NCT04994301

Brief Summary

In this observational study, the investigators evaluate the sensitivity of T1-MRI to identify lung perfusion changes in pediatric patients with CF (age = 6-11) before and after initiating FDA-approved Trikafta therapy. The investigators compare these Lung T1 MRI assessments (% Normal lung perfusion) to currently best-available assessments of lung function in CF patients (i.e., MBW (LCI( and Spirometry (FEV1 % Predicted).

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
48

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started Dec 2020

Typical duration for all trials

Geographic Reach
1 country

3 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

December 15, 2020

Completed
8 months until next milestone

First Submitted

Initial submission to the registry

July 29, 2021

Completed
8 days until next milestone

First Posted

Study publicly available on registry

August 6, 2021

Completed
2.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 15, 2023

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 15, 2023

Completed
Last Updated

September 14, 2026

Status Verified

September 1, 2026

Enrollment Period

3 years

First QC Date

July 29, 2021

Last Update Submit

September 9, 2026

Conditions

Cystic Fibrosis

Keywords

Cystic FibrosisTransmembraneConductanceRegulator

Outcome Measures

Primary Outcomes (1)

  • Lung T1-MRI

    Evaluate lung T1-MRI (% Normal Lung Perfusion) to assess lung perfusion changes associated with FDA-approved Trikafta therapy in pediatric cystic fibrosis patients.

    Visit 1: Before starting Trikafta, Visit 2: 3 months (+/- 15 days) from start of Trikafta, Visit 3: 6 months (+/- 30 days) from start of Trikafta

Secondary Outcomes (2)

  • Spirometry

    Visit 1: Before starting Trikafta, Visit 2: 3 months (+/- 15 days) from start of Trikafta, Visit 3: 6 months (+/- 30 days) from start of Trikafta

  • Multiple breath washout (MBW)

    Visit 1: Before starting Trikafta, Visit 2: 3 months (+/- 15 days) after start of Trikafta, Visit 3: 6 months (+/- 30 days) after start of Trikafta

Study Arms (1)

Patient with Cystic Fibrosis

Patients (male, female) age 5-11 with confirmed diagnosis of Cystic Fibrosis. These patients will begin clinically prescribed FDA-approved Trikafta therapy.

Diagnostic Test: Lung T1 MRI

Interventions

Lung T1 MRIDIAGNOSTIC_TEST

Evaluation of lung T1 MRI to Assess Lung Disease

Patient with Cystic Fibrosis

Eligibility Criteria

Age5 Years - 11 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)
Sampling MethodNon-Probability Sample
Study Population

Patients with cystic fibrosis (CF) who are planning to start Trikafta therapy once approved

You may qualify if:

  • Male or female individuals with a diagnosis of cystic fibrosis and have at least one copy of the F508del mutation.

You may not qualify if:

  • Subject who cannot hold their breath for up to 15 seconds.
  • Subjects who are pregnant.
  • Subjects with MRI contraindication (e.g., heart pacemaker, heart defibrillator, metal in within the body.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

Riley Hospital for Children

Indianapolis, Indiana, 46202, United States

Location

CS Mott Children's Hospital

Ann Arbor, Michigan, 48109, United States

Location

University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44106, United States

Location

Related Publications (2)

  • Nasr SZ, Flask CA, Kretzler ME, Slaven JE, Joshi A, Mahani MG, Clem C, Cooper M, Farr S, MacAskill CJ, Gupta A, Ferrebee M, McBennett KA, Ren CL. Lung T1-MRI and Multi-Breath Washout Detect Longitudinal Changes in Children 6-11 Years Old After Beginning Elexacaftor/Tezacaftor/Ivacaftor (ETI) Therapy. Pediatr Pulmonol. 2026 Apr;61(4):e71628. doi: 10.1002/ppul.71628.

    PMID: 42003453RESULT

  • Ren CL, Nasr SZ, Slaven JE, Joshi A, Mahani MG, Clem C, Cooper M, Farr S, MacAskill CJ, Keshock E, Nicholas JL, Ferrebee M, McBennett K, Flask CA. Lung T1 MRI assessments in children with mild cystic fibrosis lung disease. Pediatr Pulmonol. 2024 Oct;59(10):2464-2471. doi: 10.1002/ppul.27039. Epub 2024 May 2.

    PMID: 38695557RESULT

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, Diseases

Study Officials

  • Chris Flask, PhD

    Case Western Reserve University

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 29, 2021

First Posted

August 6, 2021

Study Start

December 15, 2020

Primary Completion

December 15, 2023

Study Completion

December 15, 2023

Last Updated

September 14, 2026

Record last verified: 2026-09

Locations

US(3)