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

NCT ID: NCT04506073

Last Updated: 2024-07-26

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

45 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-11-09

Study Completion Date

2023-07-30

Brief Summary

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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).

Detailed Description

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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.

Conditions

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Parkinson's Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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Mesenchymal Stem Cells and Placebo

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

Group Type EXPERIMENTAL

Mesenchymal Stem Cells

Intervention Type DRUG

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

Placebo

Intervention Type DRUG

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

Mesenchymal Stem Cells

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

Group Type EXPERIMENTAL

Mesenchymal Stem Cells

Intervention Type DRUG

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

Placebo

3 placebo doses administered every 4 months.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

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

Interventions

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Mesenchymal Stem Cells

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

Intervention Type DRUG

Placebo

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

Intervention Type DRUG

Other Intervention Names

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allogeneic mesenchymal stem cells

Eligibility Criteria

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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.
Minimum Eligible Age

50 Years

Maximum Eligible Age

79 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Michael J. Fox Foundation for Parkinson's Research

OTHER

Sponsor Role collaborator

The University of Texas Health Science Center, Houston

OTHER

Sponsor Role lead

Responsible Party

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Mya Schiess

Professor and Adriana Blood Chair in Neurology

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Mya C Schiess, MD

Role: PRINCIPAL_INVESTIGATOR

The University of Texas Health Science Center, Houston

Locations

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The University of Texas Health Science Center at Houston

Houston, Texas, United States

Site Status

Countries

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United States

References

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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.

Reference Type BACKGROUND
PMID: 30584159 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 12498954 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 24756517 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 20070435 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 15668963 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 25757748 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 12531233 (View on PubMed)

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

Reference Type BACKGROUND
PMID: 11128604 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 8945728 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 21082892 (View on PubMed)

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.

Reference Type DERIVED
PMID: 40993774 (View on PubMed)

Other Identifiers

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HSC-MS-20-0150

Identifier Type: -

Identifier Source: org_study_id

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