STEPS Trial - Spheramine Safety and Efficacy Study

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

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.

Study Overview

• Status: Terminated
• Conditions: Parkinson Disease
• Intervention / Treatment: Biological: Spheramine (BAY86-5280); Procedure: Placebo

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Actual): 71
• Phase: Phase 2

Contacts and Locations

Study Locations

• Germany
  • Hamburg, Germany, 22307
  • Hessen
    • Marburg, Hessen, Germany, 35039
  • Saarland
    • Homburg, Saarland, Germany, 66424
  • Sachsen
    • Dresden, Sachsen, Germany, 01307
• Spain
  • Barcelona, Spain, 08036
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35233
  • California
    • Los Angeles, California, United States, 90095
  • Florida
    • Tampa, Florida, United States, 33606
  • Georgia
    • Atlanta, Georgia, United States, 30329
  • Illinois
    • Chicago, Illinois, United States, 60612
  • Kansas
    • Kansas City, Kansas, United States, 66160
  • Massachusetts
    • Boston, Massachusetts, United States, 02118
  • New York
    • New York, New York, United States, 10029-6574
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19107
  • Texas
    • Houston, Texas, United States, 77030

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 26 years to 66 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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	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

Collaborators and Investigators

• Sponsor: Bayer
• Collaborators: Titan Pharmaceuticals
• Investigators: Study Director: Bayer Study Director, Bayer

Publications and helpful links

General Publications

• Gross RE, Watts RL, Hauser RA, Bakay RA, Reichmann H, von Kummer R, Ondo WG, Reissig E, Eisner W, Steiner-Schulze H, Siedentop H, Fichte K, Hong W, Cornfeldt M, Beebe K, Sandbrink R; Spheramine Investigational Group. Intrastriatal transplantation of microcarrier-bound human retinal pigment epithelial cells versus sham surgery in patients with advanced Parkinson's disease: a double-blind, randomised, controlled trial. Lancet Neurol. 2011 Jun;10(6):509-19. doi: 10.1016/S1474-4422(11)70097-7. Epub 2011 May 10.

Study record dates

Study Major Dates

• Study Start (Actual): January 8, 2003
• Primary Completion (Actual): May 14, 2012
• Study Completion (Actual): May 14, 2012

Study Registration Dates

• First Submitted: September 13, 2005
• First Submitted That Met QC Criteria: September 13, 2005
• First Posted (Estimated): September 21, 2005

Study Record Updates

• Last Update Posted (Actual): October 31, 2024
• Last Update Submitted That Met QC Criteria: October 29, 2024
• Last Verified: October 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Synucleinopathies; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurodegenerative Diseases; Movement Disorders; Parkinsonian Disorders; Basal Ganglia Diseases; Parkinson Disease
• Other Study ID Numbers: 91039; 305405 (Other Identifier: Company Internal)