Hydroxyurea and Magnesium Pidolate to Treat People With Hemoglobin Sickle Cell Disease
Primary Purpose
Hemoglobin SC Disease
Status
Terminated
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Hydroxyurea
Magnesium Pidolate
Placebo Pills and Placebo Liquid
Sponsored by
About this trial
This is an interventional treatment trial for Hemoglobin SC Disease focused on measuring Sickle Cell Disease, Vaso-occlusive Event, Painful Crises, Acute Chest Syndrome
Eligibility Criteria
Inclusion Criteria:
- Diagnosis of HbSC disease
- Hemoglobin level between 8 and 12.5 g/dL
- At least one vaso-occlusive event (e.g., pain, acute chest syndrome) in the 12 months prior to study entry. An episode of pain is defined as the occurrence of pain in the extremities, back, abdomen, chest, or head that lasts at least 2 hours; requires a visit to a hospital, emergency room, clinic, or provider's office; and is not explained except by SCD. Acute chest syndrome is defined as a new pulmonary infiltrate on a chest x-ray associated with a fever (greater than 38.5° C), tachypnea, wheezing, cough, or chest pain.
- Regular compliance with comprehensive care
- In a steady disease state and not experiencing an acute complication of SCD (i.e., no hospitalization, pain event, or episode of acute chest syndrome within the 1 month prior to study entry)
Exclusion Criteria:
- Previous transfusion with remaining hemoglobin A greater than 10%
- Previous treatment with hydroxyurea within the last 3 months
- Previous treatment with magnesium within the 3 months prior to study entry (including vitamins containing magnesium)
- Poor compliance with previous treatment regimens
- Liver dysfunction (SGPT greater than twice the upper limit of normal) within the 1 month prior to study entry
- Kidney dysfunction (creatinine greater than or equal to 1.0 mg/dL for participants less than 18 years of age; greater than or equal to 1.2 mg/dL for participants 18 years of age or older) within the 1 month prior to study entry
- Pregnant
- Ten or more hospital admissions for pain in the 12 months prior to study entry
- Daily use of narcotics
- Treatment with any investigational drug in the 3 months prior to study entry
- Less than 3% red blood cells with density greater than 41 g/dL (as measured by the ADVIA 120 system)
- Positive HIV test
- Other long-term illness or disorder other than SCD that could adversely affect performance in the study (e.g., tuberculosis)
Sites / Locations
- University of Alabama at Birmingham
- Children's Hospital and Research Center
- University of California Davis
- University of Colorado
- University of Miami
- Children's Healthcare of Atlanta
- University of Louisville
- Johns Hopkins University
- Boston Medical Center
- Children's Hospital Boston
- University of Mississippi Medical Center
- Montefiore Medical Center
- Duke University Medical Center
- Cincinnati Children's Hospital
- University of Oklahoma
- Saint Christopher's Hospital
- Children's Hospital of Philadelphia
- St. Jude Children's Research Hospital
- Children's Medical Center
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm Type
Placebo Comparator
Active Comparator
Active Comparator
Active Comparator
Arm Label
Placebo Pills and Placebo Liquid
Hydroxyurea Pills and Placebo Liquid
Placebo Pills and Magnesium Pidolate Liquid
Hydroxyurea Pills and Magnesium Pidolate Liquid
Arm Description
Outcomes
Primary Outcome Measures
Distribution of the Density of Hemoglobin SC Red Cells
An individuals' percentage of red blood cells with density greater than 41 g/dL as measured by Advia.
Secondary Outcome Measures
Full Information
NCT ID
NCT00532883
First Posted
September 20, 2007
Last Updated
January 14, 2013
Sponsor
St. Jude Children's Research Hospital
Collaborators
National Heart, Lung, and Blood Institute (NHLBI)
1. Study Identification
Unique Protocol Identification Number
NCT00532883
Brief Title
Hydroxyurea and Magnesium Pidolate to Treat People With Hemoglobin Sickle Cell Disease
Official Title
Effectiveness of Hydroxyurea and Magnesium Pidolate Alone and in Combination in Hemoglobin SC Disease: A Phase II Trial
Study Type
Interventional
2. Study Status
Record Verification Date
January 2010
Overall Recruitment Status
Terminated
Why Stopped
Enrollment has been terminated due to a slow rate of enrollment.
Study Start Date
January 2007 (undefined)
Primary Completion Date
March 2009 (Actual)
Study Completion Date
August 2009 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
St. Jude Children's Research Hospital
Collaborators
National Heart, Lung, and Blood Institute (NHLBI)
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited blood disease that can cause intense pain episodes. Hemoglobin SCD (HbSC) is a form of SCD that is characterized by dense red blood cells. The purpose of this study is to evaluate the safety and effectiveness of hydroxyurea and magnesium pidolate, alone and combined, at reducing red blood cell density and the frequency of pain episodes in people with HbSC.
Detailed Description
SCD is an inherited blood disorder. Symptoms include anemia, infections, organ damage, and intense episodes of pain, which are called "sickle cell crises." SCD is caused by an abnormal type of hemoglobin, which is a protein inside red blood cells that carries oxygen. HbSC is a form of SCD that is characterized by the presence of dense red blood cells. People with HbSC usually develop less severe SCD symptoms than people with the more common form of the disease. There are limited treatment approaches aimed specifically at modifying the abnormal state of red blood cells. Also, few combination therapy treatments have been studied. The medication hydroxyurea is currently used to prevent sickle cell crises and to decrease the need for blood transfusions. The dietary supplement magnesium has not been widely studied as a treatment for SCD, but it may prevent dehydration, which may decrease the frequency of sickle cell crises. The purpose of this study is to evaluate the safety and effectiveness of hydroxyurea and magnesium pidolate, alone and combined, at reducing red blood cell density and the frequency of sickle cell crises in people with HbSC.
This 1-year study will enroll people with HbSC. Participants will be randomly assigned to one of the following four treatment groups:
Group 1 participants will receive placebo pills and placebo liquid.
Group 2 participants will receive hydroxyurea pills and placebo liquid.
Group 3 participants will receive placebo pills and magnesium pidolate liquid.
Group 4 participants will receive hydroxyurea pills and magnesium pidolate liquid.
Participants will receive the hydroxyurea or placebo pills once a day and the magnesium pidolate or placebo liquid twice a day for 11 months. Study visits will occur every 2 weeks during the first 2 months of the study, once a month for the following 9 months, and then at Year 1. At each visit, a physical exam and blood collection will occur. Selected visits will also include urine collection and a pregnancy test for female participants. Throughout the study, participants will record their study medication use in a daily diary.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemoglobin SC Disease
Keywords
Sickle Cell Disease, Vaso-occlusive Event, Painful Crises, Acute Chest Syndrome
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
44 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Placebo Pills and Placebo Liquid
Arm Type
Placebo Comparator
Arm Title
Hydroxyurea Pills and Placebo Liquid
Arm Type
Active Comparator
Arm Title
Placebo Pills and Magnesium Pidolate Liquid
Arm Type
Active Comparator
Arm Title
Hydroxyurea Pills and Magnesium Pidolate Liquid
Arm Type
Active Comparator
Intervention Type
Drug
Intervention Name(s)
Hydroxyurea
Intervention Description
HU capsules (20 mg/kg/day for 11 months) Mg/Placebo liquid (0.6 mEq/kg/day for 11 months)
Intervention Type
Drug
Intervention Name(s)
Magnesium Pidolate
Intervention Description
HU/Placebo capsules (20 mg/kg/day for 11 months) Mg liquid (0.6 mEq/kg/day for 11 months)
Intervention Type
Other
Intervention Name(s)
Placebo Pills and Placebo Liquid
Intervention Description
HU/Placebo capsules (20 mg/kg/day for 11 months) Mg/Placebo liquid (0.6 mEq/kg/day for 11 months)
Primary Outcome Measure Information:
Title
Distribution of the Density of Hemoglobin SC Red Cells
Description
An individuals' percentage of red blood cells with density greater than 41 g/dL as measured by Advia.
Time Frame
measured 2 months after initiation of treatment
10. Eligibility
Sex
All
Minimum Age & Unit of Time
5 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Diagnosis of HbSC disease
Hemoglobin level between 8 and 12.5 g/dL
At least one vaso-occlusive event (e.g., pain, acute chest syndrome) in the 12 months prior to study entry. An episode of pain is defined as the occurrence of pain in the extremities, back, abdomen, chest, or head that lasts at least 2 hours; requires a visit to a hospital, emergency room, clinic, or provider's office; and is not explained except by SCD. Acute chest syndrome is defined as a new pulmonary infiltrate on a chest x-ray associated with a fever (greater than 38.5° C), tachypnea, wheezing, cough, or chest pain.
Regular compliance with comprehensive care
In a steady disease state and not experiencing an acute complication of SCD (i.e., no hospitalization, pain event, or episode of acute chest syndrome within the 1 month prior to study entry)
Exclusion Criteria:
Previous transfusion with remaining hemoglobin A greater than 10%
Previous treatment with hydroxyurea within the last 3 months
Previous treatment with magnesium within the 3 months prior to study entry (including vitamins containing magnesium)
Poor compliance with previous treatment regimens
Liver dysfunction (SGPT greater than twice the upper limit of normal) within the 1 month prior to study entry
Kidney dysfunction (creatinine greater than or equal to 1.0 mg/dL for participants less than 18 years of age; greater than or equal to 1.2 mg/dL for participants 18 years of age or older) within the 1 month prior to study entry
Pregnant
Ten or more hospital admissions for pain in the 12 months prior to study entry
Daily use of narcotics
Treatment with any investigational drug in the 3 months prior to study entry
Less than 3% red blood cells with density greater than 41 g/dL (as measured by the ADVIA 120 system)
Positive HIV test
Other long-term illness or disorder other than SCD that could adversely affect performance in the study (e.g., tuberculosis)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Winfred C. Wang, MD
Organizational Affiliation
St. Jude Children's Research Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Alabama at Birmingham
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35233
Country
United States
Facility Name
Children's Hospital and Research Center
City
Oakland
State/Province
California
ZIP/Postal Code
94609-1809
Country
United States
Facility Name
University of California Davis
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
University of Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
University of Miami
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Children's Healthcare of Atlanta
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30342-1600
Country
United States
Facility Name
University of Louisville
City
Louisville
State/Province
Kentucky
ZIP/Postal Code
40202
Country
United States
Facility Name
Johns Hopkins University
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Facility Name
Boston Medical Center
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02118
Country
United States
Facility Name
Children's Hospital Boston
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02118
Country
United States
Facility Name
University of Mississippi Medical Center
City
Jackson
State/Province
Mississippi
ZIP/Postal Code
39216
Country
United States
Facility Name
Montefiore Medical Center
City
Bronx
State/Province
New York
ZIP/Postal Code
10463
Country
United States
Facility Name
Duke University Medical Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Cincinnati Children's Hospital
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
University of Oklahoma
City
Oklahoma City
State/Province
Oklahoma
ZIP/Postal Code
73104
Country
United States
Facility Name
Saint Christopher's Hospital
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19134-1095
Country
United States
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19444
Country
United States
Facility Name
St. Jude Children's Research Hospital
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38105
Country
United States
Facility Name
Children's Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75235
Country
United States
12. IPD Sharing Statement
Citations:
PubMed Identifier
21275961
Citation
Wang W, Brugnara C, Snyder C, Wynn L, Rogers Z, Kalinyak K, Brown C, Qureshi A, Bigelow C, Neumayr L, Smith-Whitley K, Chui DH, Delahunty M, Woolson R, Steinberg M, Telen M, Kesler K. The effects of hydroxycarbamide and magnesium on haemoglobin SC disease: results of the multi-centre CHAMPS trial. Br J Haematol. 2011 Mar;152(6):771-6. doi: 10.1111/j.1365-2141.2010.08523.x. Epub 2011 Jan 31.
Results Reference
derived
Learn more about this trial
Hydroxyurea and Magnesium Pidolate to Treat People With Hemoglobin Sickle Cell Disease
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