Effectiveness of Arginine as a Treatment for Sickle Cell Anemia

This study has been completed.

Study NCT00513617 Information provided by National Heart, Lung, and Blood Institute (NHLBI)

First Received on August 6, 2007. Last Updated on June 19, 2009 History of Changes

Results First Received: February 23, 2009

Study Type:	Interventional
Study Design:	Allocation: Randomized; Endpoint Classification: Safety/Efficacy Study; Intervention Model: Parallel Assignment; Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor); Primary Purpose: Treatment
Condition:	Anemia, Sickle Cell
Interventions:	Drug: Arginine Drug: Placebo

Participant Flow

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Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Enrolled subjects at participating sites from May 2004 through July 2007. Sites consisted of sickle cell treatment centers from across the United States.

Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
All subjects were to be without hydroxyurea, transfusion, and arginine for 90 days prior to enrollment. Prior to randomization, blood was drawn for baseline efficacy and safety measurements.

Reporting Groups

	Description
Low Dose	0.05 g/kg/day of Arginine in capsule form
High Dose	0.10 g/kg/day of Arginine in capsule form
Placebo	No text entered.

Participant Flow: Overall Study

	Low Dose	High Dose	Placebo
STARTED	36	35	38
COMPLETED	25	25	30
NOT COMPLETED	11	10	8

Baseline Characteristics

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Reporting Groups

	Description
Low Dose	0.05 g/kg/day of Arginine in capsule form
High Dose	0.10 g/kg/day of Arginine in capsule form
Placebo	No text entered.

Baseline Measures

	Low Dose	High Dose	Placebo	Total
Number of Participants [units: participants]	36	35	38	109
Age [units: participants]
<=18 years	14	18	18	50
Between 18 and 65 years	22	17	20	59
>=65 years	0	0	0	0
Age [units: years] Mean ± Standard Deviation	24.5 ± 12.85	20.0 ± 10.01	21.0 ± 11.49	23.2 ± 11.75
Gender [units: participants]
Female	21	19	20	60
Male	15	16	18	49
Region of Enrollment [units: participants]
United States	36	35	38	109
Genotype of SCD [units: participants]
Sickle cell S-Beta Thalassemia (SB0)	2	3	2	7
Sickle cell Anemia (SS)	34	32	36	102

Outcome Measures

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1. Primary:

Gardos Channel Activity [ Time Frame: 12 weeks after randomization ]

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2. Primary:

Nitric Oxide [ Time Frame: 12 weeks after randomization ]

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3. Primary:

Mean Corpuscular Hemoglobin Concentration [ Time Frame: 12 weeks after randomization ]

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4. Secondary:

Soluble Vascular Cell Adhesion Molecule [ Time Frame: 12 weeks after randomization ]

Results not yet posted. Anticipated Posting Date: No text entered. Safety Issue: No

5. Secondary:

8-iso-PGF2a [ Time Frame: 12 weeks after randomization ]

Results not yet posted. Anticipated Posting Date: No text entered. Safety Issue: No

6. Secondary:

Endothelin-1 [ Time Frame: 12 weeks after randomization ]

Results not yet posted. Anticipated Posting Date: No text entered. Safety Issue: No

7. Secondary:

Fetal Hemoglobin [ Time Frame: 12 weeks after randomization ]

Results not yet posted. Anticipated Posting Date: No text entered. Safety Issue: No

Serious Adverse Events

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Other Adverse Events

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More Information

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Certain Agreements:

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
Adult enrollment was slower than expected. Adult subjects were more likely than peds to drop-out due to SCD pain crisis. Higher doses of arginine (such as used in animal studies) were limited by number of daily pills required.

Results Point of Contact:

Name/Title: Karen Kesler, PhD
Organization: Rho Federal Systems Division
phone: 919-408-8000 ext 244
e-mail: karen_kesler@rhoworld.com

No publications provided

Responsible Party:	Lori Styles/Principal Investigator, Childrens Hospital of Oakland and Research Institute
ClinicalTrials.gov Identifier:	NCT00513617 History of Changes
Other Study ID Numbers:	485, U54 HL070587-04
Study First Received:	August 6, 2007
Results First Received:	February 23, 2009
Last Updated:	June 19, 2009
Health Authority:	United States: Food and Drug Administration