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NCT Number: NCT06753331

A Multicenter Study to Evaluate Safety, Tolerability, and Clinical Responses of DSP-1083 Into Subjects With Parkinson's Disease

The Goal of this study is to evaluate the safety, tolerability, and clinical responses following implantation of DSP-1083. Study enrolls both male and female patients in 2 cohorts. This study will be held in approximately 5-8 study sites in United States

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Key information

Conditions

Parkinson's Disease Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Movement Disorders Nervous System Diseases Neurodegenerative Diseases Parkinson Disease Parkinsonian Disorders Synucleinopathies

Age range

40 year–72 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

University of Kentucky Medical Center, Lexington, Kentucky, United States

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About this study

This is a multicenter first-in-human (FIH) study designed to evaluate the safety, tolerability, and clinical responses following implantation of dopaminergic progenitor cells derived from induced pluripotent stem cells (DSP-1083) compared with sham surgery. Safety is measured based on adverse events, changes in neuropsychiatric/cognition status, and serial neuroimaging (ie, engraftment status, graft expansion, rejection) over 104 weeks.

Cohort 1 sentinel subject (SS1) will undergo 2 unilateral surgical procedures separated by approximately 28 weeks, whereas SS2 and all subsequent subjects will undergo 1 bilateral surgical procedure.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Men or women aged ≥ 40 and ≤ 72 years at the time of informed consent with a clinically established diagnosis of Parkinson's disease in accordance with the Movement Disorder Society (MDS) Clinical Diagnostic Criteria for Parkinson's Disease.
  • Subject has a clinically established diagnosis of PD for ≥ 4 years.
  • Subject has suboptimal control of PD symptoms, with optimized oral antiparkinsonian medication regimen including levodopa/carbidopa monotherapy or levodopa/carbidopa plus antiparkinsonian medications, with stable dosing for ≥ 2months prior to screening.
  • Subject has a L-DOPA response of ≥ 30% without the influence of antiparkinsonian medications at Screening.
  • Subject has a Modified Hoehn and Yahr stage 3 - 4 in the Off state.
  • Subject has a pretreatment 18F-DOPA PET scan consistent with PD.
  • Subject has both On and Off states as demonstrated by the MDS-UPDRS Part III/IV and the Hauser patient daily diary.
  • Subjects must meet the following race criteria: 2 of the up to 5 sentinel subjects will be of Asian race, defined as having at least 2 grandparents who are Japanese, Taiwanese, Korean, or Chinese. Subjects in Cohort 2 can be of any race.
  • Subject is approved by the Enrollment Authorization Eligibility Committee following review of all required information collected during Screening.

Exclusion criteria

  • Subject has atypical parkinsonian syndrome (eg, progressive supranuclear palsy [PSP], multiple system atrophy [MSA], dementia with Lewy bodies [DLB], corticobasal degeneration, Parkinson-plus syndrome, vascular parkinsonism, secondary parkinsonism, hereditary parkinsonism).
  • Subject has non-PD neurological symptoms or evidence of non-PD brain disease (eg, tumor, inflammation, active or history of vascular disorder, history of cerebral hemorrhage, Alzheimer's disease, or other neurodegenerative disorder) based on neuroimaging and/or medical history that would preclude study participation.
  • Subject has psychiatric symptoms, cognitive impairment, depression, dementia, or other behavioral disorder that would preclude study participation based on Investigator decision.
  • Subject has received previous striatal or other extrapyramidal system PD treatments, including deep-brain stimulation, central nervous system (CNS) ablation (eg, pallidotomy, thalamotomy), implanted cell, or gene therapy, and/or focused ultrasound therapy.
  • Subject has peak-dose dyskinesia of sufficient severity that precludes study participation, defined as any item score of ≥ 3 (moderate dyskinesia) on the UDysRS Part 1B (Patient Dyskinesia Questionnaire) AND/OR any item score of ≥ 2 (moderate dyskinesia) on Part 3 (Objective Evaluation of Dyskinesia Disability) Intensity Scale: Impairment. Subject has another type (eg, diphasic dyskinesia) or an unusual pattern of dyskinesia.
  • Subject has a history of, or concurrent abnormal immune function that may adversely affect the engraftment of the cell implants and use of adjunctive immunosuppressants.
  • The subject has the following clinical laboratory test results at Screening:
  • Neutrophil count < 2,000/μL.
  • Platelet count < 5.0 × 104/μL.
  • Aspartate aminotransferase (AST), alanine aminotransferase (ALT) > 3.0 × upper limit of normal.
  • Total bilirubin > 1.5 × upper limit of normal.
  • Persistent estimated glomerular filtration rate (eGFR) < 60 mL/min/1.73 m2.
  • Poorly controlled blood glucose in diabetic subjects (glycosylated hemoglobin > 9.0%, or fasting serum glucose ≥ 200mg/dL).
  • Subject has any disorder that would contraindicate general anesthesia, conscious sedation or stereotactic surgery.
  • Subject has any clinically significant unstable medical condition or any clinically significant chronic disease that would pose a risk to the subject or that might confound the results of the study. In cases in which the impact of the condition upon risk to subject or study results is unclear, the Medical Monitor should be consulted.

Treatment and study plan

DSP-1083 implantation

Combination Product

DSP-1083 subjects will receive 2.7M viable cells per hemisphere; 5.4M total cell dose as implants.

Sham surgery treatment

Procedure

Sham surgery subjects will undergo a partial thickness burr hole surgical procedure on each side of the skull with no DSP-1083 administration.

Primary outcomes

  1. Incidence and severity of Adverse Events.

    Time frame: Up to 104 weeks

  2. Incidence of Serious Adverse Events (SAE).

    Time frame: Up to 104 weeks

  3. Incidence and severity of Adverse Events of Special Interest (AESI).

    Time frame: Up to 104 weeks

  4. Incidence and severity of Adverse Events leading to study discontinuation.

    Time frame: Up to 104 weeks

  5. Change from baseline in cognition and neuropsychiatric status as assessed by Montreal Cognitive Assessment (MoCA).

    Time frame: Up to 104 weeks

    The MoCA is a widely used, sensitive, validated screening test for detecting mild cognitive impairment and can also predict the presence of cognitive deterioration (ie, progression from mild cognitive impairment to dementia) in PD patients.

  6. Change from baseline in cognition and neuropsychiatric status as assessed by Mattis Dementia Rating Scale (MDRS).

    Time frame: Up to 104 weeks

    The MDRS has been utilized for early detection of dementia, differential diagnosis between Alzheimer's disease and other dementias. The 144-point scale is an aggregate score of 5 subscales: attention, initiation/perseveration (I/P), construction, conceptualization, and memory.The total score ranges from 0 to 144, with lower scores indicating greater cognitive impairment.

  7. Change from baseline in Head Magnetic Resonance Imaging (MRI) (graft expansion/rejection) neuroimaging parameters.

    Time frame: Up to 104 weeks

    Safety MRIs - are conducted to assess the safety of DSP-1083 including rejection, abnormal growth, and formation of mass lesions, which could indicate teratoma formation

  8. Change from baseline in Fluorodopa (F-DOPA) uptake (graft function) neuroimaging parameters.

    Time frame: Up to 104 weeks

    PET scans using F-DOPA will be performed at Screening and then post-surgery to follow the course of graft development and to provide a measure of dopaminergic nerve terminals in the striatum.

  9. Frequency of subjects with suicidal ideation or suicidal behavior using the Columbia Suicide Severity Scale (C-SSRS).

    Time frame: Up to 104 weeks

  10. Observed values and change from baseline in clinical laboratory tests.

    Time frame: Up to 104 weeks

    Laboratory results at each time point will be summarized using descriptive statistics (mean, standard deviation (SD), median, minimum, and maximum) by treatment group.

  11. Observed values and change from baseline in Heart Rate (HR).

    Time frame: Up to 104 weeks

    12-lead ECG parameters ventricular HR at each time point will be summarized by treatment group. All ECG diagnostic findings will be summarized on the basis of incidence rates by treatment group.

  12. Observed values and change from baseline in QT interval.

    Time frame: Up to 104 weeks

    12-lead ECG parameters QT interval at each time point will be summarized by treatment group. All ECG diagnostic findings will be summarized on the basis of incidence rates by treatment group.

  13. Observed values and change from baseline in PR interval.

    Time frame: Up to 104 weeks

    12-lead ECG parameters PR interval at each time point will be summarized by treatment group. All ECG diagnostic findings will be summarized on the basis of incidence rates by treatment group.

  14. Observed values and change from baseline in QRS duration.

    Time frame: Up to 104 weeks

    12-lead ECG parameters QRS duration at each time point will be summarized by treatment group. All ECG diagnostic findings will be summarized on the basis of incidence rates by treatment group.

  15. Observed values and change from baseline in RR interval.

    Time frame: Up to 104 weeks

    12-lead ECG parameters RR interval at each time point will be summarized by treatment group. All ECG diagnostic findings will be summarized on the basis of incidence rates by treatment group.

  16. Observed values and change from baseline in QTcF interval.

    Time frame: Up to 104 weeks

    12-lead ECG parameters QTcF interval at each time point will be summarized by treatment group. All ECG diagnostic findings will be summarized on the basis of incidence rates by treatment group.

  17. Observed values and change from baseline in body temperature.

    Time frame: Up to 104 weeks

    Vital signs parameter body temperature will be summarized using descriptive statistics at each time point by treatment group.

  18. Observed values and change from baseline in respiratory rate.

    Time frame: Up to 104 weeks

    Vital signs parameter supine respiratory rate will be summarized using descriptive statistics at each time point by treatment group.

  19. Observed values and change from baseline in pulse rate.

    Time frame: Up to 104 weeks

    Vital signs parameter supine and standing pulse will be summarized using descriptive statistics at each time point by treatment group.

  20. Observed values and change from baseline in Systolic Blood Pressure.

    Time frame: Up to 104 weeks

    Vital signs parameter Systolic Blood Pressure will be summarized using descriptive statistics at each time point by treatment group.

  21. Observed values and change from baseline in Diastolic Blood Pressure.

    Time frame: Up to 104 weeks

    Vital signs parameter Diastolic Blood Pressure will be summarized using descriptive statistics at each time point by treatment group.

Sponsors and collaborators

Lead sponsor

Sumitomo Pharma America, Inc.

Industry

Registry information

Official study title

A Multicenter, Sham-controlled, Randomized Study to Evaluate the Safety, Tolerability, and Clinical Responses Following Stereotactic Intracranial Implantation of DSP-1083 Into Subjects With Parkinson's Disease

Important dates

Study start
2024
Primary completion
2030
Study completion
2030
First posted
Dec 31, 2024
Registry last updated
Mar 2, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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