Trial Outcomes & Findings for A Phase 1 Study of Continuous Intravenous L-citrulline During Sickle Cell Pain Crisis or Acute Chest Syndrome (NCT NCT02697240)

Results Overview

The number and severity of adverse event will be determined according to the NCI Common Terminology Criteria for Adverse Events (CTCAE)

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

4 participants

Primary outcome timeframe

30 days

Results posted on

2019-06-25

Participant Flow

Participant milestones

Participant milestones
Measure	Intravenous Citrulline Intravenous citrulline at a bolus dose over 5 minutes and then a continuous rate for the next 23 hours. Intravenous citrulline: Intravenous citrulline at a bolus dose of 20 mg/kg over 5 minutes and then continuous at a rate of 7 mg/kg/hour for the next 23 hours for the first 7 participants, and depending on safety and pharmacokinetic profile, increase or decrease the continuous rate to 9 or 5 mg/kg/hour for the next 7 participants.
Overall Study STARTED	4
Overall Study COMPLETED	4
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Intravenous Citrulline n=4 Participants Intravenous citrulline at a bolus dose over 5 minutes and then a continuous rate for the next 23 hours. Intravenous citrulline: Intravenous citrulline at a bolus dose of 20 mg/kg over 5 minutes and then continuous at a rate of 7 mg/kg/hour for the next 23 hours for the first 7 participants, and depending on safety and pharmacokinetic profile, increase or decrease the continuous rate to 9 or 5 mg/kg/hour for the next 7 participants.
Age, Categorical <=18 years	0 Participants n=4 Participants
Age, Categorical Between 18 and 65 years	4 Participants n=4 Participants
Age, Categorical >=65 years	0 Participants n=4 Participants
Age, Continuous	19.5 years n=4 Participants
Sex: Female, Male Female	3 Participants n=4 Participants
Sex: Female, Male Male	1 Participants n=4 Participants
Region of Enrollment United States	4 Participants n=4 Participants

PRIMARY outcome

Timeframe: 30 days

The number and severity of adverse event will be determined according to the NCI Common Terminology Criteria for Adverse Events (CTCAE)

Outcome measures

Outcome measures
Measure	Intravenous Citrulline n=4 Participants Intravenous citrulline at a bolus dose over 5 minutes and then a continuous rate for the next 23 hours. Intravenous citrulline: Intravenous citrulline at a bolus dose of 20 mg/kg over 5 minutes and then continuous at a rate of 7 mg/kg/hour for the next 23 hours for the first 7 participants, and depending on safety and pharmacokinetic profile, increase or decrease the continuous rate to 9 or 5 mg/kg/hour for the next 7 participants.
Number of Participants With Adverse Events	4 Participants

PRIMARY outcome

Timeframe: Plasma citrulline levels will be evaluated at the following time points: trough level, 10 minutes (peak level), 30 minutes, 1 hour, 2 hours, 3 hours, 4 hours, 6 hours, 8 hours, 12 hours, 24 hours and 48 hours, to evaluate the pharmacokinetic profile

Outcome measures

Outcome measures
Measure	Intravenous Citrulline n=4 Participants Intravenous citrulline at a bolus dose over 5 minutes and then a continuous rate for the next 23 hours. Intravenous citrulline: Intravenous citrulline at a bolus dose of 20 mg/kg over 5 minutes and then continuous at a rate of 7 mg/kg/hour for the next 23 hours for the first 7 participants, and depending on safety and pharmacokinetic profile, increase or decrease the continuous rate to 9 or 5 mg/kg/hour for the next 7 participants.
Plasma Citrulline Level	5 micromol/hr/kg Standard Deviation 2.2

Adverse Events

Intravenous Citrulline

Serious events: 1 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Intravenous Citrulline n=4 participants at risk Intravenous citrulline at a bolus dose over 5 minutes and then a continuous rate for the next 23 hours. Intravenous citrulline: Intravenous citrulline at a bolus dose of 20 mg/kg over 5 minutes and then continuous at a rate of 7 mg/kg/hour for the next 23 hours for the first 7 participants, and depending on safety and pharmacokinetic profile, increase or decrease the continuous rate to 9 or 5 mg/kg/hour for the next 7 participants.
Hepatobiliary disorders transaminitis	25.0% 1/4 • Number of events 1 • Adverse events were collected up to 4 weeks from study drug administration

Other adverse events

Adverse event data not reported

Additional Information

Suvankar Majumdar

Children's National Medical Center

Phone: 2024763800

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place