Parkinson’s disease MedDRA version: 21.1 Level: LLT Classification code 10013113 Term: Disease Parkinson's System Organ Class: 100000004852
Conditions
Interventions
Product Name: STEM-PD Product
Product Code: STEM-PD Product
Pharmaceutical Form: Suspension for injection
INN or Proposed INN: INN not available at this time
CAS Number: n/a
Other descriptive name: Al
Sponsors
Region Skåne – Skåne University Hospital
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: •Patient has given written informed consent to participate in the trial •Diagnosed with Parkinson’s disease 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 Principal Investigator (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 (including UK participants) •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 •Be approved by the TMG clinical sub-group for trial participation Are the trial subjects under 18? no Number of subjects for this age range: 0 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 5 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 3
Exclusion criteria
Exclusion criteria: •Tremor dominant disease, as assessed by 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 (Montgomery-Åsberg Depression Rating Scale [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 Montreal Cognitive Assessment (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-human leukocyte antigen (HLA) antibodies to the cell product •Thiopurine methyltransferase (TPMT) deficiency < 10 pmol/h/mg Hb •History of documented severe/significant allergy requiring treatment •Pregnant or breastfeeding female participants •Received an investigational drug (including investigational vaccines) or used an invasive investigational medical device within 4 weeks of the screening assessment, or is currently enrolled in an interventional investigational trial •Female participant of childbearing potential or male participant 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the safety, tolerability and feasibility of intraputamenal transplantation of the STEM-PD product in patients with moderate PD.;Secondary Objective: •To evaluate the course and efficacy on clinical features following intraputamenal transplantation of the STEM-PD product in patients with moderate PD. •To assess the survival of dopamine cells following transplantation of the STEM-PD product in patients with moderate PD using PET imaging. •To determine the safety and clinical efficacy between doses (if dose escalation is undertaken) of the STEM-PD product including assessment of whether there is a dose response effect.;Primary end point(s): The primary outcome measures are: •The number and nature of adverse events (AEs) and serious adverse events (SAEs) in the first 12 months following transplantation •Absence of space occupying masses on cranial MRI in the first 12 months following transplantation ;Timepoint(s) of evaluation of this end point: AEs and SAEs from the day of surgery (day 0), and all trial visits up to and including 12 months post-surgery will inform the first primary outcome measure. The second primary outcome measure is evaluated at 12 months post-surgery. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): •Clinical effects at 36 months following transplantation (including emergence of new neurological features, including graft-induced dyskinesias (GIDs), global cognitive changes, and changes in non-motor/quality of life (QOL) assessments) •Changes in motor features in the OFF state •Change in anti-Parkinson medication as measured by levodopa equivalent dose (LED) •Changes in F-DOPA uptake and dopamine transporter (DAT) binding at 36 months on PET imaging with [18F]-fluorodopa (F-DOPA) and [18F]FE-PE2i compared to screening •The number and nature of SAEs and AEs in the 12 to 36 months period following transplantation ;Timepoint(s) of evaluation of this end point: All timepoints between baseline and 36 months post-surgery. | — |
Countries
Sweden, United Kingdom
Contacts
Public ContactResearch coordinator
Skåne University Hospital, Section Neurology
Outcome results
None listed