Study on Doses of Inhaled ALX1 in Adults With Bronchiectasis
A Phase 2a, Placebo-Controlled, Single-Blind, Dose Range-Finding Study of Inhaled ALX1 in Adults With Bronchiectasis
Study Overview
Status
Status
Conditions
Conditions
Intervention / Treatment
Intervention / Treatment
Detailed Description
Study Type
Study Type
Enrollment (Estimated)
Enrollment
Phase
Phase
- Phase 2
Contacts and Locations
Study Contact
Study Contact
- Name: Paul Bruinenberg, MD
- Phone Number: +1 201-312-0988
- Email: pbruinenberg@vasttherapeutics.com
Study Contact Backup
- Name: Laura MacLean
- Email: LMacLean@vasttherapeutics.com
Participation Criteria
Eligibility Criteria
Eligibility Criteria
Ages Eligible for Study
- Adult
- Older Adult
Accepts Healthy Volunteers
Description
Inclusion Criteria:
- Current sputum producer with a history of chronic expectoration that, in the opinion of the Investigator, will be able to continue to reliably provide sputum throughout the study.
- Confirmed diagnosis of BE per high-resolution computed tomography (HRCT) prior to Screening due to any of the following: NCFB, CF, primary ciliary dyskinesia, or COPD.
- Clinical history consistent with BE (cough, daily chronic sputum production, and/or recurrent respiratory infections).
- FEV1 ≥ 40% of predicted values at Screening.
- Able to reproducibly perform spirometry manoeuvres (i.e., able to perform at least 3 acceptable forced expiratory curves based on the PI's assessment).
- History of at least one exacerbation treated with a course of antibiotics (inhaled, oral or intravenous [IV]) within the 24 months prior to Screening
- Woman of childbearing potential (WOCBP) or fertile man (see definitions in Section 5.3) agrees to use an acceptable method of contraception from the start of Screening until 90 days after the last dose of IP.
Exclusion Criteria:
- Negative sputum NEATstik result for neutrophil elastase at Pre-screening.
- History of Burkholderia cepacia complex within 2 years prior to Pre-screening and/or detection of any Burkholderia spp. in sputum culture or by polymerase chain reaction (PCR) at Screening.
- History of Aspergillus fumigatus requiring treatment within 12 months prior to Pre-screening.
- History of non-tuberculosis mycobacteria (NTM) infection requiring treatment within 12 months prior to Screening, or detection of one or more NTM species in sputum by PCR at Screening.
- History of bronchospasm with inhaled antibiotics or hypertonic saline.
- Haemoptysis exceeding 50 mL of blood from the respiratory tract at any time within 30 days prior to IP administration (Day 1).
- Initiated macrolide therapy within 90 days before Screening. Existing stable maintenance with inhaled macrolides is permitted if initiated more than 90 days prior to Screening.
- Received inhaled anti-pseudomonal therapy within the last 14 days before Pre-screening. Must be willing to refrain from use of inhaled anti-pseudomonal therapy during the study until completion of the Follow-up video/telephone call.
- Received oral antibiotics other than macrolide within 30 days prior to Screening. Must be willing to refrain from use of oral antibiotics during the study until completion of the Follow-up video/telephone call.
- Received IV antibiotics within 60 days prior to Screening
- Initiation of, or increase in the dose of, inhaled corticosteroids within 90 days prior to Screening. Note: participants may be taking stable inhaled corticosteroids at the time of enrolment but must have initiated treatment more than 90 days prior to Screening
- Started any of the following muco-corrective therapies (e.g., nebulised saline, N-acetyl cysteine, Pulmozyme®, etc.) within 30 days prior to Screening. Maintenance with these muco-corrective therapies is permitted if initiated 30 days prior to Screening.
Any of the following laboratory abnormalities at Screening:
- Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 2.5 × upper limit of normal (ULN)
- Creatinine > 1.5 × ULN
- QT interval corrected by Fridericia's formula (QTcF) interval > 450 ms for males or > 470 ms for females at Screening, or history of prolonged QT syndrome. PR interval < 200 ms at Screening. Out-of-range values may be repeated twice for confirmation. The mean QTcF and PR intervals of the triplicate ECG recordings will be used to determine qualification.
- Positive test for hepatitis C antibody (HCV), hepatitis B surface antigen (HBsAg), or human immunodeficiency virus (HIV) antibody at Screening
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Single
Number of Arms
Arms and Interventions
Participant Group / ArmParticipant Group / Arm |
Intervention / TreatmentIntervention / Treatment |
|---|---|
|
Placebo Comparator: Placebo
|
Dose Formulation: Solution for inhalation Dose Strength: 0.9% sodium chloride Route of Administration: Inhalation via nebulizer
|
|
Experimental: ALX1
|
Dose Formulation: Solution for inhalation Dose Strength: 28 mg/mL Route of Administration: Inhalation via nebulizer
|
What is the study measuring?
Primary Outcome Measures
Primary Outcome Measures
Outcome Measure |
Time Frame |
|---|---|
|
Proportion of participants with a metHb value ≥ 5% per dose level
Time Frame: From Day 1 to Day 14 (EOT visit)
|
From Day 1 to Day 14 (EOT visit)
|
Secondary Outcome Measures
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
|
Incidence of TEAEs
Time Frame: From Day 1 to Day 14 (EOT visit)
|
From Day 1 to Day 14 (EOT visit)
|
|
|
Incidence of SAEs
Time Frame: From Day 1 to Day 14 (EOT visit)
|
From Day 1 to Day 14 (EOT visit)
|
|
|
Proportion of participants with abnormal vital signs
Time Frame: From Day 1 to Day 14 (EOT visit)
|
From Day 1 to Day 14 (EOT visit)
|
|
|
Proportion of participants with abnormal Laboratory parameters
Time Frame: From Day 1 to Day 14 (EOT visit)
|
From Day 1 to Day 14 (EOT visit)
|
|
|
Proportion of participants with abnormal ECG readings
Time Frame: From Day 1 to Day 14 (EOT visit)
|
From Day 1 to Day 14 (EOT visit)
|
|
|
Proportion of participants with abnormal SpO2
Time Frame: From Day 1 to Day 14 (EOT visit)
|
From Day 1 to Day 14 (EOT visit)
|
|
|
Proportion of participants with abnormal Spirometry Value
Time Frame: From Day 1 to Day 14 (EOT visit)
|
From Day 1 to Day 14 (EOT visit)
|
|
|
Mean change in sputum inflammatory biomarkers per dose level
Time Frame: From Day 1 to Day 14 (EOT visit)
|
Active neutrophil elastase, IL-1β, IL-6, IL-8, and TNF-alpha biomarkers assessed via quantitative immunoassay
|
From Day 1 to Day 14 (EOT visit)
|
|
Mean change in total bacterial load of pathogens per dose level
Time Frame: From Day 1 to Day 14 (EOT visit)
|
Assessed by sputum culture and quantitative polymerase chain reaction (qPCR)
|
From Day 1 to Day 14 (EOT visit)
|
|
Proportion of participants achieving a microbiological culture of pathogens change of at least 1-log CFU/g per dose level
Time Frame: From Day 1 to Day 14 (EOT visit)
|
From Day 1 to Day 14 (EOT visit)
|
Collaborators and Investigators
Sponsor
Sponsor
Study record dates
Study Major Dates
Study Start (Estimated)
Study Start
Primary Completion (Estimated)
Primary Completion
Study Completion (Estimated)
Study Completion
Study Registration Dates
First Submitted
First Submitted
First Submitted That Met QC Criteria
First Submitted That Met QC Criteria
First Posted (Actual)
First Posted
Study Record Updates
Last Update Posted (Actual)
Last Update Posted
Last Update Submitted That Met QC Criteria
Last Update Submitted That Met QC Criteria
Last Verified
Last Verified
More Information
Terms related to this study
Other Study ID Numbers
Other Study ID Numbers
- VST-201
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
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