An Investigator-initiated Study to Evaluate Safety and Efficacy of ARINA-1 in People With a Tracheostomy

NCT05658029 | Withdrawn Phase 1 FDA Drug

• 1 other identifier: WMD-001
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this investigator-initiated, open label study is to evaluate the safety and efficacy of ARINA-1 in people with a tracheostomy. Participants will attend study visits at Screening, Baseline, Day 14, Day 28, and Day 56. There will be 3 safety phone calls at Days 2, 7, and 21. Participants will nebulize the ARINA-1 solution twice daily for 28 days

Conditions

Tracheostomy

Outcome Measures

Primary Outcomes (1)

• Incidence of adverse events / serious adverse events

  Baseline to day 28

Secondary Outcomes (6)

• Change from baseline to day 28 for Composite Tracheostomy Questionnaire and the Clinical Global Impression of Change (CGI-C)

  Baseline to day 28

• Change in mucus rheology

  Baseline to day 28

• Change in bacterial culture at 28 days

  Baseline to day 28

• Changes in blood CRP over 28 days

  Baseline to day 28

• Ventilator settings / respiratory system compliance (for subjects on intermittent chronic ventilatory support)

  Baseline to day 28

Study Arms (1)

Open label treatment group

EXPERIMENTAL

Drug: ARINA-1

Interventions

ARINA-1 DRUG

Fixed-dose solution of ascorbic acid and reduced glutathione nebulized twice daily for 4 weeks (28 days)

Open label treatment group

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Tracheostomy for ≥6 months with no anticipated changes to tracheostomy status within the next 3 months from screening visit
• Males or females 18-75 years old at time of consent
• Willing and able to comply with the protocol and visit schedule
• Subject or legal authorized representative capable of giving informed consent. Determination of subject capacity to give informed consent will be determined by investigators and guided by principles set forth in the Declaration of Helsinki (World Medical Association, 2013) and by WakeMed Health \& Hospitals guidance (Patient Competency and Decisional Capacity - Legal Affairs Tip Sheet/FAQ).

You may not qualify if:

• Inability to speak or understand English
• Positive urine pregnancy test at screening and/or baseline visit, if applicable
• Active breastfeeding status
• Diagnosis of cystic fibrosis or primary ciliary dyskinesia
• History of lung transplant
• Listed for lung transplant
• Inability to tolerate nebulized treatments
• Planned decannulation before completion of this study
• Exacerbation or infections requiring any acute antibiotics, urgent care visit, emergency department visit or hospitalization within 14 days of screening visit
• Previous intolerance to hypertonic saline (HTS)
• Initiating a chronic azithromycin or any new inhaled maintenance therapy \< 28 days prior to baseline visit
• Initiating any N-acetyl-cysteine-containing (NAC), ascorbic acid or glutathione (GSH)-containing therapy (oral or nebulized) \< 28 days prior to baseline visit
• Intolerance to NAC or GSH
• Intolerance to bronchodilator (e.g., Albuterol)
• Significant comorbidities that in the opinion of the investigator would reduce the safety of the subject or interfere with the ability to interpret study data

Sponsors & Collaborators

• Matthew Bruehl: lead
• Renovion, Inc.: collaborator

Study Sites (1)

WakeMed Health & Hospitals

Raleigh, North Carolina, 27610, United States

Location

Study Officials

• Matthew Bruehl, MD

  WakeMed Health & Hospitals

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: M.D.

Study Record Dates

• First Submitted: November 30, 2022
• First Posted: December 20, 2022
• Study Start: May 23, 2023
• Primary Completion: September 9, 2023
• Study Completion: September 9, 2023
• Last Updated: May 14, 2024

Record last verified: 2024-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)