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New treatment option for Cystic Fibrosis
Official title BEGIN Novel ImagiNG Biomarkers
ClinicalTrials.gov ID: NCT05517655
What this study is testing
What is 129Xe?
129Xe is an investigational medicine, being studied as a potential treatment for cystic fibrosis.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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).
- Phase 4: studies an already-approved treatment
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 6 to 8
You may be able to join 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...
- Sweat chloride equal to or greater than 60 mEq/liter by quantitative pilocarpine iontophoresis test
You likely can't join if
- Individuals currently on ivacaftor therapy (including Kalydeco, Orkambi, and Symdeko) and with at least one gating mutation. Gating mutations include...
- 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).
See the full eligibility criteria
- 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
- 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...
- 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...
The study team makes the final eligibility decision.
Where it's taking place
- Kansas City, Kansas, United States
- Cincinnati, Ohio, United States
- Charlottesville, Virginia, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 6 years to 8 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Kansas City, Kansas, United States; Cincinnati, Ohio, United States; Charlottesville, Virginia, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.