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BEGIN Novel ImagiNG Biomarkers (BEGINNING)

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ClinicalTrials.gov Identifier: NCT05517655
Recruitment Status : Recruiting
First Posted : August 26, 2022
Last Update Posted : August 26, 2022
Sponsor:
Collaborators:
University of Kansas
University of Iowa
University of Virginia
Information provided by (Responsible Party):
Jason Woods, Children's Hospital Medical Center, Cincinnati

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).

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: 129Xe Phase 4

Detailed 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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 44 participants
Allocation: Non-Randomized
Intervention Model: Crossover Assignment
Masking: Single (Outcomes Assessor)
Primary Purpose: Diagnostic
Official Title: BEGIN Novel ImagiNG Biomarkers
Actual Study Start Date : May 1, 2022
Estimated Primary Completion Date : November 1, 2023
Estimated Study Completion Date : November 1, 2024

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Pre Trikafta
129Xe MRI
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.

Experimental: Post Trikafta
129Xe MRI
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 :
  1. 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.

  2. 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.


Secondary Outcome Measures :
  1. Abdominal T1 values [ Time Frame: 1 year ]
    Changes in MRI T1 average in the liver and pancreas, from baseline to follow up at 1 year

  2. Lung reader score [ Time Frame: 1 year ]
    Changes in reader score for visible structural defects from proton MRI, from baseline to follow up at 1 year



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Ages Eligible for Study:   6 Years to 8 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05517655


Contacts
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Contact: Priyanka Desirazu 513-803-4781 Priyanka.Desirazu@cchmc.org

Locations
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United States, Kansas
University of Kansas Medical Center Active, not recruiting
Kansas City, Kansas, United States, 66160
United States, Ohio
Cincinnati Children's Hospital Medical Center Active, not recruiting
Cincinnati, Ohio, United States, 45229
United States, Virginia
University of Virginia Recruiting
Charlottesville, Virginia, United States, 22903
Contact: Jamie Mata         
Principal Investigator: Jamie Mata         
Principal Investigator: Deborah Froh         
Sponsors and Collaborators
Children's Hospital Medical Center, Cincinnati
University of Kansas
University of Iowa
University of Virginia
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Responsible Party: Jason Woods, Principal Investigator, Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier: NCT05517655    
Other Study ID Numbers: 2021-0325
First Posted: August 26, 2022    Key Record Dates
Last Update Posted: August 26, 2022
Last Verified: August 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Cystic Fibrosis
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases