Glutamine Therapy for Hemolysis-Associated Pulmonary Hypertension
Status:
Completed
Trial end date:
2014-03-01
Target enrollment:
Participant gender:
Summary
The primary hypothesis of this study is that glutamine supplementation will improve the
erythrocyte glutamine/glutamate ratio, a biomarker of oxidative stress, hemolysis and
pulmonary hypertension (PH) in sickle cell disease (SCD) and thalassemia (Thal) patients with
PH. PH is defined as a tricuspid regurgitant jet velocity (TRV) on Doppler echocardiography >
2.5 m/s. We also predict that glutamine therapy will increase arginine bioavailability and
subsequently alter sickle red cell endothelial interaction that can be identified using
endo-PAT technology through nitric oxide (NO) generation, leading to changes in biological
markers, and clinical outcome. Specifically our second hypothesis is that oral glutamine will
decrease biomarkers of hemolysis and adhesion molecules, and improve the imbalanced
arginine-to-ornithine ratio that occurs in hemolytic anemias, leading to improved arginine
bioavailability and clinical endpoints of endothelial dysfunction and PH in patients with SCD
and Thal.
Phase:
Phase 2
Details
Lead Sponsor:
Children's Hospital & Research Center Oakland UCSF Benioff Children's Hospital Oakland