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The PROMISE Pediatric Study 6 to 11 Years Old

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04613128
Recruitment Status : Recruiting
First Posted : November 3, 2020
Last Update Posted : April 14, 2022
Sponsor:
Collaborator:
Cystic Fibrosis Foundation
Information provided by (Responsible Party):
Nicole Hamblett, Seattle Children's Hospital

Brief Summary:
This is a prospective, multi-center observational study. The study is designed to measure the clinical effectiveness of elexacaftor, tezacaftor and ivacaftor (ETI) triple combination therapy in children (6-11 years of old) with one or more copies of the F508del mutation, study the effects of ETI across a number of CF disease manifestations, and collect specimens for future research. Subjects in the study will have one "before ETI" visit within 30 days before initiation of the therapy and five "after ETI" visits over a 24-month follow-up period. The duration of participation for each subject is 25 months. NOTE: Study will not begin unless and until FDA approval for ETI in the 6-11 age group is granted.

Condition or disease
Cystic Fibrosis Cystic Fibrosis in Children

Detailed Description:

While nearly 2000 mutations have been described, the most common disease causing CFTR mutation is F508del, which is found in >85% of patients followed in the US CF Patient Registry. Two CFTR corrector drugs (elexacaftor and tezacaftor) plus the potentiator ivacaftor have been developed and approved as a triple combination therapy for CF patients (12 years old and above) with one or two copies of the F508del mutation. We predict that over 90% of pediatric CF patients (age 6-11 y/o) will be eligible for highly effective CFTR modulator therapy in the U.S.

The PROMISE Pediatric Study is designed to measure the direct and indirect CFTR dependent anion secretion by collecting and analyzing clinical research outcomes and biomarkers on pediatric patients both before and after they begin treatment with ETI. This study will investigate the impact of ETI across a wide range of CF disease manifestations and organ systems. While specific biomarkers of special interest have been selected for detailed analysis in this study, an additional important goal is to collect blood, urine, stool, and airway epithelial cell specimens for long-term storage in a biorepository to enable future research. These samples can be made available for research beyond the current scope of work. The PROMISE Pediatric Study will provide a coordinated collection of clinical research outcomes data that can be linked with these specimens.

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Study Type : Observational
Estimated Enrollment : 180 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function (the PROMISE Pediatric 6 to 11 Years Old Study)
Actual Study Start Date : June 11, 2021
Estimated Primary Completion Date : July 1, 2023
Estimated Study Completion Date : July 1, 2023

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Group/Cohort
Pediatric
Cystic Fibrosis pediatric patients (6-11 years old) prescribed ETI CFTR modulator Therapy.



Primary Outcome Measures :
  1. Sweat Chloride at 6 months [ Time Frame: 6 months ]
    Change in sweat chloride from Baseline to 6 months.

  2. Sweat Chloride at 24 months [ Time Frame: 24 months ]
    Change sweat chloride from Baseline to 24 months.

  3. Forced expiratory volume at one second (FEV1) at 6 months [ Time Frame: 6 months ]
    Change in FEV1 from Baseline to 6 months.

  4. Forced expiratory volume at one second (FEV1) at 24 months [ Time Frame: 24 months ]
    Change in FEV1 from Baseline to 24 months.

  5. Lung Clearance Index (LCI) at 6 months [ Time Frame: 6 months ]
    Change Lung Clearance Index (LCI) from baseline to 6 months.

  6. Lung Clearance Index (LCI) at 24 months [ Time Frame: 24 months ]
    Change Lung Clearance Index (LCI) from baseline to 24 months.


Secondary Outcome Measures :
  1. Weight at 6 Months [ Time Frame: 6 months ]
    Change in weight from Baseline to 6 months.

  2. Weight at 24 Months [ Time Frame: 24 months ]
    Change in weight from Baseline to 24 months.

  3. BMI at 6 Months [ Time Frame: 6 months ]
    Change in BMI from Baseline to 6 months.

  4. BMI at 24 Months [ Time Frame: 24 months ]
    Change in BMI from Baseline to 24 months.

  5. Cystic Fibrosis Questionnaire Revised (CFQ-R) at 6 Months [ Time Frame: 6 months ]
    Change in CFQ-R (respiratory domain) from Baseline to 6 months.

  6. Cystic Fibrosis Questionnaire Revised (CFQ-R) at 24 Months [ Time Frame: 24 months ]
    Change in CFQ-R (respiratory domain) from Baseline to 24 months.


Biospecimen Retention:   Samples With DNA
Serum, plasma, buffy coat, urine, sputum, oropharyngeal swab (throat swab), stool, sweat


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   6 Years to 11 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Cystic Fibrosis, 6-11 years old prescribed ETI.
Criteria

Inclusion Criteria:

  1. Written parental informed consent and assent obtained from subject and the subject's legal guardian.
  2. Be willing and able to adhere to the study visit schedule and other protocol requirements.
  3. All genders 6-11 years old on Day 1.
  4. Diagnosis of CF.
  5. CFTR mutations consistent with the FDA labeled indication for the ETI.
  6. Physician intent to prescribe the ETI.
  7. Able to attempt the testing and procedures required for this study, as judged by the investigator.
  8. Enrolled in the Cystic Fibrosis Foundation Patient Registry.
  9. Clinically stable with no significant changes in health status within the 14 days prior to Visit 1 (and inclusive of Visit 1).

Exclusion Criteria:

  1. Has 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.
  2. Use of any ETI within the 180 days prior to Visit 1.
  3. Any acute use of antibiotics (oral, inhaled or IV) or acute use of systemic corticosteroids within the 14 days prior to Visit 1 (inclusive of Visit 1) for lower respiratory tract symptoms.
  4. Initiation of any new chronic therapy (e.g., ibuprofen, Pulmozyme®, hypertonic saline, azithromycin, inhaled tobramycin, Cayston®, Kalydeco, Orkambi®, Symdeko®) within the 4 weeks prior to Visit 1 (inclusive of Visit 1).
  5. Use of an investigational agent within the 28 days prior to Visit 1.
  6. Use of chronic oral corticosteroids (equivalent to 10 mg. or more per day of prednisone) within the 28 days prior to Visit 1.
  7. Treatment for nontuberculous mycobacterial (NTM) infection, consisting of ≥ two antibiotics (oral, IV, and/or inhaled) within the 28 days prior to Visit 1.
  8. History of lung or liver transplantation,or listing for organ transplantation.

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): NCT04613128


Contacts
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Contact: Laurel Couture 206-884-2649 laurel.couture@seattlechildrens.org
Contact: Anna Mead 206-884-7531 anna.mead@seattlechildrens.org

Locations
Show Show 20 study locations
Sponsors and Collaborators
Nicole Hamblett
Cystic Fibrosis Foundation
Investigators
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Principal Investigator: Steven Rowe, MD University of Alabama at Birmingham
Principal Investigator: David Nichols, MD Seattle Children's Hospital
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Responsible Party: Nicole Hamblett, Professor of Pediatrics, Division of Pulmonary and Sleep Medicine, University of Washington School of Medicine Adjunct Professor, Biostatistics, University of Washington School of Medicine Co-Executive Director, Cystic Fibrosis Therapeutics Development, Seattle Children's Hospital
ClinicalTrials.gov Identifier: NCT04613128    
Other Study ID Numbers: PROMISE-OB-18 Pediatric Study
First Posted: November 3, 2020    Key Record Dates
Last Update Posted: April 14, 2022
Last Verified: April 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Nicole Hamblett, Seattle Children's Hospital:
CF
Cystic Fibrosis
CFTR Modulator
Trikafta
Triple Combination Therapy
Pediatric
ETI
elexacaftor/texacaftor/ivacaftor
Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases