Phase 2
N=109
Effectiveness of Arginine as a Treatment for Sickle Cell Anemia
Anemia, Sickle Cell
Bottom Line
View on ClinicalTrials.gov: NCT00513617 ↗Enrolled (actual)
109
Serious AEs
—
Results posted
Aug 2009
Primary outcome: Primary: Gardos Channel Activity — -0.0342; 0.0043; 0.1076 mmol/10^13 cells x min — p=0.080
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Arginine (Drug); Placebo (Drug)
- Age
- Pediatric, Adult, Older Adult · 5+ yrs
- Sex
- All
- Sponsor
- UCSF Benioff Children's Hospital Oakland
- Primary completion
- Sep 2007
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Gardos Channel Activity |
-0.0342; 0.0043; 0.1076 | 0.080 |
| PRIMARY Nitric Oxide |
-3.8697; 2.9250; -3.0265 | 0.915 |
| PRIMARY Mean Corpuscular Hemoglobin Concentration |
-1.8485; 0.7692; -0.0705 | 0.015 sig |
| SECONDARY Soluble Vascular Cell Adhesion Molecule |
— | — |
| SECONDARY 8-iso-PGF2a |
— | — |
| SECONDARY Endothelin-1 |
— | — |
| SECONDARY Fetal Hemoglobin |
— | — |
Summary
Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited genetic disease that can cause intense pain episodes. This study will evaluate the effectiveness of the nutritional supplement arginine at improving blood cell function and disease symptoms in people with SCD.
Eligibility Criteria
Inclusion Criteria
- Established diagnosis of H SS or S-beta thalassemia
- History of at least one vaso-occlusive pain event in the 12 months prior to study entry
- Regular compliance with comprehensive medical care
- In a steady disease state and not in the midst of any acute complication due to SCD at study entry
Exclusion Criteria
- Inability to take or tolerate oral medications
- Liver dysfunction (i.e., SGPT level greater than or equal to two times the normal limit and albumin level less than or equal to 3.2 g/dL)
- Kidney dysfunction ( i.e., creatinine level greater than or equal to 1.2 mg/dL for children and greater than or equal to 1.4 mg/dL for adults)
- Allergy to arginine
- Pregnant
- Received a blood transfusion within the 90 days prior to study entry
- More than 10 hospital admissions for pain in the 12 months prior to study entry
- Daily use of opioids and experiencing unstable pain that interferes with work or daily routine
- Required more than 3 hospital admissions and more than 10 emergency department/day hospital visits in the 12 months prior to study entry
- Received treatment with hydroxyurea within the 90 days prior to study entry
- Received treatment with any investigational drug in the 90 days prior to study entry
Data sourced from ClinicalTrials.gov (NCT00513617). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.