Phase 3 Cystic Fibrosis Trial, Completed NCT03691779 Sponsor: Vertex Pharmaceuticals Incorporated Condition: Cystic Fibrosis
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Trial results

AI generated results 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

According to the results reported on ClinicalTrials.gov, this trial (NCT03691779) looked at a combination medicine made up of three components — elexacaftor, tezacaftor, and ivacaftor (referred to together as ELX/TEZ/IVA) — and was run in two separate parts. Part A enrolled 16 participants and focused on measuring how the medicine and its breakdown products moved through the body over a 15-day period, including how much of the medicine reached the bloodstream and how it was processed. Part B enrolled 66 participants (64 of whom completed it) and ran for 24 weeks, with its main focus on tracking unwanted or unexpected health events that occurred while taking the medicine. The reported data shows that in Part A, the highest recorded levels of the three active components in the blood (a measure called peak concentration) were 6.13, 6.93, and 1.01 micrograms per millilitre respectively. The levels present in the blood just before each dose (a measure of how much remained overnight) were 2.86, 1.06, and 0.297 micrograms per millilitre. A measure of total drug exposure over a 24-hour period came in at 107, 58.4, and 8.12 hour-micrograms per millilitre for each component. Similar blood-level figures were also reported for the breakdown products the body creates from each component. For Part B, the reported data shows that 65 out of 66 participants experienced at least one treatment-emergent adverse event (an unwanted health event that occurred after starting the medicine), and 1 participant experienced a serious adverse event. It is worth noting that the data as submitted does not include details about what those adverse events were, how severe they were, or what happened as a result, so those specifics were not reported in the structured results available here. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

These are the results as reported to ClinicalTrials.gov, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.

Phase 3 Cystic Fibrosis Trial, Completed

NCT03691779
Completed Phase 3 🇦🇺 Australian site

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:

  • You carry a specific gene change related to cystic fibrosis called "F508del" — either two copies of it, or one copy alongside certain other gene changes (confirm with trial site)
  • Your lung function test result (measuring how much air you can breathe out in one second) is at least 40% of the normal expected level for your age, sex, and height

Who may not be able to join:

  • You have significant scarring of the liver, with or without increased blood pressure in the liver's blood vessels
  • You currently have a lung infection caused by certain germs known to cause faster decline in lung health (confirm with trial site for specific organisms)
  • You have previously received a transplant of a solid organ (such as a kidney, liver, or lung) or of blood/bone marrow cells

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
Phase 3: approximately ~65% of drugs entering this phase reach regulatory approval, based on published industry-wide historical data. This is not specific to this trial.
Phase success rates shown are historical industry-wide averages based on published data. They are not a prediction for this specific trial or your individual situation.

Contact this trial

Australian sites

Queensland Children's Hospital, South Brisbane,
The Children's Hospital at Westmead, Westmead,

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.

Trial details

Status
Completed
Phase
Phase 3
Registry
ClinicalTrials.gov
Start date
2 October 2018
Est. completion
7 August 2020

Where this trial is recruiting

🇦🇺 Australia 🇨🇦 Canada 🇮🇪 Ireland 🇬🇧 United Kingdom 🇺🇸 United States

2 site(s) in Australia. Confirm current status and contact details directly with the trial site.

Primary endpoints

Part A: Maximum Observed Plasma Concentration (Cmax) of ELX, TEZ, and IVA; Part A: Observed Pre-dose Plasma Concentration (Ctrough) of ELX, TEZ, and IVA; Part A: Area Under the Concentration Versus Time Curve From 0 to 24 Hours (AUC0-24h) of ELX, TEZ, and IVA; Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

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