Oral Magnesium Pidolate, Hemoglobin SC Disease, MG Pidolate

NCT00040456 | Terminated Phase 2 DMC

• 2 other identifiers: H9251 MG PidolateMG Pidolate
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 2

Brief 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). 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).

Conditions

HEMOGLOBIN SC DISEASE

Keywords

Hemoglobin SC disease; sickle cell-hemoglobin

Outcome Measures

Primary Outcomes (1)

• 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%.

  64 weeks

Secondary Outcomes (3)

• Evaluation of tolerance of long-term treatment with oral Mg pidolate.

  16 months

• To find out if treatment with oral Mg pidolate increases the intracellular Mg content of erythrocytes.

  16 months

• Evaluations of the effect of Mg pidolate therapy on the K-Cl cotransport activity and RBC hydration status

  16 months

Study Arms (2)

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.

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.

Drug: Mg Pidolate

Interventions

Mg Pidolate DRUG

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).

Also known as: Magnesium Pidolate

MG Pidolate Administration

Placebo DRUG

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).

Placebo

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Baylor College of Medicine: lead
• Boston Children's Hospital: collaborator

Study Sites (2)

Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Texas Children's Hospital

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Hemoglobin SC Disease

Interventions

Pyrrolidonecarboxylic Acid

Condition Hierarchy (Ancestors)

Anemia, Sickle Cell; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Pyrrolidinones; Pyrrolidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Glutamates; Amino Acids, Acidic; Amino Acids; Amino Acids, Peptides, and Proteins; Imino Acids; Amino Acids, Cyclic

Study Officials

• Brigitta Mueller, MD

  Baylor College of Medicine

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Pediatrics-Hem-Oncology

Study Record Dates

• First Submitted: June 26, 2002
• First Posted: June 28, 2002
• Study Start: January 1, 2001
• Study Completion: May 1, 2006
• Last Updated: November 19, 2012

Record last verified: 2012-11

Locations

US (2)