Aztreonam Aerosol to Treat Cystic Fibrosis Nasal Disease

NCT02730793 | Terminated Phase 2 Results FDA Drug

• 1 other identifier: HM20005657
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

Brief Summary

This study is designed as a masked, two center, randomized, placebo-controlled pilot study to evaluate the safety and efficacy of nasal and oral inhalation of 75 mg aztreonam in subjects with CF and lung infection due to PA. The study will involve two sites: Virginia Commonwealth University Medical Center (VCU) and Eastern Virginia Medical School (EVMS). Potential subjects will be identified in each site's CF clinic.

Conditions

Cystic Fibrosis

Keywords

Cystic Fibrosis; P. aeruginosa (PA)

Outcome Measures

Primary Outcomes (1)

• Number of Protocol-defined Pulmonary Exacerbations Treated With IV Anti-pseudomonal Antibiotics on Day 140

  Protocol-Defined Pulmonary Exacerbation includes events that are characterized by change or worsening pulmonary symptoms (increased cough, increased sputum production and chest congestion, decreased appetite and exercise tolerance), loss of weight, and lung function decline that prompt initation of antibiotic therapy. Protocol-defined exacerbations in subjects that warrant treatment with IV antibiotics will be determined from the medical record during the course of this study.

  140 days

Secondary Outcomes (8)

• Time to First Protocol-defined Pulmonary Exacerbation Treated With IV Anti-pseudomonal Antibiotics

  Up to 6 months

• Change in Sinus and Nasal QoL Questionnaire (SNOT-20) on Day 140 and Day 168

  Day 140 and Day 168

• Change in Cystic Fibrosis QoL Score (CFQ-R) on Day 140

  Day 140

• Change in Pulmonary Function (FVC and FEV1 Percent Predicted) on Day 140

  Day 140

• Change in Paired Sputum Cultures and Nasal Swabs for Bacteria and Antibiotic Resistance

  1 year

Study Arms (2)

Standard Therapy

ACTIVE COMPARATOR

Oral Inhalation via PARI Altera Device: Standard Therapy Comparator (Oral Aztreonam 75mg three times a day) AND Nasal Inhalation via PARI PAS Device: Nasal Placebo-Normal Saline twice per day

Drug: Oral Aztreonam; Drug: Nasal Placebo

Study Therapy

EXPERIMENTAL

Oral Inhalation via PARI Altera Device: Standard Therapy Comparator (Oral Aztreonam 75mg three times a day) AND Nasal Inhalation via PARI PAS Device: Experimental- Nasal Aztreonam 75 mg twice per day

Drug: Oral Aztreonam; Drug: Nasal Aztreonam

Interventions

Oral Aztreonam DRUG

Standard Therapy Comparator (Oral Cayston 75mg three times a day)

Also known as: Cayston, Azactam

Standard Therapy; Study Therapy

Nasal Aztreonam DRUG

Study Therapy (nasal Aztreonam 75mg twice per day)

Also known as: Cayston, Azactam

Study Therapy

Nasal Placebo DRUG

Placebo (nasal saline twice per day)

Also known as: Saline

Standard Therapy

Eligibility Criteria

• Age: 7 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Males or females 7 years of age or older and able to perform pulmonary function testing
• Confirmed diagnosis of CF by the 1997 CF Consensus Conference criteria and followed by the VCU or EVMS CF clinic
• Presence of PA in 2 lower respiratory tract (sputum) cultures in the 24 months before screening
• Subjects and/or parent guardian must be able to give written informed consent prior to any study related procedure
• All sexually active female subjects who are of childbearing potential must agree to use an effective method of contraception (i.e.condoms or abstinence).
• All sexually active female subjects must have a negative pregnancy test at screening (V0).
• Clinically stable determined by the study physician with no significant new respiratory symptoms.
• Presence of PA in nasal culture (swab or secretion) or sinus culture obtained in the 12 months before screening or at screening visit

You may not qualify if:

• Use of oral, IV or inhaled antibiotics within 0 days before study other than low dose azithromycin
• Severe pulmonary disease with FEV1\<30% predicted of baseline SpO2\<0.90
• ENT surgery within 6 months of screening
• Allergy or documented adverse reaction to aztreonam
• Epistaxis or significant (\>30mL) hemoptysis in the past 6 months
• Frequent (weekly or more frequently) or severe headaches
• Subject is unlikely to comply with the procedures scheduled in the protocol
• Subject participates in another clinical trial within 30 days prior to study entry
• Subjects who have had a lung transplant will be excluded
• Prisoners will be excluded
• Non-English Speaking patients will be excluded

Sponsors & Collaborators

• Virginia Commonwealth University: lead
• Eastern Virginia Medical School: collaborator

Study Sites (1)

Children's Hospital of Richmond at VCU

Richmond, Virginia, 23298, United States

Location

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

Aztreonam; Sodium Chloride

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

Intervention Hierarchy (Ancestors)

Monobactams; beta-Lactams; Lactams; Amides; Organic Chemicals; Sulfur Compounds; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Chlorides; Hydrochloric Acid; Chlorine Compounds; Inorganic Chemicals; Sodium Compounds

Results Point of Contact

• Title: Bruce K. Rubin MEngr, MD, MBA, FRCPC
• Organization: Virginia Commonwealth University

bruce.rubin@vcuhealth.org

804 828 9604

Study Officials

• Bruce K Rubin, MD

  Virginia Commonwealth University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 3, 2015
• First Posted: April 6, 2016
• Study Start: January 1, 2017
• Primary Completion: September 19, 2019
• Study Completion: September 19, 2019
• Last Updated: January 28, 2021
• Results First Posted: January 12, 2021

Record last verified: 2021-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)