Pulmonary Arterial Hypertension (PAH)SAVOIRNCT07120789
Validation of a Patient Knowledge Questionnaire for Pulmonary Hypertension
A study testing Application of Patient Knowledge Questionnaire on Pulmonary Hypertension in people with Pulmonary Arterial Hypertension (PAH) and Pulmonary Arterial Hypertension (PAH) (WHO Group 1 PH), run by University of Sao Paulo General Hospital at 1 site in Brazil.
Trial summary
This methodological study aims to develop and validate a questionnaire named for the Assessment of Patient Knowledge in Pulmonary Arterial Hypertension. The study will be conducted in four phases: (1) questionnaire development based on guidelines and literature; (2) content validation by expert judges; (3) semantic… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Pulmonary Arterial Hypertension (PAH), Pulmonary Arterial Hypertension (PAH) (WHO Group 1 PH), Pulmonary Hypertension
- What is being tested?
- Application of Patient Knowledge Questionnaire on Pulmonary Hypertension
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 215 participants
- Where
- 1 siteBrazil
- Study dates
- 1 Sep 2025to Dec 2026
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adults aged 18 years or older
- Confirmed diagnosis of Group 1 Pulmonary Arterial Hypertension (PAH)
- Ability to read and understand the questionnaire language
- Provided informed consent to participate in the study
- For expert judges (content validation phase): professionals with recognized expertise in pulmonary hypertension, with academic or clinical experience in the field
Who cannot join
- Cognitive, visual, or auditory impairments that hinder questionnaire comprehension
- Refusal to participate or withdrawal of informed consent
- Inability to complete the questionnaire due to clinical instability
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Brazil1 site, 1 recruiting
- Recruiting
Instituto do Coração (InCor), Hospital das Clínicas HCFMUSP, Faculdade de Medicina, Universidade de São Paulo
São Paulo, São Paulo, 05403-900
Caio Fernandes, PhD, +55 11 2661 5034, caio.cesar@hc.fm.usp.br
About this study
Official title: Content, Semantic, and Psychometric Validation of a Patient Knowledge Questionnaire for Pulmonary Hypertension
This methodological study aims to develop and validate a questionnaire named for the Assessment of Patient Knowledge in Pulmonary Arterial Hypertension. The study will be conducted in four phases: (1) questionnaire development based on guidelines and literature; (2) content validation by expert judges; (3) semantic validation with patients; and (4) psychometric testing in a sample of up to 200 patients with confirmed pulmonary arterial hypertension (PAH).
In addition to validation, the study will collect clinical and functional data from medical records, including risk stratification using the COMPERA 2.0 method The final instrument is expected to support patient education strategies and contribute to improved clinical management of PAH.
Read the detailed description
This is a methodological study with a quantitative approach, developed in four main phases, aimed at constructing and validating a questionnaire to assess the knowledge of patients with pulmonary arterial hypertension (PAH). The study will be conducted at a reference outpatient center in Brazil and will follow established methodological guidelines for the development and validation of health-related instruments.
Phase 1 - Questionnaire Development:
The initial version of the questionnaire, will be created based on the 2022 ESC/ERS Guidelines for PAH, a review of the scientific literature on patient knowledge in chronic diseases, and validated instruments in other conditions (e.g., chronic kidney disease, inflammatory bowel disease, rheumatoid arthritis). Items will cover the following domains: basic concepts, signs and symptoms, diagnosis and exams, available treatments and their mechanisms, adherence and self-care, and side effect management. Items will be multiple-choice with the option "I don't know".
Phase 2 - Content Validation:
At least five expert judges with experience in PAH, health education, and instrument development will evaluate each item for clarity, relevance, representativeness, and appropriateness using a 4-point Likert scale. The Content Validity Index (CVI) will be calculated for each item (I-CVI) and for the total instrument (S-CVI). Items with I-CVI ≥ 0.78 will be retained.
Phase 3 - Semantic Validation:
A sample of at least 10 patients diagnosed with PAH will be asked to complete the questionnaire while verbalizing their understanding ("think-aloud" technique). Revisions will be made based on participant feedback to ensure clarity and comprehension.
Phase 4 - Pilot Study and Psychometric Analysis:
The refined questionnaire will be applied to a sample of up to 200 patients with confirmed PAH. Psychometric analysis will include internal consistency (Cronbach's alpha) and, if applicable, test-retest reliability (intraclass correlation coefficient - ICC). Exploratory factor analysis may be used to assess internal structure.
In addition to validation, the study will collect clinical and functional data from medical records, including:
Risk stratification using COMPERA 2.0 methodology
The relationship between knowledge scores (SAVOIR) and these outcomes will be analyzed, aiming to explore whether higher patient knowledge is associated with better risk status, lower hospitalization rates, and higher quality of life.
This instrument is expected to help identify educational gaps, support targeted health education strategies, and contribute to improved clinical outcomes and shared decision-making in the care of PAH patients.
- Conditions
- Pulmonary Arterial Hypertension (PAH), Pulmonary Arterial Hypertension (PAH) (WHO Group 1 PH), Pulmonary Hypertension
- Being tested
- Application of Patient Knowledge Questionnaire on Pulmonary Hypertension
- Keywords
- Pulmonary Hypertension, Patient Knowledge, Questionnaire Validation, Content Validity, Semantic Analysis, Psychometric Validation, Health Education, Patient Education, Chronic Disease
- Medical subject headings
- Pulmonary Arterial Hypertension, Hypertension, Pulmonary, Health Education, Chronic Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Application of Patient Knowledge Questionnaire on Pulmonary HypertensionOther
Participants will complete one version of the SAVOIR questionnaire, developed to assess patient knowledge about pulmonary arterial hypertension. Depending on the study phase, participants will contribute to content validation (expert judges), semantic validation (patients), or psychometric validation (patients). In the final phase, additional data including risk stratification (COMPERA 2.0) will be collected for correlational analysis.
| Study group | Role | What they receive |
|---|---|---|
| Single Group | Experimental | Participants in this arm will take part in one of three phases of the questionnaire validation study:
1. content validation by expert judges,
2. semantic validation by patients with pulmonary arterial hypertension (PAH),
3. psychometric validation in a larger sample of up to 200 patients with confirmed PAH.
In the psychometric phase, participants will complete the final version of the SAVOIR questionnaire. Additional clinical and functional data will be collected from medical records, including:
risk stratification using the COMPERA 2.0 method,
These data will be used to explore associations between patient knowledge and clinical/functional outcomes. Receives: Other: Application of Patient Knowledge Questionnaire on Pulmonary Hypertension |
What is being measured
The primary outcome is the main question the study is designed to answer.
Content Validity Index (CVI) of the Questionnaire Items
Proportion of agreement among expert judges regarding the relevance and clarity of each item. Items with CVI ≥ 0.80 will be considered valid.
Measured: 4 weeks after expert panel review
Semantic Clarity Rating by Patients
Assessment of the clarity and comprehension of each item by patients using a structured form. Items with ≥ 85% of patients rating them as clear or very clear will be considered semantically valid.
Measured: 2 weeks after patient interview
Internal Consistency of the Questionnaire (Cronbach's Alpha)
Psychometric evaluation of the final version of the SAVOIR questionnaire using Cronbach's alpha. A coefficient ≥ 0.70 will be considered acceptable. The analysis will be conducted in a sample of up to 200 patients.
Measured: 4 weeks after questionnaire completion
Secondary outcomes (6)
Test-Retest Reliability (Intraclass Correlation Coefficient - ICC)
Stability of the questionnaire scores over time will be assessed by reapplying the instrument after 1 to 2 weeks in a subsample of patients. ICC ≥ 0.80 will be considered acceptable.
Measured: 2 weeks after first questionnaire administration
Completion Rate and Acceptability of the Questionnaire
Percentage of participants who complete the questionnaire without missing data and report that the items are clear and easy to understand.
Measured: At the time of first questionnaire administration (Day 1)
6. Association Between questionnaire Score and Risk Stratification (COMPERA 2.0)
Association between total DICTA score and patient risk status based on the COMPERA 2.0 stratification tool.
Measured: Within 4 weeks of questionnaire completion
Association Between questionnaire Score and Number of PAH-related Hospitalizations
Correlation between questionnaire score and number of PAH-related hospitalizations in the 12 months prior to questionnaire administration.
Measured: Within 4 weeks of questionnaire completion
Association Between the questionnaire Score (being developed) and Quality of Life Measures (EMPHASIS-10)
Association between questionnaire score and scores on quality of life tools.
Measured: Within 4 weeks of questionnaire completion
Association Between Patient Knowledge and Treatment Adherence using Martin-Bayarre-Grau (MBG Score)
Assess the association between the questionnaire scores and treatment adherence, as measured by the Martin-Bayarre-Grau (MBG) scale. The goal is to explore whether higher knowledge levels are correlated with greater adherence to treatment in patients with pulmonary arterial hypertension.
Measured: Within 4 weeks of questionnaire completion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Aug 2025
- Study start1 Sep 2025
- Primary completionDec 2026 (estimated)
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Pulmonary Arterial Hypertension (PAH)Studies of the same condition, open and under way.
- More trials run by University of Sao Paulo General HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Health services research
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- This is a methodological study for the development and validation of a questionnaire. The model includes sequential phases: construction, content validation by expert judges, semantic validation with patients, and psychometric analysis in a cross-sectional design. During the final phase, clinical and functional data will be collected from medical records to assess associations between knowledge scores and patient outcomes such as risk stratification (COMPERA 2.0). The study does not involve any intervention or assignment of participants to treatment arms.
- Masking
- None (open label)
- Enrolment
- 215 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Sao Paulo General Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Caio Júlio César dos Santos Fernandes, University of Sao Paulo General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07120789
- Sponsor's protocol ID
- SGP 27073
- Acronym
- SAVOIR
- Status last verified
- Mar 2026
- First submitted
- 4 Jul 2025
- Last updated
- 10 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Nascimento I, Carvalho GG, Rossetto NZ, Cauzzo IS, Milan MM, Jardim CVP, Fernandes CJCDS. The SAVOIR study: protocol for the development and validation of a PAH knowledge questionnaire. Respir Med. 2026 Jul 10;261:109028. doi: 10.1016/j.rmed.2026.109028. Online ahead of print.
PubMed 42425312
Study at a glance
From the registry record, last updated by the sponsor on 10 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Content, Semantic, and Psychometric Validation of a Patient Knowledge Questionnaire for Pulmonary Hypertension
- ClinicalTrials.gov ID
- NCT07120789
- Acronym / sponsor ID
- SAVOIR / SGP 27073
- Conditions
- Pulmonary Arterial Hypertension (PAH), Pulmonary Arterial Hypertension (PAH) (WHO Group 1 PH), Pulmonary Hypertension
- Keywords
- Pulmonary Hypertension, Patient Knowledge, Questionnaire Validation, Content Validity, Semantic Analysis, Psychometric Validation, Health Education, Patient Education
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 215 participants (estimated)
- Lead sponsor
- University of Sao Paulo General HospitalUniversity, hospital or other
- Study start
- 1 Sep 2025 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 13 Aug 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This methodological study aims to develop and validate a questionnaire named for the Assessment of Patient Knowledge in Pulmonary Arterial Hypertension. The study will be conducted in four phases: (1) questionnaire development based on guidelines and literature; (2) content validation by expert judges; (3) semantic validation with patients; and (4) psychometric testing in a sample of up to 200 patients with confirmed pulmonary arterial hypertension (PAH).
In addition to validation, the study will collect clinical and functional data from medical records, including risk stratification using the COMPERA 2.0 method The final instrument is expected to support patient education strategies and contribute to improved clinical management of PAH.
Detailed description
This is a methodological study with a quantitative approach, developed in four main phases, aimed at constructing and validating a questionnaire to assess the knowledge of patients with pulmonary arterial hypertension (PAH). The study will be conducted at a reference outpatient center in Brazil and will follow established methodological guidelines for the development and validation of health-related instruments.
Phase 1 - Questionnaire Development:
The initial version of the questionnaire, will be created based on the 2022 ESC/ERS Guidelines for PAH, a review of the scientific literature on patient knowledge in chronic diseases, and validated instruments in other conditions (e.g., chronic kidney disease, inflammatory bowel disease, rheumatoid arthritis). Items will cover the following domains: basic concepts, signs and symptoms, diagnosis and exams, available treatments and their mechanisms, adherence and self-care, and side effect management. Items will be multiple-choice with the option "I don't know".
Phase 2 - Content Validation:
At least five expert judges with experience in PAH, health education, and instrument development will evaluate each item for clarity, relevance, representativeness, and appropriateness using a 4-point Likert scale. The Content Validity Index (CVI) will be calculated for each item (I-CVI) and for the total instrument (S-CVI). Items with I-CVI ≥ 0.78 will be retained.
Phase 3 - Semantic Validation:
A sample of at least 10 patients diagnosed with PAH will be asked to complete the questionnaire while verbalizing their understanding ("think-aloud" technique). Revisions will be made based on participant feedback to ensure clarity and comprehension.
Phase 4 - Pilot Study and Psychometric Analysis:
The refined questionnaire will be applied to a sample of up to 200 patients with confirmed PAH. Psychometric analysis will include internal consistency (Cronbach's alpha) and, if applicable, test-retest reliability (intraclass correlation coefficient - ICC). Exploratory factor analysis may be used to assess internal structure.
In addition to validation, the study will collect clinical and functional data from medical records, including:
Risk stratification using COMPERA 2.0 methodology
The relationship between knowledge scores (SAVOIR) and these outcomes will be analyzed, aiming to explore whether higher patient knowledge is associated with better risk status, lower hospitalization rates, and higher quality of life.
This instrument is expected to help identify educational gaps, support targeted health education strategies, and contribute to improved clinical outcomes and shared decision-making in the care of PAH patients.
Study design
- Primary purpose
- Health services research
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- This is a methodological study for the development and validation of a questionnaire. The model includes sequential phases: construction, content validation by expert judges, semantic validation with patients, and psychometric analysis in a cross-sectional design. During the final phase, clinical and functional data will be collected from medical records to assess associations between knowledge scores and patient outcomes such as risk stratification (COMPERA 2.0). The study does not involve any intervention or assignment of participants to treatment arms.
| Arm | Type | Receives |
|---|---|---|
| Single Group | Experimental | Other: Application of Patient Knowledge Questionnaire on Pulmonary HypertensionParticipants in this arm will take part in one of three phases of the questionnaire validation study: 1. content validation by expert judges, 2. semantic validation by patients with pulmonary arterial hypertension (PAH), 3. psychometric validation in a larger sample of up to 200 patients with confirmed PAH. In the psychometric phase, participants will complete the final version of the SAVOIR questionnaire. Additional clinical and functional data will be collected from medical records, including: risk stratification using the COMPERA 2.0 method, These data will be used to explore associations between patient knowledge and clinical/functional outcomes. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Adults aged 18 years or older
- Confirmed diagnosis of Group 1 Pulmonary Arterial Hypertension (PAH)
- Ability to read and understand the questionnaire language
- Provided informed consent to participate in the study
- For expert judges (content validation phase): professionals with recognized expertise in pulmonary hypertension, with academic or clinical experience in the field
Key exclusion criteria (3 listed)
- Cognitive, visual, or auditory impairments that hinder questionnaire comprehension
- Refusal to participate or withdrawal of informed consent
- Inability to complete the questionnaire due to clinical instability
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Application of Patient Knowledge Questionnaire on Pulmonary HypertensionParticipants will complete one version of the SAVOIR questionnaire, developed to assess patient knowledge about pulmonary arterial hypertension. Depending on the study phase, participants will contribute to content validation (expert judges), semantic validation (patients), or psychometric validation (patients). In the final phase, additional data including risk stratification (COMPERA 2.0) will be collected for correlational analysis. | Other | Single Group |
Primary outcome
Content Validity Index (CVI) of the Questionnaire Items
Time frame: 4 weeks after expert panel review
Proportion of agreement among expert judges regarding the relevance and clarity of each item. Items with CVI ≥ 0.80 will be considered valid.
Semantic Clarity Rating by Patients
Time frame: 2 weeks after patient interview
Assessment of the clarity and comprehension of each item by patients using a structured form. Items with ≥ 85% of patients rating them as clear or very clear will be considered semantically valid.
Internal Consistency of the Questionnaire (Cronbach's Alpha)
Time frame: 4 weeks after questionnaire completion
Psychometric evaluation of the final version of the SAVOIR questionnaire using Cronbach's alpha. A coefficient ≥ 0.70 will be considered acceptable. The analysis will be conducted in a sample of up to 200 patients.
6 secondary outcomes
Test-Retest Reliability (Intraclass Correlation Coefficient - ICC)
Time frame: 2 weeks after first questionnaire administration
Completion Rate and Acceptability of the Questionnaire
Time frame: At the time of first questionnaire administration (Day 1)
6. Association Between questionnaire Score and Risk Stratification (COMPERA 2.0)
Time frame: Within 4 weeks of questionnaire completion
Association Between questionnaire Score and Number of PAH-related Hospitalizations
Time frame: Within 4 weeks of questionnaire completion
Association Between the questionnaire Score (being developed) and Quality of Life Measures (EMPHASIS-10)
Time frame: Within 4 weeks of questionnaire completion
Association Between Patient Knowledge and Treatment Adherence using Martin-Bayarre-Grau (MBG Score)
Time frame: Within 4 weeks of questionnaire completion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Mar 2026.
- Derived from this study: PubMed 42425312Nascimento I, Carvalho GG, Rossetto NZ, Cauzzo IS, Milan MM, Jardim CVP, Fernandes CJCDS. The SAVOIR study: protocol for the development and validation of a PAH knowledge questionnaire. Respir Med. 2026 Jul 10;261:109028. doi: 10.1016/j.rmed.2026.109028. Online ahead of print.
- Individual participant dataSharing planned: No
Related trials for Pulmonary Arterial Hypertension (PAH)
Active, not recruitingPhase 1
A Pilot Study of Microbiota Transplant With Antibiotic Preconditioning and Fiber Supplementation in Pulmonary Arterial Hypertension (GUT RESTORE-PAH)
Pulmonary Arterial Hypertension
RecruitingObservational study
Natural History Study of Biomarkers in Pulmonary Arterial Hypertension
Pulmonary Disease, Pulmonary Hypertension
Active, not recruitingPhase 1
Study of Intravenous ZMA001 in Healthy Subjects
Pulmonary Arterial Hypertension PAH
RecruitingPhase 4
Sotatercept in Pulmonary Arterial Hypertension
Pulmonary Artery Hypertension
Not yet recruiting
Home-based Inspiratory Muscle Training in Pulmonary Hypertension
Pulmonary Arterial Hypertension, Chronic Thromboembolic Pulmonary Hypertension
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Application of Patient Knowledge Questionnaire on Pulmonary Hypertension in people with Pulmonary Arterial Hypertension (PAH) and Pulmonary Arterial Hypertension (PAH) (WHO Group 1 PH), run by University of Sao Paulo General Hospital at 1 site in Brazil.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 10 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Brazil. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Caio Fernandes Principal Investigato, PhD (+55 11 2661 5034, caio.cesar@hc.fm.usp.br), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Content Validity Index (CVI) of the Questionnaire Items (time frame: 4 weeks after expert panel review). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT07120789, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Mar 2026.