Pilot Study "Ready to Sail 2": Evaluation of the Feasibility and Safety of Sail-Assisted Telerehabilitation in Patients With Rare Skeletal Diseases - Assessment of Motor, Psychosocial, and Quality of Life Outcomes
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 24
- 试验地点
- 1
- 主要终点
- Acceptability of the Intervention
研究概览
简要总结
"Ready to Sail 2 - EXTEND" is a pilot randomized interventional trial designed to assess the feasibility, safety, and preliminary impact of an innovative rehabilitation model combining sailing therapy (velatherapy) with a remote telerehabilitation maintenance program in young individuals with rare skeletal disorders. Building on the promising results of the previous Ready to Sail feasibility study (NCT06397443), this study aims to validate and compare two rehabilitative approaches.
A total of 24 patients aged 12-30 years with a confirmed diagnosis of a rare skeletal disorder will be randomly assigned (1:1) to either:
- Group 1 (Experimental): a 5-day intensive sailing therapy intervention followed by a 3-month personalized telerehabilitation program at home; or
- Group 2 (Active Control): a 3-month telerehabilitation-only program with identical frequency and progression.
The primary objective is to evaluate the feasibility of the experimental program, in terms of adherence, acceptability, and safety. Secondary outcomes include motor functionality (measured by inertial sensors), psychosocial well-being (assessed through validated PROMs), health-related quality of life, and pain perception. The study also explores the medium-term sustainability of rehabilitation outcomes and the influence of individual variables (e.g., age, gender, disease subtype).
The intervention is delivered through a multidisciplinary, patient-centered approach involving rare disease experts, physiatrists, biomechanical engineers, and partner sailing organizations. Sail training activities are adapted for safety and inclusion, and are conducted in two coastal settings (Marina di Ravenna and Palermo), ensuring geographical balance for participant accessibility.
This trial seeks to strengthen the evidence base for integrated, inclusive, and scalable rehabilitation models that address both motor and psychosocial dimensions in rare skeletal conditions.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Supportive Care
- 盲法
- Single (Outcomes Assessor)
入排标准
- 年龄范围
- 12 Years 至 30 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Individuals aged 12 to 30 years, of any sex
- •Clinical and/or molecular genetic diagnosis of RSD per Orphanet classification
- •No orthopedic surgery within the past 6 months, unless deemed clinically irrelevant to study participation by the investigator
- •Ability to understand and comply with study procedures
- •Signed informed consent obtained from the participant or a legal guardian (for minors)
排除标准
- •Severe or unstable medical conditions that could interfere with participation (e.g., uncontrolled cardiac or respiratory disease)
- •Cognitive or neurological impairments that may hinder adherence to the study protocol
- •Participation in similar intensive rehabilitation programs within the past 3 months
- •Contraindications to virtual or sailing-based activities, including but not limited to: (a) Severe motion sickness; (b) Vertigo or vestibular disorders; (c) Photosensitive epilepsy
- •Musculoskeletal injuries (e.g., fractures or major sprains) occurring within the past 12 months
- •Any condition or circumstance that, in the opinion of the investigator, may compromise safety or study compliance
- •Decline or inability to provide informed consent
研究组 & 干预措施
Home-Based Telerehabilitation Program
Participants in this arm will undergo a 3-month home-based telerehabilitation program. The intervention consists of two weekly remote sessions conducted via an accredited digital platform. Each session includes warm-up, structured motor and postural exercises, and cool-down/stretching phases. Exercises are progressive and tailored to individual functional capacities, with the goal of improving balance, coordination, and physical function. No sailing therapy is included in this arm.
干预措施: Home-Based Telerehabilitation (Behavioral)
Sailing Intervention Followed by Home-Based Telerehabilitation Program
Participants assigned to this arm will receive a 5-day intensive sailing intervention program, followed by a 3-month home-based telerehabilitation program. The sailing includes daily group-based sailing activities and debriefing activities aimed at enhancing motor skills, coordination, and psychosocial well-being. After the sailing phase, participants will engage in remote physical therapy sessions twice a week via a supervised digital platform. The telerehabilitation component includes progressive exercises targeting balance, proprioception, and functional strength.
干预措施: Sailing Intervention Plus Home-Based Telerehabilitation (Behavioral)
结局指标
主要结局
Acceptability of the Intervention
时间窗: At 6 months after baseline (end of intervention period)
Defined by recruitment rate ≥80% and dropout rate \<20%. Unit of Measure: Recruitment rate (%), Dropout rate (%)
Adherence to Sailing and Home-Based Telerehabilitation
时间窗: At 6 months after baseline (end of intervention period)
Defined as the percentage of scheduled sessions completed by each participant. Adherence is considered achieved if ≥75% of scheduled sessions are completed. Unit of Measure: Percentage of participants meeting adherence threshold
Safety of the Intervention
时间窗: At 6 months after baseline (end of intervention period)
Defined as the absence of serious adverse events related to the intervention. Unit of Measure: Number of participants experiencing a serious adverse event
次要结局
- Change in Motor Function Measured by Inertial Measurement Units(Baseline (T0), up to 1-day after intervention (T1-only for experimental arm), and at 3-Month Follow-Up (T2))
- Change in Health-Related Quality of Life Measured by EQ-5D-5L(Baseline (T0), up to 1-day after intervention (T1-only for experimental arm), and at 3-Month Follow-Up (T2))
- Change in Psychosocial Functioning Measured by BESSI-45(Baseline (T0), up to 1-day after intervention (T1-only for experimental arm), and at 3-Month Follow-Up (T2))
- Change in Functional Outcomes Measured by PODCI(Baseline (T0), up to 1-day after intervention (T1-only for experimental arm), and at 3-Month Follow-Up (T2))
- Change in Pain Intensity Measured by Visual Analogue Scale (VAS)(Baseline (T0), up to 1-day after intervention (T1-only for experimental arm), and at 3-Month Follow-Up (T2))
- Change in Motor Function Measured by Inertial Measurement Units(Baseline (T0), up to 1-day after intervention (T1-only for experimental arm), at 3-Month Follow-Up (T2), and at 6-Month Follow-Up (T3))
- Change in Health-Related Quality of Life Measured by EQ-5D-5L(Baseline (T0), up to 1-day after intervention (T1-only for experimental arm), at 3-Month Follow-Up (T2), and at 6-Month Follow-Up (T3))
- Change in Psychosocial Functioning Measured by BESSI-45(Baseline (T0), up to 1-day after intervention (T1-only for experimental arm), at 3-Month Follow-Up (T2), and at 6-Month Follow-Up (T3))
- Change in Functional Outcomes Measured by PODCI(Baseline (T0), up to 1-day after intervention (T1-only for experimental arm), at 3-Month Follow-Up (T2), and at 6-Month Follow-Up (T3))
- Change in Pain Intensity Measured by Visual Analogue Scale (VAS)(Baseline (T0), up to 1-day after intervention (T1-only for experimental arm), at 3-Month Follow-Up (T2), and at 6-Month Follow-Up (T3))
- Change in Kinesiophobia Measured by Tampa Scale of Kinesiophobia (TSK)(Baseline (T0), up to 1-day after intervention (T1-only for experimental arm), at 3-Month Follow-Up (T2), and at 6-Month Follow-Up (T3))
研究者
Luca Sangiorgi
Director of Rare Skeletal Disorders Department
Istituto Ortopedico Rizzoli