Ascending Single Dose Study of Rhu-pGelsolin in Patients With Decreased Gelsolin Levels

Critical Biologics

Status and phase: Completed; Phase 2; Phase 1

Conditions: Infection; Trauma; Burns; Low Gelsolin

Treatments: Drug: Placebo; Drug: rhu-pGelsolin

Study type: Interventional

Funder types: Industry

Identifiers

NCT00671307

CBC101

Details and patient eligibility

About

This study is designed to assess the pharmacokinetics and safety of an infusion of a single dose of recombinant plasma gelsolin (rhu-pGelsolin) when given to patients admitted to the Intensive Care Unit with documented low levels of natural gelsolin. It is believed that this drug will raise the gelsolin levels in these patients and decrease the probability that they will develop complications from their underlying disease such as organ system failure or death.

Full description

This is a randomized, double-blind, placebo-controlled, ascending single dose of rhu-pGelsolin infusion study. Thirteen subjects (10 active and 3 placebo) will be dosed at 3 mg/kg and 5 subjects (3 active and 2 placebo) will be dosed at 6 mg/kg. In addition, twenty-two subjects (18 active and 4 placebo) will be dosed at 6 mg/kg per day for 3 days. The Data Safety Monitoring Committee (DSMC) and Steering Committee will review the preceding dose safety data, and agree the tolerability prior to the enrolment for next higher dose. Blood samples will be collected during 1 hour infusion through 72 hours post dose for PK assessment after each dose.

Enrollment: 40 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• >18 years of age
• Documented gelsolin level <100 mg/mL
• Admission to ICU
• Women of child-bearing age have a negative pregnancy test
• Multiple Organ Failure score < 4
• Catheter present through which blood samples can be taken
• Written Informed Consent obtained

Exclusion criteria

• Participation in other investigational treatment protocols
• Patients <18 years of age
• Patients who have a modified Multiple Organ Failure score of >=4
• Patient, who in the opinion of the Principal Investigator, are unlikely to be in the ICU for >48 hours

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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40 participants in 4 patient groups, including a placebo group

Placebo

Placebo Comparator group

Description:

Placebo

Treatment:

Drug: Placebo

Low dose

Experimental group

Description:

3 mg/kg of rhu-pGelsolin given as an IV infusion over 1 hour

Treatment:

Drug: rhu-pGelsolin

Mid-dose

Experimental group

Description:

6 mg/kg of rhu-pGelsolin given as an IV infusion over 1 hour

Treatment:

Drug: rhu-pGelsolin

High dose

Experimental group

Description:

6 mg/kg of rhu-pGelsolin given a an IV infusion over 1 hour once a day for 3 days

Treatment:

Drug: rhu-pGelsolin