Hyperpolarized Imaging for New Treatments

Summary

The introduction of triple combination CFTR modulator therapy for patients with Cystic Fibrosis (CF) with at least one copy of the deltaF508 mutation is expected to provide major health benefits, but will also require novel outcome measures that can detect CF lung disease at an early stage, capture the efficacy of new therapies when disease manifestations are limited, as well as determine whether stopping existing chronic maintenance therapies does not have negative effects.

In the past decade, research has focused on the multiple breath washout (MBW) test, as a sensitive outcome measure, especially if highly-effective modulator therapies are initiated in early childhood. Even LCI, however, may not adequately capture early lung function changes, thus warranting investigation of even more sensitive outcome measures.

Magnetic resonance imaging (MRI) has the advantage of being a radiation-free modality, making it more suitable for assessing response to therapy in a shorter time frame with repeated imaging. Inhalation of a hyperpolarized gas enables the visualization and quantification of regional ventilation in the lung and can be combined with structural MRI to assess both structure and function in parallel.

The main Investigator and others have recently formed an international consortium (the 129Xe MRI Clinical Trial Consortium), comprised of both imaging experts and pulmonary clinicians to standardize imaging procedures, thus facilitating multi-site implementations. Data from this proposed study (HyPOINT; Hyperpolarized Imaging for New Treatments) will inform the future utility of MRI for both longitudinal studies to track disease progression over time as well as for future interventional trials. Further, the current study could inform the design of future trials of interventions of patients for whom currently no effective CFTR modulator therapy is available and for patients with rare genotypes thus laying the groundwork for a more personalized medicine approach in the near-term future.

Conditions

• Cystic Fibrosis

Interventions

DRUG

Initiation of CFTR Modulator

Inhaled contrast for MRI occurring at each 4 visits.

Sponsors & Collaborators

• University of Virginia

  collaborator OTHER

• University of Wisconsin, Madison

  collaborator OTHER

• The Hospital for Sick Children

  collaborator OTHER

• Children's Hospital Medical Center, Cincinnati

  lead OTHER

Principal Investigators

• Jason Woods, PhD · Children's Hospital Medical Center, Cincinnati

Study Design

Allocation

NON_RANDOMIZED

Purpose

DIAGNOSTIC

Masking

SINGLE

Model

CROSSOVER

Eligibility

Min Age

6 Years

Max Age

18 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2020-01-13

Primary Completion

2026-05-01

Completion

2026-12-31

FDA Drug

Yes

Countries

• United States
• Canada

Study Locations