Evaluating the safety and effects of DSP-1083 in Parkinson's disease patients

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

This study is testing a new treatment called DSP-1083 to see if it can safely help people with Parkinson's disease feel better over two years.

Quick facts

• Phase: Phase1; Phase2
• Study type: Interventional
• Enrollment: 25 (estimated)
• Ages: 40 Years to 72 Years
• Sex: All
• Sponsor: Sumitomo Pharma America, Inc. (Industry-sponsored)
• Locations: 2 sites (Lexington, Kentucky and 1 other locations)
• Trial ID: NCT06753331 on ClinicalTrials.gov

What this trial studies

This multicenter, first-in-human study aims to assess the safety, tolerability, and clinical responses of DSP-1083 implantation in patients with Parkinson's disease. Participants will be divided into cohorts receiving either unilateral or bilateral surgical procedures, with a comparison to sham surgery. The study will monitor adverse events, neuropsychiatric changes, and neuroimaging results over a period of 104 weeks to evaluate the treatment's effectiveness. The trial will enroll both male and female patients aged 40 to 70 with a history of Parkinson's disease.

Who should consider this trial

Good fit: Ideal candidates are men and women aged 40 to 70 with a clinically established diagnosis of Parkinson's disease for at least 5 years and suboptimal control of symptoms.

Not a fit: Patients who have not been diagnosed with Parkinson's disease or those with early-stage disease may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide improved symptom control for patients with Parkinson's disease who have not responded adequately to current medications.

How similar studies have performed: While this approach is novel, similar studies evaluating surgical interventions for Parkinson's disease have shown promise in improving patient outcomes.

Eligibility criteria

Inclusion Criteria:

1. 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.
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 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).
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.

Where this trial is running

Lexington, Kentucky and 1 other locations

• University of Kentucky Medical Center — Lexington, Kentucky, United States (Recruiting)
• New York Presbyterian Hospital-Columbia University Medical Center — New York, New York, United States (Recruiting)

Conditions Parkinson's Disease