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Rethinking Early Airway Clearence Therapies

ConditionCystic Fibrosis (CF)
Trial statusNot yet recruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age3-16
SponsorNicole Hamblett

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial design

Design model

Parallel

Treatments tested in this trial

  • As-needed hypertonic saline (HS)

    Other intervention

    As-needed hypertonic saline (HS) therapy during the 52-week study period.

  • Twice daily hypertonic saline (HS)

    Other intervention

    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).

  • As-needed dornase alfa (DA)

    Other intervention

    As-needed dornase alfa (DA) therapy during the 52-week study period.

  • Daily dornase alfa (DA)

    Other intervention

    Daily dornase alfa (DA) during the 52-week study period.

Treatment groups

405 Participants
are divided into 4 treatment groups
Group A: As-Needed HSExperimental treatment 1 intervention
Group B: Daily HSActive comparator 1 intervention
Group C: As-Needed DAExperimental treatment 1 intervention
Group D: Daily DAActive comparator 1 intervention

Trial outcomes

Primary outcomes

1

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.

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).

Time frame
52 weeks

Secondary outcomes

1

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.

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).

Time frame
52 weeks
2

Rate of Protocol-Defined Pulmonary Exacerbations (PEx) through Week 52 in Hypertonic Saline (HS Study) and Dornase Alfa (DA Study) Therapy Arms.

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).

Time frame
52 weeks
3

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

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).

Time frame
52 weeks
4

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.

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).

Time frame
6 weeks

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Nicole Hamblett

Lead sponsor

Seattle Children's Hospital

Sponsor institution

Cystic Fibrosis Foundation

Collaborator

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: