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).
Phase:
Phase 2
Details
Lead Sponsor:
Baylor College of Medicine
Collaborators:
Boston Children's Hospital Boston Children’s Hospital Texas Children's Hospital