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Glutamine Therapy for Hemolysis-Associated Pulmonary Hypertension

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01048905
Recruitment Status : Completed
First Posted : January 14, 2010
Results First Posted : June 10, 2021
Last Update Posted : June 10, 2021
Sponsor:
Collaborator:
Food and Drug Administration (FDA)
Information provided by (Responsible Party):
UCSF Benioff Children's Hospital Oakland

Brief 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.

Condition or disease Intervention/treatment Phase
Pulmonary Hypertension Sickle Cell Disease Thalassemia Drug: L-Glutamine Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 13 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase 2 Trial for Glutamine Therapy for Hemolysis-Associated Pulmonary Hypertension
Study Start Date : March 2009
Actual Primary Completion Date : March 2014
Actual Study Completion Date : March 2014


Arm Intervention/treatment
Experimental: Treatment: L-glutamine
Patients will receive an 8-week course of oral L-glutamine 10 grams TID
Drug: L-Glutamine
Oral L-glutamine 10 grams TID or (0.1g/kg TID) for children < 15 years of age.




Primary Outcome Measures :
  1. Erythrocyte Glutamine/Glutamate Ratio at 8 Weeks [ Time Frame: 8 weeks ]
    Erythrocyte Glutamine/Glutamate Ratio: a novel biomarker of oxidative stress


Secondary Outcome Measures :
  1. Plasma Glutamine [ Time Frame: 8 weeks ]
  2. Tricuspid Regurgitant Jet Velocity on Doppler Echocardiography [ Time Frame: 8 week ]
    Tricuspid Regurgitant Jet Velocity was measured using Doppler Echocardiography in meters per second.

  3. 6 Minute Walk Distance [ Time Frame: 8 weeks ]
    The six-minute walk test (6MWT) measures the distance in meters an individual is able to walk over a total of six minutes on a hard, flat surface.

  4. Liver Function Tests [ Time Frame: 8 weeks ]
    Alanine aminotransferase (ALT) Aspartate aminotransferase (AST)

  5. Renal Function Tests [ Time Frame: 8 weeks ]
    Creatinine Blood urea nitrogen (BUN)



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Ages Eligible for Study:   4 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

Inclusion Criteria:

  • Established diagnosis of SCD (Hb SS, SC or SBeta- thalassemia) or Thal
  • PH documented by echocardiography, defined as at TRV greater than 2.5 m/s
  • Age greater than or equal to 4 years

Exclusion Criteria:

  • Inability to take or tolerate oral medication
  • Acute crisis or hospitalization within 1 month of enrollment
  • Hepatic dysfunction (SGPT greater than 3X normal)
  • Renal dysfunction (Creatinine greater than 2X normal)
  • Allergy to glutamine
  • Pregnancy or breastfeeding
  • Patients on sildenafil (Viagra), calcium channel blockers, or amino acid/protein supplements (other therapies acceptable if stable more than 3 months)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01048905


Locations
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United States, California
Children's Hospital & Research Center Oakland
Oakland, California, United States, 94608
Sponsors and Collaborators
UCSF Benioff Children's Hospital Oakland
Food and Drug Administration (FDA)
Investigators
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Principal Investigator: Claudia Morris, MD Emory University
Principal Investigator: Augusta Saulys, MD Children's Hosptial & Research Center Oakland
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Responsible Party: UCSF Benioff Children's Hospital Oakland
ClinicalTrials.gov Identifier: NCT01048905    
Other Study ID Numbers: 1R01FD003531-01 ( U.S. FDA Grant/Contract )
IRB 2008-059 ( Other Identifier: Institutional Review Board )
1R01FD003531-01 ( U.S. FDA Grant/Contract )
First Posted: January 14, 2010    Key Record Dates
Results First Posted: June 10, 2021
Last Update Posted: June 10, 2021
Last Verified: June 2021
Keywords provided by UCSF Benioff Children's Hospital Oakland:
Pulmonary Hypertension
Sickle Cell Disease
Thalassemia
Additional relevant MeSH terms:
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Hypertension, Pulmonary
Hypertension
Anemia, Sickle Cell
Thalassemia
Hemolysis
Vascular Diseases
Cardiovascular Diseases
Lung Diseases
Respiratory Tract Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Pathologic Processes