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Completed

NCT Number: NCT04506073

Phase IIa Randomized Placebo Controlled Trial: Mesenchymal Stem Cells as a Disease-modifying Therapy for Idiopathic Parkinson's Disease

The purpose of this study is to select the safest and most effective number of repeat doses of allogeneic bone marrow-derived mesenchymal stem cell (MSC) infusions to slow the progression of Parkinson's disease (PD).

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Key information

Conditions

Parkinson's Disease Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Movement Disorders Nervous System Diseases Neurodegenerative Diseases Parkinson Disease Parkinsonian Disorders Synucleinopathies

Age range

50 year–79 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

The University of Texas Health Science Center at Houston

Houston, Texas, 77030, United States

About this study

Single site phase IIa study of allogeneic MSC in a double blind randomized control trial as disease modifying therapy for PD. The design includes three treatment arms with 45 patients.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Diagnosis of Parkinson's disease by the UK brain bank criteria including the presence of 2 cardinal signs of PD plus bradykinesia.
  • Mild microsomia to anosmia.
  • A modified Hoehn and Yahr stage of 3 or less.
  • Date of diagnosis of PD between 3 to 10 years
  • Robust response to dopaminergic therapy.

Exclusion criteria

  • Atypical, vascular, or drug-induced Parkinsonism.
  • An atypical DAT scan or MRI supporting an alternative explanation for PD symptoms.
  • Patient not on levodopa containing medications.
  • Clinical features of psychosis or refractory hallucinations.
  • A Montreal Cognitive Assessment (MoCA) score of less than 25.
  • Uncontrolled seizure disorder.
  • Abnormal Kidney and liver function.
  • Presence of clinically refractory orthostatic hypotension at the screening or baseline visit.
  • Body mass index of greater than or equal to 35.
  • Cardiac disease: History of congestive heart failure, clinically significant bradycardia, presence of 2nd, or 3rd-degree atrioventricular block.
  • Pulmonary disease: COPD with oxygen-requirement at rest or with ambulation; or moderate to severe asthma.
  • Active malignancy or diagnosis of malignancy within 5 years prior to the start of screening
  • Any current suicidal ideation or behaviors.
  • Any diagnosis of autoimmune disease or immunocompromised state
  • History of medium or large size vessel cerebrovascular accidents.
  • History of traumatic brain injury with loss of consciousness and residual neurologic symptoms.
  • Major surgery within the previous 3 months or planned in the ensuing 6 months.
  • History of use of an investigational drug within 90 days prior to the screening visit.
  • History of brain surgery for PD.
  • Substance abuse disorder.
  • Active anticoagulation treatment and/or abnormal INR.

Treatment and study plan

Mesenchymal Stem Cells

Drug

1 dose is 10 X 10^6 MSC/kg

Other names: allogeneic mesenchymal stem cells

Placebo

Drug

Placebo will be identical to the investigational product but will not contain mesenchymal stem cells (MSCs).

Primary outcomes

  1. Safest number of effective doses of MSC as measured by the Part III of the Movement Disorder Society Unified Parkinson's disease Rating Scale (MDS-UPDRS) scale

    Time frame: Screening

  2. Safest number of effective doses of MSC as measured by the Part III of the Movement Disorder Society Unified Parkinson's disease Rating Scale (MDS-UPDRS) scale

    Time frame: Week 7, post infusion #1

  3. Safest number of effective doses of MSC as measured by the Part III of the Movement Disorder Society Unified Parkinson's disease Rating Scale (MDS-UPDRS) scale

    Time frame: Week 20, post infusion #2

  4. Safest number of effective doses of MSC as measured by the Part III of the Movement Disorder Society Unified Parkinson's disease Rating Scale (MDS-UPDRS) scale

    Time frame: Week 29, post-infusion #3

  5. Safest number of effective doses of MSC as measured by the Part III of the Movement Disorder Society Unified Parkinson's disease Rating Scale (MDS-UPDRS) scale

    Time frame: Week 39 follow-up

  6. Safest number of effective doses of MSC as measured by the Part III of the Movement Disorder Society Unified Parkinson's disease Rating Scale (MDS-UPDRS) scale

    Time frame: Week 52 follow-up

  7. Safest number of effective doses of MSC as measured by the Part III of the Movement Disorder Society Unified Parkinson's disease Rating Scale (MDS-UPDRS) scale

    Time frame: Week 78 follow-up

Secondary outcomes

  1. Safety and tolerability as measured by serious adverse reactions.

    Time frame: Baseline,week 7,week 20,week 29,week 39,week 78

  2. Safety and tolerability as measured by immunologic responses.

    Time frame: Baseline,week 7,week 20,week 29,week 39,week 78

    Donor specific antibodies (DSA) in serum of patients will be measured and compared to baseline to determine if there is a development of antibody response to donor HLA (human leukocyte antigen). This will determine if there is a possibility of anti-donor alloimmune response after the first or second infusion of MSC, which might lead to a subsequent antibody-mediated rejection (AMR) with the following infusion. Testing will be done at the these time points to determine if 2nd or 3rd infusions will continue.

  3. Motor function as measured by the Timed-Up-and-Go (TUG) scale

    Time frame: Baseline,week 7,week 20,week 29,week 39,week 52,week 78

    This test uses the time that a person takes to rise from a chair, walk three meters, turn around, walk back to the chair, and sit down. A longer duration of time indicates a worse outcome

  4. Global measurement of disability as measured by the change in the screening "Off" modified Hoehn and Yahr (H&Y)

    Time frame: Baseline,week 7,week 20,week 29,week 39,week 52,week 78

  5. Quality of life as measured by the modified Schwab and England activities of daily living scale (ADL)

    Time frame: Baseline,week 7,week 20,week 29,week 39,week 52,week 78

  6. Quality of life as measured by the Parkinson's Disease Questionnaire 39 (PDQ-39)

    Time frame: Baseline,week 7,week 20,week 29,week 39,week 52,week 78

  7. Quality of life as measured by the EuroQol- 5 Dimension (EQ-5D)

    Time frame: Baseline,week 7,week 20,week 29,week 39,week 52,week 78

  8. Non-motor symtoms as measured by the The University of Pennsylvania Smell Identification Test (UPSIT- 40 odor test booklet).

    Time frame: Baseline,week 29,week 78

  9. Cognitive function as measured by the the change in Montreal Cognitive Assessment (MoCA)

    Time frame: Baseline,week 29,week 78

  10. Behavioral changes as measured by the Columbia Suicide Severity Rating Scale (C-SSRS)

    Time frame: Baseline,week 7,week 20,week 29,week 39,week 52,week 78

    The Columbia-Suicide Severity Rating Scale (C-SSRS) is an assessment tool that evaluates suicidal ideation and behavior. It rates an individual's degree of suicidal ideation on a scale, ranging from "wish to be dead" to "active suicidal ideation with specific plan and intent". Exclusionary criteria views 1 or more positive answers in the last month as not eligible for participation. In follow up, one positive response requires further investigation.

  11. Behavioral changes as measured by the Geriatric Depression Scale-Short Form (GDS-SF)

    Time frame: Baseline,week 7,week 20,week 29,week 39,week 52,week 78

    The Geriatric Depression Scale Short form (GDS-SF) is a 15-item screening tool that is used to identify depression in older adults. Answers indicating depression are in bold and italicized; score one point for each bold one selected. A score of 0 to 5 is normal. A score greater than 5 suggests depression and requires evaluation, whereas a score of 10 or greater would require evaluation for treatment .

  12. Behavioral changes as measured by the Parkinson Anxiety Scale (PAS)

    Time frame: Baseline,week 7,week 20,week 29,week 39,week 52,week 78

  13. Measurement of putative paracrine mechanism of MSCs using neuroimaging

    Time frame: Baseline,week 29,week 78

  14. Measurement of putative paracrine mechanism of MSCs as measured by concentration of cytokines in patient blood sample.

    Time frame: Baseline,week 7,week 20,week 29,week 39,week 78

    Examples of these are IL-1beta, IL-6, TNF-alpha, COX-2 and PGE-2. These are measured by Magnetic Bead Panel - Multiplex Assay or ELISA, allowing for specificity in the blood.

  15. Measurement of putative paracrine mechanism of MSCs as measured by concentration of chemokines in patient blood sample.

    Time frame: Baseline,week 7,week 20,week 29,week 39,week 78

    Examples of these are CCL2 (MCP-1), CCL7 (MCP-3), CCL11 (Eotaxin) CCL2 (MDC), CXCL10 (IP-10), CX3CL1 (Fractalkine). These will be measured by Magnetic Bead Panel - Multiplex Assay or ELISA, allowing for specificity in the blood.

  16. Measurement of putative paracrine mechanism of MSCs as measured by concentration of growth factors

    Time frame: Baseline,week 7,week 20,week 29,week 39,week 78

    Examples of these are Glial Derived Neurotrophic Factor, Brain Derived Neurotrophic Factor and Vascular Epidermal Growth Factor . These will be measured by ELISA specific to blood and CSF.

  17. Measurement of putative paracrine mechanism of MSCs as measured by concentration of neurotransmitters

    Time frame: Baseline,week 7,week 20,week 29,week 39,week 78

    Examples of these are homovanillic acid and 5-hydroxytryptamine. These will be measured in CSF and blood by ELISA.

  18. Measurement of putative paracrine mechanism of MSCs as measured by alpha-synuclein oligomers in the blood (serum or plasma)

    Time frame: Basleline,week 29,week 39,week 78

  19. Measurement of putative paracrine mechanism of MSCs as measured by alpha-synuclein oligomers in the cerebral spinal fluid

    Time frame: Baseline,week 39

Sponsors and collaborators

Lead sponsor

The University of Texas Health Science Center, Houston

Other

Collaborators

  • Michael J. Fox Foundation for Parkinson's Research

Registry information

Official study title

A Randomized, Double-blind, Placebo-controlled Trial of Allogeneic Bone Marrow-derived Mesenchymal Stem Cells as a Disease-modifying Therapy for Idiopathic Parkinson's Disease

Important dates

Study start
2020
Primary completion
2023
Study completion
2023
First posted
Aug 10, 2020
Registry last updated
Jul 26, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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