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

NCT02611167 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: HSC-MS-16-0026
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

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).

Conditions

Parkinson's Disease

Keywords

mesenchymal stem cells

Outcome Measures

Primary Outcomes (4)

• Safety of allogeneic MSC therapy in patients with iPD as indicated by the presence of adverse events that are confirmed to be related to the therapy

  Adverse events include renal failure, liver failure, and hemolytic anemia.

  3 weeks after the first infusion

• Safety of allogeneic MSC therapy in patients with iPD as indicated by the presence of adverse events that are confirmed to be related to the therapy

  Adverse events include renal failure, liver failure, and hemolytic anemia.

  12 weeks after the first infusion

• Safety of allogeneic MSC therapy in patients with iPD as indicated by the presence of adverse events that are confirmed to be related to the therapy

  Adverse events include renal failure, liver failure, and hemolytic anemia.

  24 weeks after the first infusion

• Safety of allogeneic MSC therapy in patients with iPD as indicated by the presence of adverse events that are confirmed to be related to the therapy

  Adverse events include renal failure, liver failure, and hemolytic anemia.

  52 weeks after the first infusion

Secondary Outcomes (37)

• Change in motor function as assessed by the Unified Parkinson's Disease Rating Scale (UPDRS) Total score

  baseline, 3 weeks

• Change in motor function as assessed by the Unified Parkinson's Disease Rating Scale (UPDRS) Total score

  baseline, 12 weeks

• Change in motor function as assessed by the Unified Parkinson's Disease Rating Scale (UPDRS) Total score

  baseline, 24 weeks

• Change in motor function as assessed by the Unified Parkinson's Disease Rating Scale (UPDRS) Total score

  baseline, 52 weeks

• Change in motor function as assessed by the UPDRS Motor score

  baseline, 3 weeks

Study Arms (4)

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

EXPERIMENTAL

Allogeneic bone marrow-derived MSCs will be delivered intravenously.

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

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

EXPERIMENTAL

Allogeneic bone marrow-derived MSCs will be delivered intravenously.

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

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

EXPERIMENTAL

Allogeneic bone marrow-derived MSCs will be delivered intravenously.

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

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

EXPERIMENTAL

Allogeneic bone marrow-derived MSCs will be delivered intravenously.

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

Interventions

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

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

Eligibility Criteria

• Age: 45 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• The University of Texas Health Science Center, Houston: lead

Study Sites (1)

The University of Texas Health Science Center at Houston

Houston, Texas, 77030, United States

Location

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 Schiess, MD

  The University of Texas Health Science Center, Houston

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor and Adriana Blood Chair in Neurology

Study Record Dates

• First Submitted: November 11, 2015
• First Posted: November 20, 2015
• Study Start: November 1, 2017
• Primary Completion: September 18, 2019
• Study Completion: September 18, 2019
• Last Updated: November 29, 2023

Record last verified: 2023-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)