← Back to Search

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 28 days

Awards & highlights

HyPOINT 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.Check my eligibility

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.See study design

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.

HyPOINT Trial Eligibility Criteria

Inclusion Criteria

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

Select...

I can follow the study's schedule and requirements.

HyPOINT Trial 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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

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

Xe VDP Assessment

Secondary outcome measures

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

HyPOINT 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 / 1Eligibility criteria met

Find a Location

Who is running the clinical trial?

Children's Hospital Medical Center, CincinnatiLead Sponsor

815 Previous Clinical Trials

6,531,532 Total Patients Enrolled

22 Trials studying Cystic Fibrosis

1,121 Patients Enrolled for Cystic Fibrosis

University of VirginiaOTHER

753 Previous Clinical Trials

1,244,967 Total Patients Enrolled

5 Trials studying Cystic Fibrosis

321 Patients Enrolled for Cystic Fibrosis

University of Wisconsin, MadisonOTHER

1,182 Previous Clinical Trials

3,167,323 Total Patients Enrolled

Media Library

Initiation of CFTR Modulator Clinical Trial Eligibility Overview. Trial Name: NCT04259970 — Phase 4

Cystic Fibrosis Research Study Groups: Phase 1, Initiation of CFTR Modulator

Cystic Fibrosis Clinical Trial 2023: Initiation of CFTR Modulator Highlights & Side Effects. Trial Name: NCT04259970 — Phase 4

Initiation of CFTR Modulator 2023 Treatment Timeline for Medical Study. Trial Name: NCT04259970 — Phase 4

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many sites are administering the clinical trial?

"4 sites are currently running this clinical trial, situated in Charlottesville, Toronto, Madison and other locations. To minimize travelling requirements for the patient, it is most beneficial to seek out a clinic located nearby."

Answered by AI

Is there eligibility criteria for joining this medical trial?

"This clinical trial seeks 64 individuals between the ages of 6 to 18 who have cystic fibrosis. The applicants must also demonstrate their willingness and capability in following the visit schedule, possess a certain genotype, take specific medications, and meet age requirements depending on which phase they are applying for."

Answered by AI

Is there an ongoing process of enlisting participants in this experiment?

"This medical trial, first posted on January 13th 2020 and last updated on August 29th 2022, is not recruiting at present. However, there are 413 other clinical trials that are actively seeking participants."

Answered by AI