Studies
| Study First Submitted Date | 2022-04-06 |
| Study First Posted Date | 2022-08-26 |
| Last Update Posted Date | 2022-08-26 |
| Start Month Year | May 1, 2022 |
| Primary Completion Month Year | November 1, 2023 |
| Verification Month Year | August 2022 |
| Verification Date | 2022-08-31 |
| Last Update Posted Date | 2022-08-26 |
Detailed Descriptions
| Sequence: | 20814942 |
| Description | The overall hypothesis is that multi-organ MRI will provide more sensitive, robust outcome measures in young CF patients than traditional measures employed in the BEGIN study and that these novel measures will be more sensitive to treatment effects, tested here by comparison before and after triple-combination modulator therapy. By understanding the nature of early lung obstruction and characteristic changes in the liver and pancreas over time, we continue to lay the groundwork for more personalized medicine in the future. Assessing treatment response and clinical benefit in children with CF who are clinically normal per standard outcomes (e.g., spirometry, pancreatic function) will become paramount as triplecombination therapy is extended to younger patients with milder CF clinical presentation than their historic peers. Here the sensitivity and profile free of ionizing-radiation exposure of MRI can be leveraged to follow an individual with CF over time to quantify changes with therapy-with additional spatial resolution unavailable from standard clinical testing. |
Facilities
| Sequence: | 200885681 | Sequence: | 200885682 | Sequence: | 200885683 |
| Status | Active, not recruiting | Status | Active, not recruiting | Status | Recruiting |
| Name | University of Kansas Medical Center | Name | Cincinnati Children's Hospital Medical Center | Name | University of Virginia |
| City | Kansas City | City | Cincinnati | City | Charlottesville |
| State | Kansas | State | Ohio | State | Virginia |
| Zip | 66160 | Zip | 45229 | Zip | 22903 |
| Country | United States | Country | United States | Country | United States |
Facility Contacts
| Sequence: | 28241715 |
| Facility Id | 200885683 |
| Contact Type | primary |
| Name | Jamie Mata |
Facility Investigators
| Sequence: | 18416613 | Sequence: | 18416614 |
| Facility Id | 200885683 | Facility Id | 200885683 |
| Role | Principal Investigator | Role | Principal Investigator |
| Name | Jamie Mata | Name | Deborah Froh |
Conditions
| Sequence: | 52409218 |
| Name | Cystic Fibrosis |
| Downcase Name | cystic fibrosis |
Id Information
| Sequence: | 40326866 |
| Id Source | org_study_id |
| Id Value | 2021-0325 |
Countries
| Sequence: | 42749991 |
| Name | United States |
| Removed | False |
Design Groups
| Sequence: | 55856654 | Sequence: | 55856655 |
| Group Type | Experimental | Group Type | Experimental |
| Title | Pre Trikafta | Title | Post Trikafta |
| Description | 129Xe MRI | Description | 129Xe MRI |
Interventions
| Sequence: | 52717263 |
| Intervention Type | Drug |
| Name | 129Xe |
| Description | 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. |
Design Outcomes
| Sequence: | 178258927 | Sequence: | 178258928 | Sequence: | 178258929 | Sequence: | 178258930 |
| Outcome Type | primary | Outcome Type | primary | Outcome Type | secondary | Outcome Type | secondary |
| Measure | Ventilation Defect Percentage change from baseline | Measure | Pancreas volume | Measure | Abdominal T1 values | Measure | Lung reader score |
| Time Frame | 1 year | Time Frame | 1 year | Time Frame | 1 year | Time Frame | 1 year |
| Description | 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. | Description | 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. | Description | Changes in MRI T1 average in the liver and pancreas, from baseline to follow up at 1 year | Description | Changes in reader score for visible structural defects from proton MRI, from baseline to follow up at 1 year |
Browse Conditions
| Sequence: | 194390286 | Sequence: | 194390287 | Sequence: | 194390288 | Sequence: | 194390289 | Sequence: | 194390290 | Sequence: | 194390291 | Sequence: | 194390292 |
| Mesh Term | Cystic Fibrosis | Mesh Term | Pancreatic Diseases | Mesh Term | Digestive System Diseases | Mesh Term | Lung Diseases | Mesh Term | Respiratory Tract Diseases | Mesh Term | Genetic Diseases, Inborn | Mesh Term | Infant, Newborn, Diseases |
| Downcase Mesh Term | cystic fibrosis | Downcase Mesh Term | pancreatic diseases | Downcase Mesh Term | digestive system diseases | Downcase Mesh Term | lung diseases | Downcase Mesh Term | respiratory tract diseases | Downcase Mesh Term | genetic diseases, inborn | Downcase Mesh Term | infant, newborn, diseases |
| Mesh Type | mesh-list | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor |
Sponsors
| Sequence: | 48540656 | Sequence: | 48540657 | Sequence: | 48540658 | Sequence: | 48540659 |
| Agency Class | OTHER | Agency Class | OTHER | Agency Class | OTHER | Agency Class | OTHER |
| Lead Or Collaborator | lead | Lead Or Collaborator | collaborator | Lead Or Collaborator | collaborator | Lead Or Collaborator | collaborator |
| Name | Children's Hospital Medical Center, Cincinnati | Name | University of Kansas | Name | University of Iowa | Name | University of Virginia |
Central Contacts
| Sequence: | 12071627 |
| Contact Type | primary |
| Name | Priyanka Desirazu |
| Phone | 513-803-4781 |
| [email protected] | |
| Role | Contact |
Design Group Interventions
| Sequence: | 68470816 | Sequence: | 68470817 |
| Design Group Id | 55856655 | Design Group Id | 55856654 |
| Intervention Id | 52717263 | Intervention Id | 52717263 |
Eligibilities
| Sequence: | 30902331 |
| Gender | All |
| Minimum Age | 6 Years |
| Maximum Age | 8 Years |
| Healthy Volunteers | No |
| Criteria | Inclusion 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 Exclusion Criteria: 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. |
| Adult | False |
| Child | True |
| Older Adult | False |
Calculated Values
| Sequence: | 254156627 |
| Number Of Facilities | 3 |
| Registered In Calendar Year | 2022 |
| Were Results Reported | False |
| Has Us Facility | True |
| Has Single Facility | False |
| Minimum Age Num | 6 |
| Maximum Age Num | 8 |
| Minimum Age Unit | Years |
| Maximum Age Unit | Years |
| Number Of Primary Outcomes To Measure | 2 |
| Number Of Secondary Outcomes To Measure | 2 |
Designs
| Sequence: | 30648050 |
| Allocation | Non-Randomized |
| Intervention Model | Crossover Assignment |
| Observational Model | |
| Primary Purpose | Diagnostic |
| Time Perspective | |
| Masking | Single |
| Outcomes Assessor Masked | True |
Responsible Parties
| Sequence: | 29014690 |
| Responsible Party Type | Principal Investigator |
| Name | Jason Woods |
| Title | Principal Investigator |
| Affiliation | Children's Hospital Medical Center, Cincinnati |