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Rethinking Early Airway Clearence Therapies (REACT)

2026. július 6. frissítette: Nicole Hamblett

The REACT trial consists of two parallel, randomized studies; the Hypertonic Saline Study and the Dornase Alfa Study.

Health outcomes among people with cystic fibrosis (CF) have been steadily improving, most recently with the advent of highly effective modulator therapy (HEMT). While therapies like hypertonic saline (HS) and dornase alfa (DA) improved outcomes in the past, they are often burdensome. Now that almost 90% of the North American CF population is being treated with elexacaftor/tezacaftor/ivacaftor (ETI) or vanzacaftor/tezacaftor/deutivacaftor (VTD), this trial will evaluate whether these newer treatments make daily HS or DA unnecessary. The trial begins with a 6-week run-in period where participants continue ETI or VTD but stop using HS and DA. Eligible participants are then assigned to either the HS Study or the DA Study for one year. Within those groups, they are randomized to either daily use of HS or DA or as needed use only during respiratory illnesses. The study aims to find out if lung health is similar between children and teens taking HEMT who use HS or DA treatments daily and those who use HS or DA treatments only when they are sick.

A tanulmány áttekintése

Állapot

Még nincs toborzás

Körülmények

Beavatkozás / kezelés

Részletes leírás

Health outcomes among people with cystic fibrosis (CF) have been steadily improving for decades through guideline-directed multi-disciplinary clinical care models, expanding CF-specific therapies, and, most recently, the advent of highly effective modulator therapies (HEMT). Chronic therapies such as hypertonic saline (HS) and dornase alfa (DA) were associated with improved outcomes in the pre-modulator era but are also burdensome and costly. Now that almost 90% of the North American CF population is being treated with elexacaftor/tezacaftor/ivacaftor (ETI) or vanzacaftor/tezacaftor/deutivacaftor (VTD), many in the CF community are asking if chronic inhaled therapies such as HS or DA can be stopped or not started (in young children). Indeed, many people with CF stably on ETI are already stopping or reducing these chronic inhaled mucoactive therapies (CIMT) without an evidence base to guide shared decision-making.

The REACT trial is a platform trial consisting of two parallel prospective, multicenter, randomized, open-label studies: the Hypertonic Saline (HS) Study and the Dornase Alfa (DA) Study. In the Hypertonic Saline (HS) Study, participants will be randomized to twice-daily inhaled HS or as-needed HS (with acute respiratory illnesses, if considered indicated) for one year. In the Dornase Alfa (DA) Study, participants will be randomized to daily inhaled DA or as-needed DA (with acute respiratory illnesses, if considered indicated) for one year.

Study participation will begin with a 6-week run-in period, during which participants who currently use HS, DA, or both will be instructed to stop these therapies; those who do not use chronic inhaled mucoactive therapies (CIMT) will be instructed to remain off these therapies. At the end of the run-in, eligible participants will be enrolled and assigned first to the HS or DA Study and then randomized to study arm. Participants who use only HS or no CIMT at study entry will be assigned to the HS Study. Those who use DA only at study entry will be assigned to the DA Study. Those who use both HS and DA will be randomly assigned to the HS or DA Study. Participants will be instructed to continue their mechanical airway clearance and inhaled antibiotics (if applicable) as prescribed at study entry. Those who were on both HS and DA at study entry will be instructed to only use the inhaled mucoactive agent to which they have been assigned (HS or DA). Participants randomized to the as-needed arm will be allowed to use the study inhaled agent (HS or DA) temporarily, if considered indicated, with acute respiratory illnesses. Participants in either arm will be allowed to (re)introduce chronic daily therapy if felt to be indicated by the treating physician.

Tanulmány típusa

Beavatkozó

Beiratkozás (Becsült)

405

Fázis

  • Nem alkalmazható

Kapcsolatok és helyek

Ez a rész a vizsgálatot végzők elérhetőségeit, valamint a vizsgálat lefolytatásának helyére vonatkozó információkat tartalmazza.

Tanulmányi kapcsolat

Tanulmányozza a kapcsolattartók biztonsági mentését

Tanulmányi helyek

  • Egyesült Államok
    • Alabama
      • Birmingham, Alabama, Egyesült Államok, 35233
        • The Children's Hospital Alabama, University of Alabama at Birmingham
    • Arizona
      • Tucson, Arizona, Egyesült Államok, 85724
        • Tucson Cystic Fibrosis Center
    • California
      • Los Angeles, California, Egyesült Államok, 90027
        • Childrens Hospital Los Angeles
      • Orange, California, Egyesült Államok, 92868
        • CHOC Children's Hospital
      • Palo Alto, California, Egyesült Államok, 94025
        • Stanford University Medical Center
    • Colorado
      • Aurora, Colorado, Egyesült Államok, 80045
        • Children's Hospital Colorado
    • Florida
      • St. Petersburg, Florida, Egyesült Államok, 33701
        • All Children's Hospital
    • Georgia
      • Atlanta, Georgia, Egyesült Államok, 30322
        • Children's Healthcare of Atlanta and Emory University
    • Illinois
      • Chicago, Illinois, Egyesült Államok, 60611
    • Indiana
      • Indianapolis, Indiana, Egyesült Államok, 46202
        • Riley Hospital for Children
    • Iowa
      • Iowa City, Iowa, Egyesült Államok, 52242
        • University of Iowa
    • Maryland
      • Baltimore, Maryland, Egyesült Államok, 21287
        • John Hopkins Hospital
    • Massachusetts
      • Boston, Massachusetts, Egyesült Államok, 02115
        • Boston Children's Hospital
    • Michigan
      • Ann Arbor, Michigan, Egyesült Államok, 48109
        • University of Michigan, Michigan Medicine
        • Kapcsolatba lépni:
    • Minnesota
      • Minneapolis, Minnesota, Egyesült Államok, 55404
      • Minneapolis, Minnesota, Egyesült Államok, 55455
        • The Minnesota Cystic Fibrosis Center
    • Missouri
      • Kansas City, Missouri, Egyesült Államok, 64108
        • Children's Mercy Kansas City
        • Kapcsolatba lépni:
      • St Louis, Missouri, Egyesült Államok, 63110
        • St. Louis Children's Hospital
    • New York
      • Rochester, New York, Egyesült Államok, 14642
        • University of Rochester Medical Center Strong Memorial
    • North Carolina
      • Chapel Hill, North Carolina, Egyesült Államok, 27599
    • Ohio
      • Cincinnati, Ohio, Egyesült Államok, 45229
        • Cincinnati Children's Hospital Medical Center
      • Cleveland, Ohio, Egyesült Államok, 44106
        • Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
      • Columbus, Ohio, Egyesült Államok, 43205
        • Nationwide Children's Hospital
      • Dayton, Ohio, Egyesült Államok, 45404
        • Dayton Children's Hospital
    • Oregon
      • Portland, Oregon, Egyesült Államok, 97239
        • Oregon Health & Sciences University
    • Pennsylvania
      • Philadelphia, Pennsylvania, Egyesült Államok, 19104
        • Children's Hospital of Philadelphia
      • Pittsburgh, Pennsylvania, Egyesült Államok, 15224
    • South Carolina
      • Charleston, South Carolina, Egyesült Államok, 29425
        • Medical University of South Carolina
        • Kapcsolatba lépni:
    • Texas
      • Dallas, Texas, Egyesült Államok, 75207
        • University of Texas Southwestern / Children's Health
      • Houston, Texas, Egyesült Államok, 77030
        • Baylor College of Medicine
    • Virginia
      • Charlottesville, Virginia, Egyesült Államok, 22904
        • University of Virginia
      • Richmond, Virginia, Egyesült Államok, 23219
        • Virginia Commonwealth University
    • Washington
      • Seattle, Washington, Egyesült Államok, 98105
      • Spokane, Washington, Egyesült Államok, 99204
        • Providence Medical Group, Cystic Fibrosis Clinic - Pediatrics
        • Kapcsolatba lépni:
    • Wisconsin
      • Madison, Wisconsin, Egyesült Államok, 53792
        • University of Wisconsin
      • Milwaukee, Wisconsin, Egyesült Államok, 53226
        • Children's Wisconsin
        • Kapcsolatba lépni:
  • Kanada
    • British Columbia
      • Vancouver, British Columbia, Kanada, V6H3V4
        • CF Centre BC Children's Hospital (Vancouver, Canada)
        • Kapcsolatba lépni:
    • Nova Scotia
      • Halifax, Nova Scotia, Kanada
        • Queen Elizabeth II Hospital Halifax Adult CF Centre
        • Kapcsolatba lépni:
    • Ontario
      • Toronto, Ontario, Kanada, M5G1X8

Részvételi kritériumok

A kutatók olyan embereket keresnek, akik megfelelnek egy bizonyos leírásnak, az úgynevezett jogosultsági kritériumoknak. Néhány példa ezekre a kritériumokra a személy általános egészségi állapota vagy a korábbi kezelések.

Jogosultsági kritériumok

Tanulmányozható életkorok

  • Gyermek

Egészséges önkénteseket fogad

Nem

Leírás

Inclusion Criteria at Screening:

  • All genders ≥ 3 and ≤ 16 years of age
  • Documentation of a CF diagnosis
  • If capable of completing spirometry, forced expiratory volume in 1 second (FEV1) ≥ 70 % predicted at the Screening Visit
  • Clinically stable with no significant changes in health status within the 28 days prior to and including Screening Visit
  • MBW test meets acceptability criteria at the Screening Visit
  • On elexacaftor/tezacaftor/ivacaftor (ETI) or vanzacaftor/tezacaftor/deutivacaftor (VTD) for at least 90 days prior to and including Screening (modified dose permissible) and willing to continue daily use of either ETI or VTD for the duration of the study

Inclusion Criteria at Randomization:

  • Clinically stable with no significant changes in health status for 28 days prior to Visit 1
  • MBW test meets acceptability at Visit 1
  • Completed at least 60% of weekly electronic treatment diaries
  • Take at least one dose of ETI or VTD per weekly electronic treatment diaries

Exclusion Criteria at Screening:

  • No use of an investigational drug within 28 days prior to and including Screening Visit
  • No initiation of new chronic therapy (e.g., azithromycin, inhaled tobramycin, inhaled aztreonam) within 28 days prior to and including Screening Visit
  • No acute use of antibiotics (oral, inhaled, or IV) or acute use of systemic corticosteroids for respiratory tract symptoms within 28 days prior to and including Screening Visit
  • No antibiotic treatment for nontuberculous mycobacteria (NTM) within 28 days prior to and including the Screening Visit

Exclusion Criteria at Visit 1:

  • No acute use of antibiotics (oral, inhaled or IV), systemic corticosteroids, hypertonic saline, or dornase alfa for respiratory tract symptoms within 28 days prior to and including Visit 1
  • No absolute decrease in FEV1 % predicted of ≥10% from the Screening Visit to Visit 1 (in participants who performed acceptable and reproducible spirometry at both visits)

Tanulási terv

Ez a rész a vizsgálati terv részleteit tartalmazza, beleértve a vizsgálat megtervezését és a vizsgálat mérését.

Hogyan készül a tanulmány?

Tervezési részletek

  • Elsődleges cél: Kezelés
  • Kiosztás: Véletlenszerűsített
  • Beavatkozó modell: Párhuzamos hozzárendelés
  • Maszkolás: Nincs (Open Label)

Fegyverek és beavatkozások

Résztvevő csoport / kar
Beavatkozás / kezelés
Kísérleti: As-Needed HS
As-needed hypertonic saline (HS) therapy in the HS Study
Egyéb: As-needed hypertonic saline (HS)
As-needed hypertonic saline (HS) therapy during the 52-week study period.
Aktív összehasonlító: Daily HS
Twice daily hypertonic saline (HS) therapy in the HS Study
Egyéb: Twice daily hypertonic saline (HS)
Twice daily hypertonic saline (HS) therapy during the 52-week study period. The concentration of HS is according to clinical prescription (e.g., 7% sodium chloride).
Kísérleti: As-Needed DA
As-needed dornase alfa (DA) therapy in the DA Study
Egyéb: As-needed dornase alfa (DA)
As-needed dornase alfa (DA) therapy during the 52-week study period.
Aktív összehasonlító: Daily DA
Daily dornase alfa (DA) therapy in the DA Study
Egyéb: Daily dornase alfa (DA)
Daily dornase alfa (DA) during the 52-week study period.

Mit mér a tanulmány?

Elsődleges eredményintézkedések

Eredménymérő
Intézkedés leírása
Időkeret
Absolute Change in Lung Clearance Index (LCI) through Week 52, Relative to Week 0, in Hypertonic Saline (HS Study) and Dornase Alfa (DA Study) Therapy Arms.
Időkeret: 52 weeks
Difference in the change in lung clearance index through Week 52, relative to Week 0, between hypertonic saline (HS) therapy arms (twice daily HS - as-needed HS Entire Concurrently Eligible (ECE) cohort) and between dornase alfa (DA) therapy arms (daily DA - as-needed DA ECE cohort). Participants included in the as-needed HS ECE are those who had a non-zero probability of being assigned to the HS Study and were randomized to either the as-needed HS arm (HS Study) or the as-needed DA arm (DA Study).
52 weeks

Másodlagos eredményintézkedések

Eredménymérő
Intézkedés leírása
Időkeret
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) through Week 52, Relative to Week 0, in Hypertonic Saline (HS Study) and Dornase Alfa (DA Study) Therapy Arms.
Időkeret: 52 weeks
Difference in the change in percent predicted forced expiratory volume in 1 second through Week 52, relative to Week 0, between hypertonic saline (HS) therapy arms (twice daily HS - as-needed HS Entire Concurrently Eligible (ECE) cohort) and between dornase alfa (DA) therapy arms (daily DA - as-needed DA ECE cohort). Participants included in the as-needed HS ECE are those who had a non-zero probability of being assigned to the HS Study and were randomized to either the as-needed HS arm (HS Study) or the as-needed DA arm (DA Study).
52 weeks
Rate of Protocol-Defined Pulmonary Exacerbations (PEx) through Week 52 in Hypertonic Saline (HS Study) and Dornase Alfa (DA Study) Therapy Arms.
Időkeret: 52 weeks
Difference in the rate of protocol-defined pulmonary exacerbations (PEx) through Week 52 between hypertonic saline (HS) therapy arms (twice daily HS - as-needed HS Entire Concurrently Eligible (ECE) cohort) and between dornase alfa (DA) therapy arms (daily DA - as-needed DA ECE cohort). Participants included in the as-needed HS ECE are those who had a non-zero probability of being assigned to the HS Study and were randomized to either the as-needed HS arm (HS Study) or the as-needed DA arm (DA Study).
52 weeks
Absolute Change in Respiratory Symptoms, as Measured by the Cystic Fibrosis Questionnaire - Revised Respiratory Domain (CFQ-R RD), through Week 52, Relative to Week 0, in Hypertonic Saline (HS Study) and Dornase Alfa (DA Study) Therapy Arms
Időkeret: 52 weeks
Difference in the change in the Cystic Fibrosis Questionnaire - Revised Respiratory Domain (CFQ-R RD) through Week 52, relative to Week 0, between hypertonic saline (HS) therapy arms (twice daily HS - as-needed HS Entire Concurrently Eligible (ECE) cohort) and between dornase alfa (DA) therapy arms (daily DA - as-needed DA ECE cohort). Participants included in the as-needed HS ECE are those who had a non-zero probability of being assigned to the HS Study and were randomized to either the as-needed HS arm (HS Study) or the as-needed DA arm (DA Study).
52 weeks
Absolute Change in Lung Clearance Index (LCI) from Week 0 to Week 6 in Hypertonic Saline (HS Study) and Dornase Alfa (DA Study) Therapy Arms.
Időkeret: 6 weeks
Difference in the change in the lung clearance index (LCI) from Week 0 to Week 6 between hypertonic saline (HS) therapy arms (twice daily HS - as-needed HS Entire Concurrently Eligible (ECE) cohort) and between dornase alfa (DA) therapy arms (daily DA - as-needed DA ECE cohort). Participants included in the as-needed HS ECE are those who had a non-zero probability of being assigned to the HS Study and were randomized to either the as-needed HS arm (HS Study) or the as-needed DA arm (DA Study).
6 weeks
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) from Week 0 to Week 6 in Hypertonic Saline (HS Study) and Dornase Alfa (DA Study) Therapy Arms.
Időkeret: 6 weeks
Difference in the change in the percent predicted forced expiratory volume in 1 second (ppFEV1) from Week 0 to Week 6 between hypertonic saline (HS) therapy arms (twice daily HS - as-needed HS Entire Concurrently Eligible (ECE) cohort) and between dornase alfa (DA) therapy arms (daily DA - as-needed DA ECE cohort). Participants included in the as-needed HS ECE are those who had a non-zero probability of being assigned to the HS Study and were randomized to either the as-needed.
6 weeks
Absolute Change in Respiratory Symptoms, as Measured by the Cystic Fibrosis Questionnaire - Revised Respiratory Domain (CFQ-R RD), from Week 0 to Week 6 in Hypertonic Saline (HS Study) and Dornase Alfa (DA Study) Therapy Arms.
Időkeret: 6 weeks
Difference in the change in the Cystic Fibrosis Questionnaire - Revised Respiratory Domain (CFQ-R RD) from Week 0 to Week 6 between hypertonic saline (HS) therapy arms (twice daily HS - as-needed HS Entire Concurrently Eligible (ECE) cohort) and between dornase alfa (DA) therapy arms (daily DA - as-needed DA ECE cohort). Participants included in the as-needed HS ECE are those who had a non-zero probability of being assigned to the HS Study and were randomized to either the as-needed HS arm (HS Study) or the as-needed DA arm (DA Study).
6 weeks
Absolute Change in Treatment Burden, as Measured by the Cystic Fibrosis Questionnaire - Revised Treatment Burden Domain, through Week 52, Relative to Week 0, in Hypertonic Saline (HS Study) and Dornase Alfa (DA Study) Therapy Arms.
Időkeret: 52 weeks
Difference in the change in the Cystic Fibrosis Questionnaire - Revised Treatment Burden domain through Week 52, relative to Week 0, between hypertonic saline (HS) therapy arms (twice daily HS - as-needed HS Entire Concurrently Eligible (ECE) cohort) and between dornase alfa (DA) therapy arms (daily DA - as-needed DA ECE cohort). Participants included in the as-needed HS ECE are those who had a non-zero probability of being assigned to the HS Study and were randomized to either the as-needed HS arm (HS Study) or the as-needed DA arm (DA Study).
52 weeks
Absolute Change in Family Impact, as Measured by the Pediatric Quality of Life Inventory (PedsQL) Family Impact Module, through Week 52, Relative to Week 0, in Hypertonic Saline (HS Study) and Dornase Alfa (DA Study) Therapy Arms.
Időkeret: 52 weeks
Difference in the change in the Pediatric Quality of Life Inventory (PedsQL) Family Impact Module through Week 52, relative to Week 0, between hypertonic saline (HS) therapy arms (twice daily HS - as-needed HS Entire Concurrently Eligible (ECE) cohort) and between dornase alfa (DA) therapy arms (daily DA - as-needed DA ECE cohort). Participants included in the as-needed HS ECE are those who had a non-zero probability of being assigned to the HS Study and were randomized to either the as-needed HS arm (HS Study) or the as-needed DA arm (DA Study).
52 weeks
Healthcare Resource Utilization (HCRU) through Week 52 in Hypertonic Saline (HS Study) and Dornase Alfa (DA Study) Therapy Arms.
Időkeret: 52 weeks
Difference in the Healthcare Resource Utilization (HCRU) through Week 52 between hypertonic saline (HS) therapy arms (twice daily HS - as-needed HS) and between dornase alfa (DA) therapy arms (daily DA - as-needed DA).
52 weeks
Incremental cost-effectiveness ratios (ICERs) through Week 52 in Hypertonic Saline (HS Study) and Dornase Alfa (DA Study) Therapy Arms.
Időkeret: 52 weeks
Difference in the change in the Incremental cost-effectiveness ratios (ICERs) through Week 52 between hypertonic saline (HS) therapy arms (twice daily HS - as-needed HS) and between dornase alfa (DA) therapy arms (daily DA - as-needed DA).
52 weeks

Együttműködők és nyomozók

Itt találhatja meg a tanulmányban érintett személyeket és szervezeteket.

Szponzor

Nicole Hamblett

Együttműködők

Cystic Fibrosis Foundation

Nyomozók

  • Kutatásvezető: Margaret Rosenfeld, MD, MPH, University of Washington, Seattle Children's Research Institute
  • Kutatásvezető: Felix Ratjen, MD, PhD, University of Toronto, SickKids Research Institute
  • Kutatásvezető: Jonathan Rayment, MDCM, MSc, FRCPC, University of British Columbia, BC Children's Hospital

Tanulmányi rekorddátumok

Ezek a dátumok nyomon követik a ClinicalTrials.gov webhelyre benyújtott vizsgálati rekordok és összefoglaló eredmények benyújtásának folyamatát. A vizsgálati feljegyzéseket és a jelentett eredményeket a Nemzeti Orvostudományi Könyvtár (NLM) felülvizsgálja, hogy megbizonyosodjon arról, hogy megfelelnek-e az adott minőség-ellenőrzési szabványoknak, mielőtt közzéteszik őket a nyilvános weboldalon.

Tanulmány főbb dátumok

Tanulmány kezdete (Becsült)

2026. szeptember 15.

Elsődleges befejezés (Becsült)

2030. február 28.

A tanulmány befejezése (Becsült)

2030. február 28.

Tanulmányi regisztráció dátumai

Először benyújtva

2026. július 6.

Először nyújtották be, amely megfelel a minőségbiztosítási kritériumoknak

2026. július 6.

Első közzététel (Tényleges)

2026. július 10.

Tanulmányi rekordok frissítései

Utolsó frissítés közzétéve (Tényleges)

2026. július 10.

Az utolsó frissítés elküldve, amely megfelel a minőségbiztosítási kritériumoknak

2026. július 6.

Utolsó ellenőrzés

2026. július 1.

Több információ

A tanulmányhoz kapcsolódó kifejezések

Kulcsszavak

További vonatkozó MeSH feltételek

Egyéb vizsgálati azonosító számok

  • REACT-IP-25

Terv az egyéni résztvevői adatokhoz (IPD)

Tervezi megosztani az egyéni résztvevői adatokat (IPD)?

NEM

Gyógyszer- és eszközinformációk, tanulmányi dokumentumok

Egy amerikai FDA által szabályozott gyógyszerkészítményt tanulmányoz

Nem

Egy amerikai FDA által szabályozott eszközterméket tanulmányoz

Nem

Ezt az információt közvetlenül a clinicaltrials.gov webhelyről szereztük be, változtatás nélkül. Ha bármilyen kérése van vizsgálati adatainak módosítására, eltávolítására vagy frissítésére, kérjük, írjon a következő címre: register@clinicaltrials.gov. Amint a változás bevezetésre kerül a clinicaltrials.gov oldalon, ez a webhelyünkön is automatikusan frissül. .

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