BEGIN Novel ImagiNG Biomarkers

Recruiting now · Phase 4

Conditions studied: Cystic Fibrosis

In brief

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).

Key facts

Study ID

NCT05517655

Run by

Children's Hospital Medical Center, Cincinnati

People needed

44

Starts

2022-05-01

Expected to finish

2028-11-01

Last updated by the study team

2024-11-21

Who can join

Age: 6 and older, up to 8. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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
• 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

You may not qualify if…

• 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).
• 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.

Where it is running

• University of Virginia — Charlottesville, Virginia, United States (enrolling)
• University of Kansas Medical Center — Kansas City, Kansas, United States
• Carrie Stevens — Cincinnati, Ohio, United States

Full record on ClinicalTrials.gov

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