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Real-world orkambi study tracks 852 cystic fibrosis patients

NCT ID NCT03475381

Disease control Sponsor: Assistance Publique - Hôpitaux de Paris Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study followed 852 cystic fibrosis patients aged 12 and older who started taking Orkambi outside of a clinical trial. Researchers tracked how many stopped treatment, why, and measured lung function and nutrition over one year. The goal was to see how the drug performs in real-life settings.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Ivacaftor+lumacaftor (Orkambi)
What this could lead to
If successful, this could confirm that Orkambi helps control cystic fibrosis symptoms and improve lung function in daily use.
What could go wrong
This is an observational study, not a controlled trial, so results may be less reliable. It only follows patients for one year, so long-term effects are unknown.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

852 people

The number who actually took part.

Started

Jan 2016

Finished

Dec 2018

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Patient with Cystic Fibrosis with presence of two mutations DF508 in the CFTR gene and treated by Orkambi in usual care

Ages

12 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Patient aged 12 years or older. * Patient with Cystic Fibrosis with presence of two mutations DF508 in the CFTR gene * Patient treated with ivacaftor+lumacaftor (Orkambi) Exclusion Criteria: * Refusal to participate in the study * Start of Orkambi as part of a clinical trial

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Conditions

The condition(s) this trial relates to.

cystic fibrosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Cystic fibrosis

Contacts and locations

Locations

  • Adult CF center, Service de Pneumologie, Cochin Hospital

    Paris, Paris, 75006, France

More trials for these conditions

Other studies related to the condition(s) this trial covers.