Parkinson's DiseasePhase 1NCT00252850
Safety of CERE-120 (AAV2-NTN) in Subjects With Idiopathic Parkinson's Disease
A Phase 1 study testing CERE-120: AAV2-NTN in people with Parkinson's Disease, run by Sangamo Therapeutics at 2 sites in United States.
Trial summary
This is a Phase I dose escalating open-label study designed to assess the safety, tolerability and biologic activity of an in vivo AAV2 mediated delivery of the gene encoding NTN (CERE-120). Read more about this study
- Who is it for?
- Ages 35 to 75 years
- Conditions the study is for
- Parkinson's Disease
- What is being tested?
- CERE-120: AAV2-NTN
- How many people
- 12 participants
- Where
- 2 sitesUnited States
- Study dates
- Jun 2005to Mar 2007
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 35 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of bilateral idiopathic Parkinson's Disease of at least 5 years duration since diagnosis with motor fluctuations, despite adequate oral antiparkinsonian therapy.
- Diagnosis of moderate to severe Parkinson's Disease based on clinical rating scales.
- Males or nonpregnant females 35-75 years of age, inclusive.
- Stable medication requirements, and clear response to antiparkinsonian medications during the 60-day eligibility evaluation period.
- No conditions that would render the subject unsuitable for surgery, or that would interfere with any of the assessments of safety or efficacy in this trial.
- Subject's informed consent prior to the performance of any study-specific procedure.
Who cannot join
- A history of any clinically significant medical, psychiatric, or laboratory abnormality for which participation in the study would pose a safety risk to the subject.
- History of treatment of Parkinson's disease by any procedure involving intracranial surgery or implantation of a device.
- MRI of the brain within 12 months before the anticipated dosing procedure that indicates the presence of an abnormality that may interfere with the assessments of safety or efficacy or would, in the judgment of the investigator, present a surgical risk to the subject.
- Any disorder that precludes a surgical procedure or alters wound healing.
- A score of less than or equal to 25 on the Folstein Mini-Mental examination performed during the eligibility evaluation period.
- Chemotherapy, cytotoxic therapy, or immunotherapy within 6 weeks prior to CERE-120 administration.
Show all 12Show fewer
- Vaccinations within 30 days prior to CERE-120 administration.
- History, within two years before the anticipated dosing procedure, of drug or alcohol abuse.
- Treatment with nonantiparkinsonian agents that may affect symptoms of Parkinson's disease within 60 days before the anticipated dosing procedure.
- Any medical disability that would interfere with the assessment of safety and efficacy in this trial or would compromise the ability of the subject to undergo study procedures (e.g. MRI, PET) or to give informed consent.
- History of prior gene transfer therapy.
- Treatment with an investigational agent within 60 days before the anticipated dosing procedure.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Rush University Medical Center
Chicago, Illinois, 60612
University of California, San Francisco
San Francisco, California, 94143
About this study
Official title: A Phase I, Open-Label Study of CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin[NTN] to Assess the Safety and Tolerability of Intrastriatal Delivery to Subjects With Idiopathic Parkinson's Disease
This is a Phase I dose escalating open-label study designed to assess the safety, tolerability and biologic activity of an in vivo AAV2 mediated delivery of the gene encoding NTN (CERE-120).
Twelve (up to 18) subjects will receive one of two open-label doses of CERE-120 via bilateral stereotactic injections targeting the putaminal region of the brain. Subjects will be enrolled in one of two cohorts, a low-dose cohort of six subjects followed by a high dose cohort of six subjects.
The design of this study is such that the primary objective, the evaluation of safety and tolerability, will be assessed by frequent observations for adverse events, clinical laboratory test results, imaging (MRI), neuropsychometric testing, and evaluations of disease status.
- Conditions
- Parkinson's Disease
- Being tested
- CERE-120: AAV2-NTN
- Keywords
- Parkinson's Disease, Gene Transfer
- Medical subject headings
- Parkinson Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CERE-120: AAV2-NTNGenetic
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Nov 2005 (estimated)
- Study startJun 2005
- Primary completionMar 2007
- Study completionMar 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Parkinson's DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants
Sponsor and investigators
- Lead sponsor
- Sangamo Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Ceregene
- Investigators
- William J Marks, Jr., M.D., Principal investigator, University of California, San FranciscoLeo Verhagen Metman, M.D., Ph.D., Principal investigator, Rush University Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00252850
- Sponsor's protocol ID
- CERE-120-01
- Status last verified
- Sep 2016
- First submitted
- 11 Nov 2005
- Last updated
- 10 Nov 2022
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Marks WJ Jr, Ostrem JL, Verhagen L, Starr PA, Larson PS, Bakay RA, Taylor R, Cahn-Weiner DA, Stoessl AJ, Olanow CW, Bartus RT. Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial. Lancet Neurol. 2008 May;7(5):400-8. doi: 10.1016/S1474-4422(08)70065-6. Epub 2008 Apr 2.
PubMed 18387850
Study at a glance
From the registry record, last updated by the sponsor on 10 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I, Open-Label Study of CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin[NTN] to Assess the Safety and Tolerability of Intrastriatal Delivery to Subjects With Idiopathic Parkinson's Disease
- ClinicalTrials.gov ID
- NCT00252850
- Acronym / sponsor ID
- CERE-120-01
- Conditions
- Parkinson's Disease
- Keywords
- Parkinson's Disease, Gene Transfer
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants
- Lead sponsor
- Sangamo TherapeuticsIndustry (pharma, biotech, devices)
- Collaborators
- Ceregene
- Study start
- Jun 2005
- Primary completion
- Mar 2007 (actual)
- Study completion
- Mar 2007 (actual)
- First posted
- 15 Nov 2005 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a Phase I dose escalating open-label study designed to assess the safety, tolerability and biologic activity of an in vivo AAV2 mediated delivery of the gene encoding NTN (CERE-120).
Twelve (up to 18) subjects will receive one of two open-label doses of CERE-120 via bilateral stereotactic injections targeting the putaminal region of the brain. Subjects will be enrolled in one of two cohorts, a low-dose cohort of six subjects followed by a high dose cohort of six subjects.
The design of this study is such that the primary objective, the evaluation of safety and tolerability, will be assessed by frequent observations for adverse events, clinical laboratory test results, imaging (MRI), neuropsychometric testing, and evaluations of disease status.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 35 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Diagnosis of bilateral idiopathic Parkinson's Disease of at least 5 years duration since diagnosis with motor fluctuations, despite adequate oral antiparkinsonian therapy.
- Diagnosis of moderate to severe Parkinson's Disease based on clinical rating scales.
- Males or nonpregnant females 35-75 years of age, inclusive.
- Stable medication requirements, and clear response to antiparkinsonian medications during the 60-day eligibility evaluation period.
- No conditions that would render the subject unsuitable for surgery, or that would interfere with any of the assessments of safety or efficacy in this trial.
Key exclusion criteria (12 listed)
- A history of any clinically significant medical, psychiatric, or laboratory abnormality for which participation in the study would pose a safety risk to the subject.
- History of treatment of Parkinson's disease by any procedure involving intracranial surgery or implantation of a device.
- MRI of the brain within 12 months before the anticipated dosing procedure that indicates the presence of an abnormality that may interfere with the assessments of safety or efficacy or would, in the judgment of the investigator, present a surgical risk to the subject.
- Any disorder that precludes a surgical procedure or alters wound healing.
- A score of less than or equal to 25 on the Folstein Mini-Mental examination performed during the eligibility evaluation period.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CERE-120: AAV2-NTN | Genetic | – |
Primary outcome
Not reported in the source record.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Nov 2022.
- Results publication: PubMed 18387850Marks WJ Jr, Ostrem JL, Verhagen L, Starr PA, Larson PS, Bakay RA, Taylor R, Cahn-Weiner DA, Stoessl AJ, Olanow CW, Bartus RT. Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial. Lancet Neurol. 2008 May;7(5):400-8. doi: 10.1016/S1474-4422(08)70065-6. Epub 2008 Apr 2.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
William J Marks, Jr., M.D. principal investigator
University of California, San Francisco
Leo Verhagen Metman, M.D., Ph.D. principal investigator
Rush University Medical Center
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CERE-120: AAV2-NTN in people with Parkinson's Disease, run by Sangamo Therapeutics at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Nov 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00252850, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Nov 2022.