Safety and Tolerability Study of Liquid Alpha1 Proteinase Inhibitor (API) in Subjects With Cystic Fibrosis

CSL Behring

Status and phase: Completed; Phase 1

Conditions: Cystic Fibrosis

Treatments: Biological: Placebo; Biological: CR002 Liquid API

Study type: Interventional

Funder types: Industry

Identifiers

NCT01347190

CR002_1004

2010-022671-60 (EudraCT Number)

Details and patient eligibility

About

Study to assess safety and tolerability of a single dose of study-drug administered to Cystic Fibrosis (CF) patients.

Enrollment: 25 patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Have a confirmed diagnosis of CF: one or more clinical findings consistent with CF and at least one of the following:

  1. Sweat chloride ≥ 60 mEq/L by quantitative pilocarpine iontophoresis test OR
  2. A genotype with two identifiable mutations consistent with CF (∆F508 homozygous or two alleles known to cause a class I, II, or III mutation)

• Have an FEV1 ≥30% of predicted normal as defined by age, gender, and height

Exclusion criteria

• Oxygen saturation <90%
• Changed in treatment regimen within 2 weeks prior to screening
• Antibiotics regimen change < 4 weeks before screening
• Persistent colonization with Burkholderia cepacia
• Serum IgA < 50% of lower level of normal

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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25 participants in 2 patient groups, including a placebo group

Liquid API

Experimental group

Treatment:

Biological: CR002 Liquid API

Placebo

Placebo Comparator group

Treatment:

Biological: Placebo