Phase 1
Completed N=4
A Dose Escalation Study of Intravenous L-citrulline in Steady-state Sickle Cell Disease
Source: ClinicalTrials.gov NCT02314689 ↗Enrolled (actual)
4
Serious AEs
0.0%
Results posted
Mar 2016
Primary outcomePrimary: Number of Participants With Grade 2 or Higher Adverse Event According to NCI Criteria — 0 participants
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Grade 2 or Higher Adverse Event According to NCI Criteria |
— | — |
Eligibility Criteria
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 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
Data sourced from ClinicalTrials.gov (NCT02314689). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.