PK and PD Responses to Oral L-Citrulline in Patients With Sickle Cell Disease

NCT00343954 | Unknown Phase 1

• 1 other identifier: ANGX-3227-01
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

To evaluate PK and PD responses to L-citrulline given orally for four weeks to patients with sickle cell disease who are otherwise healthy.

Conditions

Sickle Cell Disease

Keywords

sickle cell disease

Outcome Measures

Primary Outcomes (2)

• PK and PD responses to L-citrulline

• Endpoints will be determined as change from baseline

Secondary Outcomes (1)

• Assessment of safety and tolerability of L-citrulline,

Interventions

L-citrulline tablets, 1000 mg DRUG

Eligibility Criteria

• Age: 10 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female, 10 years of age or older on the day of dosing, and generally healthy as determined by medical history, physical examination, and laboratory test values
• Diagnosis of sickle cell anemia (Hb SS)
• For female of child-bearing potential, a negative serum pregnancy test and using an adequate method of contraception
• Has signed and received a copy of the written informed consent form approved by the investigator's Institutional Review Board (IRB), understands the purposes and risks of the study and agrees to follow the restrictions and schedule of procedures as defined by this protocol

You may not qualify if:

• History of sickle-cell-related pain crisis within two weeks of study
• Pregnant or breast feeding
• Transfusion within last 90 days
• Creatinine \>1.5 X upper limit of normal
• SGPT \> 2 X upper limit of normal
• History of allergic reaction to arginine or citrulline product
• Requires chronic medication other than study drug that cannot be discontinued during the study period
• Unable to take or tolerate oral medications
• Unreliable venous access
• Noncompliant with regular care
• Participation in an investigational drug or medical device study within previous 30 days
• In the opinion of the investigator is not a good candidate for participation in the study

Sponsors & Collaborators

• Angiogenix: lead

Study Sites (1)

Children's Hospital of Pittsburgh of the University of Pittsburgh Medical Center Health System

Pittsburgh, Pennsylvania, 15213, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

Citrulline

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Amino Acids, Diamino; Amino Acids; Amino Acids, Peptides, and Proteins

Study Officials

• Lakshmanam Krishnamurti, MD

  Children's Hospital of Pittsburgh, Univ. of Pittsburgh Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY

Study Record Dates

• First Submitted: June 21, 2006
• First Posted: June 23, 2006
• Last Updated: June 23, 2006

Record last verified: 2006-06

Locations

US (1)