NCT05517655 · Children's Hospital Medical Center, Cincinnati
BEGIN Novel ImagiNG Biomarkers
(BEGINNING)
What this study is about
To determine the treatment effect of triple-two or more treatments used together in 6-8 year olds after presumed FDA approval, using rapid structural and functional pulmonary and abdominal MRI (UTE and 129Xe).
View original scientific description
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).
Interventions
DRUG
129Xe
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.
Primary outcome measures
Ventilation Defect Percentage change from baseline
Time frame: 1 year
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.
Pancreas volume
Time frame: 1 year
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.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Written informed consent (and assent where appropriate) obtained from the subject or subject's legal representative. 2. Willingness to adhere to the study-visit schedule and other protocol requirements. 3. Ages 6-8 years old at baseline MRI visit (may be enrolled up to 60 days before 6th birthday). 4. 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: 1. Sweat chloride equal to or greater than 60 mEq/liter by quantitative pilocarpine iontophoresis test 2. Two well-characterized mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene 5. Physician intent to prescribe triple-combination therapy 6. Clinically-stable with no respiratory tract infection at the time of enrollment. 7. No change in chronic maintenance therapies in the 28 days prior to enrollment. 8. Ability to cooperate with MRI procedures
Where
- Kansas City, Kansas
- Cincinnati, Ohio
- Charlottesville, Virginia
Collaborators
University of Kansas, University of Iowa, University of Virginia
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Nov 21, 2024 · Source of record for eligibility and locations