Gene Therapy Communication: Use of a Needs Assessment to Drive Decision-AIDS for Gene Therapy for Rare Diseases (GENETX)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 145
- 试验地点
- 1
- 主要终点
- Use of semi-structured interviews to assess the beliefs, attitudes, and informational needs around gene therapy among patients and families with rare genetic diseases.
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 8 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •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.
- •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.
排除标准
- •(for all 7 groups):
- •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).
研究组 & 干预措施
Young Adult
15-25 parents/families of children (patients aged 8 and above) with rare genetic diseases, who have recently received gene therapy
干预措施: Interview (Other)
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.
干预措施: Interview (Other)
Healthcare Worker
10-20 health care workers' who provide care to patients receiving gene therapy.
干预措施: Interview (Other)
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
干预措施: Interview (Other)
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
干预措施: Interview (Other)
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
干预措施: Interview (Other)
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.
干预措施: Interview (Other)
结局指标
主要结局
Use of semi-structured interviews to assess the beliefs, attitudes, and informational needs around gene therapy among patients and families with rare genetic diseases.
时间窗: 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.
次要结局
未报告次要终点