This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. This is a randomized, double-blind, placebo-controlled phase II study with cross-over design as shown below. After meeting eligibility criteria, patients will be randomly assigned to study medication (Mg pidolate or placebo) for 24 weeks, followed by a washout period of 8 weeks. After the washout period, each patient will receive a further 24 weeks of study medication. The study will be conducted at Texas Children's Cancer Center and Hematology Service and Children's Hospital, Boston. It is expected that 20 patients will be enrolled over four years, with each patient on study for 16 months. 21 clinic visits will be required, with interval history and physical examination (including a urine pregnancy tests for all post-menarchal females due to the questionable effects of magnesium on pregnant women and fetuses) at each clinic visit in order to monitor for and evaluate any toxicities. Blood tests will be taken throughout the study to monitor hematology, serum chemistry, renal function and liver function. At 8 visits, blood will also be drawn for membrane transport studies and assessment of morphologic sickling in vitro and in vivo, and at 4 visits, blood will also be drawn for serum storage. The total amount of blood collected per patient during this study is 120ml (24 teaspoons) - 2-3ml (half teaspoon) at each of 13 visits, 5ml (I teaspoon) at each of 4 visits and 15ml (3 teaspoons) on 4 visits. Urine will be collected on 4 visits for urinalysis. The patient (or their parent, if appropriate) will be asked to keep a written record (in the form of a diary) of all episodes of pain or any other changes that are notice. Patients who are responding to the study medication will be allowed to continue treatment with the study medication until the study is closed. The study medication will be a liquid containing either 0.6 meq Mg pidolate/kg body weight per day or an equivalent amount of placebo, divided into 2 daily doses. This dose is slightly higher than physiologic magnesium supplementation (5 mg/kg/day) but lower than the doses used for Mg deficiency treatment (>10 mg/kg/day), and is approximately equivalent to 3 Mylanta double strength tablets per day. The Mg pidolate (45 g) will be distributed as a pre-mixed powder containing KoolAid Tropical Punch powder (9 gm), and sucrose (67 gm). This breaks down to (per 1/8 teaspoon) Magnesium pidolate 0.29 gm (23.7 mg Mg 2+) = 1.97 meq Mg 2+ = 1 mmol Mg. The powder is easily dissolved in water and has a pleasant taste. The placebo will have the same amount of sucrose and Tropical Punch powder as well as 45 g of lactose.
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