Ph 1/2 Study Evaluating Safety and Tolerability of Inhaled AP-PA02 in Subjects With Chronic Pseudomonas Aeruginosa Lung Infections and Cystic Fibrosis

NCT04596319 | Completed Phase 1 Results DMC FDA Drug

• 1 other identifier: AP-PA02-101
• Study Type: interventional
• Target: 29
• Locations: 1 country
• Sites: 20

Brief Summary

Phase 1b/2a, double-blind, randomized, placebo-controlled, single and multiple ascending dose study to evaluate the safety, tolerability and phage recovery profile of AP-PA02 multi-bacteriophage therapeutic candidate administered by inhalation in subjects with cystic fibrosis and chronic pulmonary Pseudomonas aeruginosa (PA) infection.

Conditions

Cystic Fibrosis; Pseudomonas Aeruginosa; Pseudomonas; Lung Infection; Lung Infection Pseudomonal

Keywords

phage; bacteriophage; cystic fibrosis; Pseudomonas; Pseudomonas aeruginosa

Outcome Measures

Primary Outcomes (1)

• Incidence and Severity Treatment Emergent Adverse Events (TEAEs)

  Incidence and severity of treatment emergent adverse events of single and multiple doses of AP-PA02 administered by inhalation

  Day 1 pre-dose through End of Study Visit (28 days post last dose of study drug), up to 4 weeks for single ascending dose and up to 5.5 weeks for multiple ascending dose.

Study Arms (2)

AP-PA02

EXPERIMENTAL

Anti-pseudomonal bacteriophage

Biological: AP-PA02

Placebo

PLACEBO COMPARATOR

Inactive isotonic solution

Other: Placebo

Interventions

AP-PA02 BIOLOGICAL

Bacteriophage administered via inhalation

AP-PA02

Placebo OTHER

Inactive Placebo administered via inhalation

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• ≥ 18 years old
• Body mass index (BMI) of ≥ 18 kg/m2
• Documented diagnosis of CF
• Evidence of chronic pulmonary Pseudomonas aeruginosa infection
• Willing to undergo sputum induction procedures at designated study visits, and willing to provide expectorated sputum samples at all other timepoints (for subjects who are able to expectorate)
• For SAD: FEV1 ≥ 60% of predicted normal \[per Global Lung Function Initiative (GLI) standards\] at Screening
• For MAD: FEV1 ≥ 40% of predicted normal \[per Global Lung Function Initiative (GLI) standards\] at Screening
• Adequate renal function

You may not qualify if:

• Recent significant weight loss
• Abnormal vital signs at Screening
• History of prolonged QT syndrome
• Use of supplemental oxygen during the day at rest
• Abnormal liver function tests greater than 3X the upper limit of normal (ULN)
• Recent oral or IV antibiotics received for acute pulmonary exacerbation. Inhaled antibiotic use for chronic suppression of P. aeruginosa is acceptable.
• Recent clinically significant infection requiring systemic antimicrobial therapy
• Currently receiving anti-pseudomonal antibiotic treatment for acute sinusitis.
• Currently receiving systemic corticosteroids
• Currently receiving treatment for active infection with nontuberculous mycobacteria (NTM), Staphylococcus aureus, or Burkholderia cepacia complex lung infection
• Currently receiving treatment for aspergillosis or ABPA (allergic bronchopulmonary aspergillosis)
• Initiation of a CFTR potentiator/corrector therapy, such as Trikafta®, less than 90 days prior to Screening
• Acquired or primary immunodeficiency syndromes
• Active pulmonary malignancy (primary or metastatic)
• History of lung transplantation

Sponsors & Collaborators

• Armata Pharmaceuticals, Inc.: lead
• Cystic Fibrosis Foundation: collaborator

Study Sites (20)

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

University of South Florida

Tampa, Florida, 33606, United States

Location

St. Luke's Cystic Fibrosis Center of Idaho

Boise, Idaho, 83712, United States

Location

Northwestern University

Chicago, Illinois, 60208, United States

Location

University of Iowa

Iowa City, Iowa, 52242, United States

Location

The University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

Johns Hopkins University

Baltimore, Maryland, 21205, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Harper University Hospital

Detroit, Michigan, 48201, United States

Location

Rutgers Robert Wood Johnson Medical School

New Brunswick, New Jersey, 08901, United States

Location

New York Medical College

Valhalla, New York, 10595, United States

Location

University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44106, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

The Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

University of Texas Southwestern

Dallas, Texas, 75390, United States

Location

University of Washington

Seattle, Washington, 98195, United States

Location

University of Wisconsin

Madison, Wisconsin, 53792-9988, United States

Location

Related Links

• Armata Pharmaceuticals, Inc.

MeSH Terms

Conditions

Cystic Fibrosis; Pseudomonas Infections

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases; Gram-Negative Bacterial Infections; Bacterial Infections; Bacterial Infections and Mycoses; Infections

Results Point of Contact

• Title: Mina Pastagia, MD
• Organization: Armata Pharmaceuticals, Inc.

mpastagia@armatapharma.com

(310) 665-2928

Study Officials

• Mina Pastagia, MD, MS

  Armata Pharmaceuticals, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Randomized, double-blind, placebo-controlled

Study Record Dates

• First Submitted: October 15, 2020
• First Posted: October 22, 2020
• Study Start: December 22, 2020
• Primary Completion: December 14, 2022
• Study Completion: December 14, 2022
• Last Updated: January 31, 2024
• Results First Posted: January 31, 2024

Record last verified: 2024-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (20)