Sickle Cell DiseaseNCT05810181
Gene Therapy Communication: Use of a Needs Assessment to Drive Decision-AIDS for Gene Therapy for Rare Diseases (GENETX)
An observational study of Sickle Cell Disease, run by St. Jude Children's Research Hospital at 1 site in United States.
Trial summary
This prospective mixed-method interview study aims to qualitatively describe the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases among patients and parents of children with a rare disease targeted for treatment using gene therapy techniques. Read more about this study
- Who is it for?
- Ages 8 years and olderHealthy volunteers can take part
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Interview
- How many people
- 145 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Jun 2023to Dec 2027
- Phase
- Observational
- 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 8 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: About 105 patients (age 8 and above) or parents of children verified to have a rare disease targeted for treatment using gene therapy techniques and 10-20 healthcare workers who provide care to patients receiving gene therapy (including an additional 10-20 healthcare workers treating patients with a bone marrow failure condition) .
Who may be able to join
- For Group 1 participants only (Undergone Gene Therapy):
- Parent/caregiver whose child has undergone gene therapy. OR Parent/caregiver of a child who died after receiving gene therapy at least 6 months prior to enrollment, but no more than 24 months prior to enrollment, to be contacted no sooner than 3 months after the death has occurred and no longer than 2 years. OR Patients age 8 and above who have undergone gene therapy.
- Willingness to participate in one-on-one video interview with a study team member using a personal mobile device or computer with working internet connection.
- Must be willing to provide verbal informed consent.
- Release of information form signed by participant providing our study team with permission to contact healthcare provider to verify their diagnosis and receipt of gene therapy (if received).
- Successful verification of diagnosis of rare genetic disease targeted for treatment using gene therapy.
Show all 40Show fewer
- A positive confirmation on receipt of gene therapy and type received from their healthcare provider (only for those received gene therapy).
- For Group 2 participants only (Offered, but did not Undergo Gene Therapy):
- Parent/caregiver of children (or patients 8 and above ) with a rare genetic disease who had been offered but were not eligible for a trial or decided against receiving gene therapy.
- Willingness to participate in one-on-one video interview with a study team member using a personal mobile device or computer with working internet connection.
- Must be willing to provide verbal informed consent.
- Signed release of information form providing GeneTx study team with permission to contact participant's healthcare provider to verify the diagnosis.
- Successful verification of diagnosis of rare genetic disease targeted for treatment using gene therapy.
- For Group 3 participants only (Provider Interviews):
- Healthcare worker who has provided care to ≥ 2 patients receiving gene therapy.
- Willingness to participate in one-on-one video (or in-person) interview with a study team member using a personal mobile device or computer with working internet connection.
- Informed consent from a study participant.
- For Group 4 participants only (Undergone Gene Therapy for Bone Marrow Failure Condition):
- Parent/caregiver whose child has undergone gene therapy. OR Parent/caregiver of a child who died after receiving gene therapy at least 6 months prior to enrollment, but no more than 24 months prior to enrollment, to be contacted no sooner than 3 months after the death has occurred and no longer than 2 years. OR Patients age 8 and above who have undergone gene therapy.
- Willingness to participate in one-on-one video interview with a study team member using a personal mobile device or computer with working internet connection.
- Must be willing to provide verbal informed consent.
- Release of information form signed by participant providing our study team with permission to contact healthcare provider to verify their diagnosis and receipt of gene therapy (if received).
- Successful verification of diagnosis of rare genetic disease targeted for treatment using gene therapy.
- A positive confirmation on receipt of gene therapy and type received from their healthcare provider (only for those received gene therapy).
- For Group 5 participants only (Offered, but did not Undergo Gene Therapy for Bone Marrow Failure Condition ):
- Parent/caregiver of children (or patients 8 and above ) with a bone marrow failure disease who had been offered but were not eligible for a trial or decided against receiving gene therapy.
- Willingness to participate in one-on-one video interview with a study team member using a personal mobile device or computer with working internet connection.
- Must be willing to provide verbal informed consent.
- Signed release of information form providing GeneTx study team with permission to contact participant's healthcare provider to verify the diagnosis.
- Successful verification of diagnosis of rare genetic disease targeted for treatment using gene therapy.
- For Group 6 participants only (Never offered gene therapy for Bone Marrow Failure Condition):
- Parent/caregiver of children (or patients 8 and above ) with a bone marrow failure disease who had not been offered gene therapy.
- Willingness to participate in one-on-one video interview with a study team member using a personal mobile device or computer with working internet connection.
- Must be willing to provide verbal informed consent.
- Signed release of information form providing GeneTx study team with permission to contact participant's healthcare provider to verify the diagnosis.
- Successful verification of diagnosis of rare genetic disease targeted for treatment using gene therapy.
- For Group 7 participants only (Provider Interviews for Bone Marrow Failure Condition):
- Healthcare worker who has provided care to ≥ 2 patients receiving gene therapy.
- Willingness to participate in one-on-one video (or in-person) interview with a study team member using a personal mobile device or computer with working internet connection.
- Informed consent from a study participant.
Who cannot join
- Participants who are unable to converse fluently in English will be excluded.
- Inability or unwillingness of research participant to give verbal informed consent.
- Participants who lack access to a computer or mobile device that supports video communications will be excluded.
- Condition or chronic illness, which in the opinion of the PI/Co-I, makes participation unsafe or untenable (i.e., cognitive impairment, concurrent acute morbidity).
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
888-226-4343, referralinfo@stjude.org
About this study
This prospective mixed-method interview study aims to qualitatively describe the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases among patients and parents of children with a rare disease targeted for treatment using gene therapy techniques. Using learned insights, the team will develop an online platform providing educational content and patient decision aids for patients and their families.
Read the detailed description
The study team seeks to understand the unique needs of patients and parents of children who are considering novel experimental gene therapy treatments. The study team will engage patients, caregivers, and healthcare worker stakeholders to understand their attitudes, beliefs, and concerns surrounding these treatments. Based on these insights, the team, with the support of the above stakeholders and an external vendor, will develop web platform offering educational content and decision aids to enhance their experience and overall satisfaction with the process of making treatment decisions. This is especially important as novel therapies for SCD will increasingly include gene editing in pediatric trials over the next two years. These efforts will address significant gaps in the educational material available to those considering gene therapy and (to the team's knowledge) will create a resource that will be the first of its kind.
- Conditions
- Sickle Cell Disease
- Being tested
- Interview
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
InterviewOther
The following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases:
(1) patients and families of children with rare genetic diseases who have received gene therapy (to assess their perspectives and understand their informational needs around participating in a clinical trial using gene therapy).
InterviewOther
The following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (2) patients and families of children with rare genetic diseases who were offered but have decided against receiving gene therapy or who were never offered gene therapy or ultimately did not qualify for a trial (to elicit feedback and to evaluate whether their informational needs and communication expectations about participating in a clinical trial using gene therapy were met).
InterviewOther
The following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (3) healthcare workers who provide care to patients receiving gene therapy (to assess their perspective regarding patient/family needs for gene therapy education and communication).
InterviewOther
The following group of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (4) parents/caregivers of children with a bone marrow failure condition, who has undergone gene therapy, OR parent/caregiver of a child who died after receiving gene therapy at least 6 months prior to enrollment, but no more than 24 months prior to enrollment OR patients age 8 and above who have undergone gene therapy (to assess their perspectives and understand their informational needs around participating in a clinical trial using gene therapy).
InterviewOther
The following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (5) parent/caregiver of children (or patients 8 and above) with bone marrow failure conditions who were offered but have decided against receiving gene therapy or who were ultimately not eligible for a clinical trial (to elicit feedback and to evaluate whether their informational needs and communication expectations about participating in a clinical trial using gene therapy were met).
InterviewOther
The following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (6) parent/caregiver of children (or patients 8 and above) with a bone marrow failure condition who were never offered gene therapy (to elicit feedback and to evaluate whether their informational needs and communication expectations about participating in a clinical trial using gene therapy)
InterviewOther
The following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (7) health care workers who provide care to patients receiving gene therapy for bone marrow failure conditions (to assess their perspective regarding patient/family needs for gene therapy education and communication).
| Study group | Role | What they receive |
|---|---|---|
| Young Adult | 15-25 parents/families of children (patients aged 8 and above) with rare genetic diseases, who have recently received gene therapy Receives: Other: Interview | |
| Parent/caregiver | 10-20 patients/families of children with rare genetic diseases who were offered but have decided against receiving gene therapy or who were ultimately not eligible for a clinical trial. Receives: Other: Interview | |
| Healthcare Worker | 10-20 health care workers' who provide care to patients receiving gene therapy. Receives: Other: Interview | |
| Bone marrow failure condition (received gene therapy) | 5-10 parents/families of children with a bone marrow failure condition, who has undergone gene therapy, OR parent/caregiver of a child who died after receiving gene therapy at least 6 months prior to enrollment, but no more than 24 months prior to enrollment OR patients age 8 and above who have undergone gene therapy Receives: Other: Interview | |
| Bone marrow failure condition (did not receive gene therapy) | 5-10 parent/caregiver of children (or patients 8 and above) with bone marrow failure conditions who were offered but have decided against receiving gene therapy or who were ultimately not eligible for a clinical trial Receives: Other: Interview | |
| Bone marrow failure condition (never offered gene therapy) | 30-40 parent/caregiver of children (or patients 8 and above) with a bone marrow failure condition who were never offered gene therapy Receives: Other: Interview | |
| Healthcare Workers (for bone marrow failure condition) | 10-20 health care workers who provide care to patients receiving gene therapy for bone marrow failure conditions. Receives: Other: Interview |
What is being measured
The primary outcome is the main question the study is designed to answer.
Use of semi-structured interviews to assess the beliefs, attitudes, and informational needs around gene therapy among patients and families with rare genetic diseases.
Trained experts will interview study participants to elucidate their beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases among the disease stakeholders. These interviews will be analyzed via the well-described rigorous methodology of semantic content analysis to identify themes through a systematic and standardized process.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Apr 2023
- Study start1 Jun 2023
- Primary completionDec 2027 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies 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
- 145 participants (estimated)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Sponsor
- Investigators
- Liza-Marie Johnson, MD, MPH, MSB, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05810181
- Sponsor's protocol ID
- GENETX
- Other IDs
- R01HL156647, U01HL163983
- Status last verified
- Aug 2026
- First submitted
- 30 Mar 2023
- Last updated
- 10 Aug 2026
- 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 10 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Gene Therapy Communication: Use of a Needs Assessment to Drive Decision-AIDS for Gene Therapy for Rare Diseases (GENETX)
- ClinicalTrials.gov ID
- NCT05810181
- Acronym / sponsor ID
- GENETX
- Conditions
- Sickle Cell Disease
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 145 participants (estimated)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- 1 Jun 2023 (actual)
- Primary completion
- Dec 2027 (estimated)
- Study completion
- Dec 2027 (estimated)
- First posted
- 12 Apr 2023 (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 prospective mixed-method interview study aims to qualitatively describe the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases among patients and parents of children with a rare disease targeted for treatment using gene therapy techniques. Using learned insights, the team will develop an online platform providing educational content and patient decision aids for patients and their families.
Detailed description
The study team seeks to understand the unique needs of patients and parents of children who are considering novel experimental gene therapy treatments. The study team will engage patients, caregivers, and healthcare worker stakeholders to understand their attitudes, beliefs, and concerns surrounding these treatments. Based on these insights, the team, with the support of the above stakeholders and an external vendor, will develop web platform offering educational content and decision aids to enhance their experience and overall satisfaction with the process of making treatment decisions. This is especially important as novel therapies for SCD will increasingly include gene editing in pediatric trials over the next two years. These efforts will address significant gaps in the educational material available to those considering gene therapy and (to the team's knowledge) will create a resource that will be the first of its kind.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 7
| Arm | Type | Receives |
|---|---|---|
| Young Adult | – | Other: Interview15-25 parents/families of children (patients aged 8 and above) with rare genetic diseases, who have recently received gene therapy |
| Parent/caregiver | – | Other: Interview10-20 patients/families of children with rare genetic diseases who were offered but have decided against receiving gene therapy or who were ultimately not eligible for a clinical trial. |
| Healthcare Worker | – | Other: Interview10-20 health care workers' who provide care to patients receiving gene therapy. |
| Bone marrow failure condition (received gene therapy) | – | Other: Interview5-10 parents/families of children with a bone marrow failure condition, who has undergone gene therapy, OR parent/caregiver of a child who died after receiving gene therapy at least 6 months prior to enrollment, but no more than 24 months prior to enrollment OR patients age 8 and above who have undergone gene therapy |
| Bone marrow failure condition (did not receive gene therapy) | – | Other: Interview5-10 parent/caregiver of children (or patients 8 and above) with bone marrow failure conditions who were offered but have decided against receiving gene therapy or who were ultimately not eligible for a clinical trial |
| Bone marrow failure condition (never offered gene therapy) | – | Other: Interview30-40 parent/caregiver of children (or patients 8 and above) with a bone marrow failure condition who were never offered gene therapy |
| Healthcare Workers (for bone marrow failure condition) | – | Other: Interview10-20 health care workers who provide care to patients receiving gene therapy for bone marrow failure conditions. |
Who may qualify
- Ages
- 8 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- About 105 patients (age 8 and above) or parents of children verified to have a rare disease targeted for treatment using gene therapy techniques and 10-20 healthcare workers who provide care to patients receiving gene therapy (including an additional 10-20 healthcare workers treating patients with a bone marrow failure condition) .
Key inclusion criteria (40 listed)
- For Group 1 participants only (Undergone Gene Therapy):
- For Group 2 participants only (Offered, but did not Undergo Gene Therapy):
- For Group 3 participants only (Provider Interviews):
- For Group 4 participants only (Undergone Gene Therapy for Bone Marrow Failure Condition):
- For Group 5 participants only (Offered, but did not Undergo Gene Therapy for Bone Marrow Failure Condition ):
Key exclusion criteria (4 listed)
- Participants who are unable to converse fluently in English will be excluded.
- Inability or unwillingness of research participant to give verbal informed consent.
- Participants who lack access to a computer or mobile device that supports video communications will be excluded.
- Condition or chronic illness, which in the opinion of the PI/Co-I, makes participation unsafe or untenable (i.e., cognitive impairment, concurrent acute morbidity).
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 |
|---|---|---|
| InterviewThe following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (1) patients and families of children with rare genetic diseases who have received gene therapy (to assess their perspectives and understand their informational needs around participating in a clinical trial using gene therapy). | Other | Young Adult |
| InterviewThe following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (2) patients and families of children with rare genetic diseases who were offered but have decided against receiving gene therapy or who were never offered gene therapy or ultimately did not qualify for a trial (to elicit feedback and to evaluate whether their informational needs and communication expectations about participating in a clinical trial using gene therapy were met). | Other | Parent/caregiver |
| InterviewThe following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (3) healthcare workers who provide care to patients receiving gene therapy (to assess their perspective regarding patient/family needs for gene therapy education and communication). | Other | Healthcare Worker |
| InterviewThe following group of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (4) parents/caregivers of children with a bone marrow failure condition, who has undergone gene therapy, OR parent/caregiver of a child who died after receiving gene therapy at least 6 months prior to enrollment, but no more than 24 months prior to enrollment OR patients age 8 and above who have undergone gene therapy (to assess their perspectives and understand their informational needs around participating in a clinical trial using gene therapy). | Other | Bone marrow failure condition (received gene therapy) |
| InterviewThe following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (5) parent/caregiver of children (or patients 8 and above) with bone marrow failure conditions who were offered but have decided against receiving gene therapy or who were ultimately not eligible for a clinical trial (to elicit feedback and to evaluate whether their informational needs and communication expectations about participating in a clinical trial using gene therapy were met). | Other | Bone marrow failure condition (did not receive gene therapy) |
| InterviewThe following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (6) parent/caregiver of children (or patients 8 and above) with a bone marrow failure condition who were never offered gene therapy (to elicit feedback and to evaluate whether their informational needs and communication expectations about participating in a clinical trial using gene therapy) | Other | Bone marrow failure condition (never offered gene therapy) |
| InterviewThe following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (7) health care workers who provide care to patients receiving gene therapy for bone marrow failure conditions (to assess their perspective regarding patient/family needs for gene therapy education and communication). | Other | Healthcare Workers (for bone marrow failure condition) |
Primary outcome
Use of semi-structured interviews to assess the beliefs, attitudes, and informational needs around gene therapy among patients and families with rare genetic diseases.
Time frame: 2 years
Trained experts will interview study participants to elucidate their beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases among the disease stakeholders. These interviews will be analyzed via the well-described rigorous methodology of semantic content analysis to identify themes through a systematic and standardized process.
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 Aug 2026.
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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.
Liza-Marie Johnson, MD, MPH, MSB principal investigator
St. Jude Children's Research Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Sickle Cell Disease, run by St. Jude Children's Research Hospital at 1 site in United States.
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 40 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: looking for participants now, as of the sponsor's last update on 10 Aug 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 United States. 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 Liza-Marie Johnson, MD, MPH, MSB (888-226-4343, referralinfo@stjude.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Use of semi-structured interviews to assess the beliefs, attitudes, and informational needs around gene therapy among patients and families with rare genetic diseases. (time frame: 2 years). 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 10 Aug 2026.