Phase IIa Randomized Placebo Controlled Trial: Mesenchymal Stem Cells as a Disease-modifying Therapy for Idiopathic Parkinson's Disease
August 11, 2026 updated by: Mya Schiess
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
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).
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Detailed Description
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.
Study Type
Interventional
Enrollment (Actual)
45
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
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Texas
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Houston, Texas, United States, 77030
- The University of Texas Health Science Center at Houston
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
50 years to 79 years (Adult, Older Adult)
Accepts Healthy Volunteers
No
Description
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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Triple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
|---|---|
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Experimental: Mesenchymal Stem Cells and Placebo
2 infusions of 10 X 10^6 MSC/kg and 1 placebo infusion, all doses administered 4 months apart.
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1 dose is 10 X 10^6 MSC/kg
Other Names:
Placebo will be identical to the investigational product but will not contain mesenchymal stem cells (MSCs).
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Experimental: Mesenchymal Stem Cells
3 infusions of 10 X 10^6 MSC/kg administered every 4 months.
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1 dose is 10 X 10^6 MSC/kg
Other Names:
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Placebo Comparator: Placebo
3 placebo doses administered every 4 months.
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Placebo will be identical to the investigational product but will not contain mesenchymal stem cells (MSCs).
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
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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.
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From Baseline to Week 62
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
|
Number of Participants With New-onset Organ Failure
Time Frame: Baseline through week 88
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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.
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Baseline through week 88
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Number of Participants Developing Donor-Specific Anti-HLA Antibodies
Time Frame: Baseline through week 88
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Baseline through week 88
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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.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Number of Participants With Lifetime Suicidal Ideation (C-SSRS)
Time Frame: Baseline
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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?"
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Baseline
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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
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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.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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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
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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.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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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
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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.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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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
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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.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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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
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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.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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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
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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.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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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
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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.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Olfactory Function as Assessed by the University of Pennsylvania Smell Identification Test (UPSIT-40) Score
Time Frame: Baseline, Week 49, Week 88
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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.
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Baseline, Week 49, Week 88
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Cognitive Function as Measured by the Montreal Cognitive Assessment (MoCA)
Time Frame: Baseline, Week 49, Week 88
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The Montreal Cognitive Assessment (MoCA) assesses cognitive function.
The total score ranges from 0 to 30, a higher score indicates better cognitive function.
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Baseline, Week 49, Week 88
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Anxiety as Assessed by the Parkinson Anxiety Scale (PAS) Score
Time Frame: Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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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.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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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
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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.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Number of Participants Reporting Suicidal Ideation Since Last Visit
Time Frame: Week 9, Week 27, Week 49, Week 62, Week 88
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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.
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Week 9, Week 27, Week 49, Week 62, Week 88
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Other Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
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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
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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.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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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
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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.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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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
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Examples of these are homovanillic acid and 5-hydroxytryptamine.
These will be measured in CSF and blood by ELISA.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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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
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Baseline, Week 40, Week 49
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Measurement of Putative Paracrine Mechanism of MSCs as Measured by Alpha-synuclein Oligomers in the Cerebral Spinal Fluid
Time Frame: Baseline,week 49
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Baseline,week 49
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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
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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.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Measurement of Putative Paracrine Mechanism of MSCs Using Neuroimaging
Time Frame: Baseline, week 40,week 88
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Baseline, week 40,week 88
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Collaborators
Investigators
- Principal Investigator: Mya C Schiess, MD, The University of Texas Health Science Center, Houston
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
General Publications
- Dorsey ER, Sherer T, Okun MS, Bloem BR. The Emerging Evidence of the Parkinson Pandemic. J Parkinsons Dis. 2018;8(s1):S3-S8. doi: 10.3233/JPD-181474.
- Braak H, Del Tredici K, Rub U, de Vos RA, Jansen Steur EN, Braak E. Staging of brain pathology related to sporadic Parkinson's disease. Neurobiol Aging. 2003 Mar-Apr;24(2):197-211. doi: 10.1016/s0197-4580(02)00065-9.
- Connolly BS, Lang AE. Pharmacological treatment of Parkinson disease: a review. JAMA. 2014 Apr 23-30;311(16):1670-83. doi: 10.1001/jama.2014.3654.
- Jellinger KA. Basic mechanisms of neurodegeneration: a critical update. J Cell Mol Med. 2010 Mar;14(3):457-87. doi: 10.1111/j.1582-4934.2010.01010.x. Epub 2010 Jan 11.
- Kortekaas R, Leenders KL, van Oostrom JC, Vaalburg W, Bart J, Willemsen AT, Hendrikse NH. Blood-brain barrier dysfunction in parkinsonian midbrain in vivo. Ann Neurol. 2005 Feb;57(2):176-9. doi: 10.1002/ana.20369.
- Gray MT, Woulfe JM. Striatal blood-brain barrier permeability in Parkinson's disease. J Cereb Blood Flow Metab. 2015 May;35(5):747-50. doi: 10.1038/jcbfm.2015.32. Epub 2015 Mar 11.
- Orr CF, Rowe DB, Halliday GM. An inflammatory review of Parkinson's disease. Prog Neurobiol. 2002 Dec;68(5):325-40. doi: 10.1016/s0301-0082(02)00127-2.
- Nagatsu T, Mogi M, Ichinose H, Togari A. Cytokines in Parkinson's disease. J Neural Transm Suppl. 2000;(58):143-51.
- Stypula G, Kunert-Radek J, Stepien H, Zylinska K, Pawlikowski M. Evaluation of interleukins, ACTH, cortisol and prolactin concentrations in the blood of patients with parkinson's disease. Neuroimmunomodulation. 1996 Mar-Jun;3(2-3):131-4. doi: 10.1159/000097237.
- Joyce N, Annett G, Wirthlin L, Olson S, Bauer G, Nolta JA. Mesenchymal stem cells for the treatment of neurodegenerative disease. Regen Med. 2010 Nov;5(6):933-46. doi: 10.2217/rme.10.72.
- Martinez-Lemus JD, Molony DA, Suescun J, Tharp E, Thomas TS, Green C, Onuigbo C, Ritter R 3rd, Schiess MC. Allogeneic bone marrow-derived mesenchymal stem cells in the aging kidney: secondary results of a Parkinson's disease clinical trial. Stem Cell Res Ther. 2025 Sep 24;16(1):493. doi: 10.1186/s13287-025-04577-y.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
November 9, 2020
Primary Completion (Actual)
July 30, 2023
Study Completion (Actual)
July 30, 2023
Study Registration Dates
First Submitted
July 16, 2020
First Submitted That Met QC Criteria
August 6, 2020
First Posted (Actual)
August 10, 2020
Study Record Updates
Last Update Posted (Actual)
September 2, 2026
Last Update Submitted That Met QC Criteria
August 11, 2026
Last Verified
August 1, 2026
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- HSC-MS-20-0150
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.