Health Outcomes of Parents With Cystic Fibrosis-Aim 2

Cystic Fibrosis Clinical Trial

— HOPeCF  

Official title:

Health Outcomes of Parents With Cystic Fibrosis-Aim 2

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06296394
• Other study ID #: STUDY23080161
• Secondary ID: 1R01HL161164-01A
• Status: Recruiting
• Start date: June 1, 2024
• Est. completion date: February 1, 2031

Study information

• Verified date: May 2024
• Source: University of Pittsburgh
• Contact: Olivia M Stransky, MPH
• Phone: 412-648-4701
• Email: stranskyom[@]upmc.edu
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 146
• Est. completion date: February 1, 2031
• Est. primary completion date: February 1, 2030
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis
• Parenthood Status

Intervention

Other:
• Parenthood
The intervention group will consist of participants who became first time parents to children under 5 years of age.

Locations

Country	Name	City	State
United States	University of Michigan	Ann Arbor	Michigan
United States	Johns Hopkins University	Baltimore	Maryland
United States	University of Alabama-Birmingham	Birmingham	Alabama
United States	Boston Children's Hospital/Brigham and Women's Hospital	Boston	Massachusetts
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	University of North Carolina	Chapel Hill	North Carolina
United States	Medical University of South Carolina	Charleston	South Carolina
United States	University of Texas-Southwestern	Dallas	Texas
United States	National Jewish Health	Denver	Colorado
United States	Northwestern University	Evanston	Illinois
United States	Indiana University	Indianapolis	Indiana
United States	University of Kansas Medical Center	Kansas City	Kansas
United States	University of Minnesota	Minneapolis	Minnesota
United States	University of Pennsylvania	Philadelphia	Pennsylvania
United States	University of Pittsburgh	Pittsburgh	Pennsylvania
United States	Oregon Health and Science University	Portland	Oregon
United States	Washington University School of Medicine	Saint Louis	Missouri
United States	University of Washington	Seattle	Washington

Sponsors (3)

Lead Sponsor	Collaborator
University of Pittsburgh	Cystic Fibrosis Foundation, National Heart, Lung, and Blood Institute (NHLBI)

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Forced Expiratory Volume (FEV1)	Rate of change in ppFEV1 as reflected in the medical record from Year 1 to Year 5	Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
Secondary	Pulmonary Function Tests (PFTs) - Forced Vital Capacity (FVC)	Occurrence of PFTs at each clinic visit/hospitalization. Measures include FVC (Forced vital capacity), and the percent predicted.	Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
Secondary	Pulmonary Function Tests (PFTs) - Forced Expiratory Volume (FEV1)	Occurrence of PFTs at each clinic visit/hospitalization. Measures include FEV1(Forced expiratory volume in one second), and the percent predicted.	Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
Secondary	Pulmonary Function Tests (PFTs) - Forced Expiratory Flow at 25 and 75 percent (FEF25-75)	Occurrence of PFTs at each clinic visit/hospitalization. Measures include FEF25-75 (forced expiratory flow at 25 percent and 75 percent of the pulmonary volume), and the percent predicted for each.	Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
Secondary	History of CF Diagnosis	Date of diagnosis, as reported in the medical record.	Year 1
Secondary	History of CF Genotype Information	Genotype information, as reported in the medical record.	Year 1
Secondary	History of CF Mutation	Mutation selection, as reported in the medical record.	Year 1
Secondary	Number of Hospitalizations	The number of hospitalizations, as reported in the medical record, will be used to compute the rate of hospitalizations.	Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
Secondary	Length of Hospitalizations	The length of each hospitalization, as reported in the medical record, will be used to compute the rate of hospitalizations.	Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
Secondary	Number of Pulmonary Exacerbations	The number of pulmonary exacerbations as reported in the medical record will be used to compute the rate of pulmonary exacerbations.; , hospitalizations (number and length)	Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
Secondary	Severity of Pulmonary Exacerbations	The severity of pulmonary exacerbations (mild, moderate, severe), as reported in the medical record.	Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
Secondary	Treatment of Pulmonary Exacerbations	The type of treatment used for pulmonary exacerbations (IV antibiotic, oral antibiotic), as reported in the medical record.	Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
Secondary	Rate of Hospitalizations Due to Pulmonary Exacerbations	The number and length of hospitalizations, combined, due to pulmonary exacerbations will be used to compute the rate of pulmonary exacerbations.	Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
Secondary	Clinic Visit Attendance	Number of outpatient clinic visits attended, as reported in the Cystic Fibrosis Foundation Patient Registry (CFFPR).	Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
Secondary	Microbiologic Profile	Number of positive screenings of specific types of bacteria, mycobacteria, fungus in cultures as reported in the CFFPR	Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
Secondary	Medication use	Number of positive screenings of medication use, as reported in the medical record, including CFTR modulator therapy, chronic antibiotic use, and home IV treatment.	Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
Secondary	History of CF-Related Diabetes Status	Instances of positive screenings for CF-Related Diabetes as reported in the medical record.	Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
Secondary	CF-Related Diabetes Control - Hemoglobin A1C	Highest Hemoglobin A1C values, as reported in the medical record.	Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
Secondary	CF-Related Diabetes Control - Glucose Tolerance	Oral glucose tolerance test results, as reported in the medical record.	Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
Secondary	CF-Related Diabetes Control - Insulin Prescription Usage	Insulin prescription use and dosage as reported in the medical record	Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
Secondary	Liver Disease Status	Liver disease status (gall stones, liver disease with cirrhosis, liver disease non-cirrhosis, acute liver failure, hepatic steatosis) as reported by the medical record.	Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
Secondary	Transplantation Status	Current transplant status of the patient (not pertinent, accepted on waiting list, evaluated with final decision pending, evaluated and rejected, or had transplantation), as reported in the medical record.	Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
Secondary	Transplantation Type	If participant had transplantation in previous years, the transplant type will also be recorded as shown in the medical record	Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
Secondary	BMI	Height and weight combined, as reported in the medical record, will be collected to calculate BMI and BMI percentile	Annually from 2 Years prior to enrollment and annually for Year 1 through Year 5
Secondary	Sleep Quality	The Pittsburgh Sleep Quality Index (PSQI) is a self-rated questionnaire which assesses sleep quality and disturbances over a 1-month period. Nineteen individual items generate seven "component" scores: subjective sleep quality, sleep latency, sleep duration, sleep efficiency, sleep disturbances, use of sleeping medication, and daytime dysfunction. The seven component scores are derived, each scored 0 (no difficulty) to 3 (severe difficulty). The component scores are summed to produce a global score (range 0 to 21). Higher scores indicate worse sleep quality.	Up to 4 times in Year 1, Up to 2 times in Year 2 through Year 5.
Secondary	Parental Stress	The Parental Stress Scale is a self-reported questionnaire which assesses feelings and perceptions about the experience of being a parent. Eighteen-item measure with 5-point Likert scale questions from 1 (strongly disagree) to 5 (strongly agree). Score is determined by the sum (possible range of 18 to 90) with higher scores indicating a higher measured level of parental stress.	Up to 4 times in Year 1, Up to 2 times in Year 2 through Year 5.
Secondary	Life Stressors	Holmes-Rahe Life Stress Inventory (HRLSI) is a forty-three-item measure which assesses life stressors by accounting for life events over a 1-year period. Each life event corresponds to a point value (range 11-100). Scores are determined by the sum of the values for all checked life events. Scores of 150 or less refer to a relatively low amount of life change and low susceptibility to stress-induced health breakdowns. Scores of 151 to 300 imply about a 50% chance of a major health breakdown in the next two years. Scores greater than 301 raise the odds of a major health breakdown to about 80%.	Up to 2 times per year in Year 1 through Year 5.
Secondary	Cystic Fibrosis Questionnaire to evaluate quality of life domains	The Cystic Fibrosis Questionnaire-Revised (CFQ-R) is a disease-specific instrument designed to measure the impact on overall health, daily life, well-being and symptoms. It consists of 50 individual measures assessing 9 quality of life domains: physical, role/school, vitality, emotion, social, body image, eating, treatment burden, and health perceptions. Each question is scored with the following scales: for questions 1 - 6: Very True = 1, Mostly True = 2, Somewhat True = 3, Not at all True = 4. For questions 7 - 17: Always = 1, Often = 2, Sometimes = 3, Never = 4. For questions 18 - 30: Very True = 1, Mostly True = 2, Somewhat True = 3, Not at all True = 4. For questions 31 - 35: Always = 1, Often = 2, Sometimes = 3, Never = 4. Scaled scores are calculated using the following formula: (Sum of responses - Minimal Possible sum (n x 1)) / (Maximum possible sum (n x 4) - Minimum possible sum (n x 1)) x 100.	Up to 2 times per year in Year 1 through Year 5.
Secondary	Parental Responsibility	Adapted Parental Responsibility Scale (APRS) is a forty-item measure which assesses responsibility forms of parental involvement. Each form of responsibility is to be rated on a 5-point Likert scale (I always do, I usually do, co-parent and I equally do, co-parent usually does, and co-parent always does) to designate who had primary responsibility for each task.	Up to 4 times in Year 1, Up to 2 times in Year 2 through Year 5.
Secondary	Daily Care Check-In	The Daily Care Check-In Tool is an 18-item measure that assesses the occurrence and frequency of facing barriers to completing treatments over a 6-month period. The Daily Care Check-In Occurrence Scale score can range from 0 (no barriers) to 18 (experiences all barriers). Each barrier is further scored based on a 5-point Likert scale (always, often, sometimes, rarely, never) to assess the frequency of each interference to treatment. Scores for the Daily Care Check-In Interference Scale are calculated by summing the point values assigned to each score. Scores can range from 0 (no barriers) to 90 (experiences all barriers and they always interfere with completing treatments).	Up to 2 times per year in Year 1 through Year 5.
Secondary	Social Support	Interpersonal Support Evaluation List-Short Form (ISEL-SF) is a 12-item measure that assesses the perceived availability of social support from family, friends, and others. Each statement is scored using a 4-point scale (definitely false, probably false, probably true, definitely true) to measure subscales of appraisal, belonging, and tangible support. All items are summed to a total score ranging from 0 to 36 with higher scores indicating greater perceived social support.	Up to 4 times in Year 1, Up to 2 times in Year 2 through Year 5.
Secondary	Anxiety	The Generalized Anxiety Disorder Assessment (GAD-7) is a seven-item measure that is used to assess the severity of generalized anxiety disorder over a 2-week period. The frequency of symptoms is evaluated using a 4-point scale (not at all, several days, more than half the days, nearly every day). The whole scale score can range from 0 to 21. Scores of 0 to 4 indicate minimal anxiety, scores 5-9 indicate mild anxiety, scores 10-15 indicate moderate anxiety and scores greater than 15 indicate severe anxiety.	Up to 4 times in Year 1, Up to 2 times in Year 2 through Year 5.
Secondary	Depression	The Patient Health Questionnaire depression scale (PHQ-8) is an 8-item screening tool used to assess depressive symptoms and severity over a 2-week period. The frequency of symptoms is evaluated using a 4-point scale (not at all, several days, more than half the days, nearly every day). Total score (range of 0 to 24) is determined by adding together the scores of each of the items. Scores of 5, 10, 15, and 20 represent the cutoff points for mild, moderate, moderately severe and severe depression, respectively.	Up to 4 times in Year 1, Up to 2 times in Year 2 through Year 5.
Secondary	Self-Reported Adherence	The Self-Reported Adherence to Highly Effective Modulator Therapy scale is a 4-item questionnaire aimed at identifying the class of modulator (ivacaftor, elexacaftor-tezacaftor-ivacaftor, tezacaftor-ivacaftor, lumacaftor-ivacaftor), presence of altered dosing schedules (specific time or days), and missed treatments (yes, no). Adherence is further evaluated by assessing the number of days that treatment occurred as prescribed (0-3 days, 4-6 days, 7-10 days, 11-14 days) over a 2-week period. This measure serves as descriptive data only, and therefore requires no scoring calculations.	Up to 2 times per year in Year 1 through Year 5.