Cystic Fibrosis
Conditions
Keywords
Cystic Fibrosis, Pseudomonas aeruginosa, Pulmonary CF
Brief summary
The primary objective of this study is to evaluate the safety and tolerability of a single dose of KB001 in Cystic Fibrosis patients infected with Pseudomonas aeruginosa (Pa)
Detailed description
CF patients often have lung infections. Pseudomonas aeruginosa (Pa) is the most significant bacteria in CF, with up to 80% of CF patients eventually becoming infected. These patients are often treated with antibiotics with variable results. This study will examine the impact of a single-dose of an investigational drug, KB001, on Pa bacteria in the CF lung.
Interventions
BIOLOGICALKB001
Single-dose, 3mg/kg or 10mg/kg dose administered intravenously
OTHERPlacebo
Placebo single-dose administered intravenously
Sponsors
Humanigen, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Written informed consent * Confirmed diagnosis of CF based on the following criteria: Positive sweat chloride greater than 60mEq/liter, and/or a genotype with 2 identifiable mutations consistent with CF, and one or more clinical features consistent with the CF phenotype * Age \>/= 18 years of age with the potential for age 12 \>/= * Screening Pa sputum culture * FEV1% predicted \>/= 40 (based on Wang's equations for Males aged 12-17 and females 12-15 years, and Hankinson's equations for all other patients)
Exclusion criteria
* Evidence of an acute respiratory infection or pulmonary exacerbation within 4 weeks prior to day 0 * Use of systemic corticosteroids or antibiotics within 4 weeks prior to day 0, or cyclic inhaled antibiotics within 14 days prior to day 0. * History of positive B. cepacia complex, organ transplantation, hepatic disease or venal dysfunction * Current cigarette smoker, history of drug addiction or alcohol abuse * Use of investigational medication or participation in an investigational study within 4 weeks prior to day 0 * Women who are pregnant or breastfeeding and patients and/or partner unwilling to use an effective form of barrier contraception throughout the study
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The Safety and Tolerability of a Single-dose of KB001. | Day 28 | Safety assessments were conducted after completion of day 28. AEs were followed through completion of day 56. |
Countries
United States
Participant flow
Recruitment details
This was a multicenter, randomized, double-blind, placebo-controlled safety and PK study.
Pre-assignment details
Two cohorts of subjects were randomized 2:1 and received a single intravenous (IV) infusion of KB001 or Placebo. Cohort 1 received KB001 at 3 mg/kg. Cohort 2 was enrolled at 10 mg/kg. The Placebo groups were combined for analysis.
Participants by arm
| Arm | Count |
|---|---|
| Placebo | 9 |
| KB001, 3 mg/kg | 10 |
| KB001, 10 mg/kg | 8 |
| Total | 27 |
Baseline characteristics
| Characteristic | Placebo | KB001, 3 mg/kg | KB001, 10 mg/kg | Total |
|---|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 2 Participants | 0 Participants | 2 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 9 Participants | 8 Participants | 8 Participants | 25 Participants |
| Region of Enrollment United States | 9 participants | 10 participants | 8 participants | 27 participants |
| Sex: Female, Male Female | 2 Participants | 5 Participants | 2 Participants | 9 Participants |
| Sex: Female, Male Male | 7 Participants | 5 Participants | 6 Participants | 18 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — | — / — |
| other Total, other adverse events | 7 / 9 | 10 / 10 | 8 / 8 |
| serious Total, serious adverse events | 1 / 9 | 1 / 10 | 0 / 8 |
Outcome results
Primary
The Safety and Tolerability of a Single-dose of KB001.
Safety assessments were conducted after completion of day 28. AEs were followed through completion of day 56.
Time frame: Day 28
Population: Safety population: all subjects randomized and receiving any study medication.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Placebo | The Safety and Tolerability of a Single-dose of KB001. | 7 Number of participants experiencing AEs |
| KB001, 3 mg/kg | The Safety and Tolerability of a Single-dose of KB001. | 10 Number of participants experiencing AEs |
| KB001, 10 mg/kg | The Safety and Tolerability of a Single-dose of KB001. | 8 Number of participants experiencing AEs |