Hemoglobin SC DiseasePhase 2NCT00532883
Hydroxyurea and Magnesium Pidolate to Treat People With Hemoglobin Sickle Cell Disease
A Phase 2 study testing Hydroxyurea and Magnesium Pidolate in people with Hemoglobin SC Disease, run by St. Jude Children's Research Hospital at 19 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 5 years and older
- Conditions the study is for
- Hemoglobin SC Disease
- What is being tested?
- Hydroxyurea and Magnesium Pidolate
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 44 participants
- Where
- 19 sitesUnited States
- Study dates
- Jan 2007to Aug 2009
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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)
Who cannot join
- 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
Show all 13Show fewer
- 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)
Trial locations and contacts
19 sites in 1 country.
19 sites in 1 country
United States19 sites
Children's Healthcare of Atlanta
Atlanta, Georgia, 30342-1600
University of Colorado
Aurora, Colorado, 80045
Johns Hopkins University
Baltimore, Maryland, 21205
University of Alabama at Birmingham
Birmingham, Alabama, 35233
Boston Medical Center
Boston, Massachusetts, 02118
Children's Hospital Boston
Boston, Massachusetts, 02118
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229
Children's Medical Center
Dallas, Texas, 75235
Duke University Medical Center
Durham, North Carolina, 27710
University of Mississippi Medical Center
Jackson, Mississippi, 39216
University of Louisville
Louisville, Kentucky, 40202
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
University of Miami
Miami, Florida, 33136
Children's Hospital and Research Center
Oakland, California, 94609-1809
University of Oklahoma
Oklahoma City, Oklahoma, 73104
Saint Christopher's Hospital
Philadelphia, Pennsylvania, 19134-1095
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19444
University of California Davis
Sacramento, California, 95817
Montefiore Medical Center
The Bronx, New York, 10463
About this study
Official title: Effectiveness of Hydroxyurea and Magnesium Pidolate Alone and in Combination in Hemoglobin SC Disease: A Phase II Trial
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.
Read the 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.
- Conditions
- Hemoglobin SC Disease
- Being tested
- Hydroxyurea, Magnesium Pidolate, Placebo Pills and Placebo Liquid
- Keywords
- Sickle Cell Disease, Vaso-occlusive Event, Painful Crises, Acute Chest Syndrome
- Medical subject headings
- Hemoglobin SC Disease, Anemia, Sickle Cell, Vaso-Occlusive Crises, Acute Chest Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HydroxyureaDrug
HU capsules (20 mg/kg/day for 11 months) Mg/Placebo liquid (0.6 mEq/kg/day for 11 months)
Magnesium PidolateDrug
HU/Placebo capsules (20 mg/kg/day for 11 months) Mg liquid (0.6 mEq/kg/day for 11 months)
Placebo Pills and Placebo LiquidOther
HU/Placebo capsules (20 mg/kg/day for 11 months) Mg/Placebo liquid (0.6 mEq/kg/day for 11 months)
| Study group | Role | What they receive |
|---|---|---|
| Placebo Pills and Placebo Liquid | Placebo comparator | Not described Receives: Other: Placebo Pills and Placebo Liquid |
| Hydroxyurea Pills and Placebo Liquid | Active comparator | Not described Receives: Drug: Hydroxyurea |
| Placebo Pills and Magnesium Pidolate Liquid | Active comparator | Not described Receives: Drug: Magnesium Pidolate |
| Hydroxyurea Pills and Magnesium Pidolate Liquid | Active comparator | Not described Receives: Drug: Hydroxyurea; Drug: Magnesium Pidolate |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: measured 2 months after initiation of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Sep 2007 (estimated)
- Study startJan 2007
- Primary completionMar 2009
- Study completionAug 2009
Why it stopped: Enrollment has been terminated due to a slow rate of enrollment.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hemoglobin SC DiseaseStudies of the same condition, open and under way.
- More trials run by St. Jude Children's Research HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 44 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Sponsor
- Investigators
- Winfred C. Wang, MD, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00532883
- Sponsor's protocol ID
- CHAMPS-St. Jude
- Other IDs
- U54HL070587
- Status last verified
- Jan 2010
- First submitted
- 20 Sep 2007
- Last updated
- 18 Jan 2013
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
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.
PubMed 21275961
Study at a glance
From the registry record, last updated by the sponsor on 18 Jan 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effectiveness of Hydroxyurea and Magnesium Pidolate Alone and in Combination in Hemoglobin SC Disease: A Phase II Trial
- ClinicalTrials.gov ID
- NCT00532883
- Acronym / sponsor ID
- CHAMPS-St. Jude
- Conditions
- Hemoglobin SC Disease
- Keywords
- Sickle Cell Disease, Vaso-occlusive Event, Painful Crises, Acute Chest Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Enrollment has been terminated due to a slow rate of enrollment.
- Enrolment
- 44 participants (actual)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- Jan 2007
- Primary completion
- Mar 2009 (actual)
- Study completion
- Aug 2009 (actual)
- First posted
- 21 Sep 2007 (estimated)
- Results
- Posted 11 May 2010
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Placebo Pills and Placebo Liquid | Placebo comparator | Other: Placebo Pills and Placebo Liquid |
| Hydroxyurea Pills and Placebo Liquid | Active comparator | Drug: Hydroxyurea |
| Placebo Pills and Magnesium Pidolate Liquid | Active comparator | Drug: Magnesium Pidolate |
| Hydroxyurea Pills and Magnesium Pidolate Liquid | Active comparator | Drug: Hydroxyurea, Drug: Magnesium Pidolate |
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- 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)
Key exclusion criteria (13 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| HydroxyureaHU capsules (20 mg/kg/day for 11 months) Mg/Placebo liquid (0.6 mEq/kg/day for 11 months) | Drug | Hydroxyurea Pills and Magnesium Pidolate Liquid, Hydroxyurea Pills and Placebo Liquid |
| Magnesium PidolateHU/Placebo capsules (20 mg/kg/day for 11 months) Mg liquid (0.6 mEq/kg/day for 11 months) | Drug | Hydroxyurea Pills and Magnesium Pidolate Liquid, Placebo Pills and Magnesium Pidolate Liquid |
| Placebo Pills and Placebo LiquidHU/Placebo capsules (20 mg/kg/day for 11 months) Mg/Placebo liquid (0.6 mEq/kg/day for 11 months) | Other | Placebo Pills and Placebo Liquid |
Primary outcome
Distribution of the Density of Hemoglobin SC Red Cells
Time frame: measured 2 months after initiation of treatment
An individuals' percentage of red blood cells with density greater than 41 g/dL as measured by Advia.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jan 2013.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 21275961Wang 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.
Related trials for Hemoglobin SC Disease
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Investigating the Mechanistic Effects of Mitapivat in Subjects With Sickle Cell Disease
Sickle Cell Anemia, Sickle Cell Thalassemia
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Sickle Cell Disease and the Genomic and Gene Therapy Needs of Stakeholders
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Winfred C. Wang, MD principal investigator
St. Jude Children's Research Hospital
19 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Hydroxyurea and Magnesium Pidolate in people with Hemoglobin SC Disease, run by St. Jude Children's Research Hospital at 19 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 18 Jan 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 19 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 11 May 2010). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Distribution of the Density of Hemoglobin SC Red Cells (time frame: measured 2 months after initiation of treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00532883, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Jan 2013.