Autologous Bone Marrow Aspirate (IV) and Bone Marrow Concentrate (Intranasal) for Parkinson's Disease and Parkinson-Plus Syndromes (SPARC-PD)

NCT07751640 | Active Not Recruiting Phase 1 FDA Drug

• 1 other identifier: BBRC-PDPILOT-01
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

This pilot study evaluated the safety, tolerability, and exploratory epigenetic outcomes of a single same-day autologous bone marrow procedure in adults with Parkinson's disease (PD) or Parkinson-plus syndromes (PPS). Ten participants underwent posterior superior iliac spine bone marrow aspiration under local anesthesia. The unprocessed aspirate (BMA) was filtered and administered intravenously via normal saline infusion on the same day. A portion of the aspirate was centrifuged to produce a bone marrow aspirate concentrate (BMAC), which was atomized intranasally using a mucosal atomization device. Cells were not expanded in culture or genetically modified. The procedure was performed under the Same Surgical Procedure exception (21 CFR 1271.15(b)); an investigational new drug application (IND 31770) was submitted to the FDA Center for Biologics Evaluation and Research (CBER). The primary outcome was safety and tolerability, assessed by treatment-emergent adverse events (TEAEs) graded per CTCAE v5.0 criteria through 12 months post-treatment. Exploratory outcomes included DNA methylation biological age (GrimAge-based composite and organ/system Systems Age clocks) measured from peripheral blood at baseline and approximately 6 months, and characterization of the delivered cell product by automated hematology and multiparameter flow cytometry.

Conditions

PARKINSON DISEASE (Disorder); Atypical Parkinsonism

Keywords

Parkinson Disease; Parkinson_Plus Syndrome; Stem Cells; MSCs; Epigenetics; Metagenomics

Outcome Measures

Primary Outcomes (1)

• Incidence of Treatment-Emergent Adverse Events (TEAEs)

  Incidence, severity, and relationship to study treatment of treatment-emergent adverse events (TEAEs) following a single same-day autologous bone marrow procedure consisting of intravenous bone marrow aspirate (BMA) and intranasal bone marrow aspirate concentrate (BMAC). Adverse events will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0 (Grade 1=mild, Grade 2=moderate, Grade 3=severe, Grade 4=life-threatening, Grade 5=death related to adverse event). Serious adverse events (SAEs) include death, life-threatening events, hospitalization or prolongation of hospitalization, persistent or significant disability, congenital anomaly, or other important medical events. TEAEs will be recorded throughout the study and assessed by the investigator for relationship to study treatment.

  Baseline through Month 12

Secondary Outcomes (40)

• Mean Change from Baseline in GrimAge Biological Age

  Baseline and 6 months

• Bone Marrow Mesenchymal Stromal Cell (MSC) Concentration

  Day 0

• Change in Movement Disorder Society Non-Motor Rating Scale (MDS-NMS) Total Score

  Baseline, Month 3, Month 6, and Month 12

• Change in Parkinson's Disease Questionnaire-39 (PDQ-39) Summary Index

  Baseline, Month 3, Month 6, and Month 12

• Change in Non-Motor Symptoms Questionnaire (NMSQ) Score

  Baseline, Month 3, Month 6, and Month 12

Study Arms (1)

Autologous BMA (IV) and BMAC (Intranasal)

EXPERIMENTAL

Participants received a single same-day autologous bone marrow procedure consisting of: (1) intravenous infusion of filtered, unprocessed bone marrow aspirate (BMA) in normal saline; and (2) intranasal atomization of bone marrow aspirate concentrate (BMAC). Both products were derived from a single posterior superior iliac spine (PSIS) aspiration performed under local anesthesia on the same day. Cells were not expanded in culture or genetically modified.

Biological: Autologous Bone Marrow Aspirate (BMA) and Bone Marrow Aspirate Concentrate (BMAC)

Interventions

Autologous Bone Marrow Aspirate (BMA) and Bone Marrow Aspirate Concentrate (BMAC) BIOLOGICAL

Same-day autologous bone marrow aspirate (BMA) administered intravenously and bone marrow aspirate concentrate (BMAC) administered intranasally via mucosal atomization. Both products derived from a single posterior superior iliac spine aspiration. No ex vivo expansion, genetic modification, or cryopreservation performed.

Autologous BMA (IV) and BMAC (Intranasal)

Eligibility Criteria

• Age: 40 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• General:
• Participants diagnosed with PD or PPS by a licensed medical professional
• Documented diagnosis of PD or PPS ≤ 6 years
• Participants with an anticipated survival of at least 3 years in the investigator's opinion
• Participants who are willing and able to give informed consent
• Participants who can comply with the study protocol over the 6-month duration
• Stable medical profile for 60 days prior to the initial intake screening
• Participants can ambulate at least 25m without assistance
• No known history of heparin-induced thrombocytopenia
• Willingness to comply with study requirements and provide informed consent
• Participants have no history of adverse reactions to heparin, e.g., HIT, local numbing medications, adhesives, or skin sterilizing agents
• Idiopathic Parkinson's disease patients who meet the MDS's Clinical Diagnostic Criteria for Parkinson's disease
• Responsive to levodopa or dopamine agonists defined by 33% improvement in "Off"/"On" symptoms by MDS-UPDRS-III
• A modified Hoehn and Yahr stage of 3 or less
• Neuroimaging findings are consistent with PD and absent of atrophy or other brain pathology inconsistent with PD

You may not qualify if:

• Other non-PD/PPS Parkinsonism (e.g., drug-induced, vascular parkinsonism)
• No strong familial history of PD/PPS not attributable to environmental exposure or any known genetic predisposition to PD/PPS
• Unable to maintain/tolerate supine position with cervical neck extension
• Active systemic infection or local infection near the lumbar pelvis region
• Any bone marrow aspiration from the pelvis within 6 months of initial screening
• Any musculoskeletal-pelvis contraindications to BMA harvest from the PSIS
• Concurrent enrollment in another PD/PPS study or having taken/received another investigational intervention within 6 weeks of initial screening
• Malignancy diagnosed 2 years prior to initial screening
• History of intracranial or nasopharyngeal surgery deemed detrimental to the participant during the trial, including brain surgery/stereotactic procedure for PD/PPS
• History of electroconvulsive therapy
• Chronic Kidney Disorder (CKD) \> Stage II or eGFR \<60 mL/min
• Autoimmune disease, including:
• Rheumatoid Arthritis (RA)
• Systemic Lupus Erythematosus (SLE)
• Any disorders of glucocorticoid excess or diseases requiring systemic steroids or immune-modulating therapies

Sponsors & Collaborators

• Apeiron Research Center: lead

Study Sites (1)

Boulder Biologics Research Center

Boulder, Colorado, 80303, United States

Location

MeSH Terms

Conditions

Parkinson Disease

Interventions

cyclomaltodextrin glucanotransferase

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Study Officials

• Jason W Glowney, MD, MSc

  Apeiron Research Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Masking Details: No masking was applied. Participants, investigators, and outcome assessors were all aware of the intervention.
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Within-subject pre/post design. Each participant serves as their own control, with baseline (Day 0) measurements compared to 6-month and 12-month follow-up assessments. No placebo or sham arm was included in this pilot phase. The single-arm design was selected because the primary objective is safety and feasibility characterization, for which an internal control is appropriate and a concurrent control group is not required.

Study Record Dates

• First Submitted: July 22, 2026
• First Posted: August 7, 2026
• Study Start: May 1, 2025
• Primary Completion (Estimated): September 4, 2026
• Study Completion (Estimated): September 4, 2026
• Last Updated: August 7, 2026

Record last verified: 2026-08

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ANALYTIC CODE
• Time Frame: Beginning 12 months after publication of primary results; available for 5 years
• Access Criteria: Qualified researchers upon reasonable written request; data use agreement required

Locations

US (1)