Pilot Study of Safety and Efficacy of Spheramine

Study Overview
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Brief Summary
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Tolerability and Efficacy of Spheramine will be evaluated after operative implantation (Stereotactic Intrastriatal Implantation) in patients with advanced Parkinson's Disease

Official Title
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Stereotactic Intrastriatal Implantation of Spheramine in Advanced Parkinson's Disease Patients: A Pilot Study of Tolerability and Efficacy.

Conditions
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Parkinson''s Disease

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


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Idiopathic Parkinson's disease (PD). As defined by: presence of bradykinesia and either rest tremor or rigidity history of asymmetry of PD signs history of progression of PD signs, and no other suspected cause of PD signs Patient is between 40 and 70 years of age, inclusive Patient is L-dopa-sensitive and has unequivocal clinical off periods as documented by home diary, completed on 4 consecutive days with an average of 1.5 hours/day in the clinically off state Patient is Hoehn & Yahr stage III or higher in the practically defined off phase All other medical problems are stable or under control and are not contraindications to general anesthesia or stereotactic neurosurgery Patient's brain magnetic imaging resonance (MRI) shows no abnormality that would contraindicate Spheramine implantation Patient does not have a major psychiatric problem or dementia Patient must give written informed consent to participate in this study Patient must be on a stable dose of antiparkinsonian medication for at least 1 month prior to completion of the home diary assessment and the baseline UPDRS assessments If the patient is a woman of childbearing potential, she must not be nursing or pregnant and must use an adequate form of birth control for the duration of the study deemed appropriate by the investigator such as oral contraceptives, IUD, or condoms Patient must be living with or in close constant contact with a person who can contribute information as to the patient's condition before and after implant and provide assistance, if necessary Exclusion Criteria: Patients with atypical Parkinson's disease (e.g. parkinsonian plus syndrome, secondary Parkinsonian syndrome) Patients exhibiting only a tremor-based symptomatology Patients exhibiting very severe dyskinesias (lasting more than 2 hour/day) exacerbated by L-dopa, evaluated as 4/4 on the dyskinesia scale (violent dyskinesias, incompatible with any normal motor task) Women of childbearing potential without contraception Patients who are non-cooperative or incapable of completing self-evaluation scales or diaries Patients presenting with known pharmacologic immunosuppression related to chemotherapeutic treatment for malignancy, autoimmune disease, allografts or other medical conditions associated with immunosuppression Patient's cardiovascular, pulmonary, renal and hematological state contraindicates use of general anesthesia Evidence of abnormal coagulation including PT > 13 sec, PT > 35 sec or platelets < 5 ml Patients having had previous stereotactic brain surgery Patients treated by apomorphine pump Patients with significant cognitive impairment, untreated or treatment resistant depression, psychosis, hallucinations or delusions that would prevent their effective participation in the study Patients who have participated in another experimental drug or device trial in the past 30 days

Ages Eligible for Study
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Minimum Age: 40 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: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Arm 1<br> | Biological: Spheramine (BAY86-5280)<br>* Stereotactic Intrastriatal Implantation<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The primary outcome measure for evaluating efficacy will be the motor subscore of the Unified Parkinson's Disease Rating Scale (UPDRS) (version 3.0) in the practically defined off state. During the Screening and at Baseline Evaluations. |  | Evaluations, and at approximately 1, 3, 6, 9, 12, 15, 18 and 24 months post-implantation and annually thereafter, the patients Parkinson's symptomatology will be evaluated both on their baseline drug therapy and in the practically defined off period. | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Involuntary Movements, time Motor Tests |  | Performed during defined off and best on states | 
| Motor Fluctuation and Percent OFF Time Evaluation |  | Daily on phases, off phases and hours of sleep per day, using Home Diary | 
| Quality of Life and Physician's and Patients Global Evaluations |  | Every visit until year 5 | 
| Neuropsychological Evaluations |  | Will occur pre-operatively and at 3, 12 and 24 months post-operatively |