Real World Outcomes of Intranasal MuSE Exosomes and Stem Cells in Neurological Regenerative Therapy

Official title

Prospective Observational Multi Cohort Study of Intranasal MuSE Cell Derived Exosomes, MuSE Stem Cells, and Combination Therapy Delivered Via the Kurve Therapeutics ViaNase Device in Patients Receiving International Regenerative Treatments

Overview

This prospective observational study collects real world data on participants receiving regenerative therapies administered internationally and delivered intranasally via the Kurve Therapeutics ViaNase device. The study does not assign treatment. Participants are enrolled after receiving, or electing to receive, therapy as part of routine clinical care outside the study. Participants are observed in one of three cohorts based on the therapy received: MuSE cell derived exosomes, MuSE stem cells, or combination therapy. The objective is to evaluate safety, tolerability, and changes in inflammatory biomarkers and clinical outcomes over time in a real world setting. The study also evaluates changes in inflammatory biomarkers, including serum tumor necrosis factor alpha (TNF-α), to better understand the biological effects of these therapies.

Detailed description

This is a prospective, multi cohort observational study designed to collect real world data on participants receiving regenerative therapies administered outside the study protocol and delivered intranasally via the Kurve Therapeutics ViaNase device.

The study does not assign interventions. Participants are enrolled after treatment decisions have been made by the treating clinician and patient as part of routine clinical care in international settings. Participants are categorized into observational cohorts based on the therapy received.

The study includes the following cohorts:

Cohort 1: Participants receiving 100 billion MuSE cell-derived exosomes administered intranasally via the ViaNase device Cohort 2: Participants receiving 50 million MuSE stem cells administered intranasally via the ViaNase device Cohort 3: Participants receiving combination therapy consisting of 100 billion MuSE cell-derived exosomes and 50 million MuSE stem cells administered intranasally via the ViaNase device

Dosing and treatment regimens are determined by the treating clinician and may vary based on individual patient factors and clinical protocols.

The primary objective is to characterize safety, tolerability, and changes in inflammatory biomarkers in a real world setting. Secondary objectives include evaluating trends in clinical outcomes, functional status, neurologic symptoms, and quality of life over time.

A key objective of this study is to evaluate changes in inflammatory biomarkers, including serum tumor necrosis factor alpha (TNF-α), measured at baseline and follow-up intervals. These biomarkers are used to explore the potential biological effects of intranasally delivered MuSE-derived therapies across treatment cohorts.

Data will be collected prospectively at baseline and predefined follow-up intervals, including approximately 1 month and 3 months post-treatment, using a combination of patient-reported outcomes, caregiver assessments, laboratory measurements, and available clinical documentation. Outcomes may include changes in functional performance, symptom burden, healthcare utilization, and biomarker levels.

This observational design enables the systematic collection of real-world evidence associated with internationally delivered regenerative therapies while maintaining separation between clinical care and research data collection.

Findings from this study may inform future controlled clinical trials and support hypothesis generation for regenerative medicine approaches targeting inflammatory and neurologic conditions.

Primary outcome measures

• Change in Serum Tumor Necrosis Factor Alpha (TNF-α) From Baseline to 1 Month [Time frame: Baseline to 1 Month]
• Incidence of Adverse Events [Time frame: Baseline to 6 Months]

Secondary outcome measures (4)

• Change in Serum TNF-α From Baseline to 3 Months [Time frame: Baseline to 3 Months]
• Caregiver or Participant Global Impression of Change [Time frame: Baseline to 3 Months]
• Change in Health Related Quality of Life [Time frame: Baseline to 3 Months]
• Change in Diagnosis Specific Functional Outcome Measures [Time frame: Baseline to 3 Months]

Eligibility criteria

Inclusion criteria

• Participant has a documented diagnosis of a neurologic condition, including but not limited to: traumatic brain injury, cerebral palsy, hypoxic-ischemic encephalopathy, stroke, or other neurologic disorders
• Participant has received or is scheduled to receive intranasal therapy using MuSE cell-derived exosomes, MuSE stem cells, or combination therapy administered via the Kurve Therapeutics ViaNase system as part of clinical care outside the study protocol
• Baseline serum TNF-α measurement is available or can be obtained prior to treatment
• Participant or legally authorized representative is able to provide informed consent
• Willingness to participate in follow-up assessments, including laboratory and clinical outcome measures
• Stable clinical status for at least 2 weeks prior to baseline assessment

Exclusion criteria

• Active systemic infection at the time of enrollment
• Use of systemic immunosuppressive or biologic anti-inflammatory therapies that may significantly alter TNF-α levels within 30 days prior to baseline measurement
• Inability to obtain baseline or follow-up TNF-α measurements
• Incomplete documentation of treatment type, dose, or administration method
• Any medical condition that, in the opinion of the investigator, would interfere with interpretation of study outcomes

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model

Cohort

Study locations

Mexico · 1 center

• Stem Solutions — Monterrey

Publications

• Lu Z, Ren S, Wang B, Shao L, Zhang Y, Wu H, Mu X, Wang Z. Intranasally administered muse cells attenuate neurodegeneration in Parkinson's disease. J Transl Med. 2025 Dec 24;23(1):1421. doi: 10.1186/s12967-025-07401-6. PMID 41444595
• Perone MJ, Gimeno ML, Fuertes F. Immunomodulatory Properties and Potential Therapeutic Benefits of Muse Cells Administration in Diabetes. Adv Exp Med Biol. 2018;1103:115-129. doi: 10.1007/978-4-431-56847-6_6. PMID 30484226
• Yamada Y, Minatoguchi S, Baba S, Shibata S, Takashima S, Wakao S, Okura H, Dezawa M, Minatoguchi S. Human Muse cells reduce myocardial infarct size and improve cardiac function without causing arrythmias in a swine model of acute myocardial infarction. PLoS One. 2022 Mar 24;17(3):e0265347. doi: 10.1371/journal.pone.0265347. eCollection 2022. PMID 35324926
• Uchida H, Niizuma K, Kushida Y, Wakao S, Tominaga T, Borlongan CV, Dezawa M. Human Muse Cells Reconstruct Neuronal Circuitry in Subacute Lacunar Stroke Model. Stroke. 2017 Feb;48(2):428-435. doi: 10.1161/STROKEAHA.116.014950. Epub 2016 Dec 20. PMID 27999136
• Nagaoki T, Kumagai G, Nitobe Y, Sasaki A, Fujita T, Fukutoku T, Saruta K, Tsukuda M, Asari T, Wada K, Dezawa M, Ishibashi Y. Comparison of the Anti-Inflammatory Effects of Mouse Adipose- and Bone-Marrow-Derived Multilineage-Differentiating Stress-Enduring Cells in Acute-Phase Spinal Cord Injury. J Neurotrauma. 2023 Dec;40(23-24):2596-2609. doi: 10.1089/neu.2022.0470. Epub 2023 May 16. PMID 37051701
• Park YJ, Niizuma K, Mokin M, Dezawa M, Borlongan CV. Cell-Based Therapy for Stroke: Musing With Muse Cells. Stroke. 2020 Sep;51(9):2854-2862. doi: 10.1161/STROKEAHA.120.030618. Epub 2020 Aug 19. PMID 32811374
• Tanaka T, Nishigaki K, Minatoguchi S, Nawa T, Yamada Y, Kanamori H, Mikami A, Ushikoshi H, Kawasaki M, Dezawa M, Minatoguchi S. Mobilized Muse Cells After Acute Myocardial Infarction Predict Cardiac Function and Remodeling in the Chronic Phase. Circ J. 2018 Jan 25;82(2):561-571. doi: 10.1253/circj.CJ-17-0552. Epub 2017 Sep 20. PMID 28931784
• Hori E, Hayakawa Y, Hayashi T, Hori S, Okamoto S, Shibata T, Kubo M, Horie Y, Sasahara M, Kuroda S. Mobilization of Pluripotent Multilineage-Differentiating Stress-Enduring Cells in Ischemic Stroke. J Stroke Cerebrovasc Dis. 2016 Jun;25(6):1473-81. doi: 10.1016/j.jstrokecerebrovasdis.2015.12.033. Epub 2016 Mar 24. PMID 27019988

Identifiers

NCT: NCT07521384 · HHI-MUSE-OBS-2026-001

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗