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

NCT07521384 | Enrolling By Invitation

• 1 other identifier: HHI-MUSE-OBS-2026-001
• Study Type: observational
• Target: 36
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Cerebral Palsy; MS (Multiple Sclerosis); Traumatic Brain Injury; Hypoxic Ischemic Encephalopathy; Neurologic Disorders; Alzheimer Dementia (AD); Parkinson Disease (PD); Stroke; Autism

Keywords

MuSE stem cells; MuSE cell-derived exosomes; Intranasal delivery; Traumatic Brain Injury; Hypoxic-Ischemic Encephalopathy; Neurologic Disorders; Real-world evidence; Stem cell therapy; Exosome therapy; Neuroregeneration; Regenerative medicine; Kurve Therapeutics; ViaNase; HIE; Cerebral Palsy; stroke; Multiple Sclerosis; Alzheimers Dementia; Parkinson Disease; Autism

Outcome Measures

Primary Outcomes (2)

• Change in Serum Tumor Necrosis Factor Alpha (TNF-α) From Baseline to 1 Month

  Serum TNF-α concentration in pg/mL measured at baseline before treatment and at 1 month after treatment; analyzed as absolute and percent change from baseline within and across treatment cohorts.

  Baseline to 1 Month

• Incidence of Adverse Events

  Number of participants with treatment-emergent adverse events after intranasal administration, categorized by seriousness, severity, and relationship to treatment as assessed from available clinical documentation.

  Baseline to 6 Months

Secondary Outcomes (4)

• Change in Serum TNF-α From Baseline to 3 Months

  Baseline to 3 Months

• Caregiver or Participant Global Impression of Change

  Baseline to 3 Months

• Change in Health Related Quality of Life

  Baseline to 3 Months

• Change in Diagnosis Specific Functional Outcome Measures

  Baseline to 3 Months

Other Outcomes (3)

• Change in Gross Motor Function Measure-66 Score in Participants With Cerebral Palsy

  Baseline to 3 Months

• Change in Glasgow Outcome Scale-Extended Score in Participants With Traumatic Brain Injury

  Baseline to 3 Months

• Change in Modified Rankin Scale Score in Participants With Stroke

  Baseline to 3 Months

Study Arms (3)

MuSE Cell Derived Exosomes

Participants in this observational cohort receive MuSE cell derived exosomes administered intranasally via the Kurve Therapeutics ViaNase system as part of routine clinical care outside the study protocol. The study does not assign treatment. Participants are followed prospectively to evaluate safety and tolerability and to assess changes in serum tumor necrosis factor alpha (TNF-α) as a biomarker of systemic inflammation. Exploratory outcomes include changes in neurologic function, symptom burden, and health-related quality of life over time.

MuSE Stem Cells

Participants in this observational cohort receive MuSE stem cells administered intranasally via the Kurve Therapeutics ViaNase system as part of routine clinical care outside the study protocol. The study does not assign treatment. Participants are followed prospectively to evaluate safety and tolerability and to assess changes in serum tumor necrosis factor alpha (TNF-α) as a biomarker of systemic inflammation. Exploratory outcomes include changes in neurologic function, symptom burden, and health-related quality of life over time.

Combination MuSE Exosomes Plus MuSE Stem Cells

Participants in this observational cohort receive combination therapy consisting of MuSE cell derived exosomes and MuSE stem cells administered intranasally via the Kurve Therapeutics ViaNase system as part of routine clinical care outside the study protocol. The study does not assign treatment. Participants are followed prospectively to evaluate safety and tolerability and to assess changes in serum tumor necrosis factor alpha (TNF-α) as a biomarker of systemic inflammation. Exploratory outcomes include changes in neurologic function, symptom burden, and health-related quality of life over time.

Eligibility Criteria

• Age: 4 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

This study includes pediatric and adult participants with neurologic conditions receiving intranasal regenerative therapies administered outside the study protocol. Participants are enrolled into observational cohorts based on treatment type and followed prospectively to evaluate changes in inflammatory biomarkers, including TNF-α, as well as safety and clinical outcomes over time.

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Healing Hope International: lead
• Kurve Technology Inc.: collaborator

Study Sites (1)

Stem Solutions

Monterrey, Nuevo León, Mexico

Location

Related Publications (13)

• Lu Z,Ren S,Wang B,Shao L,Zhang Y,Wu H,Mu X,Wang Z

  BACKGROUND

• 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 BACKGROUND

• 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 BACKGROUND

• 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 BACKGROUND

• 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 BACKGROUND

• 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 BACKGROUND

• 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 BACKGROUND

• 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 BACKGROUND

• Kuroda Y, Wakao S, Kitada M, Murakami T, Nojima M, Dezawa M. Isolation, culture and evaluation of multilineage-differentiating stress-enduring (Muse) cells. Nat Protoc. 2013;8(7):1391-415. doi: 10.1038/nprot.2013.076. Epub 2013 Jun 20.

  PMID: 23787896 BACKGROUND

• Abe T, Aburakawa D, Niizuma K, Iwabuchi N, Kajitani T, Wakao S, Kushida Y, Dezawa M, Borlongan CV, Tominaga T. Intravenously Transplanted Human Multilineage-Differentiating Stress-Enduring Cells Afford Brain Repair in a Mouse Lacunar Stroke Model. Stroke. 2020 Feb;51(2):601-611. doi: 10.1161/STROKEAHA.119.026589. Epub 2019 Dec 12.

  PMID: 31826733 BACKGROUND

• Suzuki T, Sato Y, Kushida Y, Tsuji M, Wakao S, Ueda K, Imai K, Iitani Y, Shimizu S, Hida H, Temma T, Saito S, Iida H, Mizuno M, Takahashi Y, Dezawa M, Borlongan CV, Hayakawa M. Intravenously delivered multilineage-differentiating stress enduring cells dampen excessive glutamate metabolism and microglial activation in experimental perinatal hypoxic ischemic encephalopathy. J Cereb Blood Flow Metab. 2021 Jul;41(7):1707-1720. doi: 10.1177/0271678X20972656. Epub 2020 Nov 22.

  PMID: 33222596 BACKGROUND

• Hori Y, Kitani T, Yanishi K, Suga T, Kogure M, Kusaba T, Kushida Y, Dezawa M, Matoba S. Intravenous administration of human Muse cells recovers blood flow in a mouse model of hindlimb ischemia. Front Cardiovasc Med. 2022 Nov 11;9:981088. doi: 10.3389/fcvm.2022.981088. eCollection 2022.

  PMID: 36440014 BACKGROUND

• Chen X, Yin XY, Zhao YY, Wang CC, Du P, Lu YC, Jin HB, Yang CC, Ying JL. Human Muse cells-derived neural precursor cells as the novel seed cells for the repair of spinal cord injury. Biochem Biophys Res Commun. 2021 Sep 3;568:103-109. doi: 10.1016/j.bbrc.2021.06.070. Epub 2021 Jun 30.

  PMID: 34214874 BACKGROUND

Related Links

• Kurve Therapeutics

MeSH Terms

Conditions

Brain Injuries, Traumatic; Cerebral Palsy; Hypoxia-Ischemia, Brain; Nervous System Diseases; Multiple Sclerosis; Alzheimer Disease; Parkinson Disease; Stroke; Autistic Disorder

Condition Hierarchy (Ancestors)

Brain Injuries; Brain Diseases; Central Nervous System Diseases; Craniocerebral Trauma; Trauma, Nervous System; Wounds and Injuries; Brain Damage, Chronic; Brain Ischemia; Cerebrovascular Disorders; Hypoxia, Brain; Vascular Diseases; Cardiovascular Diseases; Hypoxia; Signs and Symptoms, Respiratory; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases; Dementia; Tauopathies; Neurodegenerative Diseases; Neurocognitive Disorders; Mental Disorders; Parkinsonian Disorders; Basal Ganglia Diseases; Movement Disorders; Synucleinopathies; Autism Spectrum Disorder; Child Development Disorders, Pervasive; Neurodevelopmental Disorders

Study Officials

• Dr. Glen Cronett, PhD/MD

  Kurve Therapeutics

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 3, 2026
• First Posted: April 9, 2026
• Study Start: June 1, 2026
• Primary Completion (Estimated): January 1, 2028
• Study Completion (Estimated): January 30, 2029
• Last Updated: April 9, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: Individual participant data and supporting documents will be made available beginning 6 months following completion of primary data collection and will remain available for a period of 5 years.
• Access Criteria: Access to de identified individual participant data will be provided to qualified researchers upon reasonable request. Requests must include a research proposal outlining the intended use of the data. Access will be granted following review and approval by the study sponsor or designated review body and may require a data use agreement. Only de-identified data necessary for the approved research purpose will be shared. Data will be provided in a secure manner that protects participant confidentiality and complies with applicable ethical and regulatory standards.

Locations

ME (1)