STEPS Trial - Spheramine Safety and Efficacy Study

Study Overview
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Brief Summary
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The purpose of the study is to explore the safety, tolerability and efficacy of Spheramine (cultured human retinal pigment epithelial cells on microcarriers) in Parkinson's Disease patients with advanced disease who have insufficient symptom control by optimum oral medication. Patients are randomized to receive Spheramine injections into both hemispheres or a sham surgical procedure in a ratio of 1:1. A three month pretreatment period must be completed prior to surgery. Time to endpoint is 24 months.

Detailed Description
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This study has previously been posted by Berlex, Inc. and Schering AG, Germany.Berlex, Inc. has been renamed to Bayer HealthCare Pharmaceuticals, Inc., Schering AG Germany has been renamed to Bayer Schering Pharma AG, Germany. Bayer HealthCare Pharmaceuticals, Inc.and Bayer Schering Pharma AG, Germany are the sponsors of the trial.

Official Title
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Study of the Safety, Tolerability and Efficacy of Spheramine Implanted Bilaterally Into the Postcommissural Putamen of Patients With Advanced Parkinson's Disease

Conditions
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Parkinson Disease

Intervention / Treatment
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* Biological: Spheramine (BAY86-5280)
* Procedure: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Advanced Parkinson's disease for at least 5 years Good response to L-dopa Age 30 to 70 years Optimum oral therapy Exclusion Criteria: Tremor only Dementia Very severe dyskinesia Previous brain surgery including deep brain stimulation Malignant disease

Ages Eligible for Study
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Minimum Age: 30 Years
Maximum Age: 70 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Arm 1<br> | Biological: Spheramine (BAY86-5280)<br>* Bilateral implantation of Spheramine into the postcommissural putamen, each side at a dose of 325,000 cells<br>|
| Sham Comparator: Arm 2<br> | Procedure: Placebo<br>* Sham surgery procedure without penetration of the dura mater. Nothing wil be implanted into the brain.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in UPDRS part III (Motor Score) in the defined medication at 12 months post surgery |  | 12 months post surgery | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in total UPDRS in ON and OFF at 12 months post surgery |  | 12 months post surgery | 
| Change in UPDRS Part III in ON at 12 months post surgery |  | 12 months post surgery | 
| Amount of L-dopa reduction at 12 months post surgery |  | 12 months post surgery | 
| Activities of Daily Living subscore of the UPDRS at 12 months post surgery |  | 12 months post surgery | 
| Quality of Life as assessed by PDQ-39, SF-36 and EQ-5D at 12 months post surgery |  | 12 months post surgery | 
| Percent time spent in ON and OFF at 12 months post surgery |  | 12 months post surgery |