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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)
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 1 | Experimental | KB001, a monoclonal antibody |
|
| 2 | Placebo Comparator | Placebo |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| KB001 | Biological | Single-dose, 3mg/kg or 10mg/kg dose administered intravenously |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Day 28 |
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Inclusion 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
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Carlos Milla, MD | Stanford University | Principal Investigator |
| Nestor A. Molfino, MD, MSc | Humanigen, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama | Birmingham | Alabama | 35233 | United States | ||
| Lucille Packard Children's Hospital at Stanford |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 24019259 | Derived | Milla CE, Chmiel JF, Accurso FJ, VanDevanter DR, Konstan MW, Yarranton G, Geller DE; KB001 Study Group. Anti-PcrV antibody in cystic fibrosis: a novel approach targeting Pseudomonas aeruginosa airway infection. Pediatr Pulmonol. 2014 Jul;49(7):650-8. doi: 10.1002/ppul.22890. Epub 2013 Sep 9. |
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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.
This was a multicenter, randomized, double-blind, placebo-controlled safety and PK study.
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| ID | Title | Description |
|---|---|---|
| FG000 | Placebo | |
| FG001 | KB001, 3 mg/kg | |
| FG002 | KB001, 10 mg/kg |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Placebo | Other | Placebo single-dose administered intravenously |
|
| Stanford |
| California |
| 94305 |
| United States |
| University of Colorado | Aurora | Colorado | 80045 | United States |
| Johns Hopkins University | Baltimore | Maryland | 21287 | United States |
| University of Minnesota | Minneapolis | Minnesota | 55455 | United States |
| St. Louis Children's Hospital | St Louis | Missouri | 63110 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| Case Western Reserve University | Cleveland | Ohio | 44106 | United States |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States |
| Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15213 | United States |
| University of Utah | Salt Lake City | Utah | 84132 | United States |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | Placebo | |
| BG001 | KB001, 3 mg/kg | |
| BG002 | KB001, 10 mg/kg | |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
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| Sex: Female, Male | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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. | Safety population: all subjects randomized and receiving any study medication. | Posted | Number | Number of participants experiencing AEs | Day 28 |
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|
56 Days
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Placebo | 1 | 9 | 7 | 9 | |||
| EG001 | KB001, 3 mg/kg | 1 | 10 | 10 | 10 | |||
| EG002 | KB001, 10 mg/kg | 0 | 8 | 8 | 8 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Bronchitis | Infections and infestations | MedDRA (10.1) | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA (10.1) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal Pain Upper | Gastrointestinal disorders | MedDRA (10.1) | Systematic Assessment | Stomach pain |
|
| Anxiety | Psychiatric disorders | MedDRA (10.1) | Systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA (10.1) | Systematic Assessment | Exacerbation of asthma |
|
| Blood Pressure Increased | Investigations | MedDRA (10.1) | Systematic Assessment |
| |
| Breath Sounds Abnormal | Investigations | MedDRA (10.1) | Systematic Assessment |
| |
| Cheilitis | Gastrointestinal disorders | MedDRA (10.1) | Systematic Assessment | Angular stromatitis |
|
| Chest Discomfort | General disorders | MedDRA (10.1) | Systematic Assessment | Chest soreness |
|
| Chills | General disorders | MedDRA (10.1) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (10.1) | Systematic Assessment |
| |
| Crepitations | General disorders | MedDRA (10.1) | Systematic Assessment |
| |
| Cystic Fibrosis Lung | Congenital, familial and genetic disorders | MedDRA (10.1) | Systematic Assessment | Cystic Fibrosis pumonary exacerbation |
|
| Depression | Psychiatric disorders | MedDRA (10.1) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (10.1) | Systematic Assessment |
| |
| Dysphonia | Respiratory, thoracic and mediastinal disorders | MedDRA (10.1) | Systematic Assessment | Hoarse voice |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (10.1) | Systematic Assessment | Shortness of breath |
|
| Fatigue | General disorders | MedDRA (10.1) | Systematic Assessment |
| |
| Flushing | Vascular disorders | MedDRA (10.1) | Systematic Assessment |
| |
| Forced Expiratory Volume Decreased | Investigations | MedDRA (10.1) | Systematic Assessment |
| |
| Haemoptysis (hemoptysis) | Respiratory, thoracic and mediastinal disorders | MedDRA (10.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (10.1) | Systematic Assessment | Intermittent headache |
|
| Heart Rate Increased | Investigations | MedDRA (10.1) | Systematic Assessment |
| |
| Infusion Related Reaction | General disorders | MedDRA (10.1) | Systematic Assessment | Adverse infusion reaction |
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| Infusion Site Discolouration | General disorders | MedDRA (10.1) | Systematic Assessment |
| |
| Infusion Site Rash | General disorders | MedDRA (10.1) | Systematic Assessment | Faint scattered erythematous macules at prior infusion site |
|
| Irritability | General disorders | MedDRA (10.1) | Systematic Assessment |
| |
| Nasal Congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (10.1) | Systematic Assessment | Increased nasal congestion |
|
| Nausea | Respiratory, thoracic and mediastinal disorders | MedDRA (10.1) | Systematic Assessment | Nausea |
|
| Oral Pruritus | Skin and subcutaneous tissue disorders | MedDRA (10.1) | Systematic Assessment |
| |
| Pancreatitis | Gastrointestinal disorders | MedDRA (10.1) | Systematic Assessment | Pancreatitis, mild |
|
| Pharyngolaryingeal Pain | Respiratory, thoracic and mediastinal disorders | MedDRA (10.1) | Systematic Assessment | Sore throat |
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| Procedural Pain | Injury, poisoning and procedural complications | MedDRA (10.1) | Systematic Assessment | Mouth pain from wisdom tooth extraction |
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| Productive Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (10.1) | Systematic Assessment | Increased sputum production |
|
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (10.1) | Systematic Assessment | Feet itching |
|
| Pyrexia (fever) | General disorders | MedDRA (10.1) | Systematic Assessment |
| |
| Rales | Respiratory, thoracic and mediastinal disorders | MedDRA (10.1) | Systematic Assessment |
| |
| Respiratory Tract Congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (10.1) | Systematic Assessment | Chest congestion |
|
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (10.1) | Systematic Assessment | Runny nose |
|
| Sinus Congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (10.1) | Systematic Assessment |
| |
| Sinus Headache | Nervous system disorders | MedDRA (10.1) | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA (10.1) | Systematic Assessment | Sinus infection |
|
| Stress | Psychiatric disorders | MedDRA (10.1) | Systematic Assessment |
| |
| Sunburn | Injury, poisoning and procedural complications | MedDRA (10.1) | Systematic Assessment |
| |
| Throat Irritation | Respiratory, thoracic and mediastinal disorders | MedDRA (10.1) | Systematic Assessment | Itchy throat |
|
| Tinnitus | Ear and labyrinth disorders | MedDRA (10.1) | Systematic Assessment | Patient hears ear clicking |
|
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA (10.1) | Systematic Assessment | Urticaria right anterior abdomen |
|
| Vomiting | Gastrointestinal disorders | MedDRA (10.1) | Systematic Assessment |
| |
| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA (10.1) | Systematic Assessment |
|
The sponsor has right of first publication of trial results, which will be a joint, multi-center publication of the study results from all appropriate sites contributing data, analysis and comments.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Nestor A. Molfino, MD., MSc | KaloBios Pharmaceuticals, Inc. | 650-243-3103 | nmolfino@kalobios.com |
| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| D011552 | Pseudomonas Infections |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
| D016905 | Gram-Negative Bacterial Infections |
| D001424 | Bacterial Infections |
| D001423 | Bacterial Infections and Mycoses |
| D007239 | Infections |
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| Between 18 and 65 years |
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| >=65 years |
|
| Male |
|