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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified June 2006 by Angiogenix.
Recruitment status was  Not yet recruiting
Sponsor:
Information provided by:
Angiogenix
ClinicalTrials.gov Identifier:
NCT00343954
First received: June 21, 2006
Last updated: NA
Last verified: June 2006
History: No changes posted
  Purpose

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


Condition Intervention Phase
Sickle Cell Disease
 Drug: L-citrulline tablets, 1000 mg
 Phase 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Pharmacokinetics/Dynamics Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Phase 1 Pharmacokinetic and Pharmacodynamic Responses to Oral L-Citrulline in Patients With Sickle Cell Disease

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Angiogenix:

Primary Outcome Measures:
  • PK and PD responses to L-citrulline
  • Endpoints will be determined as change from baseline

Secondary Outcome Measures:
  • Assessment of safety and tolerability of L-citrulline,

Estimated Enrollment: 12
Detailed Description:

Phase 1, single center, open label, multiple-dose study consisting of a screening period followed by four weeks of treatment. At least 12 male and female patients, 10 years of age or older, with sickle cell disease but otherwise healthy, will be enrolled in the study.

PK measurements include plasma concentrations of L-citrulline, L-arginine, L-ornithine, and L-proline following the first dose of orally administered L-citrulline and after four weeks of twice daily administration of the drug.

PD measurements include intercellular and vascular adhesion molecules (ICAM, VCAM, and E-selectin), surrogate markers of sickle cell disease activity, and PAT, a measurement of vascular function.

  Eligibility

Ages Eligible for Study:   10 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  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

Exclusion Criteria:

  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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00343954

Locations
United States, Pennsylvania
Children's Hospital of Pittsburgh of the University of Pittsburgh Medical Center Health System Not yet recruiting
Pittsburgh, Pennsylvania, United States, 15213
Contact: Lakshmanan Krishnamurti, MD     412-692-7192        
Contact: Sidney Morris, PhD     (412) 648-9338        
Sponsors and Collaborators
Angiogenix
Investigators
Principal Investigator: Lakshmanam Krishnamurti, MD Children's Hospital of Pittsburgh, Univ. of Pittsburgh Medical Center
  More Information

No publications provided

ClinicalTrials.gov Identifier: NCT00343954     History of Changes
Other Study ID Numbers: ANGX-3227-01
Study First Received: June 21, 2006
Last Updated: June 21, 2006
Health Authority: United States: Food and Drug Administration

Keywords provided by Angiogenix:
sickle cell disease

Additional relevant MeSH terms:
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
 Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on May 19, 2013