Trial Outcomes & Findings for A Dose Escalation Study of Intravenous L-citrulline in Steady-state Sickle Cell Disease (NCT NCT02314689)

Last Updated: 2019-09-03

Results Overview

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

4 participants

Primary outcome timeframe

12 months

Results posted on

2019-09-03

Participant milestones
Measure	Intravenous (IV) Citrulline Intravenous (IV) citrulline 20 mg/kg bolus with dose escalation of 10 mg/kg to target citrulline concentration of 100 µmol/L with a maximum dose of 60 mg/kg.
Overall Study STARTED	4
Overall Study COMPLETED	4
Overall Study NOT COMPLETED	0

Withdrawal data not reported

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

Baseline characteristics by cohort
Measure	Intravenous (IV) Citrulline n=4 Participants Intravenous (IV) citrulline 20 mg/kg bolus with dose escalation of 10 mg/kg to target citrulline concentration of 100 µmol/L with a maximum dose of 60 mg/kg.
Age, Continuous	22.8 years n=99 Participants
Sex: Female, Male Female	2 Participants n=99 Participants
Sex: Female, Male Male	2 Participants n=99 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=99 Participants
Race (NIH/OMB) Asian	0 Participants n=99 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=99 Participants
Race (NIH/OMB) Black or African American	4 Participants n=99 Participants
Race (NIH/OMB) White	0 Participants n=99 Participants
Race (NIH/OMB) More than one race	0 Participants n=99 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants
Region of Enrollment United States	4 participants n=99 Participants

Timeframe: 12 months

Outcome measures
Measure	IV Citrulline n=4 Participants IV citrulline 20 mg/kg bolus with dose escalation of 10 mg/kg to target citrulline concentration of 100 µmol/L with a maximum dose of 60 mg/kg. Intravenous (IV) citrulline
Number of Participants With Grade 2 or Higher Adverse Event According to NCI Criteria	0 participants

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Adverse event data not reported

Other adverse events
Measure	Intravenous (IV) Citrulline n=4 participants at risk Intravenous (IV) citrulline 20 mg/kg bolus with dose escalation of 10 mg/kg to target citrulline concentration of 100 µmol/L with a maximum dose of 60 mg/kg.
Vascular disorders Greater than 20% drop in blood pressure	25.0% 1/4 • Number of events 1 • 1 week

University of Mississippi Medical Center

Phone: 6019845220