RECRUITING

BEGIN Novel ImagiNG Biomarkers

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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).

Official Title

BEGIN Novel ImagiNG Biomarkers

Quick Facts

Study Start:2022-05-01

Study Completion:2028-11-01

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT05517655

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:6 Years to 8 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD

Inclusion Criteria	Exclusion 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	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.

Inclusion Criteria

Exclusion 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

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.

Contacts and Locations

Study Contact

Carrie Stevens, BS

CONTACT

(513) 636-9973

carrie.stevens@cchmc.org

Penny New, BS

CONTACT

(513) 636-9973

Penny.New@cchmc.org

Principal Investigator

Jason Woods, PhD

PRINCIPAL_INVESTIGATOR

Children's Hospital Medical Center, Cincinnati

Study Locations (Sites)

University of Kansas Medical Center

Kansas City, Kansas, 66160

United States

Carrie Stevens

Cincinnati, Ohio, 45229

United States

University of Virginia

Charlottesville, Virginia, 22903

United States

Collaborators and Investigators

Sponsor: Children's Hospital Medical Center, Cincinnati

Jason Woods, PhD, PRINCIPAL_INVESTIGATOR, Children's Hospital Medical Center, Cincinnati

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2022-05-01

Study Completion Date2028-11-01

Study Record Updates

Study Start Date2022-05-01

Study Completion Date2028-11-01

Terms related to this study

Additional Relevant MeSH Terms

Cystic Fibrosis