A Dose Escalation Study of Intravenous L-citrulline in Steady-state Sickle Cell Disease

Sickle Cell Disease Clinical Trial

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02314689
• Other study ID #: SCD-124700.
• Status: Completed
• Phase: Phase 1
• Start date: January 2015
• Est. completion date: December 2015

Study information

• Verified date: July 2019
• Source: University of Mississippi Medical Center
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to assess the maximum tolerated dose, safety and pharmacokinetics of an investigational drug, intravenous (IV) citrulline, in subjects in steady-state sickle cell disease.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 4
• Est. completion date: December 2015
• Est. primary completion date: December 2015
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 25 Years

Eligibility

Inclusion Criteria:

• Sickle cell disease genotypes (HbSS, HbS/ß° thalassemia, HbS/ß+thalassemia, HbSC)

Exclusion Criteria:

• Presence of any acute illness defined by fever >100.4° F within the past 48 hours

• Presence of sickle cell pain crisis defined by the presence of pain requiring oral or parental opioid therapy.

• Presence of acute chest syndrome or presence of any other complication related to sickle cell disease requiring hospitalization such as splenic sequestration, hepatic sequestration, stroke, avascular necrosis of the hip/shoulder, acute priapism, and patients with diabetes etc.

• Severe anemia (hemoglobin < 5g/dL)

• History of red blood cell transfusion within the last 14 days

• Systemic steroid therapy within the last 48 hours

• Pregnant (as confirmed by a negative urine pregnancy test) or lactating female

• Alanine/aspartate transferase >2x upper limit of normal laboratory range for age.

• Elevated serum creatinine >1.5mg/dL

• Patients with an inability to give consent will be excluded

• Medications that are known to be contra-indicated with use of L-citrulline (concurrent use of hydroxyurea will be allowed).

• History of diabetes due to risk of electrolyte imbalance

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

Intervention

Drug:
• Intravenous (IV) citrulline

Locations

Country	Name	City	State
United States	University of Mississippi Medical Center	Jackson	Mississippi

Sponsors (1)

Lead Sponsor	Collaborator
Suvankar Majumdar

Country where clinical trial is conducted

United States, 

References & Publications (1)

Barr FE, Tirona RG, Taylor MB, Rice G, Arnold J, Cunningham G, Smith HA, Campbell A, Canter JA, Christian KG, Drinkwater DC, Scholl F, Kavanaugh-McHugh A, Summar ML. Pharmacokinetics and safety of intravenously administered citrulline in children undergoing congenital heart surgery: potential therapy for postoperative pulmonary hypertension. J Thorac Cardiovasc Surg. 2007 Aug;134(2):319-26. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants With Grade 2 or Higher Adverse Event According to NCI Criteria		12 months