Cystic Fibrosis Trial, Recruiting (NCT04509050)

Cystic Fibrosis Trial, Recruiting NCT04509050 Sponsor: Sonya Heltshe Condition: Cystic Fibrosis

Printed from Voxsanity (voxsanity.com.au) — NCT04509050 — Data sourced from ClinicalTrials.gov. Verify directly with the trial site before attending. Not medical advice.

Back to Cystic Fibrosis

Who may be able to join

AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

Who might be able to join this trial:

Part A:

Children who are less than 10 years old at their first study visit
Children who have been diagnosed with cystic fibrosis (CF)

Part B:

Children who either took part in Part A, OR are less than 7 years old at their first study visit
Children who have been diagnosed with cystic fibrosis (CF)
Children whose specific CF gene changes (mutations) make them eligible for certain approved CF medications called ivacaftor or elexacaftor/tezacaftor/ivacaftor (confirm with trial site)
Children whose doctor plans to prescribe ivacaftor or elexacaftor/tezacaftor/ivacaftor

Who may not be able to join:

(Applies to both Part A and Part B)

Children who have taken part in another clinical drug trial within the 28 days before their first study visit
Children who have taken ivacaftor or elexacaftor/tezacaftor/ivacaftor within the 28 days before their first study visit
Children who have been regularly taking steroid tablets or liquid by mouth within the 28 days before their first study visit

Important: Always verify eligibility with the trial site directly before applying.

Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.

This is a simplified plain English summary of the eligibility criteria. Full criteria are set by the trial investigators and may include additional requirements not shown here. Never self-exclude from a trial based on this summary. Contact the trial site directly to confirm your eligibility.

Last synced 28 July 2026

This study is not part of the standard drug-approval phase pathway (for example an observational, device, behavioural, or registry study), so a phase success rate does not apply.

Contact this trial

Principal Investigator: Bonnie Ramsey, MD, Seattle Children's Hospital

Phone: 206-884-7517

Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.

GP referral letter

Print a one-page summary to share with your doctor.

Voxsanity

Clinical trials and treatments, explained plainly · voxsanity.com.au

Date:

To: My Doctor

Re: Clinical trial enquiry

I am writing to enquire about the following clinical trial which may be relevant to my care:

Trial: NCT04509050

Sponsor: Sonya Heltshe

Phase: Not Applicable

Status: Recruiting

Condition: Cystic Fibrosis

Trial contact: 206-884-7517

More information: https://clinicaltrials.gov/study/NCT04509050

Eligibility summary (AI generated)

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

Part A:

Children who are less than 10 years old at their first study visit
Children who have been diagnosed with cystic fibrosis (CF)

Part B:

Children who either took part in Part A, OR are less than 7 years old at their first study visit
Children who have been diagnosed with cystic fibrosis (CF)
Children whose specific CF gene changes (mutations) make them eligible for certain approved CF medications called ivacaftor or elexacaftor/tezacaftor/ivacaftor (confirm with trial site)
Children whose doctor plans to prescribe ivacaftor or elexacaftor/tezacaftor/ivacaftor

Who may not be able to join:

(Applies to both Part A and Part B)

Children who have taken part in another clinical drug trial within the 28 days before their first study visit
Children who have taken ivacaftor or elexacaftor/tezacaftor/ivacaftor within the 28 days before their first study visit
Children who have been regularly taking steroid tablets or liquid by mouth within the 28 days before their first study visit

Important: Always verify eligibility with the trial site directly before applying.

Access in Australia: A treatment being studied in a clinical trial is generally not yet listed on the PBS. Where a medicine is not approved or not available through a trial, the TGA Special Access Scheme may allow a doctor to access unapproved therapeutic goods for individual patients. See voxsanity.com.au/sas-navigator/ for a plain English explainer.

This information was sourced from ClinicalTrials.gov via Voxsanity (data last synced 28 July 2026). It is not medical advice. Please verify the trial's current status directly with the trial site before acting.

Trial details

Status

Recruiting

Phase

Not Applicable

Sponsor

Sonya Heltshe

Registry

ClinicalTrials.gov

Start date

18 November 2020

Est. completion

31 December 2027

Where this trial is recruiting

🇺🇸 United States

Primary endpoints

Part A Primary Outcome Measure: Change in weight-for-age z-scores; Part A Primary Outcome Measure: Change in height-for-age z-scores; Part B Primary Outcome Measure: Change in weight-for-age z-scores; Part B Primary Outcome Measure: Change in height-for-age z-scores

Can't join this trial?

Expanded access pathways

If this trial is not available to you, other access pathways may exist. In Australia, the TGA Special Access Scheme allows access to unapproved therapeutic goods for individual patients.

TGA Special Access Scheme information

Find other recruiting trials on ClinicalTrials.gov

Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.

Trial recruitment status can change without notice between our nightly data updates. Always contact the trial site directly to confirm current recruitment status before making any decisions or travel arrangements.

View original record on ClinicalTrials.gov