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Prospective Treatment Efficacy in IPF Using Genotype for Nac Selection (PRECISIONS) Trial (PRECISIONS)

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ClinicalTrials.gov Identifier: NCT04300920
Recruitment Status : Recruiting
First Posted : March 9, 2020
Last Update Posted : March 22, 2023
Sponsor:
Collaborators:
University of Virginia
University of Michigan
Pulmonary Fibrosis Foundation
University of Washington
National Heart, Lung, and Blood Institute (NHLBI)
Three Lakes Foundation
Information provided by (Responsible Party):
Weill Medical College of Cornell University

Study Description
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Brief Summary:

The purpose of this study is to compare the effect of n-acetylcysteine (NAC) plus standard care with matched placebo plus standard of care in patients diagnosed with idiopathic pulmonary fibrosis (IPF) who have the TOLLIP rs3750920 TT genotype. The study will compare the time to a composite endpoint of relative decline in lung function [10% relative decline in forced vital capacity (FVC), first respiratory hospitalization, lung transplantation, or all-cause mortality]

The secondary objectives will be to examine the effect of NAC on the components of the primary composite endpoint, the rates of clinical events, change in physiology, change in health status, and change in respiratory symptoms.


Condition or disease Intervention/treatment Phase
Idiopathic Pulmonary Fibrosis Drug: N-acetyl cysteine Drug: Placebo Phase 3

Detailed Description:

This is a multi-center, randomized, double-blind, placebo-controlled trial of NAC or placebo in about 200 participants with IPF with a TOLLIP rs3750920 TT genotype.

Eligible participants will be randomized in a 1:1 fashion to NAC or placebo, stratified by stable concomitant IPF therapy use (i.e., pirfenidone or nintedanib administered for at least 6 weeks prior to screening) versus no pirfenidone or nintedanib use. Participants will receive 600 mg NAC orally or matched placebo to take three times daily for 24 months.

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 200 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Eligible participants will be randomized in a 1:1 fashion to NAC or placebo, stratified by stable concomitant IPF therapy use (i.e., pirfenidone or nintedanib administered for at least 6 weeks prior to screening) versus no pirfenidone or nintedanib use. Participants will receive 600 mg NAC orally three times daily or matched placebo for 24 months.
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: The participant and site personnel will not know which study treatment the participant is receiving.
Primary Purpose: Treatment
Official Title: Prospective Treatment Efficacy in IPF Using Genotype for Nac Selection (PRECISIONS) Trial
Actual Study Start Date : December 17, 2020
Estimated Primary Completion Date : December 31, 2025
Estimated Study Completion Date : December 31, 2025

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: N-acetylcysteine
600 mg oral N-acetylcysteine (NAC) three times daily for 24 months.
 Drug: N-acetyl cysteine
600 mg N-acetylcysteine (NAC) oral tablets three times daily for 24 months.
Other Name: NAC
Placebo Comparator: Placebo
Placebo tablet three times daily for 24 months.
 Drug: Placebo
Matching oral placebo tablet three times daily for 24 months.


Outcome Measures
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Primary Outcome Measures :
  1. Time to one of the following composite endpoint criteria: 10% relative decline in forced vital capacity (FVC), first respiratory hospitalization, lung transplant or death from any cause. [ Time Frame: 24 months ]
    This is a composite endpoint of time to 10% relative decline in forced vital capacity (FVC), first respiratory hospitalization, lung transplant or death from any cause. Respiratory hospitalizations will be determined by a blinded clinical events classification (adjudication) committee.


Secondary Outcome Measures :
  1. Time to one of the following composite criteria: 10% relative decline in FVC % predicted, first respiratory hospitalization, lung transplant or death from any cause. [ Time Frame: 24 months ]
    This is a composite endpoint of time to 10% relative decline in FVC % predicted, based on the global lung initiative (GLI) reference equation, first respiratory hospitalization, lung transplant or death from any cause. Respiratory hospitalizations will be determined by a blinded clinical events classification (adjudication) committee.

  2. Time to death from any cause [ Time Frame: 24 months ]
  3. Time to first respiratory hospitalization [ Time Frame: 24 months ]
    Respiratory hospitalizations will be determined by a blinded clinical events classification (adjudication) committee.

  4. Time to 10% relative decline in FVC [ Time Frame: 24 months ]
  5. Time to lung transplant [ Time Frame: 24 months ]
  6. Time to 10% relative decline in FVC %predicted [ Time Frame: 24 months ]
  7. Time to first all-cause hospitalization [ Time Frame: 24 months ]
  8. Annualized rate of respiratory hospitalizations [ Time Frame: 24 months ]
  9. Annualized rate of non-elective, all-cause hospitalizations [ Time Frame: 24 months ]
  10. Proportion of participants undergoing lung transplant during follow-up [ Time Frame: 24 months ]
  11. Change in FVC from randomization at 12 months [ Time Frame: 12 months ]
  12. Change in FVC % predicted from randomization at 12 months [ Time Frame: 12 months ]
  13. Change in FVC from randomization at 24 months [ Time Frame: 24 months ]
  14. Change in FVC % predicted from randomization at 24 months [ Time Frame: 24 months ]
  15. Change in diffusing capacity of the lung for carbon monoxide (DLCO) uncorrected for hemoglobin from randomization at 12 months [ Time Frame: 12 months ]
  16. Change in DLCO from randomization at 24 months [ Time Frame: 24 months ]
  17. Change in patient reported outcomes scores for the Leicester Cough Questionnaire (LCQ) from randomization at 12 months. [ Time Frame: 12 months ]
  18. Change in patient reported outcomes scores for the EuroQoL EQ-5D Questionnaire from randomization at 12 months. [ Time Frame: 12 months ]
  19. Change in patient reported outcomes scores for the University of California, San Diego Shortness of Breath (UCSD-SOB) Questionnaire from randomization at 12 months. [ Time Frame: 12 months ]
  20. Change in patient reported outcomes scores for the King's Brief Interstitial Lung Disease (K-BILD) Questionnaire from randomization at 12 months. [ Time Frame: 12 months ]
  21. Change in patient reported outcomes scores for the St. George's Respiratory Questionnaire (SGRQ) from randomization at 12 months. [ Time Frame: 12 months ]
  22. Change in patient reported outcomes scores for the Leicester Cough Questionnaire (LCQ) from randomization at 24 months [ Time Frame: 24 months ]
  23. Change in patient reported outcomes scores for the EuroQoL EQ-5D Questionnaire from randomization at 24 months [ Time Frame: 24 months ]
  24. Change in patient reported outcomes scores for the University of California, San Diego Shortness of Breath (UCSD-SOB) Questionnaire from randomization at 24 months [ Time Frame: 24 months ]
  25. Change in patient reported outcomes scores for the King's Brief Interstitial Lung Disease (K-BILD) Questionnaire from randomization at 24 months [ Time Frame: 24 months ]
  26. Change in patient reported outcomes scores for the St. George's Respiratory Questionnaire (SGRQ) from randomization at 24 months [ Time Frame: 24 months ]
  27. Proportion of participants with and number of treatment-emergent adverse events, serious adverse events, adverse events leading to discontinuation, and unanticipated problems [ Time Frame: 24 months ]

Eligibility Criteria
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Ages Eligible for Study:   40 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • ≥ 40 years of age
  • Diagnosed with IPF according to 2018 ATS/ERS/JRS/ALAT, confirmed by enrolling investigator
  • Signed informed consent
  • If taking pirfenidone or nintedanib, must be on stable dose for at least 6 weeks prior to enrollment visit
  • Confirmed rs3570920 TT TOLLIP genotype

Exclusion Criteria:

  • Pregnancy or planning to become pregnant
  • Women of childbearing potential not willing to remain abstinent (refrain from heterosexual intercourse) or use two adequate methods of contraception, including at least one method with a failure rate of <1% per year during study participation
  • Significant medical, surgical or psychiatric illness that in the opinion of the investigator would affect subject safety, including liver and renal failure
  • Receipt of an investigational drug or biological agent within the previous 4 weeks of the screening visit or 5 times the half-life, if longer
  • Supplemental or prescribed NAC therapy within 60 days of enrollment
  • Listed for lung transplantation at the time of screening
  • History of lung cancer
  • Inability to perform spirometry
  • Forced vital capacity (FVC) less than 45% predicted, using the global lung function index (GLI) equation at Visit 1
  • Active respiratory infection requiring treatment with antibiotics within 4 weeks of Visit 1
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04300920


Contacts
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Contact: Betsy Peters 646-962-2742 elp2018@med.cornell.edu

Locations
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Sponsors and Collaborators
Weill Medical College of Cornell University
University of Virginia
University of Michigan
Pulmonary Fibrosis Foundation
University of Washington
National Heart, Lung, and Blood Institute (NHLBI)
Three Lakes Foundation
Investigators
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Principal Investigator: Fernando J Martinez, MD Weill Medical College of Cornell University
Principal Investigator: Imre Noth, MD University of Virginia
Principal Investigator: Kevin Flaherty, MS, MD University of Michigan
Principal Investigator: Cathie Spino, ScD University of Michigan
More Information
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Responsible Party: Weill Medical College of Cornell University
ClinicalTrials.gov Identifier: NCT04300920    
Other Study ID Numbers: 19-12021232
1U24HL145265-01A1 ( U.S. NIH Grant/Contract )
First Posted: March 9, 2020    Key Record Dates
Last Update Posted: March 22, 2023
Last Verified: March 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: The de-identified analytic data will be prepared as SAS transport files or ASCII comma-delimited files with accompanying codebooks that describe the data and data structure. The redaction will employ best practices and will be consistent with NHLBI data sharing policies.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Time Frame: 3 years after the end of the study or 2 years after the main paper reporting the results of the trial, whichever comes first.
Access Criteria: Data will be shared through the NHLBI Biologic Specimen and Data Repository Information Coordinating Center (BioLINCC).
URL: https://biolincc.nhlbi.nih.gov/home/

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Weill Medical College of Cornell University:
IPF
Pulmonary Fibrosis
n-acetylcysteine
NAC
Additional relevant MeSH terms:
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Pulmonary Fibrosis
Idiopathic Pulmonary Fibrosis
Fibrosis
Pathologic Processes
Lung Diseases, Interstitial
Lung Diseases
Respiratory Tract Diseases
Acetylcysteine
N-monoacetylcystine
Antiviral Agents
 Anti-Infective Agents
Expectorants
Respiratory System Agents
Free Radical Scavengers
Antioxidants
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs
Antidotes