Clinical trial NCT03431077: A Study of LJPC-501 in Pediatric Patients With Hypotension
Official title: An Open-Label, Multi-Center Study of LJPC-501 in Pediatric Patients Who Remain Hypotensive Despite Receiving Fluid Therapy and Vasopressor Therapy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Catecholamine-resistant Hypotension (CRH)
Distributive Shock
High Output Shock
Sepsis
Lead sponsor: La Jolla Pharmaceutical Company
Sponsor class: INDUSTRY
First posted: 2018-02-13
Enrollment: 2
Sex: ALL
Minimum age (years): 2
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The objective of this study is to evaluate the effect of LJPC-501 infusion on mean arterial pressure (MAP) or reduction in sum norepinephrine (NE) equivalent dosing, at Hour 2 after the start of LJPC-501, in pediatric patients who remain hypotensive despite receiving fluid therapy and vasopressor therapy. In addition, this study will evaluate the safety and tolerability of LJPC-501 in pediatric patients, the change in MAP over 24 hours after the start of LJPC-501, the change in serum lactate concentrations, and the change in Pediatric Logistic Organ Dysfunction (PELOD) scores.


Eligibility criteria: Inclusion Criteria:

1. Pediatric patients 2-17 years of age.
2. Patients requiring a total sum NE equivalent dose of \> 0.1 μg/kg/min for a minimum of 2 hours and a maximum of 48 hours prior to initiation of LJPC-501 dosing.
3. Patients must have clinical diagnosis of distributive shock in the opinion of the treating team and the Investigator.
4. Patients are required to have central venous access, which is expected to remain present for the duration of LJPC-501 treatment.
5. Patients are required to have an indwelling arterial line, which is expected to remain present for at least the first 24 hours of LJPC-501 treatment.
6. Patients must have received at least 40 mL/kg of crystalloid or colloid equivalent over the initial 24-hour resuscitation period, and are adequately volume resuscitated in the opinion of the Investigator.
7. Parent or legal guardian is willing and able to provide informed consent and assist the patient in complying with all protocol requirements.

Exclusion Criteria:

1. Patients who are \< 2 years of age or ≥ 18 years of age.
2. Patients with a standing Do Not Resuscitate order.
3. Patients diagnosed with acute occlusive coronary syndrome requiring pending intervention.
4. Patients on veno-arterial (VA) extracorporeal membrane oxygenation (ECMO).
5. Patients who have been on veno-venous (VV) ECMO for less than 6 hours.
6. Patients with a clinical suspicion of cardiogenic shock.
7. Patients who have a history of asthma or are currently experiencing bronchospasm requiring the use of inhaled bronchodilators and who are not mechanically ventilated.
8. Patients with acute mesenteric ischemia or a history of mesenteric ischemia.
9. Patients with active bleeding AND an anticipated need of multiple transfusions (within 48 hours of Screening).
10. Patients with active bleeding AND hemoglobin \< 7 g/dL or any other condition that would contraindicate serial blood sampling.
11. Patients with an absolute neutrophil count (ANC) of \< 500 cells/mm3.
12. Patients requiring more than 5 mg/kg daily of hydrocortisone or equivalent glucocorticoid medication as a standing dose.
13. Patients with an expected lifespan of \< 12 hours or withdrawal of life support within 24 hours of Screening.
14. Patients with a known allergy to mannitol.
15. Patients who are currently participating in another investigational clinical trial.
16. Patients of childbearing potential who are known to be pregnant at the time of Screening.

Primary outcomes:
measure: Effect of LJPC-501 on mean arterial pressure (MAP)
timeFrame: 2 hours
measure: Effect of LJPC-501 on sum norepinephrine (NE) equivalent dose
timeFrame: 2 hours
Secondary outcomes:
measure: Effect of LJPC-501 on the incidence of treatment-emergent adverse events
timeFrame: Day 7
measure: Effect of LJPC-501 on mean arterial pressure (MAP)
timeFrame: 24 hours
measure: Effect of LJPC-501 on serum lactate
timeFrame: 2 hours and 24 hours
measure: Effect of LJPC-501 on Pediatric Logistic Organ Dysfunction (PELOD)
timeFrame: 24 hours
measure: Effect of LJPC-501 on clinical chemistry laboratory parameters
timeFrame: 24 hours
measure: Effect of LJPC-501 on hematology laboratory parameters
timeFrame: 24 hours
measure: Effect of LJPC-501 on blood pressure
timeFrame: Day 7
measure: Effect of LJPC-501 on heart rate
timeFrame: Day 7
measure: Effect of LJPC-501 on body temperature
timeFrame: Hour 24
measure: Effect of LJPC-501 on general health
timeFrame: Day 7
measure: Effect of LJPC-501 on electrical activity of the heart
timeFrame: Hour 24
measure: Effect of LJPC-501 on urine output
timeFrame: Day 7
Other outcomes:
Locations:
city: Charlotte
country: United States
facility: Investigational Site
state: North Carolina
city: Cincinnati
country: United States
facility: Investigational Site
state: Ohio
city: San Antonio
country: United States
facility: Investigational Site
state: Texas
Study officials:
References: