A Randomized, Double-Blind, Placebo-Controlled Trial to Evaluate the Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell-Derived Exosomes Via Intranasal Administration in Patients With Frontotemporal Dementia
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 入组人数
- 33
研究概览
简要总结
This study is testing a new treatment for Frontotemporal Dementia (FTD) - a progressive brain disease that affects personality, behavior, and language. Currently, there is no cure for FTD and no approved medication that can slow down or stop the disease. Existing treatments only help manage some symptoms temporarily.
The investigational treatment in this study is made from exosomes - tiny particles naturally released by umbilical cord stem cells. Exosomes act like "message carriers" between cells. Researchers believe they may help protect brain cells, reduce harmful protein buildup, and improve brain function.
The exosomes will be given as a nasal spray (sprayed into the nose). This method may allow the treatment to reach the brain directly without needing to pass through the blood-brain barrier (a natural protective layer that often blocks medications from entering the brain).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Outcomes Assessor)
入排标准
- 年龄范围
- 30 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of probable frontotemporal dementia (FTD), including behavioral variant (bvFTD), semantic variant primary progressive aphasia (svPPA), or non-fluent variant primary progressive aphasia (nfvPPA), according to established diagnostic criteria, with supportive neuroimaging evidence showing frontal and/or temporal lobe atrophy score of 2 or higher on brain CT or MRI.
- •Age between 30 and 80 years (inclusive) at screening.
- •Study partner who agrees to participate in the study, provides at least 3 hours of daily care or visits, and can manage all study medication.
- •Frontotemporal Lobar Degeneration Clinical Dementia Rating (FTLD-CDR) score of 0-2 and Mini-Mental State Examination (MMSE) score greater than 10 at screening.
- •Stable use of cognition- or behavior-related medications (e.g., cholinesterase inhibitors, memantine, antidepressants, antipsychotics, mood stabilizers, benzodiazepines) for at least 30 days before baseline.
- •Signed informed consent form.
排除标准
- •History of stroke or other neurological or psychiatric disorder (other than FTD) that is considered the primary cause of behavioral symptoms.
- •Pregnancy or breastfeeding, or plan to become pregnant during the study period.
- •Use of any investigational or experimental drug or device within 60 days or 5 half-lives (whichever is longer) prior to screening.
- •Presence of speech or language impairment that severely affects the implementation of neuropsychological assessments or safety evaluations per the study protocol.
- •History of cancer, unless: (a) considered cured; (b) not actively receiving anti-cancer therapy or radiation and the investigator judges that treatment is unlikely to be needed in the next 5 years; or (c) for prostate cancer or basal cell carcinoma, no significant progression in the past 2 years.
- •Any clinically significant hematologic, endocrine, cardiovascular, renal, hepatic, gastrointestinal, or neurological disease that would interfere with study participation. Participants may be included if the condition has been stable for at least one year and the investigator judges that it does not affect participation.
- •Known hypersensitivity to the study drug or any of its excipients.
研究组 & 干预措施
Placebo Comparator
Participants randomized to the placebo group will receive intranasal spray of normal saline (0.9% sodium chloride), 1 mL per administration, twice weekly for 24 consecutive weeks. The placebo is identical in appearance, packaging, and administration procedure to the active investigational product to maintain blinding.
干预措施: Placebo Comparator (0.9% NaCl) (Other)
Low-Dose Exosome Group
Participants randomized to the low-dose group will receive intranasal spray of human umbilical cord mesenchymal stem cell-derived exosomes (MSC-Exos), 1 mL per administration containing 24 × 10⁹ particles, twice weekly for 24 consecutive weeks.
干预措施: Umbilical Cord Mesenchymal Stem Cell-Derived Exosomes （Low-Dose） (Biological)
High-Dose MSC-Exosome
Participants randomized to the high-dose group will receive intranasal spray of human umbilical cord mesenchymal stem cell-derived exosomes (MSC-Exos), 1 mL per administration containing 36 × 10⁹ particles, twice weekly for 24 consecutive weeks. Enrollment in this arm will only begin after the first 3 participants in the low-dose arm complete the 4-week safety lead-in without dose-limiting toxicity or serious adverse events.
干预措施: Umbilical Cord Mesenchymal Stem Cell-Derived Exosomes （High-Dose） (Biological)
研究者
Shengdi Chen
Chief Physician
Ruijin Hospital