Regional Monitoring of CF Lung Disease

NCT06339593 | Recruiting Early Phase 1 DMC FDA Drug

• 1 other identifier: 2023-0391
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

Brief Summary

The main reason for this research study is to learn more about some new tests that are being developing for patients with Cystic Fibrosis (CF) to measure changes in the lungs. In this study, the focus will be to learn how stopping Airway Clearance (ACT) and re-starting ACT can affect these tests. These new tests include using a breathable gas called Xenon (Xe) with MRI (magnetic resonance imaging) to improve the pictures of changes in the lungs. The Xenon (Xe) gas that has been treated to have a larger MRI signal (also called hyperpolarized). The other new test is called LCI (Lung Clearance Index) that can measure how well the lungs are working. The MRI machine used in this study has been approved by the U.S. Food and Drug Administration (FDA) and is commercially available for sale in the USA. Hyperpolarized Xe gas is an FDA-approved, inhaled contrast agent for lung ventilation MRI. The new Xe MRI techniques that are being developed and used for this research study are investigational, meaning these new Xe MRI techniques are not FDA approved, but they are similar to FDA-approved techniques that are used clinically at Cincinnati Children's Hospital Medical Center (CCHMC). Xe gas and the new MRI techniques used in this research study have been used for many years in research, including in many research studies conducted at CCHMC like this one.

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

• Assessing functional lung abnormalities via Xenon MRI

  We will be using Hyperpolarized Xenon MRI images of participants with Cystic Fibrosis

  two weeks

Secondary Outcomes (3)

• Assessing patients who have self-withdrawn airway clearance treatment to determine ventilation abnormalities.

  two weeks

• Assessing patients who have self-withdrawn airway clearance treatment to determine ventilation abnormalities.

  two weeks

• Assessing patients who have self-withdrawn airway clearance treatment to determine

  2 weeks

Other Outcomes (4)

• Change in Xenon Ventilation Defect Process (VDP)

  4 weeks

• Change in Xenon Ventilation Defect Process (VDP)

  4 weeks

• Changes in Xenon Ventilation Defect Process (VDP)

  4 weeks

Study Arms (3)

CF pts on MACT

OTHER

Males and females from 12 to 21 years old who have been prescribed Mechanical Airway Clearance Therapy for CF.

Drug: Hyperpolarized Xe129

Self withdrawn ACT

OTHER

To perform a stepwise ACT re-initiation trial in fifteen 12-21 y.o. patients who have self-withdrawn airway clearance treatment (defined as ≤ 3x/week). UTE and Xe MRI, spirometry, and multiple-breath washout will be performed at baseline, after increasing treatment to 7x/week for 1 week and then 14x/week for 2 weeks, with daily logging to aid compliance and study engagement.

Procedure: Airway-clearance vest; Drug: Hyperpolarized Xe129

Low MRI abnormalities and high FEV1

OTHER

To perform a stepwise ACT withdrawal trial in fifteen 12-21 y.o. patients who have low MRI abnormalities and high FEV1. Patients will be studied at baseline, after decreasing ACT to 7x/week for 1 week, and after decreasing ACT to 3x/week for 1 week.

Procedure: Airway-clearance vest; Drug: Hyperpolarized Xe129

Interventions

Airway-clearance vest PROCEDURE

Airway clearance devices will be re-initiated for those who have self withdrawn and will be withdrawn temporarily for those who are using them regularly and have high FEV1 and low Xe MRI ventilation defects.

Low MRI abnormalities and high FEV1; Self withdrawn ACT

Hyperpolarized Xe129 DRUG

Hyperpolarized Xe MRI is FDA Approved for evaluation of ventilation in adults and children 12 and above and will be used as a diagnostic test here. Earlier studies using Xe MRI have shown its efficacy in exploring CF and other lung diseases in pediatric and adult populations, and it has been found to be much more sensitive to early lung disease than traditional metrics like spirometry and MBW.

CF pts on MACT; Low MRI abnormalities and high FEV1; Self withdrawn ACT

Eligibility Criteria

• Age: 12 Years - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Written informed consent (and assent when applicable) obtained from subject or subject's legal representative.
• Use of highly effective modulators for more than 30 days (ie. Trikafta) 3 Willingness and ability to adhere to the study visit schedule and other protocol requirements.
• Documentation of a CF diagnosis with prescription of Mechanical ACT 5 Ages 12-21 inclusive, at the time of consent. 6 Clinically stable with no respiratory tract infection or recent exacerbations. 7 Treating CF physician agreeable to study procedures. Only applicable to Aim 3.
• No change in chronic maintenance therapies in the 28 days prior to enrollment.
• Ability to cooperate with MRI procedures.

You may not qualify if:

• For females of childbearing potential: Positive urine pregnancy test or Lactating.
• Acute respiratory symptoms (e.g., wheezing) at the time of the MRI
• Chronic lung or liver or pancreatic disease not related to CF.
• Any other condition that, in the opinion of the Investigator, would preclude informed consent or assent, make study participation unsafe, complicate interpretation of study outcome data, or otherwise interfere with achieving the study objectives.

Sponsors & Collaborators

• Children's Hospital Medical Center, Cincinnati: lead

Study Sites (1)

Cincinnati Children's Hospital

Cincinnati, Ohio, 45229, United States

RECRUITING

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Jason C Woods, PhD

  Cincinnati Children's Hospital Medica Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Carrie Stevens, BS

CONTACT

(513) 636-9973; carrie.stevens@cchmc.org

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: PhD, Director, Center for Pulmonary Medicine Imaging Research

Study Record Dates

• First Submitted: February 6, 2024
• First Posted: April 1, 2024
• Study Start: January 5, 2024
• Primary Completion (Estimated): January 1, 2028
• Study Completion (Estimated): January 1, 2028
• Last Updated: January 14, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)