Cystic FibrosisBEGINNCT04509050
Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children
An observational study of Cystic Fibrosis, run by Sonya Heltshe at 35 sites in United States.
Trial summary
This is a two-part, multi-center, prospective longitudinal, exploratory study of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators and their impact on children with cystic fibrosis (CF). Read more about this study
- Who is it for?
- Ages Up to 10 years
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Ivacaftor or elexacaftor/tezacaftor/ivacaftor
- How many people
- 210 participants
- Where
- 35 sitesUnited States
- Study dates
- 18 Nov 2020to 31 Dec 2027
- Phase
- Observational
- 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 Up to 10 years
- All sexes
- No healthy volunteers
- Child
Study population: Part A - children with confirmed diagnosis of cystic fibrosis who are less than 10 years of age and not on ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy Part B - children with confirmed diagnosis of cystic fibrosis who are less than 7 years of age (or participated in Part A of the study) with an intention to start ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy
Who may be able to join
- Part A:
- Less than 10 years of age at the first study visit.
- Documentation of a CF diagnosis.
- Part B:
- Participated in Part A OR less than 7 years of age at the first study visit.
- Documentation of a CF diagnosis.
- CFTR mutations consistent with FDA labeled indication of highly effective modulator therapy (ivacaftor or elexacaftor/tezacaftor/ivacaftor).
- Physician intent to prescribe ivacaftor or elexacaftor/tezacaftor/ivacaftor.
Who cannot join
- Part A and Part B:
- Use of an investigational drug within 28 days prior to and including the first study visit.
- Use of ivacaftor or elexacaftor/tezacaftor/ivacaftor within the 28 days prior to and including the first study visit.
- Use of chronic oral corticosteroids within the 28 days prior to and including the first study visit.
Trial locations and contacts
35 sites in 1 country. Contact the study team or a site near you to ask about taking part.
35 sites in 1 country
United States35 sites, 35 recruiting
- Recruiting
University of Michigan, Michigan Medicine
Ann Arbor, Michigan, 48109
Dawn Kruse, dmkruse@med.umich.edu
- Recruiting
Children's Hospital Colorado
Aurora, Colorado, 80045
Mary Cross, mary.cross@childrenscolorado.org
- Recruiting
The Children's Hospital Alabama, University of Alabama at Birmingham
Birmingham, Alabama, 35233
Heather Hathorne, hyhathorne@uabmc.edu
- Recruiting
Boston Children's Hospital
Boston, Massachusetts, 02115
Robert Fowler, Robert.fowler@childrens.harvard.edu
- Recruiting
The Cystic Fibrosis Center of Western New York
Buffalo, New York, 14203
Christine Roach, croach@upa.chob.edu
- Recruiting
Vermont Children's Hospital
Burlington, Vermont, 05401
Julie Sweet, julie.sweet@uvmhealth.org
- Recruiting
University of Virginia
Charlottesville, Virginia, 22903
Erica Tharrington, SFV6AB@hscmail.mcc.virginia.edu
- Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Kelly Thornton, Kelly.Thornton@cchmc.org
- Recruiting
Nationwide Children's Hospital
Columbus, Ohio, 43205
Diana Gilmore, Diana.Gilmore@nationwidechildrens.org
- Recruiting
University of Texas Southwestern / Children's Health
Dallas, Texas, 75207
Keianna Brown, Keianna.brown@utsouthwestern.edu
- Recruiting
Cook Children's Medical Center
Fort Worth, Texas, 76104
Anna Reyes, anna.reyes@cookchildrens.org
- Recruiting
Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503
Andrew James, andrew.james@corewellhealth.org
- Recruiting
Hershey Medical Center Pennsylvania State University
Hershey, Pennsylvania, 17033
Diane M Kitch, dkitch@pennstatehealth.psu.edu
- Recruiting
Baylor College of Medicine
Houston, Texas, 77030
Tracy Mosely, tlmosely@texaschildrens.org
- Recruiting
Riley Hospital for Children
Indianapolis, Indiana, 46202
Lisa Bendy, lbendy@iu.edu
- Recruiting
University of Iowa
Iowa City, Iowa, 52242
Mary Teresi, mary-teresi@uiowa.edu
- Recruiting
Nemours Children's Clinic
Jacksonville, Florida, 32207
Jennifer (Jenn) Gafford, Jennifer.gafford@nemours.org
- Recruiting
University of Kansas Medical Center
Kansas City, Kansas, 66160
Lawrence Scott, lscott2@kumc.edu
- Recruiting
Children's Mercy Kansas City
Kansas City, Missouri, 64108
Candy Schmoll, cschmoll@cmh.edu
- Recruiting
University of Wisconsin
Madison, Wisconsin, 53792
Melanie Nelson, mnelson@pediatrics.wisc.edu
- Recruiting
University of Miami
Miami, Florida, 33136
Ylber (Ivan) Whitaker, yiw2@miami.edu
- Recruiting
Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, 55404
Camerone Bey, Camerone.bey@childrensmn.org
- Recruiting
The Minnesota Cystic Fibrosis Center
Minneapolis, Minnesota, 55455
CF Trials Contact, cftrials@umn.edu
- Recruiting
Children's Hospital of New York
New York, New York, 10032
Hossein Sadeghi, HS762@cumc.Columbia.edu
- Recruiting
Oklahoma Cystic Fibrosis Center
Oklahoma City, Oklahoma, 73104
CF Center Participant Contact, cfresearchteam@ouhsc.edu
- Recruiting
The Nemours Children's Clinic - Orlando
Orlando, Florida, 32827
Sherry Wooldridge, sherry.wooldridge@nemours.org
- Recruiting
Stanford University Medical Center
Palo Alto, California, 94304
Jacquelyn Spano, jmzirbes@stanford.edu
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Erin Donnelly, Donnellye4@email.chop.edu
- Recruiting
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
Elizabeth Hartigan, elizabeth.hartigan@chp.edu
- Recruiting
Oregon Health Sciences University
Portland, Oregon, 97239
Jenna Bucher, bucherj@ohsu.edu
- Recruiting
Primary Children's Cystic Fibrosis Center
Salt Lake City, Utah, 84113
Jane B Vroom, jane.vroom@hsc.utah.edu
- Recruiting
Seattle Children's Hospital
Seattle, Washington, 98105
Sharon McNamara, sharon.mcnamara@seattlechildrens.org
- Recruiting
St. Louis Children's Hospital
St Louis, Missouri, 63110
Irma Bauer, irmabauer@wustl.edu
- Recruiting
SUNY Upstate Medical University
Syracuse, New York, 13210
Mary Forell, forellm@upstate.edu
- Recruiting
New York Medical College at Westchester Medical Center
Valhalla, New York, 10595
Zachary Messer, Zachary_Messer@nymc.edu
About this study
Official title: A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function in Infants and Young Children (BEGIN Study)
This is a two-part, multi-center, prospective longitudinal, exploratory study of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators and their impact on children with cystic fibrosis (CF).
Read the detailed description
This is a two-part, multi-center, prospective longitudinal, exploratory study of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators and their impact in children with cystic fibrosis (CF) on endocrine growth factors and height, gastrointestinal function and gut microbiome, lung function and respiratory microbiome, liver and pancreatic function, sweat chloride, inflammatory markers, and bone health.
Total duration of the study is expected to be 10 years. Part A will be a prospective cross-sequential study to describe the natural history of hormonal growth factors in early childhood and assess the feasibility of additional measurements. In Part A, subjects will have up to 8 visits over a period of up to 5 years.
Part B will be a prospective longitudinal study to observe the effects of administration of either ivacaftor or elexacaftor/tezacaftor/ivacaftor (elex/tez/iva) on growth. In Part B, subjects will have 1 "before ivacaftor or elex/tez/iva" visit within 30 days before initiation of the therapy and 8 "after ivacaftor or elex/tez/iva" visits over a 60-month follow-up period.
- Conditions
- Cystic Fibrosis
- Being tested
- Ivacaftor or elexacaftor/tezacaftor/ivacaftor
- Keywords
- Cystic Fibrosis, CF, CFTR Modulator, triple combination therapy, elexacaftor, tezacaftor, ivacaftor
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ivacaftor or elexacaftor/tezacaftor/ivacaftorDrug
Also called Kalydeco or Trikafta, Vertex (VX)-770 or VX-445/VX-661/VX-770
In Part B, approved CFTR modulator as prescribed at the discretion of the treating physician -not dictated by the BEGIN investigators
| Study group | Role | What they receive |
|---|---|---|
| Part A | Children with CF not on ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy. | |
| Part B | Children with CF planning to start ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy. Participants from the Part A cohort of this study may enroll into the Part B cohort if they become eligible for these CFTR modulator therapies and plan to start them. Receives: Drug: Ivacaftor or elexacaftor/tezacaftor/ivacaftor |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part A Primary Outcome Measure: Change in weight-for-age z-scores
Weight-for-age z-scores over time
Measured: Baseline to 12 months
Part A Primary Outcome Measure: Change in height-for-age z-scores
Height-for-age z-scores over time
Measured: Baseline to 12 months
Part B Primary Outcome Measure: Change in weight-for-age z-scores
Change in weight-for-age z-scores from baseline
Measured: Baseline to 12 months
Part B Primary Outcome Measure: Change in height-for-age z-scores
Change in height-for-age z-scores from baseline
Measured: Baseline to 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Aug 2020
- Study start18 Nov 2020
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2027 (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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 210 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sonya Heltshe
- Sponsor type
- University, hospital or other
- Collaborators
- Cystic Fibrosis Foundation, University of Washington, University of Alabama at Birmingham
- Responsible party
- Sponsor-investigator, Sonya Heltshe, Seattle Children's Hospital
- Investigators
- Bonnie Ramsey, MD, Principal investigator, Seattle Children's HospitalLucas Hoffman, MD PhD, Principal investigator, University of Washington/Seattle Children'sKatie Larson Ode, MD, Principal investigator, University of Iowa
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04509050
- Sponsor's protocol ID
- BEGIN-OB-19
- Acronym
- BEGIN
- Status last verified
- Jul 2026
- First submitted
- 6 Aug 2020
- Last updated
- 8 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function in Infants and Young Children (BEGIN Study)
- ClinicalTrials.gov ID
- NCT04509050
- Acronym / sponsor ID
- BEGIN / BEGIN-OB-19
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic Fibrosis, CF, CFTR Modulator, triple combination therapy, elexacaftor, tezacaftor, ivacaftor
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 210 participants (estimated)
- Lead sponsor
- Sonya HeltsheUniversity, hospital or other
- Collaborators
- Cystic Fibrosis Foundation, University of Washington, University of Alabama at Birmingham
- Study start
- 18 Nov 2020 (actual)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 11 Aug 2020 (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.
This is a two-part, multi-center, prospective longitudinal, exploratory study of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators and their impact on children with cystic fibrosis (CF).
Detailed description
This is a two-part, multi-center, prospective longitudinal, exploratory study of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators and their impact in children with cystic fibrosis (CF) on endocrine growth factors and height, gastrointestinal function and gut microbiome, lung function and respiratory microbiome, liver and pancreatic function, sweat chloride, inflammatory markers, and bone health.
Total duration of the study is expected to be 10 years. Part A will be a prospective cross-sequential study to describe the natural history of hormonal growth factors in early childhood and assess the feasibility of additional measurements. In Part A, subjects will have up to 8 visits over a period of up to 5 years.
Part B will be a prospective longitudinal study to observe the effects of administration of either ivacaftor or elexacaftor/tezacaftor/ivacaftor (elex/tez/iva) on growth. In Part B, subjects will have 1 "before ivacaftor or elex/tez/iva" visit within 30 days before initiation of the therapy and 8 "after ivacaftor or elex/tez/iva" visits over a 60-month follow-up period.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Part A | – | –Children with CF not on ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy. |
| Part B | – | Drug: Ivacaftor or elexacaftor/tezacaftor/ivacaftorChildren with CF planning to start ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy. Participants from the Part A cohort of this study may enroll into the Part B cohort if they become eligible for these CFTR modulator therapies and plan to start them. |
Who may qualify
- Ages
- Up to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Part A - children with confirmed diagnosis of cystic fibrosis who are less than 10 years of age and not on ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy Part B - children with confirmed diagnosis of cystic fibrosis who are less than 7 years of age (or participated in Part A of the study) with an intention to start ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy
Key inclusion criteria (8 listed)
- Part A:
- Participated in Part A OR less than 7 years of age at the first study visit.
- Documentation of a CF diagnosis.
- CFTR mutations consistent with FDA labeled indication of highly effective modulator therapy (ivacaftor or elexacaftor/tezacaftor/ivacaftor).
- Physician intent to prescribe ivacaftor or elexacaftor/tezacaftor/ivacaftor.
Key exclusion criteria (4 listed)
- Part A and Part B:
- Use of an investigational drug within 28 days prior to and including the first study visit.
- Use of ivacaftor or elexacaftor/tezacaftor/ivacaftor within the 28 days prior to and including the first study visit.
- Use of chronic oral corticosteroids within the 28 days prior to and including the first study visit.
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 |
|---|---|---|
| Ivacaftor or elexacaftor/tezacaftor/ivacaftorAlso: Kalydeco or Trikafta, Vertex (VX)-770 or VX-445/VX-661/VX-770In Part B, approved CFTR modulator as prescribed at the discretion of the treating physician -not dictated by the BEGIN investigators | Drug | Part B |
Primary outcome
Part A Primary Outcome Measure: Change in weight-for-age z-scores
Time frame: Baseline to 12 months
Weight-for-age z-scores over time
Part A Primary Outcome Measure: Change in height-for-age z-scores
Time frame: Baseline to 12 months
Height-for-age z-scores over time
Part B Primary Outcome Measure: Change in weight-for-age z-scores
Time frame: Baseline to 12 months
Change in weight-for-age z-scores from baseline
Part B Primary Outcome Measure: Change in height-for-age z-scores
Time frame: Baseline to 12 months
Change in height-for-age z-scores from baseline
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 8 Jul 2026.
- 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.
Bonnie Ramsey, MD principal investigator
Seattle Children's Hospital
Lucas Hoffman, MD PhD principal investigator
University of Washington/Seattle Children's
Katie Larson Ode, MD principal investigator
University of Iowa
35 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis, run by Sonya Heltshe at 35 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 8 inclusion and 4 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: looking for participants now, as of the sponsor's last update on 8 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 35 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.
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?
Contact Rachael Buckingham (206-884-7517, rachael.buckingham@seattlechildrens.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Part A Primary Outcome Measure: Change in weight-for-age z-scores (time frame: Baseline to 12 months). The study lists 4 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 8 Jul 2026.