Phase 1 Pharmacokinetic and Pharmacodynamic Responses to Oral L-Citrulline in Patients With Sickle Cell Disease

Locations

1 United States site

Age

> 10 Years

Genotypes

SS

Phase

Phase 1

Study type

Interventional

Gender

All

Interventions

Drug

Compensation

Unknown

About the study

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

participation requirements

1. 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

2. Diagnosis of sickle cell anemia (Hb SS)

3. For female of child-bearing potential, a negative serum pregnancy test and using an
adequate method of contraception

4. 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

participation restrictions

1. History of sickle-cell-related pain crisis within two weeks of study

2. Pregnant or breast feeding

3. Transfusion within last 90 days

4. Creatinine >1.5 X upper limit of normal

5. SGPT > 2 X upper limit of normal

6. History of allergic reaction to arginine or citrulline product

7. Requires chronic medication other than study drug that cannot be discontinued during
the study period

8. Unable to take or tolerate oral medications

9. Unreliable venous access

10. Noncompliant with regular care

11. Participation in an investigational drug or medical device study within previous 30
days

12. In the opinion of the investigator is not a good candidate for participation in the
study

Locations

  • Pittsburgh, Pennsylvania, United States, Children's Hospital of Pittsburgh of the University of Pittsburgh Medical Center Health System, 15213