An Observational Study on the Safety and Outcomes of MUSE Stem Cell Therapy in Participants With Traumatic Brain Injury Receiving International Clinical Treatment
概览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- Healing Hope International
- 入组人数
- 10
- 试验地点
- 2
- 主要终点
- Change in global functional outcome (Glasgow Outcome Scale-Extended)
概览
简要总结
This prospective observational study evaluates the safety profile and patient-reported outcomes associated with MUSE (Multilineage-differentiating Stress-Enduring) stem cell therapy in individuals aged 6 to 75 with chronic traumatic brain injury (TBI). Participants independently elect to receive MUSE cell treatment through international clinical programs, and this study aims to capture real-world evidence on the potential therapeutic effects and risks of this emerging regenerative approach.
The study does not administer any intervention. Instead, it follows participants who have received, or plan to receive, MUSE cell infusions outside the United States. Over a 12-month follow-up period, data will be collected on neurological functioning, quality of life, activities of daily living, and any reported adverse events or complications. Information will be gathered through remote interviews, structured digital surveys, and review of medical documentation when available.
This research is sponsored by Healing Hope International and is intended to contribute to the ethical and responsible advancement of novel cell-based therapies by generating real-world evidence that may guide future clinical trial development and inform patient care practices.
详细描述
This observational study is designed to systematically evaluate the safety, tolerability, and potential neurological outcomes associated with MUSE (Multilineage-differentiating Stress-Enduring) stem cell therapy in individuals with chronic traumatic brain injury (TBI) who independently obtain treatment through international clinical programs.
MUSE cells are a distinct subpopulation of mesenchymal stem cells characterized by stress resilience, spontaneous triploblastic differentiation, and the capacity to home to sites of tissue injury. Preclinical research suggests that MUSE cells may contribute to neuroregeneration through differentiation into neural and glial lineages, modulation of inflammatory pathways, and repair of damaged central nervous system structures. While early findings are promising, clinical evidence remains limited, highlighting the need for structured real-world data.
This study does not randomize participants or administer any treatment. Instead, it functions as a registry-style, real-world evidence platform following individuals who have elected to receive MUSE cell therapy at licensed facilities outside the United States. Collected data will include baseline demographics, TBI history, details of the stem cell procedure (such as cell source, administered dose, and route of delivery), and longitudinal follow-up over 12 months.
Primary domains of interest include:
Neurological function, assessed with validated clinical instruments (e.g., Glasgow Outcome Scale-Extended).
Quality of life, measured using standardized patient-reported outcome tools (e.g., PROMIS-29, EQ-5D).
Functional abilities and activities of daily living, to assess practical day-to-day impact.
Safety and tolerability, including documentation of adverse events, patient-reported symptoms, and any medical complications.
Data will be obtained through remote telemedicine visits, structured digital surveys, and review of available medical records. This study does not collect biospecimens and does not involve the administration of any investigational product.
The overarching objective is to generate high-quality real-world evidence regarding the use of MUSE stem cells in chronic TBI, supporting the scientific foundation needed for future controlled clinical trials and potential compassionate use pathways. The study is conducted by Healing Hope International, a nonprofit organization dedicated to advancing ethical access and research in regenerative medicine.
研究设计
- 研究类型
- Observational
- 观察模型
- Ecologic Or Community
- 时间视角
- Prospective
入排标准
- 年龄范围
- 6 Years 至 75 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Individuals aged 6 to 75 years at the time of enrollment.
- •Documented history of traumatic brain injury (TBI) occurring at least 6 months prior to enrollment (chronic phase).
- •Participant has independently elected to receive MUSE (Multilineage-differentiating Stress-Enduring) stem cell therapy at a licensed treatment facility outside the United States.
- •Ability of the participant or legally authorized representative to provide informed consent for participation in an observational study.
- •Willingness to participate in remote or in-person follow-up assessments for up to 12 months.
- •Ability to provide medical records, laboratory reports, or treatment documentation when available.
排除标准
- •Individuals who have not received, and do not plan to receive, MUSE cell therapy as part of their independent medical care.
- •Inability or unwillingness to complete study assessments (e.g., severe communication barriers not manageable through caregiver assistance or technology).
- •Any condition that, in the opinion of the study team, would make participation in an observational registry unsafe or infeasible (e.g., inability to provide minimal required data).
- •Planned participation in another research study that would prevent collection of observational outcomes for this registry.
- •Individuals currently incarcerated or in institutional settings where research participation is restricted.
研究组 & 干预措施
MUSE Therapy Recipients With Chronic TBI
This cohort includes individuals aged 6 to 75 with chronic traumatic brain injury (TBI) who independently elect to receive MUSE (Multilineage-differentiating Stress-Enduring) stem cell therapy at licensed international treatment centers outside the United States. The study does not provide or administer the therapy; instead, it observes and documents real-world outcomes following treatment. Participants may receive MUSE cell infusions using varying doses, cell sources, and routes of administration, depending on the clinical site they choose. The study will collect longitudinal data on neurological function, quality of life, functional independence, and any adverse events over a 12-month follow-up period.
干预措施: MUSE Stem Cell Therapy (Biological)
结局指标
主要结局
Change in global functional outcome (Glasgow Outcome Scale-Extended)
时间窗: Baseline (pre-MUSE cell treatment) and 12 months after first MUSE cell treatment
The Glasgow Outcome Scale-Extended (GOSE) is an 8-point ordinal scale (1 = Death, 8 = Upper Good Recovery) used to evaluate global functional status following traumatic brain injury. Higher scores reflect better functional outcome. This observational study will measure the change in GOSE score from baseline to 12 months after the participant's first independently obtained MUSE cell therapy session. Assessments will be conducted through structured interviews via in-person visits or telehealth, following standardized GOSE scoring procedures.
次要结局
- Change in post-concussive symptoms (Rivermead Post-Concussion Symptoms Questionnaire)(Baseline; 3, 6, and 12 months after first MUSE cell treatment)
- Change in cognitive function (Montreal Cognitive Assessment)(Baseline; 6 and 12 months after first MUSE cell treatment)
- Change in Health-Related Quality of Life Measured by EQ-5D-5L Index Score(Baseline; 6 and 12 months after first MUSE cell treatment)
- Change in functional independence (Functional Independence Measure)(Baseline; 6 and 12 months after first MUSE cell treatment)
- Change in mood and anxiety symptoms (HADS)(Baseline; 6 and 12 months after first MUSE cell treatment)
- Change in Interleukin-6 (IL-6) Serum Concentration(Baseline; 3 and 6 months after first MUSE cell treatment)
- Incidence of treatment-emergent serious adverse events(From first MUSE cell treatment through 12 months of follow-up)
- Change in Self-Rated Health Measured by Visual Analogue Scale (EQ-VAS)(Baseline, 6 months, and 12 months after first MUSE cell treatment)
- Change in Tumor Necrosis Factor Alpha (TNF-α) Serum Concentration(Baseline, 3 months, and 6 months after first MUSE cell treatment)
- Change in C-Reactive Protein (CRP) Serum Concentration(Time Frame: Baseline, 3 months, and 6 months after first MUSE cell treatment)