NCT07638813 · Phase 1 · NOT YET RECRUITING
MSC-Exosome Therapy for Frontotemporal Dementia
This trial is testing whether tiny particles called exosomes, derived from umbilical cord stem cells and delivered as a nasal spray, are safe and tolerable in people with frontotemporal dementia. Two doses are compared against a saltwater placebo. It is Phase 1, meaning the main goal is safety, not yet proving the treatment works. The nasal delivery route is designed to help the exosomes reach the brain directly.
You may qualify if
- 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.
You're excluded if
- 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.
The sponsor's own eligibility wording, lightly reformatted. The study team makes the final eligibility decision — worth discussing with your doctor.
Eligibility criteria as of 2026-06-10