008 (Actual)
Study Completion Date
January 2008 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
St. Jude Children's Research Hospital
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Anemia, Sickle Cell
Keywords
Hematologic Diseases, Anemia, Sickle Cell
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
16 (Actual)
8. Arms, Groups, and Interventions
Arm Title
1
Arm Type
Other
Intervention Type
Drug
Intervention Name(s)
Magnesium Pidolate, Hydroxyurea
Intervention Description
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 Outcome Measure Information:
Title
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 Outcome Measure Information:
Title
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
Title
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
10. Eligibility
Sex
All
Minimum Age & Unit of Time
3 Years
Maximum Age & Unit of Time
15 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Winfred Wang, MD
Organizational Affiliation
St. Jude Children's Research Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
St. Jude Children's Research Hospital
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38105
Country
United States
12. IPD Sharing Statement
Links:
URL
http://www.stjude.org
Description
St. Jude Children's Research Hospital
Learn more about this trial
Use of Hydroxyurea and Magnesium Pidolate for Treatment of Sickle Cell Disease
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