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CFTR Modulator Therapy for Cystic Fibrosis (HyPOINT Trial)

Phase 4

Waitlist Available

Led By Jason Woods, PhD

Research Sponsored by Children's Hospital Medical Center, Cincinnati

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Willingness and ability to adhere to the study visit schedule and other protocol requirements

Be younger than 65 years old

Must not have

For females of childbearing potential: Positive urine pregnancy test at Screening or Visit 1 or Lactating

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 28 days

Awards & highlights

Drug Has Already Been Approved

No Placebo-Only Group

Pivotal Trial

Summary

This trial will study if magnetic resonance imaging (MRI) can be used to track the effects of new CFTR modulator therapies, which are expected to provide major health benefits to patients with Cystic Fibrosis.

Who is the study for?

This trial is for people with Cystic Fibrosis who have at least one deltaF508 mutation. They must be able to follow the study schedule, give consent, and have been stable on certain CFTR modulators or not on any such therapy. Ages vary by phase: 6-18 for Phase 1 and 9-18 for Phase 2. Participants need normal organ function and no recent acute illness.

What is being tested?

The trial tests a new way to monitor lung health in Cystic Fibrosis using MRI with hyperpolarized gas, which shows both structure and function of lungs without radiation. It aims to see if this can detect early changes better than current methods when starting triple combination CFTR modulator therapy.

What are the potential side effects?

While specific side effects are not listed here, MRI procedures are generally safe but may include discomfort from lying still during the scan or anxiety in enclosed spaces (claustrophobia). The CFTR modulators' known side effects range from chest tightness to digestive issues.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I can follow the study's schedule and requirements.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am not pregnant or breastfeeding.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 28 days

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~28 days

This trial's timeline: 3 weeks for screening, Varies for treatment, and 28 days for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

The change in Xe VDP after 28 days of triple modulator CFTR therapy

Xe VDP Assessment

Secondary study objectives

Fev1 and LCI Correlation

Overall UTE MRI reader score analysis

The change in overall MRI reader score for UTE MRI 28 days after triple combination therapy is initiated

Awards & Highlights

Drug Has Already Been Approved

The FDA has already approved this drug, and is just seeking more data.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Phase 1Experimental Treatment1 Intervention

Phase 1 will include implementation of a centralized analysis program of repeated 129Xe MRI scanning in CF patients with mild lung disease to define the intra-subject variability of the primary outcome ventilation defect percentage (VDP). Patients will undergo baseline 129Xe MRI scanning and repeated measurements the same day, as well as at 28 days (± 7 days). Phase 1 will establish the intra-subject reproducibility to facilitate future use of 129Xe MRI in multi-site studies. Furthermore, the reproducibility limits defined will inform the overall design of future studies and will compare to established pulmonary function and multiple-breath washout testing (via measurement of the lung clearance index, LCI).

Group II: Initiation of CFTR ModulatorExperimental Treatment1 Intervention

Phase 2 will be an observational study of patients assessed before and after the clinical initiation of triple-combination modulator therapy (after presumed FDA and Health Canada approval). The primary endpoint for Phase 2 is the change of VDP after 28 days of triple-combination modulator therapy. Within Phase 2, this study will also address how highly-effective modulator therapies affect lung function trajectories by measuring 129Xe MRI at 28 days (± 7 days), 6 months (± 28 days), and 12 months (± 28 days) after start of therapy (paralleling time points of the PROMISE study). Finally, to understand how 129Xe MRI can be used in combination with existing measures of lung function (e.g. spirometry, multiple breath washout), the investigators will directly compare the repeated data collected in both Phase 1 and Phase 2 to these established measures of lung function that are currently used in observational and interventional studies.

0 / 2Eligibility criteria met