NCT05517655

Brief Summary

To determine the treatment effect of triple-combination therapy in 6-8 year olds after presumed FDA approval, using rapid structural and functional pulmonary and abdominal MRI (UTE and 129Xe).

Trial Health

77
On Track

Trial Health Score

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

Enrollment
44

participants targeted

Target at P25-P50 for phase_4

Timeline
30mo left

Started May 2022

Longer than P75 for phase_4

Geographic Reach
1 country

3 active sites

Status
recruiting

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 Progress62%
May 2022Nov 2028

First Submitted

Initial submission to the registry

April 6, 2022

Completed
25 days until next milestone

Study Start

First participant enrolled

May 1, 2022

Completed
4 months until next milestone

First Posted

Study publicly available on registry

August 26, 2022

Completed
6.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2028

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2028

Last Updated

November 21, 2024

Status Verified

November 1, 2024

Enrollment Period

6.5 years

First QC Date

April 6, 2022

Last Update Submit

November 19, 2024

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (2)

  • Ventilation Defect Percentage change from baseline

    For pulmonary MRI, the primary outcome measure is the change in 129Xe ventilation defect percentage (VDP) from pre-therapy baseline to the one-year follow-up visit.

    1 year

  • Pancreas volume

    For pancreatic MRI, the primary outcome measure is change in pancreas volume normalized to BSA between pre-therapy baseline and one-year follow-up visit.

    1 year

Secondary Outcomes (2)

  • Abdominal T1 values

    1 year

  • Lung reader score

    1 year

Study Arms (2)

Pre Trikafta

EXPERIMENTAL

129Xe MRI

Drug: 129Xe

Post Trikafta

EXPERIMENTAL

129Xe MRI

Drug: 129Xe

Interventions

129XeDRUG

Rapid spatial mapping of lung, liver, and pancreatic structure and function is now possible with a combination of hyperpolarized 129Xe and traditional proton MRI, all absent sedation and ionizing radiation.

Post TrikaftaPre Trikafta

Eligibility Criteria

Age6 Years - 8 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Written informed consent (and assent where appropriate) obtained from the subject or subject's legal representative.
  • Willingness to adhere to the study-visit schedule and other protocol requirements.
  • Ages 6-8 years old at baseline MRI visit (may be enrolled up to 60 days before 6th birthday).
  • Documentation of CF diagnosis as evidenced by one or more clinical features consistent with the CF phenotype and one or more of the following criteria:
  • Sweat chloride equal to or greater than 60 mEq/liter by quantitative pilocarpine iontophoresis test
  • Two well-characterized mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene
  • Physician intent to prescribe triple-combination therapy
  • Clinically-stable with no respiratory tract infection at the time of enrollment.
  • No change in chronic maintenance therapies in the 28 days prior to enrollment.
  • Ability to cooperate with MRI procedures

You may not qualify if:

  • Individuals currently on ivacaftor therapy (including Kalydeco, Orkambi, and Symdeko) and with at least one gating mutation. Gating mutations include G551D, G178R, S549N, S549R, G551S, G970R, G1244E, S1251N, S1255P, or G1349D.
  • Acute respiratory symptoms (e.g. wheezing) at the time of the MRI.
  • Acute respiratory infection, defined as increased cough, wheezing or respiratory rate in the 28 days prior to enrollment.
  • Chronic lung disease not related to CF
  • Chronic liver disease not related to CF
  • Acute pancreatitis, defined by clinical criteria (45).
  • Chronic pancreatic disease not related to CF.
  • Physical findings that would compromise the safety of the subject or the quality of the study data as determined at the discretion of the site investigator.
  • Any other condition that, in the opinion of the Site Investigator/designee, would preclude informed consent or assent, make study participation unsafe, complicate interpretation of study outcome data, or otherwise interfere with achieving the study objectives.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

ACTIVE NOT RECRUITING

Carrie Stevens

Cincinnati, Ohio, 45229, United States

ACTIVE NOT RECRUITING

University of Virginia

Charlottesville, Virginia, 22903, United States

RECRUITING

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

  • Jason Woods, PhD

    Children's Hospital Medical Center, Cincinnati

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Carrie Stevens, BS

CONTACT

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

Penny New, BS

CONTACT

(513) 636-9973Penny.New@cchmc.org

Study Design

Study Type
interventional
Phase
phase 4
Allocation
NON RANDOMIZED
Masking
SINGLE
Who Masked
OUTCOMES ASSESSOR
Purpose
DIAGNOSTIC
Intervention Model
CROSSOVER
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Principal Investigator

Study Record Dates

First Submitted

April 6, 2022

First Posted

August 26, 2022

Study Start

May 1, 2022

Primary Completion (Estimated)

November 1, 2028

Study Completion (Estimated)

November 1, 2028

Last Updated

November 21, 2024

Record last verified: 2024-11

Locations

US(3)