Idiopathic Parkinson's DiseasePhase 2NCT00400634
Double-Blind, Multicenter, Sham Surgery Controlled Study of CERE-120 in Subjects With Idiopathic Parkinson's Disease
A Phase 2 study testing CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN]) in people with Idiopathic Parkinson's Disease, run by Sangamo Therapeutics at 9 sites in United States.
Trial summary
The purpose of this double blind study is to determine whether CERE-120 (adeno-associated virus serotype 2 [AAV2]-neurturin [NTN]) is effective and safe in the treatment of patients with idiopathic Parkinson's Disease. Read more about this study
- Who is it for?
- Ages 35 to 75 years
- Conditions the study is for
- Idiopathic Parkinson's Disease
- What is being tested?
- CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN])
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 58 participants
- Where
- 9 sitesUnited States
- Study dates
- Nov 2006to Nov 2008
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 (PD) based on UK Brain Bank criteria with motor complications despite adequate oral antiparkinsonian therapy.
- At least 5 years disease duration, relative to the anticipated date of surgery, since diagnosis of PD.
- Males or nonpregnant females 35-75 years of age, inclusive.
- A UPDRS motor scale score of 30 or greater in the practically defined off condition during the 30-day eligibility evaluation period.
- Stable doses of antiparkinsonian medications and parkinsonian features for the 60-day period preceding the surgical procedure.
- No conditions that would render the subject unsuitable for surgery, or that would interfere with any of the assessments of efficacy or safety in this trial.
- Subject's informed consent prior to the performance of any study-specific procedures.
Who cannot join
- Subjects with atypical or secondary parkinsonism.
- Any subject, in the judgment of the investigator, for whom participation in the study would pose a safety risk including, but not limited to, a history of any clinically significant medical, psychiatric, or laboratory abnormality.
- History of treatment of PD by any procedure involving intracranial surgery or implantation of a device.
- MRI of the brain within 12 months before the surgical 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, represent a surgical risk to the subject.
- Any disorder that precludes a surgical procedure (e.g., signs of sepsis or inadequately treated infection) or alters wound healing.
- Receipt of antiplatelet agents for at least 10 days prior to the surgical procedure.
Show all 14Show fewer
- A score of less than or equal to 27 on the Folstein Mini-Mental examination performed during the eligibility evaluation period or clinical evidence of cognitive impairment that would affect the subject's ability to sign the informed consent or perform any of the protocol required assessments.
- Chemotherapy, cytotoxic therapy, or immunotherapy within 6 weeks prior to the surgical procedure.
- Vaccinations within 30 days prior to the surgical procedure.
- History, within 2 years before the surgical procedure, of drug or alcohol abuse.
- Treatment with neuroleptics within 1 year before the surgical procedure.
- Any medical disability (e.g., severe degenerative arthritis, compromised nutritional state, peripheral neuropathy) that would interfere with the assessment of efficacy and safety in this trial or would compromise the ability of the subject to undergo study procedures (e.g., MRI, PET), or give informed consent.
- History of prior gene transfer therapy.
- Treatment with an investigational agent within 60 days before the surgical procedure.
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
United States9 sites
University of Alabama, Birmingham
Birmingham, Alabama, 35233
Rush University Medical Center
Chicago, Illinois, 60612
Cleveland Clinic
Cleveland, Ohio, 44195
Duke University Medical Center
Durham, North Carolina, 27705
Baylor College of Medicine
Houston, Texas, 77030
Mount Sinai School of Medicine
New York, New York, 10029
Pennsylvania Hospital
Philadelphia, Pennsylvania, 19107
Oregon Health and Science University
Portland, Oregon, 97239
University of California, San Francisco
San Francisco, California, 94143-0138
About this study
Official title: Multicenter, Randomized, Double-Blind, Sham Surgery-Controlled Study of CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN]) to Assess the Efficacy and Safety of Bilateral Intraputaminal (IPu) Delivery in Subjects With Idiopathic Parkinson's Disease
The purpose of this double blind study is to determine whether CERE-120 (adeno-associated virus serotype 2 [AAV2]-neurturin [NTN]) is effective and safe in the treatment of patients with idiopathic Parkinson's Disease. CERE-120 is administered via bilateral stereotactic injections targeting the putaminal region of the brain. The design of this study involves approximately 34 patients receiving CERE-120 treatment via stereotactic surgery and approximately 17 patients receiving sham stereotactic surgery (no CERE-120 administered).
- Conditions
- Idiopathic Parkinson's Disease
- Being tested
- CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN]), Sham Surgery
- 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 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN])Drug
CERE-120 5.4 x 10^11 vg
Sham SurgeryProcedure or surgery
Bilateral partial thickness burr holes placed, no intraparenchymal injections
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Intracerebral administration of CERE-120 Receives: Drug: CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN]) |
| 2 | Sham comparator | Sham Neurosurgery Receives: Procedure: Sham Surgery |
What is being measured
The primary outcome is the main question the study is designed to answer.
UPDRS Part III OFF
The UPDRS (Unified Parkinson's Disease Rating Scale) is a clinical rating scale that assesses the symptomatic burden of Parkinson's Disease. The scale has four main sections, and each item is scored from a 0 to a 4 (higher number is more severe manifestation). Part III is a subsection devoted to motor function, has 14 questions, resulting in a score range of 0 (unaffected) to 56 (severely affected). The scale is administered by a trained clinician, and patients were assessed in a practically defined "off" condition, 12 hours or more after the last administration of medication.
Measured: Change from Baseline to 12 Month Visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Nov 2006 (estimated)
- Study startNov 2006
- Primary completionNov 2008
- Study completionNov 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Idiopathic 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 58 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sangamo Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Ceregene
- Responsible party
- Sponsor
- Investigators
- Joao Siffert, M.D., Study director, Ceregene
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00400634
- Sponsor's protocol ID
- CERE-120-02
- Status last verified
- Aug 2019
- First submitted
- 15 Nov 2006
- Last updated
- 10 Nov 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Marks WJ Jr, Bartus RT, Siffert J, Davis CS, Lozano A, Boulis N, Vitek J, Stacy M, Turner D, Verhagen L, Bakay R, Watts R, Guthrie B, Jankovic J, Simpson R, Tagliati M, Alterman R, Stern M, Baltuch G, Starr PA, Larson PS, Ostrem JL, Nutt J, Kieburtz K, Kordower JH, Olanow CW. Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial. Lancet Neurol. 2010 Dec;9(12):1164-1172. doi: 10.1016/S1474-4422(10)70254-4. Epub 2010 Oct 20.
PubMed 20970382
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
- Multicenter, Randomized, Double-Blind, Sham Surgery-Controlled Study of CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN]) to Assess the Efficacy and Safety of Bilateral Intraputaminal (IPu) Delivery in Subjects With Idiopathic Parkinson's Disease
- ClinicalTrials.gov ID
- NCT00400634
- Acronym / sponsor ID
- CERE-120-02
- Conditions
- Idiopathic Parkinson's Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 58 participants (actual)
- Lead sponsor
- Sangamo TherapeuticsIndustry (pharma, biotech, devices)
- Collaborators
- Ceregene
- Study start
- Nov 2006
- Primary completion
- Nov 2008 (actual)
- Study completion
- Nov 2008 (actual)
- First posted
- 17 Nov 2006 (estimated)
- Results
- Posted 14 Aug 2012
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this double blind study is to determine whether CERE-120 (adeno-associated virus serotype 2 [AAV2]-neurturin [NTN]) is effective and safe in the treatment of patients with idiopathic Parkinson's Disease. CERE-120 is administered via bilateral stereotactic injections targeting the putaminal region of the brain. The design of this study involves approximately 34 patients receiving CERE-120 treatment via stereotactic surgery and approximately 17 patients receiving sham stereotactic surgery (no CERE-120 administered).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN])Intracerebral administration of CERE-120 |
| 2 | Sham comparator | Procedure: Sham SurgerySham Neurosurgery |
Who may qualify
- Ages
- 35 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Diagnosis of bilateral, idiopathic Parkinson's Disease (PD) based on UK Brain Bank criteria with motor complications despite adequate oral antiparkinsonian therapy.
- At least 5 years disease duration, relative to the anticipated date of surgery, since diagnosis of PD.
- Males or nonpregnant females 35-75 years of age, inclusive.
- A UPDRS motor scale score of 30 or greater in the practically defined off condition during the 30-day eligibility evaluation period.
- Stable doses of antiparkinsonian medications and parkinsonian features for the 60-day period preceding the surgical procedure.
Key exclusion criteria (14 listed)
- Subjects with atypical or secondary parkinsonism.
- Any subject, in the judgment of the investigator, for whom participation in the study would pose a safety risk including, but not limited to, a history of any clinically significant medical, psychiatric, or laboratory abnormality.
- History of treatment of PD by any procedure involving intracranial surgery or implantation of a device.
- MRI of the brain within 12 months before the surgical 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, represent a surgical risk to the subject.
- Any disorder that precludes a surgical procedure (e.g., signs of sepsis or inadequately treated infection) or alters wound healing.
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 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN])CERE-120 5.4 x 10^11 vg | Drug | 1 |
| Sham SurgeryBilateral partial thickness burr holes placed, no intraparenchymal injections | Procedure or surgery | 2 |
Primary outcome
UPDRS Part III OFF
Time frame: Change from Baseline to 12 Month Visit
The UPDRS (Unified Parkinson's Disease Rating Scale) is a clinical rating scale that assesses the symptomatic burden of Parkinson's Disease. The scale has four main sections, and each item is scored from a 0 to a 4 (higher number is more severe manifestation). Part III is a subsection devoted to motor function, has 14 questions, resulting in a score range of 0 (unaffected) to 56 (severely affected). The scale is administered by a trained clinician, and patients were assessed in a practically defined "off" condition, 12 hours or more after the last administration of medication.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Nov 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 20970382Marks WJ Jr, Bartus RT, Siffert J, Davis CS, Lozano A, Boulis N, Vitek J, Stacy M, Turner D, Verhagen L, Bakay R, Watts R, Guthrie B, Jankovic J, Simpson R, Tagliati M, Alterman R, Stern M, Baltuch G, Starr PA, Larson PS, Ostrem JL, Nutt J, Kieburtz K, Kordower JH, Olanow CW. Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial. Lancet Neurol. 2010 Dec;9(12):1164-1172. doi: 10.1016/S1474-4422(10)70254-4. Epub 2010 Oct 20.
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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.
Joao Siffert, M.D. study director
Ceregene
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN]) in people with Idiopathic Parkinson's Disease, run by Sangamo Therapeutics at 9 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 7 inclusion and 14 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 9 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 14 Aug 2012). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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.
What does the primary outcome measure?
UPDRS Part III OFF (time frame: Change from Baseline to 12 Month Visit). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00400634, 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.