Gene TherapyNCT05044845
Needs Assessment of Knowledge, Beliefs, and Attitudes of Patients With Hemophilia B About Gene Therapy
An observational study of Gene Therapy and Hemophilia B, run by St. Jude Children's Research Hospital at 6 sites in 6 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Gene Therapy, Hemophilia B
- What is being tested?
- Interview
- Type of treatment
- Lifestyle or therapy programme
- How many people
- 150 participants
- Where
- 6 sitesin 6 countries
- Study dates
- 18 Jan 2022to 14 Oct 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
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 12 years and older
- All sexes
- Child, Adult, Older adult
Study population: Participants who meet the eligibility criteria
Who may be able to join
- 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
- Doctors, nurses, social workers, pharmacists and educators who participate in the care of hemophilia B patients
Who cannot join
- Diagnosis of Hemophilia A
- Diagnosis of other non-Hemophilia B bleeding disorders
- Health care workers who do not participate in the care of hemophilia B patients
- Healthcare worker who is conducting the interviews
Trial locations and contacts
6 sites in 6 countries.
6 sites in 6 countries
United States1 site
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
About this study
Official title: Needs Assessment of Knowledge, Beliefs, and Attitudes of Patients With Hemophilia B About Gene Therapy Globally
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:
Read more
To assess baseline knowledge, beliefs, and attitudes about gene therapy held by hemophilia B patients globally
Secondary Objectives:
- To explore healthcare workers' (i.e., physicians, nurses, social workers, educators/academic coordinators) perspectives regarding the education needs of hemophilia B patients globally
- To explore healthcare workers beliefs and attitudes about gene therapy
- To identify preferences of patients with hemophilia B and their healthcare workers on how/by what method or pathway educational content should be provided.
Read the 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.
- Conditions
- Gene Therapy, Hemophilia B
- Being tested
- Interview
- Medical subject headings
- Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
InterviewBehavioural
Also called Questions
Semi structured interview will be conducted virtually using an online video-conferencing platform, phone or in person.
| Study group | Role | What they receive |
|---|---|---|
| Hemophilia B patients | Patients ≥12 years of age with a diagnosis of moderate (FIX ≥1% and ≤2%) or severe (<1%) hemophilia B Receives: Behavioral: Interview | |
| Parents or Caregivers | Parents or caregivers to patients with hemophilia 12-17 years of age Receives: Behavioral: Interview | |
| Healthcare Workers | Doctors, nurses, social workers, pharmacists and educators who participate in the care of hemophilia B patients Receives: Behavioral: Interview |
What is being measured
The primary outcome is the main question the study is designed to answer.
Use of semi-structured interviews to qualitatively describe their attitudes, beliefs, and knowledge of gene therapy in hemophilia B
Semi-structured interviews will be performed in patients with hemophilia B, their caregivers and healthcare workers to qualitatively describe their attitudes, beliefs, and knowledge of gene therapy in hemophilia B including concerns, expectations, and best way to receive information about gene therapy. Interviews will be audio recorded, transcribed verbatim and analyzed using semantic content analysis to identify common themes.
Measured: Day 1, or at a future visit (up to approximately 1 year)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Sep 2021
- Study start18 Jan 2022
- Primary completion14 Oct 2025
- Study completion14 Oct 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gene TherapyStudies of the same condition, open and under way.
- More trials run by St. Jude Children's Research 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 150 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Nidhi Bhatt, MD, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05044845
- Sponsor's protocol ID
- BINFORMED
- Status last verified
- Nov 2025
- First submitted
- 9 Sep 2021
- Last updated
- 14 Nov 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Needs Assessment of Knowledge, Beliefs, and Attitudes of Patients With Hemophilia B About Gene Therapy Globally
- ClinicalTrials.gov ID
- NCT05044845
- Acronym / sponsor ID
- BINFORMED
- Conditions
- Gene Therapy, Hemophilia B
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 150 participants (actual)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Study start
- 18 Jan 2022 (actual)
- Primary completion
- 14 Oct 2025 (actual)
- Study completion
- 14 Oct 2025 (actual)
- First posted
- 16 Sep 2021 (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.
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:
- To explore healthcare workers' (i.e., physicians, nurses, social workers, educators/academic coordinators) perspectives regarding the education needs of hemophilia B patients globally
- To explore healthcare workers beliefs and attitudes about gene therapy
- 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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Hemophilia B patients | – | Behavioral: InterviewPatients ≥12 years of age with a diagnosis of moderate (FIX ≥1% and ≤2%) or severe (<1%) hemophilia B |
| Parents or Caregivers | – | Behavioral: InterviewParents or caregivers to patients with hemophilia 12-17 years of age |
| Healthcare Workers | – | Behavioral: InterviewDoctors, nurses, social workers, pharmacists and educators who participate in the care of hemophilia B patients |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
- Study population
- Participants who meet the eligibility criteria
Key inclusion criteria (4 listed)
- 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
- Doctors, nurses, social workers, pharmacists and educators who participate in the care of hemophilia B patients
Key exclusion criteria (4 listed)
- Diagnosis of Hemophilia A
- Diagnosis of other non-Hemophilia B bleeding disorders
- Health care workers who do not participate in the care of hemophilia B patients
- Healthcare worker who is conducting the interviews
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 |
|---|---|---|
| InterviewAlso: QuestionsSemi structured interview will be conducted virtually using an online video-conferencing platform, phone or in person. | Behavioural | Healthcare Workers, Hemophilia B patients, Parents or Caregivers |
Primary outcome
Use of semi-structured interviews to qualitatively describe their attitudes, beliefs, and knowledge of gene therapy in hemophilia B
Time frame: Day 1, or at a future visit (up to approximately 1 year)
Semi-structured interviews will be performed in patients with hemophilia B, their caregivers and healthcare workers to qualitatively describe their attitudes, beliefs, and knowledge of gene therapy in hemophilia B including concerns, expectations, and best way to receive information about gene therapy. Interviews will be audio recorded, transcribed verbatim and analyzed using semantic content analysis to identify common themes.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Nov 2025.
- St. Jude Children's Research HospitalLink provided by the sponsor.
- ClinicalTrials Open at St. JudeLink provided by the sponsor.
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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. This study is not recruiting at the moment.
Nidhi Bhatt, MD principal investigator
St. Jude Children's Research Hospital
6 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Gene Therapy and Hemophilia B, run by St. Jude Children's Research Hospital at 6 sites in 6 countries.
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 4 inclusion and 4 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: finished, as of the sponsor's last update on 14 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States, Nepal, Peru, Sri Lanka, Thailand and 1 more country. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Use of semi-structured interviews to qualitatively describe their attitudes, beliefs, and knowledge of gene therapy in hemophilia B (time frame: Day 1, or at a future visit (up to approximately 1 year)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 14 Nov 2025; AzTrials last refreshed its copy on 8 Nov 2026.