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

NCT04506073 | Completed Phase 2 DMC FDA Drug

• 1 other identifier: HSC-MS-20-0150
• Study Type: interventional
• Target: 45
• Locations: 1 country
• Sites: 1

Brief Summary

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).

Conditions

Parkinson's Disease

Keywords

Stem Cells; Mesenchymal stem cells; Inflammatory markers

Outcome Measures

Primary Outcomes (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

  Screening

• 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

  Week 7, post infusion #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

  Week 20, post infusion #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

  Week 29, post-infusion #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

  Week 39 follow-up

• 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

  Week 52 follow-up

• 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

  Week 78 follow-up

Secondary Outcomes (19)

• Safety and tolerability as measured by serious adverse reactions.

  Baseline,week 7,week 20,week 29,week 39,week 78

• Safety and tolerability as measured by immunologic responses.

  Baseline,week 7,week 20,week 29,week 39,week 78

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

  Baseline,week 7,week 20,week 29,week 39,week 52,week 78

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

  Baseline,week 7,week 20,week 29,week 39,week 52,week 78

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

  Baseline,week 7,week 20,week 29,week 39,week 52,week 78

Study Arms (3)

Mesenchymal Stem Cells and Placebo

EXPERIMENTAL

2 infusions of 10 X 10\^6 MSC/kg and 1 placebo infusion, all doses administered 4 months apart.

Drug: Mesenchymal Stem Cells; Drug: Placebo

Mesenchymal Stem Cells

EXPERIMENTAL

3 infusions of 10 X 10\^6 MSC/kg administered every 4 months.

Drug: Mesenchymal Stem Cells

Placebo

PLACEBO COMPARATOR

3 placebo doses administered every 4 months.

Drug: Placebo

Interventions

Mesenchymal Stem Cells DRUG

1 dose is 10 X 10\^6 MSC/kg

Also known as: allogeneic mesenchymal stem cells

Mesenchymal Stem Cells; Mesenchymal Stem Cells and Placebo

Placebo DRUG

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

Mesenchymal Stem Cells and Placebo; Placebo

Eligibility Criteria

• Age: 50 Years - 79 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• The University of Texas Health Science Center, Houston: lead
• Michael J. Fox Foundation for Parkinson's Research: collaborator

Study Sites (1)

The University of Texas Health Science Center at Houston

Houston, Texas, 77030, United States

Location

Related Publications (11)

• 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.

  PMID: 30584159 BACKGROUND

• 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.

  PMID: 12498954 BACKGROUND

• 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.

  PMID: 24756517 BACKGROUND

• 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.

  PMID: 20070435 BACKGROUND

• 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.

  PMID: 15668963 BACKGROUND

• 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.

  PMID: 25757748 BACKGROUND

• 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.

  PMID: 12531233 BACKGROUND

• Nagatsu T, Mogi M, Ichinose H, Togari A. Cytokines in Parkinson's disease. J Neural Transm Suppl. 2000;(58):143-51.

  PMID: 11128604 BACKGROUND

• 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.

  PMID: 8945728 BACKGROUND

• 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.

  PMID: 21082892 BACKGROUND

• 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.

  PMID: 40993774 DERIVED

MeSH Terms

Conditions

Parkinson Disease

Condition Hierarchy (Ancestors)

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

Study Officials

• Mya C Schiess, MD

  The University of Texas Health Science Center, Houston

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor and Adriana Blood Chair in Neurology

Study Record Dates

• First Submitted: July 16, 2020
• First Posted: August 10, 2020
• Study Start: November 9, 2020
• Primary Completion: July 30, 2023
• Study Completion: July 30, 2023
• Last Updated: July 26, 2024

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)