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This study will evaluate safety and tolerability of ascending doses of nebulized KB407 in adults with cystic fibrosis.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1 (open label) | Experimental | A single administration of KB407 |
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| Cohort 2 (open label) | Experimental | Two administrations of KB407 |
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| Cohort 3 (open label) | Experimental | Four administrations of KB407 |
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| Cohort 4 (open label) | Experimental | Four consecutive administrations of KB407 followed by weekly administration for up to 6 months |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| KB407 (Nebulization) | Biological | Nebulized solution of KB407, a replication-defective HSV-1 expressing full length human CFTR |
|
| Measure | Description | Time Frame |
|---|---|---|
| To evaluate safety and tolerability of KB407 based upon assessment of adverse events (frequency, severity, relatedness), and changes from baseline in physical examinations, vital signs, ECG, and clinical laboratory test results | Number of adult subjects with treatment related adverse events as assessed by NCI-CTCAE v5 | 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| To evaluate the effects of KB407 on pulmonary function, as measured by change from baseline in absolute and percent predicted FEV1 | Assessment of forced expiratory volume, in one second (FEV1,) will be assessed by spirometry as compared to baseline | 6 months |
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Inclusion Criteria:
The subject must have read, understood, and signed an Institutional Review Board/Ethics Committee (IRB/IEC) approved Informed Consent Form and must be able to and willing to follow study procedures and instructions
Subjects aged 18 years or older at the time of Informed Consent
A confirmed diagnosis of CF as defined by clinical signs and symptoms of CF and at least one of the following:
Clinically stable in the opinion of the Investigator
Percent predicted FEV1 ≥40% and ≤90% of the predicted normal for age, gender, and height at Screening
Resting oxygen saturation ≥92% on room air at Screening
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| David Sweet, MD, PhD | Contact | 412-586-5830 | dsweet@krystalbio.com | |
| Brittani Agostini, RN, CCRC | Contact | 412-586-5830 | bagostini@krystalbio.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Florida, Gainesville | Recruiting | Gainesville | Florida | 32610 | United States |
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| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
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| St. Lukes CF Center of Idaho | Recruiting | Boise | Idaho | 83712 | United States |
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| The Cystic Fibrosis Institute | Recruiting | Northfield | Illinois | 60093 | United States |
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| New York Medical College/ Boston Children's Health Physicians | Recruiting | Hawthorne | New York | 10532 | United States |
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| Northwell Health Physicians | Recruiting | New York | New York | 10028 | United States |
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| Atrium Health Wake Forest Baptist Medical Center | Recruiting | Winston-Salem | North Carolina | 27157 | United States |
|
| D030342 |
| Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |