A Study to Evaluate the Safety of Neural Stem Cells in Patients With Parkinson's Disease

NCT02452723 | Completed Phase 1 DMC

• 1 other identifier: ISC-hpNSC
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

This study will evaluate the safety of an investigational cell transplantation therapy, ISC-hpNSC, in patients with Parkinson's disease. All patients will receive the therapy, which consists of human neural stem cells. Three dose levels will be examined in the study.

Conditions

Parkinson Disease

Outcome Measures

Primary Outcomes (1)

• Incidence of treatment-emergent adverse events (TEAEs), serious TEAEs, related TEAEs, severe TEAEs

  12 month

Secondary Outcomes (2)

• Change in UPDRS score from baseline

  Baseline and 12 months

• Proportion of patients with improvement defined as any reduction in UPDRS motor score

  12 months

Study Arms (1)

ISC-hpNSC

EXPERIMENTAL

Biological: ISC-hpNSC

Interventions

ISC-hpNSC BIOLOGICAL

ISC-hpNSC

Eligibility Criteria

• Age: 30 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Signed informed consent form (ICF) indicating the patient has been informed of the procedures to be followed, the experimental nature of the therapy, alternatives, potential benefits, side-effects, risks, and discomforts
• Patient diagnosed with idiopathic PD of ≤ 13 years duration, as defined by the United Kingdom (UK) Parkinson's Disease Society Brain Bank criteria
• Outpatients (male and female) 30 - 70 years old. Females must be of non-child bearing potential, or with a negative pregnancy test and not breast-feeding
• Patients receiving a stable dose of levodopa for at least 3 months with the expectation that the treatment will remain unchanged throughout the course of the patient's participation in the trial
• Patients receiving an anti-parkinsonian treatment at a stable dose for at least 3 months with the expectation that the treatment will remain unchanged throughout the course of the patient's participation in the trial
• Hoehn and Yahr stage II-IV during "ON" time
• Unified Parkinson's Disease Rating Scale (UPDRS) motor score (Part III) in the "OFF" state ≤ 49
• Positive dopaminergic response of ≥ 33% decrease in UPDRS motor scores between "OFF" and "ON" states at screening, and unequivocal clinical off periods
• Patient is experiencing motor fluctuations with at least two cumulative hours of daily "OFF" -time during the waking period, which is measured on at least two consecutive days
• History of anti-parkinsonian treatment with sufficient doses of levodopa
• Stable, well-controlled concomitant disorders that would not contraindicate general anesthesia or stereotactic neurosurgery
• No abnormalities on baseline brain MRI
• Insufficient control of PD symptoms or intolerable side effects with optimized oral PD therapy
• Montreal Cognitive Assessment (MOCA) score ≥ 26
• Willing to fully comply with all study procedures and requirements of the trial

You may not qualify if:

• Mild cognitive impairment of dementia (MOCA score \< 26)
• The extent or severity of the disease is not measurable
• Severe dyskinesia in the "OFF" or "ON" states (violent dyskinesias, incompatible with any normal motor task)
• Pre-existing medical conditions such as bleeding disorders, septicemia, major cardiovascular, cerebrovascular or psychiatric disease
• Any current or relevant previous history of serious, severe or unstable physical or psychiatric illness or medical disorder that may make the participant unlikely to fully complete the study (depression, anxiety, cognitive impairment or impulse control disorder)
• Clinically significant abnormal hematologic evaluation (blood count, partial thromboplastin time (PTT)), blood chemistry (glucose, blood urea nitrogen (BUN), creatinine, electrolytes), liver function tests (aspartate aminotransferase (AST), alanine aminotransferase (ALT), gamma glutamyl transpeptidase (GGTP), total protein, bilirubin)
• Any active infectious disease of any nature, including clinically active viral infections (seropositive for Human Immunodeficiency Virus (HIV), Hepatitis C Virus (HCV), Hepatitis B Virus (HBV) and Syphilis
• Severe obesity
• Previous intracranial surgery, including deep-brain stimulation
• History of seizures
• Substance abuse (recent history of alcohol abuse or other drugs such as barbiturates, cannabinoids and amphetamines)
• Use of anti-platelet agents or other anti-coagulants
• Signs of any malignant disease
• Any use of immunosuppressive drugs
• Enrollment in other investigational drug trial or has completed any trial within the last 3 months

Sponsors & Collaborators

• Cyto Therapeutics Pty Limited: lead

Study Sites (1)

Dept of Neurology, The Royal Melbourne Hospital

Melbourne, Victoria, 3050, Australia

Location

Related Publications (3)

• Garitaonandia I, Gonzalez R, Sherman G, Semechkin A, Evans A, Kern R. Novel Approach to Stem Cell Therapy in Parkinson's Disease. Stem Cells Dev. 2018 Jul 15;27(14):951-957. doi: 10.1089/scd.2018.0001.

  PMID: 29882481 DERIVED

• Garitaonandia I, Gonzalez R, Christiansen-Weber T, Abramihina T, Poustovoitov M, Noskov A, Sherman G, Semechkin A, Snyder E, Kern R. Neural Stem Cell Tumorigenicity and Biodistribution Assessment for Phase I Clinical Trial in Parkinson's Disease. Sci Rep. 2016 Sep 30;6:34478. doi: 10.1038/srep34478.

  PMID: 27686862 DERIVED

• Gonzalez R, Garitaonandia I, Poustovoitov M, Abramihina T, McEntire C, Culp B, Attwood J, Noskov A, Christiansen-Weber T, Khater M, Mora-Castilla S, To C, Crain A, Sherman G, Semechkin A, Laurent LC, Elsworth JD, Sladek J, Snyder EY, Redmond DE Jr, Kern RA. Neural Stem Cells Derived from Human Parthenogenetic Stem Cells Engraft and Promote Recovery in a Nonhuman Primate Model of Parkinson's Disease. Cell Transplant. 2016 Nov;25(11):1945-1966. doi: 10.3727/096368916X691682.

  PMID: 27213850 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 Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 18, 2015
• First Posted: May 25, 2015
• Study Start: July 1, 2016
• Primary Completion: June 18, 2025
• Study Completion: September 1, 2025
• Last Updated: January 20, 2026

Record last verified: 2026-01

Locations

AU (1)