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

N/AEnrolling By InvitationNCT07521384

Healing Hope International36 enrolled

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.

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.

Eligibility

Sex: ALLMin age: 4 YearsMax age: 80 Years

Medical Language ↔ Plain English

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

Outcomes

Primary Outcomes

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.

Time frame: 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.

Time frame: Baseline to 6 Months

Secondary Outcomes

Change in Serum TNF-α From Baseline to 3 Months

Change in serum TNF-α concentration from baseline to 3 months following intranasal therapy, analyzed by treatment cohort.

Time frame: Baseline to 3 Months

Caregiver or Participant Global Impression of Change

Caregiver- or participant-reported overall change in condition compared with baseline using a 7 point global impression of change scale.

Time frame: Baseline to 3 Months

Change in Health Related Quality of Life

Change in health related quality of life from baseline using the Pediatric Quality of Life Inventory (PedsQL) for pediatric participants or a comparable age appropriate quality of life instrument for adult participants.

Time frame: Baseline to 3 Months

Change in Diagnosis Specific Functional Outcome Measures

Change from baseline in diagnosis appropriate validated functional measures, including GMFM-66 for cerebral palsy, GOSE for traumatic brain injury, modified Rankin Scale for stroke, and other predefined condition-specific instruments where applicable.

Time frame: Baseline to 3 Months

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

Overview

Conditions

Eligibility

Locations (1)

Outcomes

Primary Outcomes

Secondary Outcomes