Clinical trial NCT06296394: Health Outcomes of Parents With Cystic Fibrosis-Aim 2
Official title: Health Outcomes of Parents With Cystic Fibrosis-Aim 2
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Cystic Fibrosis
Parenthood Status
Keywords: cystic fibrosis
parenthood
highly effective modulator therapy
Lead sponsor: University of Pittsburgh
Sponsor class: OTHER
Collaborators: name: Cystic Fibrosis Foundation
name: National Heart, Lung, and Blood Institute (NHLBI)
First posted: 2024-03-06
Enrollment: 146
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Individuals with cystic fibrosis who have become first-time parents to a child under 5 years of age.

Brief summary: The goal of this observational prospective study is to determine the health impact of parenthood on United States (US) people with CF in the era of CF transmembrane regulator protein (CFTR) modulators. The investigators will collect physical and mental health data to comprehensively evaluate the impact of parenthood in CF with widespread highly effective CFTR modulator use. The main hypotheses this study aims to examine are:

H1: Parents with CF and moderate-to-severe depression have more rapid change in ppFEV1 (percent predicted forced expiratory volume in one second) versus those with mild or no depression.

H2: Parents with CF who have more parental responsibility and/or stress have more rapid ppFEV1 (percent predicted forced expiratory volume in one second) change than those with less responsibility/stress

H3: Parents using CFTR modulators have decreased ppFEV1 (percent predicted forced expiratory volume in one second) change versus those not using CFTR modulators

Participants will complete quarterly surveys during the first year of parenthood and biannual surveys, thereafter, using the computer-based survey system on an iPad protected for infection control or via personal device or computer via emailed survey link.

Detailed description: The investigators will follow 146 new parents of children \<5 years of age at 18 participating US adult CF centers to assess the primary outcome of percent predicted forced expiratory volume in one second (ppFEV1) up to 5 years after becoming a parent. A prospective approach will capture the immediate and long-term impact of the use of the highly effective CFTR modulator ETI (elexacaftor/tezacaftor/ivacaftor) by \~90 percent of US adults with CF. By combining objective health measures and participant surveys, the investigators can comprehensively assess the psychosocial impacts of parenthood and explore the interplay between the parenting role and physical and mental health. The investigators anticipate identifying modifiable factors that may ameliorate negative health impacts of parenthood. The investigators will conduct hypothesis-generating, semi-structured dyadic interviews with a subset of parents and their key supports (partner/family/friend) to inform future interventions. The investigators have selected qualitative methodology to avoid preconceived theories/hypotheses.

Eligibility criteria: Inclusion Criteria:

* Diagnosed with cystic fibrosis via sweat test or genotype analysis
* Became a first-time parent (including foster parent, step parent, adoptive parent, or legal guardian) to a child under 5 years of age within the last 90 days

Exclusion Criteria:

* Undergone a lung transplant
* Does not speak/read English or Spanish

Primary outcomes:
measure: Forced Expiratory Volume (FEV1)
timeFrame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
Secondary outcomes:
measure: Pulmonary Function Tests (PFTs) - Forced Vital Capacity (FVC)
timeFrame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
measure: Pulmonary Function Tests (PFTs) - Forced Expiratory Volume (FEV1)
timeFrame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
measure: Pulmonary Function Tests (PFTs) - Forced Expiratory Flow at 25 and 75 percent (FEF25-75)
timeFrame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
measure: History of CF Diagnosis
timeFrame: Year 1
measure: History of CF Genotype Information
timeFrame: Year 1
measure: History of CF Mutation
timeFrame: Year 1
measure: Number of Hospitalizations
timeFrame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
measure: Length of Hospitalizations
timeFrame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
measure: Number of Pulmonary Exacerbations
timeFrame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
measure: Severity of Pulmonary Exacerbations
timeFrame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
measure: Treatment of Pulmonary Exacerbations
timeFrame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
measure: Rate of Hospitalizations Due to Pulmonary Exacerbations
timeFrame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
measure: Clinic Visit Attendance
timeFrame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
measure: Microbiologic Profile
timeFrame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
measure: Medication use
timeFrame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
measure: History of CF-Related Diabetes Status
timeFrame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
measure: CF-Related Diabetes Control - Hemoglobin A1C
timeFrame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
measure: CF-Related Diabetes Control - Glucose Tolerance
timeFrame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
measure: CF-Related Diabetes Control - Insulin Prescription Usage
timeFrame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
measure: Liver Disease Status
timeFrame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
measure: Transplantation Status
timeFrame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
measure: Transplantation Type
timeFrame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
measure: BMI
timeFrame: Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
measure: Sleep Quality
timeFrame: Up to 4 times in Year 1, Up to 2 times in Year 2 through Year 5.
measure: Parental Stress
timeFrame: Up to 4 times in Year 1, Up to 2 times in Year 2 through Year 5.
measure: Life Stressors
timeFrame: Up to 2 times per year in Year 1 through Year 5.
measure: Cystic Fibrosis Questionnaire to evaluate quality of life domains
timeFrame: Up to 2 times per year in Year 1 through Year 5.
measure: Parental Responsibility
timeFrame: Up to 4 times in Year 1, Up to 2 times in Year 2 through Year 5.
measure: Daily Care Check-In
timeFrame: Up to 2 times per year in Year 1 through Year 5.
measure: Social Support
timeFrame: Up to 4 times in Year 1, Up to 2 times in Year 2 through Year 5.
measure: Anxiety
timeFrame: Up to 4 times in Year 1, Up to 2 times in Year 2 through Year 5.
measure: Depression
timeFrame: Up to 4 times in Year 1, Up to 2 times in Year 2 through Year 5.
measure: Self-Reported Adherence
timeFrame: Up to 2 times per year in Year 1 through Year 5.
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama-Birmingham
state: Alabama
city: Denver
country: United States
facility: National Jewish Health
state: Colorado
city: Evanston
country: United States
facility: Northwestern University
state: Illinois
city: Indianapolis
country: United States
facility: Indiana University
state: Indiana
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Baltimore
country: United States
facility: Johns Hopkins University
state: Maryland
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Boston
country: United States
facility: Boston Children's Hospital/Brigham and Women's Hospital
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: Chapel Hill
country: United States
facility: University of North Carolina
state: North Carolina
city: Portland
country: United States
facility: Oregon Health and Science University
state: Oregon
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Dallas
country: United States
facility: University of Texas-Southwestern
state: Texas
city: Seattle
country: United States
facility: University of Washington
state: Washington
Study officials:
affiliation: Faculty
name: Traci M Kazmerski, MD
role: PRINCIPAL_INVESTIGATOR
References: