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

Suvankar Majumdar

Status and phase

Completed

Phase 1

Conditions

Sickle Cell Disease

Treatments

Drug: Intravenous (IV) citrulline

Study type

Interventional

Funder types

Other

Identifiers

NCT02314689

SCD-124700.

Details and patient eligibility

About

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.

Enrollment

4 patients

Sex

All

Ages

18 to 25 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Sickle cell disease genotypes (HbSS, HbS/β° thalassemia, HbS/β+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