Cystic FibrosisPhase 4BEGINNINGNCT05517655
BEGIN Novel ImagiNG Biomarkers
A Phase 4 study testing 129Xe in people with Cystic Fibrosis, run by Children's Hospital Medical Center, Cincinnati at 3 sites in United States.
Trial summary
To determine the treatment effect of triple-combination therapy in 6-8 year olds after presumed FDA approval, using rapid structural and functional pulmonary and abdominal MRI (UTE and 129Xe). Read more about this study
- Who is it for?
- Ages 6 to 8 years
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- 129Xe
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- 3 sitesUnited States
- Study dates
- 1 May 2022to 31 Dec 2029
- Phase
- Phase 4
- Are there results?
- Not yet: the study is under way
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 6 to 8 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Written informed consent (and assent where appropriate) obtained from the subject or subject's legal representative.
- Willingness to adhere to the study-visit schedule and other protocol requirements.
- Ages 6-8 years old at baseline MRI visit (may be enrolled up to 60 days before 6th birthday).
- Documentation of CF diagnosis as evidenced by one or more clinical features consistent with the CF phenotype and one or more of the following criteria:
- Sweat chloride equal to or greater than 60 mEq/liter by quantitative pilocarpine iontophoresis test
- Two well-characterized mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene
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- Physician intent to prescribe triple-combination therapy
- Clinically-stable with no respiratory tract infection at the time of enrollment.
- No change in chronic maintenance therapies in the 28 days prior to enrollment.
- Ability to cooperate with MRI procedures
Who cannot join
- Individuals currently on ivacaftor therapy (including Kalydeco, Orkambi, and Symdeko) and with at least one gating mutation. Gating mutations include G551D, G178R, S549N, S549R, G551S, G970R, G1244E, S1251N, S1255P, or G1349D.
- Acute respiratory symptoms (e.g. wheezing) at the time of the MRI.
- Acute respiratory infection, defined as increased cough, wheezing or respiratory rate in the 28 days prior to enrollment.
- Chronic lung disease not related to CF
- Chronic liver disease not related to CF
- Acute pancreatitis, defined by clinical criteria (45).
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- Chronic pancreatic disease not related to CF.
- Physical findings that would compromise the safety of the subject or the quality of the study data as determined at the discretion of the site investigator.
- Any other condition that, in the opinion of the Site Investigator/designee, would preclude informed consent or assent, make study participation unsafe, complicate interpretation of study outcome data, or otherwise interfere with achieving the study objectives.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
University of Virginia
Charlottesville, Virginia, 22903
Carrie Stevens
Cincinnati, Ohio, 45229
University of Kansas Medical Center
Kansas City, Kansas, 66160
About this study
To determine the treatment effect of triple-combination therapy in 6-8 year olds after presumed FDA approval, using rapid structural and functional pulmonary and abdominal MRI (UTE and 129Xe).
Read the detailed description
The overall hypothesis is that multi-organ MRI will provide more sensitive, robust outcome measures in young CF patients than traditional measures employed in the BEGIN study and that these novel measures will be more sensitive to treatment effects, tested here by comparison before and after triple-combination modulator therapy. By understanding the nature of early lung obstruction and characteristic changes in the liver and pancreas over time, we continue to lay the groundwork for more personalized medicine in the future.
Assessing treatment response and clinical benefit in children with CF who are clinically normal per standard outcomes (e.g., spirometry, pancreatic function) will become paramount as triplecombination therapy is extended to younger patients with milder CF clinical presentation than their historic peers. Here the sensitivity and profile free of ionizing-radiation exposure of MRI can be leveraged to follow an individual with CF over time to quantify changes with therapy-with additional spatial resolution unavailable from standard clinical testing.
- Conditions
- Cystic Fibrosis
- Being tested
- 129Xe
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
129XeDrug
Rapid spatial mapping of lung, liver, and pancreatic structure and function is now possible with a combination of hyperpolarized 129Xe and traditional proton MRI, all absent sedation and ionizing radiation.
| Study group | Role | What they receive |
|---|---|---|
| Pre Trikafta | Experimental | 129Xe MRI Receives: Drug: 129Xe |
| Post Trikafta | Experimental | 129Xe MRI Receives: Drug: 129Xe |
What is being measured
The primary outcome is the main question the study is designed to answer.
Ventilation Defect Percentage change from baseline
For pulmonary MRI, the primary outcome measure is the change in 129Xe ventilation defect percentage (VDP) from pre-therapy baseline to the one-year follow-up visit.
Measured: 1 year
Pancreas volume
For pancreatic MRI, the primary outcome measure is change in pancreas volume normalized to BSA between pre-therapy baseline and one-year follow-up visit.
Measured: 1 year
Secondary outcomes (2)
Abdominal T1 values
Changes in MRI T1 average in the liver and pancreas, from baseline to follow up at 1 year
Measured: 1 year
Lung reader score
Changes in reader score for visible structural defects from proton MRI, from baseline to follow up at 1 year
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Aug 2022
- Study start1 May 2022
- Primary completion1 Dec 2028 (estimated)
- Study completion31 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- 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 4
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Collaborators
- University of Kansas, University of Iowa, University of Virginia
- Responsible party
- Principal investigator, Jason Woods, Children's Hospital Medical Center, Cincinnati
- Investigators
- Jason Woods, PhD, Principal investigator, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05517655
- Sponsor's protocol ID
- 2021-0325
- Acronym
- BEGINNING
- Status last verified
- Sep 2026
- First submitted
- 6 Apr 2022
- Last updated
- 22 Sep 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 22 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- BEGIN Novel ImagiNG Biomarkers
- ClinicalTrials.gov ID
- NCT05517655
- Acronym / sponsor ID
- BEGINNING / 2021-0325
- Conditions
- Cystic Fibrosis
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 19 participants (actual)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Collaborators
- University of Kansas, University of Iowa, University of Virginia
- Study start
- 1 May 2022 (actual)
- Primary completion
- 1 Dec 2028 (estimated)
- Study completion
- 31 Dec 2029 (estimated)
- First posted
- 26 Aug 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To determine the treatment effect of triple-combination therapy in 6-8 year olds after presumed FDA approval, using rapid structural and functional pulmonary and abdominal MRI (UTE and 129Xe).
Detailed description
The overall hypothesis is that multi-organ MRI will provide more sensitive, robust outcome measures in young CF patients than traditional measures employed in the BEGIN study and that these novel measures will be more sensitive to treatment effects, tested here by comparison before and after triple-combination modulator therapy. By understanding the nature of early lung obstruction and characteristic changes in the liver and pancreas over time, we continue to lay the groundwork for more personalized medicine in the future.
Assessing treatment response and clinical benefit in children with CF who are clinically normal per standard outcomes (e.g., spirometry, pancreatic function) will become paramount as triplecombination therapy is extended to younger patients with milder CF clinical presentation than their historic peers. Here the sensitivity and profile free of ionizing-radiation exposure of MRI can be leveraged to follow an individual with CF over time to quantify changes with therapy-with additional spatial resolution unavailable from standard clinical testing.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Pre Trikafta | Experimental | Drug: 129Xe129Xe MRI |
| Post Trikafta | Experimental | Drug: 129Xe129Xe MRI |
Who may qualify
- Ages
- 6 to 8 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (10 listed)
- Written informed consent (and assent where appropriate) obtained from the subject or subject's legal representative.
- Willingness to adhere to the study-visit schedule and other protocol requirements.
- Ages 6-8 years old at baseline MRI visit (may be enrolled up to 60 days before 6th birthday).
- Documentation of CF diagnosis as evidenced by one or more clinical features consistent with the CF phenotype and one or more of the following criteria:
- Physician intent to prescribe triple-combination therapy
Key exclusion criteria (9 listed)
- Individuals currently on ivacaftor therapy (including Kalydeco, Orkambi, and Symdeko) and with at least one gating mutation. Gating mutations include G551D, G178R, S549N, S549R, G551S, G970R, G1244E, S1251N, S1255P, or G1349D.
- Acute respiratory symptoms (e.g. wheezing) at the time of the MRI.
- Acute respiratory infection, defined as increased cough, wheezing or respiratory rate in the 28 days prior to enrollment.
- Chronic lung disease not related to CF
- Chronic liver disease not related to CF
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 |
|---|---|---|
| 129XeRapid spatial mapping of lung, liver, and pancreatic structure and function is now possible with a combination of hyperpolarized 129Xe and traditional proton MRI, all absent sedation and ionizing radiation. | Drug | Post Trikafta, Pre Trikafta |
Primary outcome
Ventilation Defect Percentage change from baseline
Time frame: 1 year
For pulmonary MRI, the primary outcome measure is the change in 129Xe ventilation defect percentage (VDP) from pre-therapy baseline to the one-year follow-up visit.
Pancreas volume
Time frame: 1 year
For pancreatic MRI, the primary outcome measure is change in pancreas volume normalized to BSA between pre-therapy baseline and one-year follow-up visit.
2 secondary outcomes
Abdominal T1 values
Time frame: 1 year
Lung reader score
Time frame: 1 year
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Sep 2026.
- Individual participant dataSharing planned: Undecided
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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.
Jason Woods, PhD principal investigator
Children's Hospital Medical Center, Cincinnati
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing 129Xe in people with Cystic Fibrosis, run by Children's Hospital Medical Center, Cincinnati at 3 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 9 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: under way, not looking for participants, as of the sponsor's last update on 22 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
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?
Ventilation Defect Percentage change from baseline (time frame: 1 year). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 22 Sep 2026.