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

The University of Texas System (UT)

Status and phase: Completed; Phase 2

Conditions: Parkinson's Disease

Treatments: Drug: Mesenchymal Stem Cells; Drug: Placebo

Study type: Interventional

Funder types: Other

Identifiers

NCT04506073

HSC-MS-20-0150

Details and patient eligibility

About

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).

Full 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.

Enrollment: 45 patients

Sex: All

Ages: 50 to 79 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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45 participants in 3 patient groups, including a placebo group

Mesenchymal Stem Cells and Placebo

Experimental group

Description:

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

Treatment:

Drug: Placebo

Drug: Mesenchymal Stem Cells

Mesenchymal Stem Cells

Experimental group

Description:

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

Treatment:

Drug: Mesenchymal Stem Cells

Placebo

Placebo Comparator group

Description:

3 placebo doses administered every 4 months.

Treatment:

Drug: Placebo