Prospective Treatment Efficacy in IPF Using Genotype for Nac Selection (PRECISIONS) Trial
Purpose
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
- Idiopathic Pulmonary Fibrosis
Eligibility
- Eligible Ages
- Over 40 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
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
Study Design
- Phase
- Phase 3
- Study Type
- Interventional
- 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.
- Primary Purpose
- Treatment
- 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.
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental N-acetylcysteine |
600 mg oral N-acetylcysteine (NAC) three times daily for 24 months. |
|
|
Placebo Comparator Placebo |
Placebo tablet three times daily for 24 months. |
|
Recruiting Locations
More Details
- NCT ID
- NCT04300920
- Status
- Active, not recruiting
- Sponsor
- Weill Medical College of Cornell University
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.