Evaluating new MRI techniques for treating cystic fibrosis in young children

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This study is testing a new treatment for cystic fibrosis in kids aged 6-8 to see how well it works using advanced MRI scans to look at their lungs, liver, and pancreas.

Quick facts

• Phase: PHASE4
• Study type: Interventional
• Enrollment: 44 (estimated)
• Ages: 6 Years to 8 Years
• Sex: All
• Sponsor: Children's Hospital Medical Center, Cincinnati (other)
• Drugs / interventions: radiation
• Locations: 3 sites (Kansas City, Kansas and 2 other locations)
• Trial ID: NCT05517655 on ClinicalTrials.gov

What this trial studies

This study aims to assess the effectiveness of a triple-combination therapy in children aged 6-8 years with cystic fibrosis using advanced MRI techniques. The research will compare traditional outcome measures with novel multi-organ MRI assessments to better understand treatment effects on lung, liver, and pancreas function. By leveraging MRI's sensitivity and spatial resolution, the study seeks to provide more personalized treatment insights for young patients with cystic fibrosis. The goal is to enhance the understanding of early disease progression and treatment response in this population.

Who should consider this trial

Good fit: Ideal candidates are children aged 6-8 years diagnosed with cystic fibrosis who are clinically stable and eligible for triple-combination therapy.

Not a fit: Patients with severe respiratory infections or those unable to undergo MRI procedures may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to more effective monitoring and treatment strategies for young cystic fibrosis patients.

How similar studies have performed: Other studies have shown promise in using advanced imaging techniques for monitoring cystic fibrosis, but this specific approach is relatively novel.

Eligibility criteria

Inclusion Criteria:

1. Written informed consent (and assent where appropriate) obtained from the subject or subject's legal representative.
2. Willingness to adhere to the study-visit schedule and other protocol requirements.
3. Ages 6-8 years old at baseline MRI visit (may be enrolled up to 60 days before 6th birthday).
4. 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:

   1. Sweat chloride equal to or greater than 60 mEq/liter by quantitative pilocarpine iontophoresis test
   2. Two well-characterized mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene
5. Physician intent to prescribe triple-combination therapy
6. Clinically-stable with no respiratory tract infection at the time of enrollment.
7. No change in chronic maintenance therapies in the 28 days prior to enrollment.
8. Ability to cooperate with MRI procedures

Exclusion Criteria:

1. 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.
2. Acute respiratory symptoms (e.g. wheezing) at the time of the MRI.
3. Acute respiratory infection, defined as increased cough, wheezing or respiratory rate in the 28 days prior to enrollment.
4. Chronic lung disease not related to CF
5. Chronic liver disease not related to CF
6. Acute pancreatitis, defined by clinical criteria (45).
7. Chronic pancreatic disease not related to CF.
8. 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.
9. 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 this trial is running

Kansas City, Kansas and 2 other locations

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

Study contacts

• Principal investigator: Jason Woods, PhD — Children's Hospital Medical Center, Cincinnati
• Study coordinator: Carrie Stevens, BS

Conditions: Cystic Fibrosis