| NCT ID | NCT05517655 |
| Status | Recruiting |
| Phase | Phase 4 |
| Sponsor | Children's Hospital Medical Center, Cincinnati |
| Condition | Cystic Fibrosis |
| Study Type | INTERVENTIONAL |
| Enrollment | 44 participants |
| Start Date | 2022-05-01 |
| Primary Completion | 2028-11-01 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 4 studies follow an already-approved treatment in real-world conditions to monitor long-term safety and effectiveness.
This trial targets 44 participants in total. It began in 2022-05-01 with a primary completion date of 2028-11-01.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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).
Eligibility Criteria
Inclusion Criteria: 1. 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 Exclusion Criteria: 1. 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. 2. Acute respiratory symptoms (e.g. wheezing) at the time of the MRI. 3. Acute respiratory infection, defined as increased cough, wheezing or respiratory rate in the 28 days prior to enrollment. 4. Chronic lung disease not related to CF 5. Chronic liver disease not related to CF 6. Acute pancreatitis, defined by clinical criteria (45). 7. Chronic pancreatic disease not related to CF. 8. 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. 9. 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 & Investigator
Jason Woods, PhD
PRINCIPAL INVESTIGATOR
Children's Hospital Medical Center, Cincinnati
Frequently Asked Questions
Who can join the NCT05517655 clinical trial?
This trial is open to participants of all sexes, aged 6 Years or older, up to 8 Years, studying Cystic Fibrosis. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT05517655 trial and what does that mean for participants?
Phase 4 studies are conducted after a treatment has been approved. They monitor long-term safety and real-world effectiveness in a broader patient population.
Is NCT05517655 currently recruiting?
Yes, NCT05517655 is actively recruiting participants. Contact the research team at carrie.stevens@cchmc.org for enrollment information.
Where is the NCT05517655 trial being conducted?
This trial is being conducted at Kansas City, United States, Cincinnati, United States, Charlottesville, United States.
Who is sponsoring the NCT05517655 clinical trial?
NCT05517655 is sponsored by Children's Hospital Medical Center, Cincinnati. The principal investigator is Jason Woods, PhD at Children's Hospital Medical Center, Cincinnati. The trial plans to enroll 44 participants.