HEMOGLOBIN SC DISEASEPhase 2NCT00040456
Oral Magnesium Pidolate, Hemoglobin SC Disease, MG Pidolate
A Phase 2 study testing Mg Pidolate in people with HEMOGLOBIN SC DISEASE, run by Baylor College of Medicine at 2 sites in United States.
Trial summary
Subjects have a form of sickle cell disease, called hemoglobin SC disease. This results in abnormally shaped red blood cells that get 'stuck' in blood vessels and then results in episodes of severe pain (pain crises). Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- HEMOGLOBIN SC DISEASE
- What is being tested?
- Mg Pidolate
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 12 participants
- Where
- 2 sitesUnited States
- Study dates
- Jan 2001to May 2006
- Phase
- Phase 2
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- This protocol is open to male and female patients of all races with a diagnosis of severe sickle hemoglobinopathy providing they also satisfy the following eligibility criteria:
- Adult and pediatric patients with Hb SC disease who are able to swallow the Mg pidolate preparation and who have had at least one prior painful crisis.
Who cannot join
- Patients transfused within 90 days of study entry,
- Patients with elevated (>1.5 times upper limit of normal for age) BUN, creatinine, or liver function tests > 3 times the upper limit of normal for age,
- Patients who take a magnesium-containing medication (Mylanta, Maalox, etc.) on a regular (i.e., more than 2 days per week) basis.
- Patients with progressive neuromuscular disease or patients who are treated with a calcium channel blocker.
- Pregnancy
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Children's Hospital
Boston, Massachusetts, 02115
Texas Children's Hospital
Houston, Texas, 77030
About this study
Official title: The Effect of Oral Magnesium Pidolate on Incidence of Painful Crises in Patients With Hemoglobin SC Disease
Subjects have a form of sickle cell disease, called hemoglobin SC disease. This results in abnormally shaped red blood cells that get 'stuck' in blood vessels and then results in episodes of severe pain (pain crises). Patients with the more common form of sickle cell disease, called hemoglobin SS disease, also suffer from pain crises. Treatment with the drug hydroxyurea is available to help prevent the pain crises in hemoglobin SS disease, but there is no good treatment to help prevent the pain crises in hemoglobin SC disease.
It has been shown that one of the reasons for the formation of the abnormally shaped red blood cells in patients with SC disease is the fact that these cells do not contain enough water; they are dehydrated. Drinking more water will not increase the amount of water in the cells. Certain salts and minerals can however have an effect on the amount of water in the red blood cells. One of the most important minerals influencing this is called magnesium. Magnesium is present in food and also in certain medications used to treat heartburn. Magnesium has been used successfully both in animals and people to increase the amount of water in the red blood cells and is very well tolerated by most people.
Read more
Investigators are using a new form of magnesium known as magnesium pidolate because this form of magnesium may help with the symptoms of disease without causing diarrhea (a common side effect of magnesium products).
Purpose The purpose of this study is to find out whether treatment with magnesium pidolate will increase the amount of water in the red blood cell and result in fewer painful crises in patients with hemoglobin SC disease while not causing diarrhea.
The study will last for about 64 weeks (about 16 months).
Read the detailed description
This is a placebo-controlled study which means that the effectiveness of the magnesium medication (the chemical form of which is known as Mg pidolate) will be compared to placebo. A placebo looks like the drug that is being studied (in this case magnesium) but does not contain any active drug of any kind. Comparing magnesium with placebo will help decide whether magnesium is better than treatment with placebo.
The study lasts for 64 weeks (about 16 months) and is divided into 2 parts, each part lasting for 32 weeks (8 months). During the first part study medication (magnesium or placebo) will be taken twice daily for 24 weeks (about 6 months), followed by no study medication for 8 weeks. These 8 weeks are called a washout period, during which time the effects of the study medication are 'washed-out' of the body. After the washout 8 weeks, patients will enter the second part of the study. During this second part, the study medication (magnesium or placebo) will again be taken twice daily for 24 weeks. During the second part of the study participants will receive whichever study medication that was not taken during the first part of the study. Following the second 24 weeks, there will be an observation period of 8 weeks (during which time no study medication is taken). The study is completed at the end of the 8-week observation period. Participants will take magnesium for 24 weeks and placebo for 24 weeks. The decision whether participants will receive magnesium or placebo first will be made randomly by a computer. This is a double-blind study which means that neither the participant nor the study doctor will know which study medication is taking during each part of the study. However, the study may be unblinded for safety issues if necessary.
If the participant responds to the study medication, it can be continued after completion of the 24 weeks.
The study medication is in the form of a liquid, taken two times a day (morning and evening) with the actual amount taken depending on the participant's weight.
During this study the participant will be seen in clinic on a regular basis; initially every 2 weeks, later once a month. There will be a total of 21 clinic visits during the study. Three of these are clinic visits that would normally take place in the routine management of this disease. The other 18 visits are required for this study. At each visit participants will have blood tests to measure the amount of magnesium in the blood, especially the red blood cells, but also to look for any other changes that might occur. The total amount of blood collected during the 16 month study is 120 ml (24 teaspoons), which is a safe amount. Three ml (half teaspoon) will be drawn at 13 visits, 5ml (1 teaspoon) at 4 visits, and 15ml (3 teaspoons) at a further 4 visits. At 4 of the clinic visits a urine test will be performed.
During the study participants will be asked to keep a record of all episodes of pain, or any other changes that are noticed, in the form of a diary.
- Conditions
- HEMOGLOBIN SC DISEASE
- Being tested
- Mg Pidolate, Placebo
- Keywords
- Hemoglobin SC disease, sickle cell-hemoglobin
- Medical subject headings
- Hemoglobin SC Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Mg PidolateDrug
Also called Magnesium Pidolate
Mag 2 will be used (magnesium pidolate, a granular powder, containing 0.184 g of Mg/packet, equal to 7.6 mmol or 15.2 meq Mg), since this preparation has less side effects such as diarrhea than other Mg preparations. The study medication will be a liquid containing 0.6 meq Mg pidolate/kg body weight per day, divided into 2 daily doses. The Mg pidolate (45 g) will be distributed as a pre-mixed powder containing Koolaid Tropical Punch powder (9 gm), and sucrose (67 gm).
PlaceboDrug
The study medication will be a liquid containing an equivalent amount of placebo to the study medication, divided into 2 daily doses.The placebo will have the same amount of sucrose and Tropical Punch powder as the study drug, MG Pidolate, as well as 45 g of lactose. Subjects will continue with the same assignment for 6 months and then switch to the other arm (after a 2-month wash-out period).
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | A computer-generated randomization list will be created by a Baylor College of Medicine statistician (unrelated to study) prior to the study.
Patients are randomly assigned to either start with Mg pidolate or placebo and will continue that therapy for 24 weeks. Then, after a 2 month wash-out period, they will be switched to the other arm and continue on that arm for another 24 weeks, followed by 8 weeks of observation off study drug. Both patient and medical care provider(s) will be blinded to treatment assignment. Mg pidolate and placebo will be distributed through the pharmacy with labels that do not indicate the assignment. Receives: Drug: Placebo |
| MG Pidolate Administration | Active comparator | A computer-generated randomization list will be created by a Baylor College of Medicine statistician (unrelated to study) prior to the study.
Patients are randomly assigned to either start with Mg pidolate or placebo and will continue that therapy for 24 weeks. Then, after a 2 month wash-out period, they will be switched to the other arm and continue on that arm for another 24 weeks, followed by 8 weeks of observation off study drug. Both patient and medical care provider(s) will be blinded to treatment assignment. Mg pidolate and placebo will be distributed through the pharmacy with labels that do not indicate the assignment. Receives: Drug: Mg Pidolate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluation of whether treatment with oral Mg pidolate decreases the number of painful crises.
The difference in frequency of painful episodes between the 2 treatment modalities will be calculated for each patient. The goal is to decrease the frequency of painful episodes by at least 50%.
Measured: 64 weeks
Secondary outcomes (3)
Evaluation of tolerance of long-term treatment with oral Mg pidolate.
Adverse events will be tabulated by body system, severity, and relation to treatment in order to determine tolerance.
Measured: 16 months
To find out if treatment with oral Mg pidolate increases the intracellular Mg content of erythrocytes.
mean values will be compared by Wilcoxon signed -rank test or the paired t-test
Measured: 16 months
Evaluations of the effect of Mg pidolate therapy on the K-Cl cotransport activity and RBC hydration status
McNemar's test will be used to assess the decrease or increase in K-CL cotransport system activity.
Measured: 16 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jun 2002 (estimated)
- Study startJan 2001
- Study completionMay 2006
Why it stopped: Lack of accrual
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HEMOGLOBIN SC DISEASEStudies of the same condition, open and under way.
- More trials run by Baylor College of MedicineThe 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
- Crossover
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baylor College of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- Boston Children's Hospital
- Responsible party
- Principal investigator, Brigitta Mueller, Baylor College of Medicine
- Investigators
- Brigitta Mueller, MD, Principal investigator, Baylor College of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00040456
- Sponsor's protocol ID
- H9251 MG Pidolate
- Other IDs
- MG Pidolate
- Status last verified
- Nov 2012
- First submitted
- 26 Jun 2002
- Last updated
- 19 Nov 2012
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 19 Nov 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Effect of Oral Magnesium Pidolate on Incidence of Painful Crises in Patients With Hemoglobin SC Disease
- ClinicalTrials.gov ID
- NCT00040456
- Acronym / sponsor ID
- H9251 MG Pidolate
- Conditions
- HEMOGLOBIN SC DISEASE
- Keywords
- Hemoglobin SC disease, sickle cell-hemoglobin
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Lack of accrual
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Baylor College of MedicineUniversity, hospital or other
- Collaborators
- Boston Children's Hospital
- Study start
- Jan 2001
- Study completion
- May 2006 (actual)
- First posted
- 28 Jun 2002 (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.
Subjects have a form of sickle cell disease, called hemoglobin SC disease. This results in abnormally shaped red blood cells that get 'stuck' in blood vessels and then results in episodes of severe pain (pain crises). Patients with the more common form of sickle cell disease, called hemoglobin SS disease, also suffer from pain crises. Treatment with the drug hydroxyurea is available to help prevent the pain crises in hemoglobin SS disease, but there is no good treatment to help prevent the pain crises in hemoglobin SC disease.
It has been shown that one of the reasons for the formation of the abnormally shaped red blood cells in patients with SC disease is the fact that these cells do not contain enough water; they are dehydrated. Drinking more water will not increase the amount of water in the cells. Certain salts and minerals can however have an effect on the amount of water in the red blood cells. One of the most important minerals influencing this is called magnesium. Magnesium is present in food and also in certain medications used to treat heartburn. Magnesium has been used successfully both in animals and people to increase the amount of water in the red blood cells and is very well tolerated by most people.
Investigators are using a new form of magnesium known as magnesium pidolate because this form of magnesium may help with the symptoms of disease without causing diarrhea (a common side effect of magnesium products).
Purpose The purpose of this study is to find out whether treatment with magnesium pidolate will increase the amount of water in the red blood cell and result in fewer painful crises in patients with hemoglobin SC disease while not causing diarrhea.
The study will last for about 64 weeks (about 16 months).
Detailed description
This is a placebo-controlled study which means that the effectiveness of the magnesium medication (the chemical form of which is known as Mg pidolate) will be compared to placebo. A placebo looks like the drug that is being studied (in this case magnesium) but does not contain any active drug of any kind. Comparing magnesium with placebo will help decide whether magnesium is better than treatment with placebo.
The study lasts for 64 weeks (about 16 months) and is divided into 2 parts, each part lasting for 32 weeks (8 months). During the first part study medication (magnesium or placebo) will be taken twice daily for 24 weeks (about 6 months), followed by no study medication for 8 weeks. These 8 weeks are called a washout period, during which time the effects of the study medication are 'washed-out' of the body. After the washout 8 weeks, patients will enter the second part of the study. During this second part, the study medication (magnesium or placebo) will again be taken twice daily for 24 weeks. During the second part of the study participants will receive whichever study medication that was not taken during the first part of the study. Following the second 24 weeks, there will be an observation period of 8 weeks (during which time no study medication is taken). The study is completed at the end of the 8-week observation period. Participants will take magnesium for 24 weeks and placebo for 24 weeks. The decision whether participants will receive magnesium or placebo first will be made randomly by a computer. This is a double-blind study which means that neither the participant nor the study doctor will know which study medication is taking during each part of the study. However, the study may be unblinded for safety issues if necessary.
If the participant responds to the study medication, it can be continued after completion of the 24 weeks.
The study medication is in the form of a liquid, taken two times a day (morning and evening) with the actual amount taken depending on the participant's weight.
During this study the participant will be seen in clinic on a regular basis; initially every 2 weeks, later once a month. There will be a total of 21 clinic visits during the study. Three of these are clinic visits that would normally take place in the routine management of this disease. The other 18 visits are required for this study. At each visit participants will have blood tests to measure the amount of magnesium in the blood, especially the red blood cells, but also to look for any other changes that might occur. The total amount of blood collected during the 16 month study is 120 ml (24 teaspoons), which is a safe amount. Three ml (half teaspoon) will be drawn at 13 visits, 5ml (1 teaspoon) at 4 visits, and 15ml (3 teaspoons) at a further 4 visits. At 4 of the clinic visits a urine test will be performed.
During the study participants will be asked to keep a record of all episodes of pain, or any other changes that are noticed, in the form of a diary.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: PlaceboA computer-generated randomization list will be created by a Baylor College of Medicine statistician (unrelated to study) prior to the study. Patients are randomly assigned to either start with Mg pidolate or placebo and will continue that therapy for 24 weeks. Then, after a 2 month wash-out period, they will be switched to the other arm and continue on that arm for another 24 weeks, followed by 8 weeks of observation off study drug. Both patient and medical care provider(s) will be blinded to treatment assignment. Mg pidolate and placebo will be distributed through the pharmacy with labels that do not indicate the assignment. |
| MG Pidolate Administration | Active comparator | Drug: Mg PidolateA computer-generated randomization list will be created by a Baylor College of Medicine statistician (unrelated to study) prior to the study. Patients are randomly assigned to either start with Mg pidolate or placebo and will continue that therapy for 24 weeks. Then, after a 2 month wash-out period, they will be switched to the other arm and continue on that arm for another 24 weeks, followed by 8 weeks of observation off study drug. Both patient and medical care provider(s) will be blinded to treatment assignment. Mg pidolate and placebo will be distributed through the pharmacy with labels that do not indicate the assignment. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- This protocol is open to male and female patients of all races with a diagnosis of severe sickle hemoglobinopathy providing they also satisfy the following eligibility criteria:
- Adult and pediatric patients with Hb SC disease who are able to swallow the Mg pidolate preparation and who have had at least one prior painful crisis.
Key exclusion criteria (5 listed)
- Patients transfused within 90 days of study entry,
- Patients with elevated (>1.5 times upper limit of normal for age) BUN, creatinine, or liver function tests > 3 times the upper limit of normal for age,
- Patients who take a magnesium-containing medication (Mylanta, Maalox, etc.) on a regular (i.e., more than 2 days per week) basis.
- Patients with progressive neuromuscular disease or patients who are treated with a calcium channel blocker.
- Pregnancy
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 |
|---|---|---|
| Mg PidolateAlso: Magnesium PidolateMag 2 will be used (magnesium pidolate, a granular powder, containing 0.184 g of Mg/packet, equal to 7.6 mmol or 15.2 meq Mg), since this preparation has less side effects such as diarrhea than other Mg preparations. The study medication will be a liquid containing 0.6 meq Mg pidolate/kg body weight per day, divided into 2 daily doses. The Mg pidolate (45 g) will be distributed as a pre-mixed powder containing Koolaid Tropical Punch powder (9 gm), and sucrose (67 gm). | Drug | MG Pidolate Administration |
| PlaceboThe study medication will be a liquid containing an equivalent amount of placebo to the study medication, divided into 2 daily doses.The placebo will have the same amount of sucrose and Tropical Punch powder as the study drug, MG Pidolate, as well as 45 g of lactose. Subjects will continue with the same assignment for 6 months and then switch to the other arm (after a 2-month wash-out period). | Drug | Placebo |
Primary outcome
Evaluation of whether treatment with oral Mg pidolate decreases the number of painful crises.
Time frame: 64 weeks
The difference in frequency of painful episodes between the 2 treatment modalities will be calculated for each patient. The goal is to decrease the frequency of painful episodes by at least 50%.
3 secondary outcomes
Evaluation of tolerance of long-term treatment with oral Mg pidolate.
Time frame: 16 months
To find out if treatment with oral Mg pidolate increases the intracellular Mg content of erythrocytes.
Time frame: 16 months
Evaluations of the effect of Mg pidolate therapy on the K-Cl cotransport activity and RBC hydration status
Time frame: 16 months
Results and safety
The study was stopped early (reason given: Lack of accrual). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Nov 2012.
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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.
Brigitta Mueller, MD principal investigator
Baylor College of Medicine
2 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 Mg Pidolate in people with HEMOGLOBIN SC DISEASE, run by Baylor College of Medicine at 2 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 2 inclusion and 5 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 19 Nov 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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?
The study was stopped early (reason given: Lack of accrual). Results are not available in the source record.
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?
Evaluation of whether treatment with oral Mg pidolate decreases the number of painful crises. (time frame: 64 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 19 Nov 2012.