Cystic FibrosisPhase 2NCT03506061

Trikafta in Cystic Fibrosis Patients

A Phase 2 study testing Trikafta in people with Cystic Fibrosis, run by Emory University at 2 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

Who is it for?: Ages 12 years and older
Conditions the study is for: Cystic Fibrosis
What is being tested?: Trikafta
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 42 participants
Where: 2 sitesUnited States
Study dates: 4 Sep 2019to 13 Feb 2024
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 12 years and older
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Provision of signed and dated informed consent form or assent form
Stated willingness to comply with all study procedures and availability for the duration of the study
Male or female age ≥12
A clinical diagnosis of CF or CFTR-related disease and either: 1) evidence for a partial function mutation not currently covered or likely to be covered for treatment with a CFTR modulator (Substudy 1), or 2) N1303K CFTR and a minimal function mutation (Substudy 2)
Sweat Chloride < 80 mmol/L and/or pancreatic sufficiency (no exogenous pancreatic enzyme supplement therapy) or carrying the N1303K CFTR variant
Able to perform spirometry meeting American Thoracic Society (ATS) criteria for acceptability and repeatability

Show all 10Show fewer

Clinically stable in the past 4 weeks with no evidence of CF exacerbation (prior to screening and study Day 1)
Willingness to use at least one form of acceptable birth control including abstinence or condom with spermicide. This will include birth control for at least one month prior to screening and agreement to use such a method during study participation for an additional four weeks after the last administration of study drug
Ability to take Trikafta
Agreement to adhere to all current medical therapies as designated by the CF care center physician

Who cannot join

Documented history of drug or alcohol abuse within the last year
Subjects should not have a pulmonary exacerbation or changes in therapy for pulmonary disease in the 4 weeks prior to screening
Listed for lung or liver transplant at the time of screening
Cirrhosis or elevated liver transaminases > 3 times the upper limit of normal
Pregnant or breastfeeding
Inhibitors or inducers of CYP3A4, including certain herbal medications and grapefruit/grapefruit juice, or other medicines known to negatively influence Trikafta administration

Show all 13Show fewer

History of solid organ transplant
Active therapy for non-tuberculosis mycobacterial infection or any plan to initiate non-tuberculosis mycobacterial therapies during the study period
Known allergy to Trikafta
Treatment in the last 6 months with an approved CFTR modulator
Any other condition that in the opinion of the lead investigators might confound results of the study or pose an additional risk from administering study drug
Treatment with another investigational drug or other intervention within one month prior to enrollment, throughout the duration of study participation, and for an additional four weeks following final drug administration
Evidence of cataract/lens opacity determined to be clinically significant by an ophthalmologist at or within 3 months prior to the Screening Visit

Trial locations and contacts

2 sites in 1 country.

United States2 sites

Emory Children's Center
Atlanta, Georgia, 30322
University of Alabama Cystic Fibrosis Research Center
Birmingham, Alabama, 35233

Other trials in United States

About this study

Official title: iPS Cell Response to CFTR Modulators: Study of Trikafta in CF Patients Carrying Partial Function Mutations or N1303K CFTR

This clinical study will enroll 42 participants without the F508del mutation, carrying partial function or N1303K mutations not approved for Trikafta, and who are not expected to be approved for CFTR modulator treatment in the immediate future. Each participant will be given Trikafta for approximately four weeks. The study researchers will monitor clinical endpoints that include forced expiratory volume (FEV1) and sweat chloride. Additionally, the researchers will obtain skin biopsy material and/or blood sample from each subject so that induced pluripotent stem (iPS) cells can be modified into airway cell monolayers and tested for response to Trikafta. In this way, the study will evaluate an emerging and readily accessible in vitro endpoint as a predictor of clinical response. This study will serve as a pilot/test case for other clinical protocols relevant to patients with rare CFTR variants who do not currently receive modulator therapies.

Read the detailed description

Cystic Fibrosis (CF) is a life threatening genetic disorder resulting from mutations found in the gene known as the cystic fibrosis transmembrane conductance regulator (CFTR). Defects in this gene prevent correct chloride and bicarbonate transport in and out of cells. It has become increasingly important to develop new in vitro model systems capable of predicting in vivo clinical effectiveness of modulator therapy among patients with CF. This objective represents a significant and unmet need for advancing personalized therapeutics in the disease.

Trikafta is currently approved for patients with CF carrying at least one copy of the common F508del variant and over 250 other CFTR abnormalities. Because approximately 90% of CF patients in the United States meet these criteria, pharmacotherapies (Trikafta in particular) are now available to a sizable majority of those with the disease. However, thousands of patients harboring relatively common variants will remain without effective drug therapy. Others with ultra-rare or private CFTR mutations have forms of the disease that are very likely to benefit from available drugs, but do not have access to these therapies. It has been estimated that over 1,000 CFTR mutations are represented by less than 5 patients each. Establishing processes so that individuals with very rare and/or poorly characterized alleles can gain access to effective modulator treatment remains one of the predominant challenges in the field.

This clinical study will enroll 42 participants without the F508del mutation, carrying partial function or N1303K mutations not approved for Trikafta. Substudy 1 will comprise an open-label, two center trial of orally administered elexacaftor, tezacaftor and ivacaftor (Trikafta) that will enroll 22 patients with rare/orphan genotypes. Substudy 2 will enroll 20 participants who encode the N1303K variant as emblematic of a mutation not approved for Trikafta, but are likely to respond to the treatment.

Each participant will have clinical and/or preclinical evidence that Trikafta should offer benefit, and each will be given Trikafta for approximately four weeks. The researchers will monitor clinical endpoints that include FEV1, sweat chloride, quality of life, and weight. The study will differentiate iPS cells from each subject to generate airway epithelial monolayers that can be tested for response to Trikafta. This trial will serve as a pilot/test case for other clinical protocols relevant to patients with rare CFTR variants and evidence of residual function who do not have an approved modulator therapy, due to rarity of their mutation. It is hypothesized that a correlation will be established between in vitro Trikafta responsiveness of iPS cells and in vivo benefit (FEV1) in patients, and provide a tool for utilizing iPS cells to identify rare CF patient populations most suitable for cystic fibrosis modulator therapy.

Conditions: Cystic Fibrosis
Being tested: Trikafta
Medical subject headings: Cystic Fibrosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

TrikaftaDrug
Also called ivacaftor, tezacaftor, elexacaftor
Participants will take Trikafta which is a combination tablet comprised of 100 milligrams (mg) of elexacaftor, 50 mg of tezacaftor and 75 mg of ivacaftor (2 tablets taken in the morning), and 150 mg of ivacaftor taken in the evening.

Study groups in NCT03506061
Study group	Role	What they receive
Substudy 1 - Participants With Evidence of Partial Function	Experimental	Participants with CF with evidence of partial function (sweat chloride < 80 milliequivalents per liter (mEq/L) or pancreatic sufficiency) will receive Trikafta for 28 days. Receives: Drug: Trikafta
Substudy 2 - Participants who Encode the N1303K Variant	Experimental	Participants with CF who encode the N1303K variant will receive Trikafta for 28 days. Receives: Drug: Trikafta

What is being measured

The primary outcome is the main question the study is designed to answer.

Percent Predicted Forced Expiratory Volume in One Second (FEV1) Among Participants With Evidence of Partial Function
FEV1 provides a direct measurement of patient health among individuals with cystic fibrosis and declines in FEV1 are associated with poor outcomes among those with CF. FEV1 is measured by spirometry and is the maximum amount of air the participant can blow out in one second.
Measured: Baseline, Day 28
Sweat Chloride Among Participants Who Encode the N1303K Variant
Persons with CF have higher levels of chloride in their sweat. Sweat chloride concentrations of less than or equal to 29 mmol/L are considered normal, concentrations of 30-59 mmol/L are considered intermediate and indicate that the individual may have CF. Concentrations of 60 mmol/L and greater mean that a diagnosis of CF is likely.
Measured: Baseline, Day 28
Number of Participants With Induced Pluripotent Stem (iPS) Cells Predicting Response to Treatment Among Participants With Evidence of Partial Function
Response of iPS cells (iPSc) to treatment among participants with evidence of partial function was examined to determine whether iPS derived monolayers could predict "personalized" clinical benefit. Cutaneous punch biopsy material was collected from each participant so that iPS cells could be differentiated into airway epithelial monolayers and tested for response to treatment in vitro - as a potential way to predict improvement from Trikafta in vivo.
Measured: Baseline

Secondary outcomes (6)

Percent Predicted Forced Expiratory Volume in One Second (FEV1) Among Participants Who Encode the N1303K Variant
FEV1 provides a direct measurement of patient health among individuals with cystic fibrosis and declines in FEV1 are associated with poor outcomes among those with CF. FEV1 is measured by spirometry and is the maximum amount of air the participant can blow out in one second.
Measured: Baseline, Day 28
Sweat Chloride Among Participants With Evidence of Partial Function
Persons with CF have higher levels of chloride in their sweat. Sweat chloride concentrations of less than or equal to 29 mmol/L are considered normal, concentrations of 30-59 mmol/L are considered intermediate and indicate that the individual may have CF. Concentrations of 60 mmol/L and greater mean that a diagnosis of CF is likely.
Measured: Baseline, Day 28
Cystic Fibrosis Questionnaire - Revised (CFQ-R) Respiratory Domain Score
Participants take the CFQ-R corresponding to their age for assessing quality of life. Responses to questions are coded as 1 = very true or always, 2 = mostly true or often, 3 = somewhat true or sometimes, and 4 = not at all true or never. Some items are reverse scored so that higher scores indicate increased ability and higher quality of life. Scores for items in the respiratory domain are summed and standardized and the standardized score ranges from 1 to 100. A minimum clinically important difference (MCID) of 4 or more points represents improved respiratory-related quality of life.
Measured: Baseline, Day 28
Weight
Weight is measured in kilograms.
Measured: Baseline, Day 28
Body Mass Index (BMI)
Body mass index is calculated as weight in kilograms divided by height in meters squared.
Measured: Baseline, Day 28
Number of Participants Where the Assay Successfully Predicted Treatment Response Using iPS Cells Among Participants Encoding N1303K
Cutaneous punch biopsy material was collected from each participant so that iPS cells could be differentiated into airway epithelial monolayers and tested for response to treatment in vitro - i.e., as a potential way to predict benefit from Trikafta in vivo. By using iPS cells differentiated to exhibit a respiratory epithelial phenotype, the study aims to determine whether iPScs can be used to predict clinical improvement due to Trikafta.
Measured: Baseline

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

4 Sep 201913 Feb 2024

First posted23 Apr 2018
Study start4 Sep 2019
Primary completion13 Feb 2024
Study completion13 Feb 2024

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (2)Papers the sponsor has linked to this study.
Other trials for Cystic FibrosisStudies of the same condition, open and under way.
More trials run by Emory UniversityThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Parallel groups
Masking: None (open label)
Enrolment: 42 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03506061
Sponsor's protocol ID: IRB00108656
Other IDs: 300001205, R01HL139876
Status last verified: Jul 2026
First submitted: 13 Apr 2018
Last updated: 11 Aug 2026
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Publications

Solomon GM, Linnemann RW, Rich R, Streby A, Buehler B, Hunter E, Vijaykumar K, Hunt WR, Brewington JJ, Rab A, Bai SP, Westbrook AL, McNicholas-Bevensee C, Hong J, Manfredi C, Barilla C, Suzuki S, Davis BR, Sorscher EJ. Evaluation of elexacaftor-tezacaftor-ivacaftor treatment in individuals with cystic fibrosis and CFTRN1303K in the USA: a prospective, multicentre, open-label, single-arm trial. Lancet Respir Med. 2024 Dec;12(12):947-957. doi: 10.1016/S2213-2600(24)00205-4. Epub 2024 Aug 26.
PubMed 39208836
Linnemann RW, Solomon GM, Westbrook AL, Rich R, Streby A, Hunter E, Rab A, Bai S, Mcnicholas CM, Hong JS, Manfredi C, Hunt WR, Stecenko AA, Barilla C, Suzuki S, Davis BR, Sorscher EJ. Elexacaftor-Tezacaftor-Ivacaftor for Individuals with Residual CFTR Activity: An Open-Label, Nonrandomized Clinical Trial. Ann Am Thorac Soc. 2026 Jun 10:aaoag136. doi: 10.1093/annalsats/aaoag136. Online ahead of print. No abstract available.
PubMed 42271629

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 2
A Study of 24 to 52 Weeks Treatment to Evaluate The Efficacy And Safety of Galvokimig in Adult Study Participants With Non-Cystic Fibrosis Bronchiectasis
Non-cystic Fibrosis Bronchiectasis
Active, not recruitingObservational study
CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis
Cystic Fibrosis, Bone Loss
RecruitingObservational study
Natural History of Bronchiectasis
Bronchiectasis, Cystic Fibrosis
RecruitingObservational study
Role of Genetic Factors in the Development of Lung Disease
Cystic Fibrosis, Pulmonary Fibrosis
RecruitingPhase 2
Early Study on Tamoxifen Safety/Tolerability in Cystic Fibrosis Patients Unable to Use CFTR Modulators.
Cystic Fibrosis - Complete

All Cystic Fibrosis trials

Study at a glance

From the registry record, last updated by the sponsor on 11 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: iPS Cell Response to CFTR Modulators: Study of Trikafta in CF Patients Carrying Partial Function Mutations or N1303K CFTR
ClinicalTrials.gov ID: NCT03506061
Acronym / sponsor ID: IRB00108656
Conditions: Cystic Fibrosis
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 42 participants (actual)
Lead sponsor: Emory UniversityUniversity, hospital or other
Collaborators: National Heart, Lung, and Blood Institute (NHLBI), Cystic Fibrosis Foundation, The University of Texas Health Science Center, Houston
Study start: 4 Sep 2019 (actual)
Primary completion: 13 Feb 2024 (actual)
Study completion: 13 Feb 2024 (actual)
First posted: 23 Apr 2018 (actual)
Results: Posted 11 Sep 2025

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 2

Arm	Type	Receives
Substudy 1 - Participants With Evidence of Partial Function	Experimental	Drug: TrikaftaParticipants with CF with evidence of partial function (sweat chloride < 80 milliequivalents per liter (mEq/L) or pancreatic sufficiency) will receive Trikafta for 28 days.
Substudy 2 - Participants who Encode the N1303K Variant	Experimental	Drug: TrikaftaParticipants with CF who encode the N1303K variant will receive Trikafta for 28 days.

Who may qualify

Ages: 12 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (10 listed)

Provision of signed and dated informed consent form or assent form
Stated willingness to comply with all study procedures and availability for the duration of the study
Male or female age ≥12
A clinical diagnosis of CF or CFTR-related disease and either: 1) evidence for a partial function mutation not currently covered or likely to be covered for treatment with a CFTR modulator (Substudy 1), or 2) N1303K CFTR and a minimal function mutation (Substudy 2)
Sweat Chloride < 80 mmol/L and/or pancreatic sufficiency (no exogenous pancreatic enzyme supplement therapy) or carrying the N1303K CFTR variant

Key exclusion criteria (13 listed)

Documented history of drug or alcohol abuse within the last year
Subjects should not have a pulmonary exacerbation or changes in therapy for pulmonary disease in the 4 weeks prior to screening
Listed for lung or liver transplant at the time of screening
Cirrhosis or elevated liver transaminases > 3 times the upper limit of normal
Pregnant or breastfeeding

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
TrikaftaAlso: ivacaftor, tezacaftor, elexacaftorParticipants will take Trikafta which is a combination tablet comprised of 100 milligrams (mg) of elexacaftor, 50 mg of tezacaftor and 75 mg of ivacaftor (2 tablets taken in the morning), and 150 mg of ivacaftor taken in the evening.	Drug	Substudy 1 - Participants With Evidence of Partial Function, Substudy 2 - Participants who Encode the N1303K Variant

Primary outcome

Percent Predicted Forced Expiratory Volume in One Second (FEV1) Among Participants With Evidence of Partial Function
Time frame: Baseline, Day 28
FEV1 provides a direct measurement of patient health among individuals with cystic fibrosis and declines in FEV1 are associated with poor outcomes among those with CF. FEV1 is measured by spirometry and is the maximum amount of air the participant can blow out in one second.
Sweat Chloride Among Participants Who Encode the N1303K Variant
Time frame: Baseline, Day 28
Persons with CF have higher levels of chloride in their sweat. Sweat chloride concentrations of less than or equal to 29 mmol/L are considered normal, concentrations of 30-59 mmol/L are considered intermediate and indicate that the individual may have CF. Concentrations of 60 mmol/L and greater mean that a diagnosis of CF is likely.
Number of Participants With Induced Pluripotent Stem (iPS) Cells Predicting Response to Treatment Among Participants With Evidence of Partial Function
Time frame: Baseline
Response of iPS cells (iPSc) to treatment among participants with evidence of partial function was examined to determine whether iPS derived monolayers could predict "personalized" clinical benefit. Cutaneous punch biopsy material was collected from each participant so that iPS cells could be differentiated into airway epithelial monolayers and tested for response to treatment in vitro - as a potential way to predict improvement from Trikafta in vivo.

6 secondary outcomes

Percent Predicted Forced Expiratory Volume in One Second (FEV1) Among Participants Who Encode the N1303K Variant
Time frame: Baseline, Day 28
Sweat Chloride Among Participants With Evidence of Partial Function
Time frame: Baseline, Day 28
Cystic Fibrosis Questionnaire - Revised (CFQ-R) Respiratory Domain Score
Time frame: Baseline, Day 28
Weight
Time frame: Baseline, Day 28
Body Mass Index (BMI)
Time frame: Baseline, Day 28
Number of Participants Where the Assay Successfully Predicted Treatment Response Using iPS Cells Among Participants Encoding N1303K
Time frame: Baseline

Results and safety

Results posted to the registry on 11 Sep 2025, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Participants With Evidence of Partial Function (Sweat Chloride < 80 mEq/L or Pancreatic Sufficiency)	22	20
Participants Who Encode the N1303K Variant	20	20

Primary outcome: Percent Predicted Forced Expiratory Volume in One Second (FEV1) Among Participants With Evidence of Partial Function

Time frame: Baseline, Day 28. Least squares mean (95% Confidence Interval). Unit: percent of predicted FEV1

Measure	Participants With Evidence of Partial Function (Sweat Chloride < 80 mEq/L or Pancreatic Sufficiency) n = 20
Baseline	74.8 (72.4 to 77.2)
Day 28	76 (73.6 to 78.5)

Population analysed: The Analysis Population includes the intended use population. Two participants with evidence of partial function were found to have cystic fibrosis transmembrane conductance regulator (CFTR) genotypes that were approved by the FDA to use elexacaftor-tezacaftor-ivacaftor while the study was ongoing; these participants were removed from data analysis.

Primary outcome: Sweat Chloride Among Participants Who Encode the N1303K Variant

Time frame: Baseline, Day 28. Least squares mean (95% Confidence Interval). Unit: mmol/liter

Measure	Participants Who Encode the N1303K Variant n = 20
Baseline	109 (105.6 to 112.4)
Day 28	107.9 (104.5 to 111.3)

Population analysed: The Analysis Population includes the intended use population.

Primary outcome: Number of Participants With Induced Pluripotent Stem (iPS) Cells Predicting Response to Treatment Among Participants With Evidence of Partial Function

Time frame: Baseline. Count of participants. Unit: Participants

Measure	Participants With Evidence of Partial Function (Sweat Chloride < 80 mEq/L or Pancreatic Sufficiency) n = 8
Value	0

Population analysed: Participants with evidence of partial function were included for the primary outcome measure of examining response of iPS cell derived epithelial monolayers. This analysis includes participants from which samples were successfully obtained, differentiated, and examined using bioelectric measurements.

6 secondary outcomes are reported in the full results.

Adverse events

Time frame: Information about adverse events was collected from the time of consent through Day 56.. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Participants With Evidence of Partial Function (Sweat Chloride < 80 mEq/L or Pancreatic Sufficiency)	0 of 22 (0%)	17 of 22 (77.3%)	0 of 22
Participants Who Encode the N1303K Variant	1 of 20 (5%)	14 of 20 (70%)	0 of 20

Limitations and caveats (from the sponsor): Analysis of using iPS cells to predict individual response to Trikafta was limited due to technical difficulties when optimizing the new iPS cell-derived epithelial cell model. Challenges and delays with processing iPS cell samples to mature airway epithelial monolayers were also encountered. The study team spent an additional 16 months further refining the novel assay developed for examining iPS cell response for this study but the assay is not functioning for the intended purpose.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 11 Aug 2026.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Results publication: PubMed 39208836Solomon GM, Linnemann RW, Rich R, Streby A, Buehler B, Hunter E, Vijaykumar K, Hunt WR, Brewington JJ, Rab A, Bai SP, Westbrook AL, McNicholas-Bevensee C, Hong J, Manfredi C, Barilla C, Suzuki S, Davis BR, Sorscher EJ. Evaluation of elexacaftor-tezacaftor-ivacaftor treatment in individuals with cystic fibrosis and CFTRN1303K in the USA: a prospective, multicentre, open-label, single-arm trial. Lancet Respir Med. 2024 Dec;12(12):947-957. doi: 10.1016/S2213-2600(24)00205-4. Epub 2024 Aug 26.
Results publication: PubMed 42271629Linnemann RW, Solomon GM, Westbrook AL, Rich R, Streby A, Hunter E, Rab A, Bai S, Mcnicholas CM, Hong JS, Manfredi C, Hunt WR, Stecenko AA, Barilla C, Suzuki S, Davis BR, Sorscher EJ. Elexacaftor-Tezacaftor-Ivacaftor for Individuals with Residual CFTR Activity: An Open-Label, Nonrandomized Clinical Trial. Ann Am Thorac Soc. 2026 Jun 10:aaoag136. doi: 10.1093/annalsats/aaoag136. Online ahead of print. No abstract available.
Individual participant dataSharing planned: No

Related trials for Cystic Fibrosis

RecruitingPhase 2
A Study of 24 to 52 Weeks Treatment to Evaluate The Efficacy And Safety of Galvokimig in Adult Study Participants With Non-Cystic Fibrosis Bronchiectasis
Non-cystic Fibrosis Bronchiectasis
Active, not recruitingObservational study
CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis
Cystic Fibrosis, Bone Loss
RecruitingObservational study
Natural History of Bronchiectasis
Bronchiectasis, Cystic Fibrosis
RecruitingObservational study
Role of Genetic Factors in the Development of Lung Disease
Cystic Fibrosis, Pulmonary Fibrosis
RecruitingPhase 2
Early Study on Tamoxifen Safety/Tolerability in Cystic Fibrosis Patients Unable to Use CFTR Modulators.
Cystic Fibrosis - Complete

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Eric Sorscher, MD principal investigator

Emory University

2 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Trikafta in people with Cystic Fibrosis, run by Emory University at 2 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 10 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 11 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 11 Sep 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percent Predicted Forced Expiratory Volume in One Second (FEV1) Among Participants With Evidence of Partial Function (time frame: Baseline, Day 28). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03506061, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Aug 2026.