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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. Bayesian Mean Estimate of the Proportion of Participants Achieving a ≥5-point Improvement on MDS-UPDRS Part III From Screening to Week 62, Compared Between Each Active MSC Dose Arm and Placebo

    Time frame: From Baseline to Week 62

    The Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III assesses motor symptoms of Parkinson's disease. . A responder was defined as a participant achieving a ≥5-point improvement (decrease of at least 5 points from screening) at Week 62. Reported are the Bayesian mean estimates of the proportion of participants achieving this response. The 95% confidence interval represents the Bayesian 95% credible interval.

Secondary outcomes

  1. Number of Participants With New-onset Organ Failure

    Time frame: Baseline through week 88

    New-onset organ failure defined as a significant acute change in the kidney or liver function, leukocytosis, leukopenia or anemia sustained over 3 months; > 75 % reduction in GFR compared to baseline; altered liver function as defined by ALT >150 U/L and or total bilirubin >1.6 mg/dl; or leukopenia defined as < 4K WBC count or anemia as defined by Hgb < 12 for men and < 11 for women.

  2. Number of Participants Developing Donor-Specific Anti-HLA Antibodies

    Time frame: Baseline through week 88

  3. Motor Function as Measured by the Timed-Up-and-Go (TUG) Scale

    Time frame: Baseline, Week 9, Week 27, Week 40, Week 62, Week 88

    This test uses the time that a person takes to rise from a chair, walk 7 meters, turn around, walk back to the chair, and sit down. Time is recorded in seconds, a longer duration of time indicates a worse outcome.

  4. Number of Participants With Lifetime Suicidal Ideation (C-SSRS)

    Time frame: Baseline

    The Columbia-Suicide Severity Rating Scale (C-SSRS) is an assessment tool that evaluates suicidal ideation and behavior. Reported is the number of participants with a positive response to the lifetime suicidal ideation question, "Have you actually had any thoughts of killing yourself?"

  5. Parkinson's Disease Severity as Assessed by the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Total Score

    Time frame: Baseline, Week 9, Week 27, Week 40, Week 62, Week 88

    The Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) assesses the severity of Parkinson's disease. The total score ranges from 0 to 265, with higher scores indicating greater Parkinson's disease severity and disability.

  6. Motor Symptoms of Parkinson's Disease as Assessed by the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III Motor Examination Score

    Time frame: Baseline, Week 9, Week 27, Week 40, Week 62, Week 88

    The Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III Motor Examination assesses motor symptoms of Parkinson's disease. The total score ranges from 0 to 132, with higher scores indicating greater motor impairment and more severe Parkinson's disease motor symptoms.

  7. Global Measurement of Disability as Measured by the Change in the Screening "Off" Modified Hoehn and Yahr (H&Y)

    Time frame: Baseline, Week 9, Week 27, Week 40, Week 62, Week 88

    The Modified Hoehn and Yahr (H&Y) Scale described the progress of Parkinson's disease. Total score ranges from 0 to 5, with higher scores indicating a worse outcome. The assessment was performed in the "off" medication state, and the score will be reported.

  8. Quality of Life as Measured by the Modified Schwab and England Activities of Daily Living Scale (ADL)

    Time frame: Baseline, Week 9, Week 27, Week 40, Week 62, Week 88

    The Schwab and England Activities of Daily Living (ADL) Scale assesses functional independence in individuals with Parkinson's disease. Scores range from 0% to 100%, with higher percentages indicating greater independence and better functional ability in activities of daily living.

  9. Quality of Life as Measured by the Parkinson's Disease Questionnaire-39 (PDQ-39)

    Time frame: Baseline, Week 9, Week 27, Week 40, Week 62, Week 88

    The Parkinson's Disease Questionnaire-39 (PDQ-39) assesses health-related quality of life in individuals with Parkinson's disease. Total scores range from 0 to 100, with higher scores indicating poorer quality of life.

  10. Quality of Life as Measured by the EuroQol- 5 Dimension (EQ-5D) Index Score

    Time frame: Baseline, Week 9, Week 27, Week 40, Week 62, Week 88

    The EuroQol- 5 Dimension (EQ-5D) assesses quality of life. The total score ranges between 0 to 1, a higher score indicating better quality of life.

  11. Non- Motor Symptoms as Measured by the Non-Motor Symptoms Questionnaire (NMSQ)

    Time frame: Baseline, Week 9, Week 27, Week 40, Week 62, Week 88

    The Non-Motor Symptoms Questionnaire (NMSQ) assesses the presence of non-motor symptoms associated with Parkinson's disease. Total score ranges from 0 to 30, with a higher score indicating a greater number of non-motor symptoms.

  12. Olfactory Function as Assessed by the University of Pennsylvania Smell Identification Test (UPSIT-40) Score

    Time frame: Baseline, Week 49, Week 88

    The University of Pennsylvania Smell Identification Test (UPSIT-40) assesses olfactory function. The total score ranges from 0 to 40 with a higher score indicating better olfactory function.

  13. Cognitive Function as Measured by the Montreal Cognitive Assessment (MoCA)

    Time frame: Baseline, Week 49, Week 88

    The Montreal Cognitive Assessment (MoCA) assesses cognitive function. The total score ranges from 0 to 30, a higher score indicates better cognitive function.

  14. Anxiety as Assessed by the Parkinson Anxiety Scale (PAS) Score

    Time frame: Baseline, Week 9, Week 27, Week 40, Week 62, Week 88

    The Parkinson Anxiety Scale (PAS) assesses anxiety symptoms in individuals with Parkinson's disease. The total score ranges from 0 to 48, with higher scores indicating greater anxiety severity.

  15. Depressive Symptoms as Assessed by the Geriatric Depression Scale-Short Form (GDS-SF) Score

    Time frame: Baseline, Week 9, Week 27, Week 40, Week 62, Week 88

    The Geriatric Depression Scale-Short Form (GDS-SF) assesses depressive symptoms. The total score ranges from 0 to 15, with higher scores indicating greater depressive symptom severity.

  16. Number of Participants Reporting Suicidal Ideation Since Last Visit

    Time frame: Week 9, Week 27, Week 49, Week 62, Week 88

    The Columbia-Suicide Severity Rating Scale (C-SSRS) assesses suicidal ideation and behavior. Reported is the number of participants with a positive response to the suicidal ideation question, "Have you actually had any thoughts of killing yourself?" since the previous study visit.

Other outcomes

  1. Measurement of Putative Paracrine Mechanism of MSCs as Measured by Concentration of Growth Factors

    Time frame: Baseline, Week 9, Week 27, Week 40, Week 62, Week 88

    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.

  2. Measurement of Putative Paracrine Mechanism of MSCs as Measured by Concentration of Chemokines in Patient Blood Sample.

    Time frame: Baseline, Week 9, Week 27, Week 40, Week 62, Week 88

    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.

  3. Measurement of Putative Paracrine Mechanism of MSCs as Measured by Concentration of Neurotransmitters

    Time frame: Baseline, Week 9, Week 27, Week 40, Week 62, Week 88

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

  4. Measurement of Putative Paracrine Mechanism of MSCs as Measured by Alpha-synuclein Oligomers in the Blood (Serum or Plasma)

    Time frame: Baseline, Week 40, Week 49

  5. Measurement of Putative Paracrine Mechanism of MSCs as Measured by Alpha-synuclein Oligomers in the Cerebral Spinal Fluid

    Time frame: Baseline,week 49

  6. Measurement of Putative Paracrine Mechanism of MSCs as Measured by Concentration of Cytokines in Patient Blood Sample.

    Time frame: Baseline, Week 9, Week 27, Week 40, Week 62, Week 88

    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.

  7. Measurement of Putative Paracrine Mechanism of MSCs Using Neuroimaging

    Time frame: Baseline, week 40,week 88

Sponsors and collaborators

Lead sponsor

Mya Schiess

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
Sep 2, 2026

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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