The PROMISE Pediatric Study 6 to 11 Years Old

NCT04613128 | Completed

• 1 other identifier: PROMISE-OB-18 Pediatric Study
• Study Type: observational
• Target: 125
• Locations: 1 country
• Sites: 20

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. Participants who have participated in the original PROMISE Pediatric Sub-Study have the option of participating in a long-term extension with annual visits performed at the 36- and 48-month timepoints. The durability of the clinical and biological changes in the PROMISE Pediatric Sub-Study can be assessed with extended follow-up, which would enable the sub-studies to consider potential clinical consequences of the biological or physiological effects being studied. This work will help to inform long term prognosis and feasibility of certain clinical trials outcomes for interventional studies and may be useful when considering research priorities in drug development. The duration of participation for each subject is 24 months (with an additional 24 months if participants agree to the optional long-term extension). NOTE: FDA has granted approval for elexacaftor, tezacaftor and ivacaftor in the 6-11 age group.

Conditions

Cystic Fibrosis; Cystic Fibrosis in Children

Keywords

CF; Cystic Fibrosis; CFTR Modulator; Trikafta; Triple Combination Therapy; Pediatric; ETI; elexacaftor/texacaftor/ivacaftor

Outcome Measures

Primary Outcomes (6)

• Sweat Chloride at 6 months

  Change in sweat chloride from Baseline to 6 months.

  6 months

• Sweat Chloride at 24 months

  Change sweat chloride from Baseline to 24 months.

  24 months

• Forced expiratory volume at one second (FEV1) at 6 months

  Change in FEV1 from Baseline to 6 months.

  6 months

• Forced expiratory volume at one second (FEV1) at 24 months

  Change in FEV1 from Baseline to 24 months.

  24 months

• Lung Clearance Index (LCI) at 6 months

  Change Lung Clearance Index (LCI) from baseline to 6 months.

  6 months

• Lung Clearance Index (LCI) at 24 months

  Change Lung Clearance Index (LCI) from baseline to 24 months.

  24 months

Secondary Outcomes (6)

• Weight at 6 Months

  6 months

• Weight at 24 Months

  24 months

• BMI at 6 Months

  6 months

• BMI at 24 Months

  24 months

• Cystic Fibrosis Questionnaire Revised (CFQ-R) at 6 Months

  6 months

Study Arms (1)

Pediatric

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

Eligibility Criteria

• Age: 6 Years - 11 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

Cystic Fibrosis, 6-11 years old prescribed ETI.

You may qualify if:

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

You may not qualify if:

• 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.
• Use of any ETI within the 180 days prior to Visit 1.
• 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.
• 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).
• Use of an investigational agent within the 28 days prior to Visit 1.
• Use of chronic oral corticosteroids (equivalent to 10 mg. or more per day of prednisone) within the 28 days prior to Visit 1.
• Treatment for nontuberculous mycobacterial (NTM) infection, consisting of ≥ two antibiotics (oral, IV, and/or inhaled) within the 28 days prior to Visit 1.
• History of lung or liver transplantation,or listing for organ transplantation.

Sponsors & Collaborators

• Nicole Hamblett: lead
• Cystic Fibrosis Foundation: collaborator

Study Sites (20)

University of Alabama at Birmingham

Birmingham, Alabama, 35294, United States

Location

Stanford University Medical Center

Palo Alto, California, 94304, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

Riley Hospital for Children

Indianapolis, Indiana, 46202, United States

Location

University of Iowa

Iowa City, Iowa, 52242, United States

Location

John Hopkins University

Baltimore, Maryland, 21287, United States

Location

Boston Children's Hospital, Brigham & Women's Hospital

Boston, Massachusetts, 02115, United States

Location

The Minnesota Cystic Fibrosis Center

Minneapolis, Minnesota, 55455, United States

Location

Children's Mercy Kansas City

Kansas City, Missouri, 64108, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27599, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44146, United States

Location

Oregon Health Sciences University

Portland, Oregon, 97239, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Virginia Commonwealth University

Richmond, Virginia, 23219, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

University of Wisconsin

Madison, Wisconsin, 53792, United States

Location

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

Biospecimen

Retention: SAMPLES WITH DNA

Serum, plasma, buffy coat, urine, sputum, oropharyngeal swab (throat swab), stool, sweat

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Steven Rowe, MD

  University of Alabama at Birmingham

  PRINCIPAL INVESTIGATOR

• David Nichols, MD

  University of Washington

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: 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

Study Record Dates

• First Submitted: October 27, 2020
• First Posted: November 3, 2020
• Study Start: June 11, 2021
• Primary Completion: December 8, 2025
• Study Completion: December 8, 2025
• Last Updated: April 27, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (20)