Clinical trial NCT06753331: A Multicenter Study to Evaluate Safety, Tolerability, and Clinical Responses of DSP-1083 Into Subjects With Parkinson's Disease
Official 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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
Conditions: Parkinson's Disease
Lead sponsor: Sumitomo Pharma America, Inc.
Sponsor class: INDUSTRY
First posted: 2024-12-31
Enrollment: 23
Sex: ALL
Minimum age (years): 40
Maximum age (years): 69
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: The sentinel cohort of 3 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 purpose: TREATMENT

Brief summary: 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 5-6 study sites in North America

Detailed description: This is a multicenter first-in-human (FIH) study designed to evaluate the safety, tolerability, and clinical responses following implantation of 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.

SS1 Cohort 1 will receive 2 unilateral surgical procedures separated by approximately 28 weeks. SS2 and SS3 will undergobilateral implantation of DSP-1083 in a single surgical procedure.

Eligibility criteria: 1. Men or women aged ≥ 40 and \< 70 years with a clinically establisheddiagnosis of Parkinson's disease in accordance with the Movement Disorder Society (MDS) Clinical Diagnostic Criteria for Parkinson's Disease.
2. Subject has a diagnosis of PD for ≥ 5 years.
3. Subject has suboptimal control of PD symptoms, or intolerable side-effects with optimized oral antiparkinsonian medication regimen for ≥ 3 months, including treatment with L-DOPA and at least one medication that increases the effects of L-DOPA .
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 medication 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 and the Hauser patient daily diary.
8. Subject must meet the following ethnicity criteria:

   * SS1 will either be Asian defined as having both parents and 4 grandparents who are ethnically Asian, or non-Asian.
   * SS2 and SS3 will be Asian defined as having both parents and 4 grandparents who are ethnically Asian.
   * Cohort 2 subjects will be of any ethnicity.
9. Subject is approved by the Sponsor Eligibility Committee following review of all required information collected during Screening and prior to surgery on Day -1.

Primary outcomes:
measure: Incidence and severity of Adverse Events.
timeFrame: Up to 104 weeks
measure: Incidence of Serious Adverse Events (SAE).
timeFrame: Up to 104 weeks
measure: Incidence and severity of Adverse Events of Special Interest (AESI).
timeFrame: Up to 104 weeks
measure: Incidence and severity of Adverse Events leading to study discontinuation.
timeFrame: Up to 104 weeks
measure: Change from baseline in cognition and neuropsychiatric status as assessed by Montreal Cognitive Assessment (MoCA).
timeFrame: Up to 104 weeks
measure: Change from baseline in cognition and neuropsychiatric status as assessed by Mattis Dementia Rating Scale (MDRS).
timeFrame: Up to 104 weeks
measure: Change from baseline in Head Magnetic Resonance Imaging (MRI) (graft expansion/rejection) neuroimaging parameters.
timeFrame: Up to 104 weeks
measure: Change from baseline in Fluorodopa (F-DOPA) uptake (graft function) neuroimaging parameters.
timeFrame: Up to 104 weeks
measure: Frequency of subjects with suicidal ideation or suicidal behavior using the Columbia Suicide Severity Scale (C-SSRS).
timeFrame: Up to 104 weeks
measure: Observed values and change from baseline in clinical laboratory tests.
timeFrame: Up to 104 weeks
measure: Observed values and change from baseline in Heart Rate (HR).
timeFrame: Up to 104 weeks
measure: Observed values and change from baseline in QT interval.
timeFrame: Up to 104 weeks
measure: Observed values and change from baseline in PR interval.
timeFrame: Up to 104 weeks
measure: Observed values and change from baseline in QRS duration.
timeFrame: Up to 104 weeks
measure: Observed values and change from baseline in RR interval.
timeFrame: Up to 104 weeks
measure: Observed values and change from baseline in QTcF interval.
timeFrame: Up to 104 weeks
measure: Observed values and change from baseline in body temperature.
timeFrame: Up to 104 weeks
measure: Observed values and change from baseline in respiratory rate.
timeFrame: Up to 104 weeks
measure: Observed values and change from baseline in pulse rate.
timeFrame: Up to 104 weeks
measure: Observed values and change from baseline in Systolic Blood Pressure.
timeFrame: Up to 104 weeks
measure: Observed values and change from baseline in Diastolic Blood Pressure.
timeFrame: Up to 104 weeks
Secondary outcomes:
Other outcomes:
Locations:
city: Lexington
country: United States
facility: University of Kentucky Medical Center
state: Kentucky
city: New York
country: United States
facility: New York Presbyterian Hospital-Columbia University Medical Center
state: New York
Study officials:
References: