Allogeneic Bone Marrow-Derived Mesenchymal Stem Cell Therapy for Idiopathic Parkinson's Disease

NCT ID: NCT02611167

Last Updated: 2023-11-29

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

PHASE1

Total Enrollment

20 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-11-01

Study Completion Date

2019-09-18

Brief Summary

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The purpose of this study is to assess the safety, feasibility, and efficacy of intravenous allogeneic bone marrow-derived mesenchymal stem cell (MSC) therapy for idiopathic Parkinson's disease (iPD).

Detailed Description

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Allogeneic bone marrow-derived mesenchymal stem cells (MSCs) will be delivered intravenously at one of four doses: 1 x 10 6 MSC/kg, 3 x 10 6 MSC/kg, 6 x 10 6 MSC/kg, or 10 x 10 6 MSC/kg of body weight to a population of patients with idiopathic Parkinson's disease (iPD). The infusion will be at 1 week after the baseline visit, following two screening visits. Patients will be followed until 52 weeks after the infusion visit. The safety of the therapy, as well as the impact of the therapy on the rate of Parkinson's disease (PD) progression, will be assessed.

Conditions

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Parkinson's Disease

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Bone marrow-derived MSC transplantation (1 x 10 6 MSC/kg)

Allogeneic bone marrow-derived MSCs will be delivered intravenously.

Group Type EXPERIMENTAL

Allogeneic bone marrow-derived MSCs (1 x 10 6 MSC/kg)

Intervention Type BIOLOGICAL

Bone marrow-derived allogeneic MSCs will be delivered intravenously in escalating doses of 1 x 10 6 MSC/kg, 3 x 10 6 MSC/kg, 6 x 10 6 MSC/kg, or 10 x 10 6 MSC/kg of body weight to a population of patients with idiopathic Parkinson's disease (iPD).

Bone marrow-derived MSC transplantation (3 x 10 6 MSC/kg)

Allogeneic bone marrow-derived MSCs will be delivered intravenously.

Group Type EXPERIMENTAL

Allogeneic bone marrow-derived MSCs (3 x 10 6 MSC/kg)

Intervention Type BIOLOGICAL

Bone marrow-derived allogeneic MSCs will be delivered intravenously in escalating doses of 1 x 10 6 MSC/kg, 3 x 10 6 MSC/kg, 6 x 10 6 MSC/kg, or 10 x 10 6 MSC/kg of body weight to a population of patients with idiopathic Parkinson's disease (iPD).

Bone marrow-derived MSC transplantation (6 x 10 6 MSC/kg)

Allogeneic bone marrow-derived MSCs will be delivered intravenously.

Group Type EXPERIMENTAL

Allogeneic bone marrow-derived MSCs (6 x 10 6 MSC/kg)

Intervention Type BIOLOGICAL

Bone marrow-derived allogeneic MSCs will be delivered intravenously in escalating doses of 1 x 10 6 MSC/kg, 3 x 10 6 MSC/kg, 6 x 10 6 MSC/kg, or 10 x 10 6 MSC/kg of body weight to a population of patients with idiopathic Parkinson's disease (iPD).

Bone marrow-derived MSC transplantation (10 x 10 6 MSC/kg)

Allogeneic bone marrow-derived MSCs will be delivered intravenously.

Group Type EXPERIMENTAL

Allogeneic bone marrow-derived MSCs (10 x 10 6 MSC/kg)

Intervention Type BIOLOGICAL

Bone marrow-derived allogeneic MSCs will be delivered intravenously in escalating doses of 1 x 10 6 MSC/kg, 3 x 10 6 MSC/kg, 6 x 10 6 MSC/kg, or 10 x 10 6 MSC/kg of body weight to a population of patients with idiopathic Parkinson's disease (iPD).

Interventions

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Allogeneic bone marrow-derived MSCs (1 x 10 6 MSC/kg)

Bone marrow-derived allogeneic MSCs will be delivered intravenously in escalating doses of 1 x 10 6 MSC/kg, 3 x 10 6 MSC/kg, 6 x 10 6 MSC/kg, or 10 x 10 6 MSC/kg of body weight to a population of patients with idiopathic Parkinson's disease (iPD).

Intervention Type BIOLOGICAL

Allogeneic bone marrow-derived MSCs (3 x 10 6 MSC/kg)

Bone marrow-derived allogeneic MSCs will be delivered intravenously in escalating doses of 1 x 10 6 MSC/kg, 3 x 10 6 MSC/kg, 6 x 10 6 MSC/kg, or 10 x 10 6 MSC/kg of body weight to a population of patients with idiopathic Parkinson's disease (iPD).

Intervention Type BIOLOGICAL

Allogeneic bone marrow-derived MSCs (6 x 10 6 MSC/kg)

Bone marrow-derived allogeneic MSCs will be delivered intravenously in escalating doses of 1 x 10 6 MSC/kg, 3 x 10 6 MSC/kg, 6 x 10 6 MSC/kg, or 10 x 10 6 MSC/kg of body weight to a population of patients with idiopathic Parkinson's disease (iPD).

Intervention Type BIOLOGICAL

Allogeneic bone marrow-derived MSCs (10 x 10 6 MSC/kg)

Bone marrow-derived allogeneic MSCs will be delivered intravenously in escalating doses of 1 x 10 6 MSC/kg, 3 x 10 6 MSC/kg, 6 x 10 6 MSC/kg, or 10 x 10 6 MSC/kg of body weight to a population of patients with idiopathic Parkinson's disease (iPD).

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

* Men and women between the ages of 45 and 70. The 45-year-old age cutoff ensures that we do not enroll juvenile PD patients.
* Diagnosis of Parkinson disease by the United Kingdom (UK) brain bank criteria including the presence of 2 cardinal signs of PD plus bradykinesia. Diagnosis will be confirmed by the PI or other specialists in Movement Disorders and based on medical history, physical and neurological exams. Patients should have an asymmetric onset, unilateral symptoms and a negative pull test. (See Appendix A)
* Moderate to severe microsmia (UPSIT \<29).
* A modified Hoehn and Yahr stage of 3 or less in the levodopa OFF state. (See Appendix B)
* Diagnosis of PD between 4 to 7 years.
* Robust response to dopaminergic therapy (defined as greater than 33% reduction in symptoms (on the Unified Parkinson's Disease Rating Scale; UPDRS) when measured in the ON medicine state compared to OFF state.
* If subject is taking any central nervous system acting medications (e.g., benzodiazepines, antidepressants, hypnotics) regimen must be optimized and stable for 90 days prior to the screening visit.
* A stable Parkinson's disease symptomatic therapy for at least 90 days prior to screening and not projected to require additional Parkinson's disease symptomatic therapy for at least one year from the baseline visit.
* Women of childbearing potential will be required to use a reliable form of contraception from 30 days prior to baseline visit until 6 months after the final dose of the study drug.

Exclusion Criteria

* Atypical or drug-induced Parkinsonism.
* A UPDRS rest tremor score of 3 or greater for any limb.
* A Montreal Cognitive Assessment (MoCA) score of less than 25. (See Appendix C)
* Clinical features of psychosis or refractory hallucinations.
* Uncontrolled seizure disorder, defined as a seizure within the last 6 months.
* Developmental delay.
* Chronic kidney disease defined as glomerular filtration rate (GFR) \< 50 mL/min/m2.
* Hepatic disease or altered liver function as defined by alanine transaminase (ALT) \>150 U/L and or T. Bilirubin \>1.6 mg/dl at admission.
* Presence of clinically refractory orthostatic hypotension at the screening or baseline visit defined as greater than or equal to 20 mmHg change in systolic BP and greater than or equal to 10 mmHg change in diastolic BP from sitting position to standing after 2 minutes that does not respond to medical treatment or baseline sitting BP less than 90/60.
* History of congestive heart failure, clinically significant bradycardia, presence of 2nd or 3rd degree atrioventricular block.
* Pulmonary disease: chronic obstructive 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 (Cancer free for at least 5 years is permitted; skin cancers, except for melanoma, are permitted).
* Any diagnosis of autoimmune disease or immunocompromised state, including chemotherapy administration within last 3 years or current immunosuppression as defined by white blood cell (WBC) \<3 x 103 cells/ml.
* History of strokes or traumatic brain injury.
* Major surgery within the previous 3 months or planned in the ensuing 6 months.
* Clinically significant abnormalities in the Screening Visit laboratory studies.
* History of use of an investigational drug within 30 days prior to the screening visit.
* History of brain surgery for PD.
* Unable to return for follow-up visits for clinical evaluation, laboratory studies, or imaging evaluation.
* Substance abuse disorder.
* Active anticoagulation treatment.
* Any other condition that the investigator feels would pose a significant hazard to the patient if enrolled or complicate the study assessments.
Minimum Eligible Age

45 Years

Maximum Eligible Age

70 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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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 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

Other Identifiers

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HSC-MS-16-0026

Identifier Type: -

Identifier Source: org_study_id