NCT05517655

BEGIN Novel ImagiNG Biomarkers

• Status: Recruiting
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 44 (estimated)
• Sponsor: Children's Hospital Medical Center, Cincinnati · Academic / Other
• Sex: All
• Age: 6 Years – 8 Years
• Healthy volunteers: Not accepted

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).

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

Interventions

Type	Name	Description
DRUG	129Xe	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.

Timeline

• Start date: 2022-05-01
• Primary completion: 2028-11-01
• Completion: 2028-11-01
• First posted: 2022-08-26
• Last updated: 2024-11-21

Locations

3 sites across 1 country: United States

Regulatory

• FDA-regulated drug study