Anemia, Sickle CellPhase 1NCT00143572
Use of Hydroxyurea and Magnesium Pidolate for Treatment of Sickle Cell Disease
A Phase 1 study testing Magnesium Pidolate, Hydroxyurea in people with Anemia, Sickle Cell, run by St. Jude Children's Research Hospital at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 3 to 15 years
- Conditions the study is for
- Anemia, Sickle Cell
- What is being tested?
- Magnesium Pidolate, Hydroxyurea
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2004to Jan 2008
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 3 to 15 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
About this study
Official title: Phase I Study of Combination Treatment With Hydroxyurea and Magnesium Pidolate in Patients With 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.
Read the 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.
- Conditions
- Anemia, Sickle Cell
- Being tested
- Magnesium Pidolate, Hydroxyurea
- Keywords
- Hematologic Diseases, Anemia, Sickle Cell
- Medical subject headings
- Anemia, Sickle Cell, Hematologic Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Magnesium Pidolate, HydroxyureaDrug
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).
| Study group | Role | What they receive |
|---|---|---|
| 1 | Other | Not described Receives: Drug: Magnesium Pidolate, Hydroxyurea |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Every 2 weeks for first 8 weeks; then every 4 weeks
Secondary outcomes (2)
To document the toxicity of the combination of hydroxyurea and magnesium pidolate.
Measured: Every 2 weeks for first 8 weeks; then every 4 weeks
To investigate the effect of the combination of hydroxyurea and magnesium on hematological parameters and red cell metabolism.
Measured: 3 months, 6 months, and 9 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Sep 2005 (estimated)
- Study startNov 2004
- Primary completionJan 2008
- Study completionJan 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Anemia, Sickle CellStudies 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Winfred Wang, MD, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00143572
- Sponsor's protocol ID
- HUMG1
- Status last verified
- Feb 2010
- First submitted
- 1 Sep 2005
- Last updated
- 26 Apr 2017
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 26 Apr 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Study of Combination Treatment With Hydroxyurea and Magnesium Pidolate in Patients With Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT00143572
- Acronym / sponsor ID
- HUMG1
- Conditions
- Anemia, Sickle Cell
- Keywords
- Hematologic Diseases, Anemia, Sickle Cell
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Study start
- Nov 2004
- Primary completion
- Jan 2008 (actual)
- Study completion
- Jan 2008 (actual)
- First posted
- 2 Sep 2005 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Other | Drug: Magnesium Pidolate, Hydroxyurea |
Who may qualify
- Ages
- 3 to 15 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (8 listed)
- 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
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 |
|---|---|---|
| Magnesium Pidolate, HydroxyureaIntervention 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). | Drug | 1 |
Primary outcome
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
2 secondary outcomes
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
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Apr 2017.
- St. Jude Children's Research HospitalLink provided by the sponsor.
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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 Wang, MD principal investigator
St. Jude Children's Research Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Magnesium Pidolate, Hydroxyurea in people with Anemia, Sickle Cell, run by St. Jude Children's Research Hospital at 1 site 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 4 inclusion and 8 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: finished, as of the sponsor's last update on 26 Apr 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 26 Apr 2017.