Clinical Research Directory

Browse clinical research sites, groups, and studies.

Sites Groups Studies

Back to Studies

ACTIVE NOT RECRUITING

NCT05635409

PHASE1

A Trial to Determine the Safety and Tolerability of Transplanted Stem Cell Derived Dopamine Neurons to the Brains of Individuals With Parkinson's Disease

Sponsor: Region Skane

View on ClinicalTrials.gov

Summary

Parkinson's disease (PD) occurs when an area of the brain begins to lose nerve cells that produce a chemical called dopamine. Dopamine is an important chemical, and one of its functions is that it helps to regulate body movement. The loss of these nerve cells leads to a reduction of dopamine in the brain. Medications used to treat PD temporarily replace this lost dopamine, but they do not repair the underlying disease. One of the most promising PD therapies to date has been the transplantation of dopamine producing cells into the brain. Unlike current treatments, these therapies may be able to repair the damage caused in PD. In this trial, the investigators will transplant a new stem cell therapy, called the STEM-PD product, into the area of the brain affected in people with PD. These stem cells can develop into many different cell types, including dopamine-producing nerve cells. The investigators will transplant the stem cells using a device that has been previously used for similar transplants in Lund. This is the first time that the STEM-PD product will be given to humans. The trial aims to assess whether the STEM-PD product is safe to use in people with PD. The investigators will also be looking for preliminary signs of efficacy. The trial will recruit participants with PD from the UK and Sweden. Eight participants will undergo the STEM-PD product transplant. Participants will receive a single dose of the STEM-PD product. Participants will attend for 25 visits primarily at their local recruiting hospital. For participants from the UK, some of the imaging will be performed at Invicro (London), and the surgery (including some visits before and after) and some imaging will be performed in Lund. All participants will be followed up for 36 months following surgery

Official title: STEM-PD Trial: A Multicentre, Single Arm, First in Human, Dose-escalation Trial, Investigating the Safety and Tolerability of Intraputamenal Transplantation of Human Embryonic Stem Cell Derived Dopaminergic Cells for Parkinson's Disease (STEM-PD Product)

Key Details

Gender

All

Age Range

50 Years - 75 Years

Study Type

INTERVENTIONAL

Enrollment

Start Date

2022-11-30

Completion Date

2027-11

Last Updated

2025-12-17

Healthy Volunteers

Conditions

Parkinson Disease

Interventions

BIOLOGICAL

STEM-PD

STEM-PD is a cryopreserved cell product, consisting of ventral midbrain dopaminergic progenitor cells derived from the clinical-grade hESC line RC17. STEM-PD will be administered using a non-CE marked class III neurosurgical medical device, the Rehncrona-Legradi device, bilaterally in one surgical session to the putamen.

Inclusion Criteria: * Have given written informed consent to participate in the trial * Diagnosed with PD as defined using Queens Square Brain Bank criteria * Moderate disease as defined by having Hoehn and Yahr stage 2-3 in OFF state * Disease duration \> 10 years * Male or female, aged between 50 and 75 years (inclusive) * Have a significant response to dopamine therapies as judged by the PI or other delegated clinician * Have symptoms that are not appropriately controlled by existing oral anti-PD medications, as judged by the PI or other delegated clinician * Ability to travel to Lund for surgery * Followed up for at least 12 months prior to inclusion in this trial in the TransEUro observational study * Be fluent in English/Swedish to enable completion of questionnaires as assessed by the PI or other delegated clinician at Cambridge/Lund, respectively * Be approved by the TMG clinical sub-group for trial participation Exclusion Criteria: * Tremor dominant disease, as assessed by the PI or other delegated clinician * Significant drug induced dyskinesias as defined by a score of \> 2 in the Abnormal Involuntary Movement Scale (AIMS) dyskinesias rating scale, in any body part in the ON state * Ongoing major medical or psychiatric disorders, including depression (MADRS \> 20) and psychosis, that make participation unsuitable, as judged by the PI or other delegated clinician * Any contraindication to neurosurgery * Unable to be imaged using MRI * Extensive ventral striatal loss or normal findings on F-DOPA PET at screening * Significant cognitive impairment indicative of an incipient dementia/established dementia or values consistent with MoCA score of ≤ 24 * Unable to perform normal copying of interlocking pentagons and/or a semantic fluency score for naming animals of less than 20 over 90 seconds * Other concomitant treatment with neuroleptics (including atypical neuroleptics) and/or cholinesterase inhibitors * Previous neurosurgery to the brain, or cell or organ transplantation, or recipient of repeated blood transfusions * Any contraindication to immunosuppressive therapy, prophylactic antibiotics, and/or osteoporosis prophylaxis (refer to STEM-PD Trial Immunosuppressant Manual) * High levels of pre-formed specific anti-HLA antibodies to the cell product * Severely reduced TPMT activity (less than half of the lower normal TPMT activity level) * History of documented severe/significant allergy requiring treatment * Female who is pregnant or breastfeeding * Received an investigational drug (including investigational vaccines) or used an invasive investigational medical device within 4 weeks of the screening visit, or is currently enrolled in an interventional investigational trial * Female of childbearing potential or male unwilling to follow contraception requirements (see protocol section 12.15) * Any other condition which, in the opinion of the investigator, makes the patient inappropriate for entry into the trial

Locations (2)

Region Skåne - Skåne University Hospital

Lund, Sweden

Cambridge University Hospitals NHS Foundation Trust

Cambridge, Cambridgeshire, United Kingdom