To characterize the natural history of exocrine pancreatic function as measured by FE-1 in infants with CF
ID
Source
Brief title
Condition
- Respiratory disorders congenital
- Exocrine pancreas conditions
- Congenital respiratory tract disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Proportion of participants with FE-1 >=200 µg/g over time
Secondary outcome
FE-1 level over time
Background summary
This study is being done to learn more about the pancreas (an organ that helps
digest food) and the intestines in infants with CF who are not treated with a
Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulator.
Caregivers will collect weekly stool samples for testing of a pancreatic enzyme
called fecal elastase and a marker of inflammation of the intestines called
calprotectin.
Study objective
To characterize the natural history of exocrine pancreatic function as measured
by
FE-1 in infants with CF <12 months of age
Study design
This is a hybrid decentralized study to characterize the natural history of
exocrine
pancreatic function by measuring FE-1 in infants with CF. Fecal calprotectin (a
biomarker of gastrointestinal inflammation) will also be assessed. In the
optional
The day of enrollment into this study will be referred to as the *index date*.
Data will be collected retrospectively from medical records during the
Pre-enrollment
Period, which is defined from date of birth to index date. Data will be
collected
prospectively during the Measurement Period, which is defined from index date
to
approximately when the participant turns 12 months of age. FE-1 and fecal
calprotectin levels will be assessed in fecal samples collected during the
Measurement Period.
Intervention
NA
Study burden and risks
Stool sample: there are no known risks or discomforts from the stool sample
collection
Confidentiality: There is a possibility that your name or other personal
information could be seen by an unauthorized person.
Van Swietenlaan 6
Groningen 9728 NZ
NL
Van Swietenlaan 6
Groningen 9728 NZ
NL
Listed location countries
Age
Inclusion criteria
1. Participant*s legally appointed and authorized representative (e.g., parent
or legal guardian)
will sign and date an informed consent form (ICF).
2. Male or female participants with CF <6 months of age at the index date.
3. Participant is not eligible to receive commercial Kalydeco* (based on local
product labels)
and is not receiving Kalydeco or any other CFTR modulator.
4. As judged by the investigator, the parent or legal guardian must be able to
understand
protocol requirements, restrictions, and instructions, and the parent or legal
guardian should
be able to ensure that the participant will comply with and is likely to
complete the study as
planned.
Exclusion criteria
1. History of any illness or any clinical condition that, in the opinion of the
investigator, might
either confound the results of the study or impact participant*s ability to
participate.
2. Ongoing or any prior participation in an investigational drug study.
Note: Ongoing participation in a noninterventional study (including
observational
studies) is permitted.
3. Participant whose mother took any CFTR modulator while pregnant with the
participant, or
who has any history of exposure to a CFTR modulator (e.g., through consumption
of breast
milk from a mother on a CFTR modulator or other means).
4. A close relative of the participant is the investigator or a
subinvestigator, research assistant,
study coordinator, or other staff directly involved with the conduct of the
study.
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| CCMO | NL86940.056.24 |
| Other | To be determined |