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    Summary
    EudraCT Number:2021-001366-38
    Sponsor's Protocol Code Number:CCTU0263
    National Competent Authority:Sweden - MPA
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-12-01
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedSweden - MPA
    A.2EudraCT number2021-001366-38
    A.3Full title of the trial
    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)
    En klinisk studie för att utvärdera säkerheten av transplantation av stamcells-deriverade dopamin celler till hjärnan hos individer med Parkinsons sjukdom
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A trial to determine the safety and tolerability of transplanted stem cell-derived dopamine neurons to the brains of individuals with Parkinson’s Disease.
    A.3.2Name or abbreviated title of the trial where available
    STEM-PD Trial
    A.4.1Sponsor's protocol code numberCCTU0263
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05635409
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorRegion Skåne – Skåne University Hospital
    B.1.3.4CountrySweden
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportLund University
    B.4.2CountrySweden
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSkåne University Hospital, Section Neurology
    B.5.2Functional name of contact pointResearch coordinator
    B.5.3 Address:
    B.5.3.1Street AddressForskningsenhet Neurologi, Remissgatan 4
    B.5.3.2Town/ cityLund
    B.5.3.3Post code221 85
    B.5.3.4CountrySweden
    B.5.4Telephone number+46 724 693494
    B.5.6E-mailTrinette.vanvliet@skane.se
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameSTEM-PD Product
    D.3.2Product code STEM-PD Product
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntracerebral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNINN not available at this time
    D.3.9.1CAS number n/a
    D.3.9.3Other descriptive nameAllogeneic embryonic stem cell-derived ventral midbrain dopaminergic progenitor cells
    D.3.9.4EV Substance CodeSUB214923
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5x10^6
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Yes
    D.3.11.3.1Somatic cell therapy medicinal product Yes
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Parkinson’s disease
    E.1.1.1Medical condition in easily understood language
    Parkinson’s disease
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level LLT
    E.1.2Classification code 10013113
    E.1.2Term Disease Parkinson's
    E.1.2System Organ Class 100000004852
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the safety, tolerability and feasibility of intraputamenal transplantation of the STEM-PD product in patients with moderate PD.
    E.2.2Secondary objectives of the trial
    •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.
    E.2.3Trial contains a sub-study No
    E.3Principal 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
    E.4Principal 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
    E.5 End points
    E.5.1Primary 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
    E.5.1.1Timepoint(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.
    E.5.2Secondary 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
    E.5.2.1Timepoint(s) of evaluation of this end point
    All timepoints between baseline and 36 months post-surgery.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans Yes
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Sweden
    United Kingdom
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1Number of subjects for this age range: 0
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) No
    F.1.1.5.1Number of subjects for this age range: 0
    F.1.1.6Adolescents (12-17 years) No
    F.1.1.6.1Number of subjects for this age range: 0
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 5
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 3
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 4
    F.4.2.2In the whole clinical trial 8
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    The trial consists of a single transplant, which is intended to be a one off intervention.

    Following completion of the trial, we intend to follow participants for life. Therefore, participants will be asked to consider consenting to a separate long-term follow-up study. Participants are informed of this in the PIS.

    This will be covered by a separate ethics application and protocol. This long-term follow-up study will be in addition to the expected normal treatment.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2022-10-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-03-23
    P. End of Trial
    P.End of Trial StatusOngoing
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