Monitoring Response to Orkambi in Cystic Fibrosis Lung Disease by Inhaled Xenon MRI

• Register: ClinicalTrials.gov
• Last refreshed on: 6 November 2023
• Main ID: NCT02848560
• Date of registration: 26/07/2016
• Prospective Registration: No
• Primary sponsor: Children's Hospital Medical Center, Cincinnati
• Public title: Monitoring Response to Orkambi in Cystic Fibrosis Lung Disease by Inhaled Xenon MRI
• Scientific title: Validation of MRI as a Sensitive Tool to Longitudinally Monitor CF Lung Disease Progression and Response to CFTR Modulator Therapy in Young Children With CF
• Date of first enrolment: March 2016
• Target sample size: 38
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT02848560
• Study type: Observational

Countries of recruitment

United States

Contacts

• Name: Jason Woods, PhD
• Affiliation: Children's Hospital Medical Center, Cincinnati

Key inclusion & exclusion criteria

Inclusion Criteria:

Treatment group:

- male or female between the ages of 6 through 12 years at enrollment

- two copies of the F508del CFTR mutation

- anticipated to be a candidate for treatment with orkambi

Control group:

- male or female between the ages of 6 through 12 years at enrollment

- two non-functional CFTR mutations with one of them being F508del CFTR mutation

- not eligible for CFTR modulation therapy

Exclusion Criteria:

- FEV1 percent predicted of <60%

- standard MRI exclusions (metal implants, claustrophobia)

- pregnancy



Age minimum: 6 Years
Age maximum: 12 Years
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: Hyperpolarized Xenon

Primary Outcome(s)

Hyperpolarized 129Xe MRI Image Analysis [Time Frame: Visit 4 (year 3)]

Secondary ID(s)

1R01HL131012-01A1

CINOO1-CF Orkambi and Xe MRI

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

National Heart, Lung, and Blood Institute (NHLBI)