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

NCT ID: NCT06753331

Last Updated: 2025-12-09

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

25 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-12-18

Study Completion Date

2030-12-15

Brief Summary

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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

Detailed Description

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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.

Conditions

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Parkinson's Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

The sentinel cohort of 3 to 5 subjects will be open-label and receive DSP-1083. Cohort 2 (20 subjects) will be randomized in a 1:1 ratio to receive either DSP-1083 during a surgical procedure, or sham surgery with no DSP-1083 administered.
Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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DSP-1083

Implantation of DSP-1083 (2.7M viable cells per hemisphere; 5.4M total cell dose)

Group Type EXPERIMENTAL

DSP-1083 implantation

Intervention Type COMBINATION_PRODUCT

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

Sham Surgery

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

Group Type SHAM_COMPARATOR

Sham surgery treatment

Intervention Type PROCEDURE

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

Interventions

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DSP-1083 implantation

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

Intervention Type COMBINATION_PRODUCT

Sham surgery treatment

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

Intervention Type PROCEDURE

Eligibility Criteria

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Inclusion Criteria

1. Men or women aged ≥ 40 and \< 70 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.
2. Subject has a clinically established diagnosis of PD for ≥ 4 years.
3. 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.
4. Subject has a L-DOPA response of ≥ 30% without the influence of antiparkinsonian medications at Screening.
5. Subject has a Modified Hoehn and Yahr stage 3 - 4 in the Off state.
6. Subject has a pretreatment 18F-DOPA PET scan consistent with PD.
7. Subject has both On and Off states as demonstrated by the MDS-UPDRS Part III/IV and the Hauser patient daily diary.
8. 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.
9. Subject is approved by the Enrollment Authorization Eligibility Committee following review of all required information collected during Screening.

Exclusion Criteria

1. Subject has a typical 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).
2. 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.
3. Subject has psychiatric symptoms, cognitive impairment, depression, dementia, or other behavioral disorder that would preclude study participation based on Investigator decision.
4. 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.
5. 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.
6. 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.
7. 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).
8. Subject has any disorder that would contraindicate general anesthesia, conscious sedation or stereotactic surgery.
9. 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.
Minimum Eligible Age

40 Years

Maximum Eligible Age

69 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Sumitomo Pharma America, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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University of Kentucky Medical Center

Lexington, Kentucky, United States

Site Status RECRUITING

New York Presbyterian Hospital-Columbia University Medical Center

New York, New York, United States

Site Status RECRUITING

Countries

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United States

Facility Contacts

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Lynne Cagle

Role: primary

859-218-5443

Natasha Desai

Role: primary

347-882-4315

Other Identifiers

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DD201101

Identifier Type: -

Identifier Source: org_study_id

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