Safety Study of Gene Transfer Therapy in People with Parkinson’s Disease

Official Study Title: A Phase 1 Open-label Safety Study of Intrastriatal Infusion of Adeno-Associated Virus Encoding Human Aromatic L-Amino Acid Decarboxylase (AAV-hAADC-2) in Subjects with Mid- to Late- Stage Parkinson’s Disease

Sponsor: Genzyme
Clinicaltrials.gov ID : NCT00229736
Study ID : AAV-hAADC-2-003
Last Updated Date: 02 Mar 2007
Trial Start Date: 01 Nov 2004
Trial Post date: 02 Mar 2007
Website URL: http://www.pdtrials.org/front/trial_...p?trial_id=139

Summary

In people with Parkinson’s disease (PD), the part of the brain called the substantia nigra progressively loses the ability to send dopamine signals to the striatum, another part of the brain. As a result, dopamine levels are lacking in people diagnosed with PD.

The purpose of this study is to determine the safety of an investigational drug, AAV-hAADC-2. This gene therapy drug works to provide an essential enzyme in the brain that will enable L-dopa – a drug commonly used to treat PD- to convert to dopamine in the brain.

Participants for this study will receive a surgical injection of this drug into the brain, then undergo periodic clinical evaluations and brain scans. They will also continue to take L-dopa while taking part on this study.

The primary objective of the study is to determine the safety of intrastriatal administration of AAV-hAADC-2. A secondary objective is to determine the effect of AAV-hAADC-2 on clinical status.

Enrollment
Expected Enrollment: 15

Trial Phase
Study Phase: Phase 1

Symptoms
#Trial does not specify symptoms

Time Commitment
Length of Time Commitment: More than six months
Frequency of office visits: Every 2 weeks for the first 3 months, once a month for the next 9 months, then once every 3 months for the next 4 years.

Eligibility
Minimum Age: 40
Maximum Age: 75

Inclusion Criteria

• Childbearing potential participants must agree to use barrier contraception, until three consecutive PCR (polymerase chain reaction- the analysis of a sequence of DNA) samples are negative
• Must be able to comply with the requirements of the study, including the need for frequent and prolonged follow-up
• Documented informed consent
• Experiencing two of four cardinal features – tremor, bradykinesia (slowness of movement), rigidity and postural instability
• Early dopaminergic (relating to dopamine) responsiveness of symptoms and absence of any cardinal signs of more widespread neurological disease
• Have taken levodopa therapy for five years or more
• Positive response to dopaminergic therapy
• Candidate for surgical therapy for Parkinson’s disease
• MRI within the past two years and MRI does not show any conditions that are clinically significant with respect to risks for brain surgery

Exclusion Criteria

• Atypical Parkinsonian syndromes due to drugs. Parkinsonian syndromes can be any of a group of nervous disorders similar to Parkinson's disease, marked by muscular rigidity, tremor, and impaired motor control and often having a specific cause
• Metabolic disorders (e.g. Wilson’s disease) or degenerative diseases other than PD
• History of three (3) hours or more of intense or violent dyskinesias (uncontrollable movements) in the past six (6) months
• Previous stereotactic neurosurgery for Parkinson’s disease (pallidotomy, thalamotomy, deep brain stimulation).
• Dementia
• Hallucinations or delusions within 6 months of screening due to mental illness
• History of stroke or other clinically significant cardiovascular disease or anticoagulant therapy (something that keeps the blood from clotting)
• Certain medical conditions, including some kinds of cancer within the past 5 years, immune dysfunction, severe depression and uncontrolled hypertension
• Pregnant or breastfeeding

Primary Contact
Rowena Mah
Study Coordinator
University of California, San Francisco (UCSF)
Phone : 415-476-0947
rowenam@itsa.ucsf.edu
San Francisco, CA 94143
USA

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Full Details:

PDTrials.org http://www.pdtrials.org/front/trial_...p?trial_id=161

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ClinicalTrials.gov http://clinicaltrials.gov/ct/show/NCT00427674?order=1[LIST][*]Complete list of PD trials

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Parkinson Pipeline Project
www.pdpipeline.org
Following theapies from pre-clinical development to approval in the Parkinson Pipeline Project Database (see homepage for link to database)

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