The PROMISE Pediatric Study 6 to 11 Years Old (NCT04613128) | Clinical Trial Compass
CompletedNot Applicable
The PROMISE Pediatric Study 6 to 11 Years Old
United States125 participantsStarted 2021-06-11
Plain-language 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.
Who can participate
Age range6 Years – 11 Years
SexALL
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Inclusion criteria
✓. 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.
Exclusion criteria
✕. 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.
What they're measuring
1
Sweat Chloride at 6 months
Timeframe: 6 months
2
Sweat Chloride at 24 months
Timeframe: 24 months
3
Forced expiratory volume at one second (FEV1) at 6 months
Timeframe: 6 months
4
Forced expiratory volume at one second (FEV1) at 24 months
✕. 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.