Clinical trial NCT05044845: Needs Assessment of Knowledge, Beliefs, and Attitudes of Patients With Hemophilia B About Gene Therapy
Official title: Needs Assessment of Knowledge, Beliefs, and Attitudes of Patients With Hemophilia B About Gene Therapy Globally
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Gene Therapy
Hemophilia B
Lead sponsor: St. Jude Children's Research Hospital
Sponsor class: OTHER
First posted: 2021-09-16
Enrollment: 150
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants who meet the eligibility criteria

Brief summary: Gene therapy is a paradigm-shifting treatment for hemophilia B patients, particularly in resource-limited countries where factor availability remains low. Transparent and culturally sensitive communication around gene therapy is vital to the success of a high-quality consenting process. Current literature on knowledge, beliefs and attitudes about gene therapy in resource-limited countries is inadequate. In addition, few educational resources to explain basic gene therapy concepts exist in languages other than English. This study aims to address these gaps in knowledge and aid for the development of educational resources to assist the informed consent processes for gene therapy in resource-limited countries.

Primary Objective:

To assess baseline knowledge, beliefs, and attitudes about gene therapy held by hemophilia B patients globally

Secondary Objectives:

1. To explore healthcare workers' (i.e., physicians, nurses, social workers, educators/academic coordinators) perspectives regarding the education needs of hemophilia B patients globally
2. To explore healthcare workers beliefs and attitudes about gene therapy
3. To identify preferences of patients with hemophilia B and their healthcare workers on how/by what method or pathway educational content should be provided.

Detailed description: This prospective cohort study will use qualitative methods, specifically semi structured interviews to evaluate knowledge, beliefs, and attitude about gene therapy in patients 12 years and older with a diagnosis of moderate and severe hemophilia B (≤2% FIX activity only), caregivers/parents and healthcare workers who care for them at St. Jude and other global countries. Participants will be interviewed virtually using an online video-conferencing platform, phone or in person.

Interviews will be performed by experienced qualitative interviewers in the official language of each participating site. Interviews will last approximately 45-60 minutes and will be performed only once. All interviews will be audio recorded and obtained information will be analyzed using semantic content analysis to identify common themes.

Eligibility criteria: Inclusion Criteria:

* Patients ≥12 years of age
* Diagnosis of moderate (FIX ≥1% and ≤2%) or severe (\<1%) hemophilia B
* Parents or caregivers to patients with hemophilia 12-17 years of age

Inclusion Criteria - Healthcare worker:

- Doctors, nurses, social workers, pharmacists and educators who participate in the care of hemophilia B patients

Exclusion Criteria:

* Diagnosis of Hemophilia A
* Diagnosis of other non-Hemophilia B bleeding disorders

Exclusion Criteria - Healthcare worker:

* Health care workers who do not participate in the care of hemophilia B patients
* Healthcare worker who is conducting the interviews

Primary outcomes:
measure: Use of semi-structured interviews to qualitatively describe their attitudes, beliefs, and knowledge of gene therapy in hemophilia B
timeFrame: Day 1, or at a future visit (up to approximately 1 year)
Secondary outcomes:
Other outcomes:
Locations:
city: Memphis
country: United States
facility: St. Jude Children's Research Hospital
state: Tennessee
city: Kathmandu
country: Nepal
facility: Civil Service Hospital
city: Lima 15003
country: Peru
facility: Hospital Dos De Mayo
city: Colombo 10
country: Sri Lanka
facility: National Hospital of Sri Lanka
city: Bangkok
country: Thailand
facility: Ramathibodi Hospital
city: Hanoi
country: Vietnam
facility: National Institute of Hematology and Blood Transfusion
Study officials:
affiliation: St. Jude Children's Research Hospital
name: Nidhi Bhatt, MD
role: PRINCIPAL_INVESTIGATOR
References: