A Stepped-Wedge Cluster Randomized Trial of the TORCH Congenital Advanced Therapies Pathway for Children and Adults With Congenital Heart Disease in Latin America
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- 入组人数
- 360
- 主要终点
- Completion of Multidisciplinary Congenital Advanced-Therapy Evaluation and Documented Pathway Decision Within 90 Days
研究概览
简要总结
TORCH-LATAM-003 is a pragmatic stepped-wedge cluster randomized trial evaluating a regional pathway for children and adults with congenital heart disease and advanced cardiac and/or respiratory failure who may require heart, lung, or heart-lung transplantation, mechanical circulatory or extracorporeal support, or another regulated advanced-therapy pathway. Clusters transition from usual local pathways to TORCH-CATP. The primary outcome is completion of the prespecified multidisciplinary evaluation and documentation of an advanced-pathway decision within 90 days after enrollment. The study also evaluates barriers, authorized pathway or waiting-list activation, longitudinal status, advanced support, definitive therapy, and clinical outcomes. Donor-organ and matching events are captured only where legally authorized and do not replace national allocation systems or transplant-center decisions.
详细描述
Each TORCH regional network cluster is anchored in an authorized advanced-therapy or transplant center and may include activated evaluation, referral, congenital cardiology, pulmonary hypertension, intensive-care, mechanical-support, donor-origin, procurement, preservation, logistics, and data-coordination nodes. Children and adults with congenital heart disease may be identified at transplanting or non-transplanting centers when an authorized route to specialized evaluation exists. Authorized centers and competent authorities retain all legal and clinical authority for candidacy, waiting-list activation, organ acceptance, allocation, and transplantation.
The recipient pathway records identification, referral, enrollment, completion of multidisciplinary congenital evaluation, a documented advanced-pathway decision, barrier management, activation of an authorized pathway or official waiting list, longitudinal clinical and support status, transplantation, regulated external-protocol enrollment, or an alternative outcome. Study enrollment is analytically distinct from advanced-therapy candidacy and official wait-list status.
The pathway permits clinically documented redirection among heart, lung, heart-lung, or other authorized transplant evaluation; temporary or durable mechanical support; recovery; surveillance; palliative care; and independently governed investigational protocols. TORCH does not assign an investigational therapy, device, cell, organoid, or xenograft and does not infer efficacy or safety for such products.
Where legally authorized, donor-organ and recipient pathways converge at traceable compatibility and offer episodes. Each decision, non-acceptance reason, reassignment, urgency change, distance, logistics constraint, and final disposition is time-stamped. TORCH does not create a supranational allocation authority or override governmental or institutional systems.
Interoperability may range from audited manual transfer to structured documents, batch exchange, application programming interfaces, standards-based exchange, or validated remote-monitoring devices. Cross-border activity is activated only after applicable ethical, regulatory, institutional, data-protection, financial, and operational approvals.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Health Services Research
- 盲法
- None
入排标准
- 年龄范围
- 0 Days 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Child or adult of any age with congenital heart disease; enrollment of minors requires parent or legal-representative permission and assent when applicable.
- •Congenital heart disease with advanced cardiac and/or respiratory failure, associated pulmonary hypertension, or another protocol-defined trigger for specialized advanced-therapy evaluation.
- •Identification within an activated TORCH network cluster with an operational route to at least one authorized advanced-therapy or transplant center.
- •Ability to complete protocol-defined follow-up directly or through an authorized network pathway.
- •Provision of informed consent by the participant or legally authorized representative, and assent when applicable, according to local requirements.
排除标准
- •Absence of congenital heart disease as the underlying condition defining the advanced cardiac and/or respiratory-failure pathway.
- •No authorized route for protocol evaluation or follow-up at the time of enrollment.
- •Unable or unwilling to provide informed consent or legally authorized permission when required.
- •The prespecified multidisciplinary evaluation and advanced-pathway decision were already completed and remain clinically current before enrollment.
研究组 & 干预措施
TORCH-CATP Intervention Period
Participants enrolled while their cluster is assigned to the intervention period receive the TORCH Congenital Advanced Therapies Pathway, including protocolized identification and referral, multidisciplinary evaluation, pathway-decision adjudication, barrier navigation, pathway and waiting-list status tracking, and traceable donor-organ opportunity coordination when applicable.
干预措施: TORCH Congenital Advanced Therapies Pathway (TORCH-CATP) (Other)
Usual Local Pathway Period
Participants enrolled before their cluster transitions to TORCH-CATP receive locally available referral, evaluation, treatment, support, and transplant-candidacy processes. Study data collection does not alter the authority of local programs or competent authorities.
结局指标
主要结局
Completion of Multidisciplinary Congenital Advanced-Therapy Evaluation and Documented Pathway Decision Within 90 Days
时间窗: Within 90 days after enrollment.
Percentage of eligible enrolled participants who complete the prespecified multidisciplinary congenital advanced-therapy evaluation and have a documented advanced-pathway decision within 90 days after enrollment. APD-A, APD-B, APD-D, APD-E, APD-F, APD-G, and APD-H count as completed decisions when the minimum evaluation is complete. APD-C denotes incomplete evaluation and is not counted as success. Death, irreversible deterioration, or missing evidence before day 90 is not counted as success unless the minimum evaluation and a valid decision were documented.
次要结局
- Activation of an Authorized Advanced-Therapy Pathway or Official Waiting List(Within 180 days after the first documented advanced-pathway decision qualifying for activation.)
- Transplantation by Organ Type(Through 24 months after enrollment.)
- Time to Completed Multidisciplinary Evaluation and Advanced-Pathway Decision(From enrollment to completed evaluation and documented advanced-pathway decision, assessed through 180 days.)
- Redirection of the Advanced-Therapy Pathway(Through 24 months after enrollment.)
- Waiting-List Status or Urgency Transition(At each documented waiting-list transition through 24 months after enrollment.)
- Initiation of Temporary Mechanical Circulatory or Extracorporeal Life Support(Through 24 months after enrollment.)
- Durable Mechanical Support Implantation(Through 24 months after enrollment.)
- Survival Free From Irreversible Clinical Deterioration(At 90 days, 1 year, and 2 years after enrollment.)
- Resolution of Identified Barriers(Within 180 days after identification of each barrier.)
- Authorized Enrollment in an External Advanced-Therapy Protocol(24 months after enrollment.)
- Utilization of TORCH-Evaluated Donor Organs(At final disposition of each donor organ, during the perioperative/periprocedural period, including implantation, recovery without implantation, or documented non-use.)