Idiopathic Parkinson's DiseasePhase 1/2NCT00985517
Safety and Efficacy of CERE-120 in Subjects With Parkinson's Disease
A Phase 1/2 study testing CERE-120: Adeno-Associated Virus Delivery of Neurturin in people with Idiopathic Parkinson's Disease, run by Sangamo Therapeutics at 11 sites in United States.
Trial summary
The purpose of this study was to evaluate the safety and potential benefits of CERE-120 in the treatment of Parkinson's disease. Read more about this study
- Who is it for?
- Ages 35 to 70 years
- Conditions the study is for
- Idiopathic Parkinson's Disease
- What is being tested?
- CERE-120: Adeno-Associated Virus Delivery of Neurturin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 57 participants
- Where
- 11 sitesUnited States
- Study dates
- 29 Oct 2009to 16 Nov 2017
- Phase
- Phase 1/2
- 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 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Males and females, ages 35 to 70 years old (inclusive)
- A diagnosis of idiopathic Parkinson's disease based on UK Brain Bank criteria, including bradykinesia and at least 1 of the following PD features: resting tremor or rigidity
- A Hoehn and Yahr score of no greater than 3 in the "off" condition at Screening
- A robust response to dopaminergic therapy as judged by the investigator based on the UPDRS Part III: Motor Examination
- Experiencing motor complications despite adequate antiparkinsonian therapy
- A stable, optimized regimen of antiparkinsonian medications and stable parkinsonian features for at least 6 weeks prior to Screening
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- Subject is willing not to undergo DBS for at least 12 months after the study surgical procedure (Phase 1 subjects) or while the study is blinded (Phase 2 subjects) and the investigator believes that this is medically acceptable
- Medically fit to undergo the study surgical procedure as determined by medical history, clinical and laboratory evaluations, and any other pre-surgical evaluations that are standard at the institution where the subject will undergo surgery
- Physically and mentally capable of performing all protocol-specified assessments and complying with the study visit schedule
- Subjects must be able to travel to study visits alone or able to identify a partner or caregiver who agrees to accompany the subject to the study visits
- Females of childbearing potential must have a negative β-HCG pregnancy test at Screening and again before surgery on Day 0
- All subjects, both male and female, must agree to practice adequate barrier method contraception for at least 6 months after the surgical procedure
- Provides written informed consent to participate before any study-specific procedures are conducted
Who cannot join
- Atypical or secondary parkinsonism, including, but not limited to, multiple system atrophy (MSA) or progressive supranuclear palsy
- Any subject for whom participation in the study would pose a substantial safety risk
- Any condition that would compromise the ability of the subject to undergo study procedures, including allergy to gadolinium
- Presence of any known brain abnormality that could interfere with the assessment of safety or efficacy or represents a surgical risk to the subject
- Evidence of significant brain atrophy on the Baseline MRI
- History of any cancer other than basal or squamous cell skin cancer within the 3 years prior to Screening
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- Any chemotherapy, cytotoxic therapy, or immunotherapy (e.g., IL-2, IL-12, interferon) within the 3 months prior to Screening
- Any prior treatment for PD with a procedure involving intracranial surgery or implantation of a device (e.g. DBS, pallidotomy)
- Any prior treatment for a neurological or psychiatric disorders with a procedure involving the implantation of a device (e.g. spinal cord stimulator, vagus nerve stimulator)
- History of any prior gene transfer therapy
- Treatment with any investigational agent within the 3 months prior to Screening
- Anticipated need for antiplatelet agents or anticoagulation therapy, including gingko biloba, during the 10 days prior to the projected surgery date
- Any vaccinations within the 30 days prior to the projected surgery date Note: Vaccinations are not allowed for 30 days after the surgical procedure, unless deemed necessary by the investigator for the subject's well-being
- Not likely to be available for the duration of the trial, likely to be noncompliant with the protocol, or who are deemed unsuitable by the investigator for any other reason
- Participation in a previous surgical treatment study for Parkinson's disease
Trial locations and contacts
11 sites in 1 country.
11 sites in 1 country
United States11 sites
Emory University
Atlanta, Georgia
University of Alabama at Birmingham
Birmingham, Alabama
Rush University Medical Center
Chicago, Illinois
Duke University
Durham, North Carolina
Baylor College of Medicine
Houston, Texas
Beth Israel Medical Center
New York, New York
Columbia University Medical Center
New York, New York
Mount Sinai Medical Center
New York, New York
Stanford School of Medicine
Palo Alto, California
University of Pennsylvania
Philadelphia, Pennsylvania
University of California, San Francisco
San Francisco, California
About this study
Official title: A Phase 1/2 Trial Assessing the Safety and Efficacy of Bilateral Intraputaminal and Intranigral Administration of CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN]) in Subjects With Idiopathic Parkinson's Disease
The purpose of this study was to evaluate the safety and potential benefits of CERE-120 in the treatment of Parkinson's disease.
Read the detailed description
CERE-120 is an experimental drug that consists of an adeno-associated virus (AAV) that was engineered to carry the human gene for neurturin, a neurotrophic (growth) factor. Similar to other growth factors (such as GDNF), neurturin is capable of restoring function and protecting brain cells from further damage. The virus used in CERE-120 is not known to cause disease in people.
CERE-120 is delivered directly to the brain cells most affected in Parkinson's disease - the dopamine producing neurons. CERE-120 is injected during brain surgery. Once in place, CERE-120 continuously produces neurturin.
During the first, open-label, part of the study (Phase 1), subjects with Parkinson's disease received CERE-120 at one of two dose levels. In the second part of the study (Phase 2), subjects were randomized 1:1 to receive CERE-120 or a "sham" surgery where no medication was injected.
Participants in both phases of the study were followed for up to five years after surgery.
- Conditions
- Idiopathic Parkinson's Disease
- Being tested
- CERE-120: Adeno-Associated Virus Delivery of Neurturin, Sham Surgery
- Keywords
- Parkinson's disease, Movement Disorders, Gene Therapy, Neurotrophic Factors, GDNF, Neurturin, DBS, Dopamine
- Medical subject headings
- Parkinson Disease, Movement Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CERE-120: Adeno-Associated Virus Delivery of NeurturinBiological
Also called AAV2-Neurturin
Sham SurgeryProcedure or surgery
| Study group | Role | What they receive |
|---|---|---|
| Phase 1: Cohort 1 | Experimental | CERE-120 9.4 x 10^11 vg, injected by a neurosurgeon directly in the substantia nigra and in the putamen Receives: Biological: CERE-120: Adeno-Associated Virus Delivery of Neurturin |
| Phase 1: Cohort 2 | Experimental | CERE-120 2.4 x 10^12 vg, injected by a neurosurgeon directly in the substantia nigra and in the putamen Receives: Biological: CERE-120: Adeno-Associated Virus Delivery of Neurturin |
| Phase 2: CERE-120 | Experimental | CERE-120 2.4 x 10^12 vg, injected by a neurosurgeon directly in the substantia nigra and in the putamen Receives: Biological: CERE-120: Adeno-Associated Virus Delivery of Neurturin |
| Phase 2: Sham Surgery | Sham comparator | Neurosurgical procedure that mimics the procedure for CERE-120 delivery. No injections were administered during sham surgery. Receives: Procedure: Sham Surgery |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Who Received CERE-120 Treatment
Number of Participants who received CERE-120 Treatment
Measured: 5 years
Phase 1 and Phase 2: Change From Baseline in Motor Examination Part III Total Score in the "Off" Condition for the CERE-120 Group as Compared to the Sham Surgery Control Group at the Last Double-blind Assessment.
UPDRS is a tool to evaluate the impact of symptomatic and potential disease modifying treatments. Part III focuses on 14 motor functions, assessing each on a scale from 0 (normal) to 4 (severe) with total score 0 - 56. The 5 Feb 2003 version of the UPDRS Rating was used in this trial. The total score is the sum of 14 motor functions. Change from baseline in total score is reported.
The last double blind assessment for each subject is defined as the most recent assessment at the time when the final randomized subject completes the Month 15 Visit. The length of the double-blind follow-up period for each subject ranged from 15 to 24 months, depending on the enrollment rate.
Measured: Baseline, Months 15, 18, 21 and 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Sep 2009 (estimated)
- Study start29 Oct 2009
- Primary completion9 Nov 2014
- Study completion16 Nov 2017
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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- In the first part of the study (Phase 1), 2 escalating dose cohorts received CERE-120. In the second part of the study (Phase 2), subjects were randomized 1:1 to receive CERE-120 or Sham Surgery.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 57 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sangamo Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00985517
- Sponsor's protocol ID
- CERE-120-09
- Status last verified
- Apr 2020
- First submitted
- 25 Sep 2009
- Last updated
- 16 Apr 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Bartus RT, Baumann TL, Siffert J, Herzog CD, Alterman R, Boulis N, Turner DA, Stacy M, Lang AE, Lozano AM, Olanow CW. Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients. Neurology. 2013 Apr 30;80(18):1698-701. doi: 10.1212/WNL.0b013e3182904faa. Epub 2013 Apr 10.
PubMed 23576625
Study at a glance
From the registry record, last updated by the sponsor on 16 Apr 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2 Trial Assessing the Safety and Efficacy of Bilateral Intraputaminal and Intranigral Administration of CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN]) in Subjects With Idiopathic Parkinson's Disease
- ClinicalTrials.gov ID
- NCT00985517
- Acronym / sponsor ID
- CERE-120-09
- Conditions
- Idiopathic Parkinson's Disease
- Keywords
- Parkinson's disease, Movement Disorders, Gene Therapy, Neurotrophic Factors, GDNF, Neurturin, DBS, Dopamine
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 57 participants (actual)
- Lead sponsor
- Sangamo TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 29 Oct 2009 (actual)
- Primary completion
- 9 Nov 2014 (actual)
- Study completion
- 16 Nov 2017 (actual)
- First posted
- 28 Sep 2009 (estimated)
- Results
- Posted 26 Mar 2020
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 study was to evaluate the safety and potential benefits of CERE-120 in the treatment of Parkinson's disease.
Detailed description
CERE-120 is an experimental drug that consists of an adeno-associated virus (AAV) that was engineered to carry the human gene for neurturin, a neurotrophic (growth) factor. Similar to other growth factors (such as GDNF), neurturin is capable of restoring function and protecting brain cells from further damage. The virus used in CERE-120 is not known to cause disease in people.
CERE-120 is delivered directly to the brain cells most affected in Parkinson's disease - the dopamine producing neurons. CERE-120 is injected during brain surgery. Once in place, CERE-120 continuously produces neurturin.
During the first, open-label, part of the study (Phase 1), subjects with Parkinson's disease received CERE-120 at one of two dose levels. In the second part of the study (Phase 2), subjects were randomized 1:1 to receive CERE-120 or a "sham" surgery where no medication was injected.
Participants in both phases of the study were followed for up to five years after surgery.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
- Model description
- In the first part of the study (Phase 1), 2 escalating dose cohorts received CERE-120. In the second part of the study (Phase 2), subjects were randomized 1:1 to receive CERE-120 or Sham Surgery.
| Arm | Type | Receives |
|---|---|---|
| Phase 1: Cohort 1 | Experimental | Biological: CERE-120: Adeno-Associated Virus Delivery of NeurturinCERE-120 9.4 x 10^11 vg, injected by a neurosurgeon directly in the substantia nigra and in the putamen |
| Phase 1: Cohort 2 | Experimental | Biological: CERE-120: Adeno-Associated Virus Delivery of NeurturinCERE-120 2.4 x 10^12 vg, injected by a neurosurgeon directly in the substantia nigra and in the putamen |
| Phase 2: CERE-120 | Experimental | Biological: CERE-120: Adeno-Associated Virus Delivery of NeurturinCERE-120 2.4 x 10^12 vg, injected by a neurosurgeon directly in the substantia nigra and in the putamen |
| Phase 2: Sham Surgery | Sham comparator | Procedure: Sham SurgeryNeurosurgical procedure that mimics the procedure for CERE-120 delivery. No injections were administered during sham surgery. |
Who may qualify
- Ages
- 35 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- Males and females, ages 35 to 70 years old (inclusive)
- A diagnosis of idiopathic Parkinson's disease based on UK Brain Bank criteria, including bradykinesia and at least 1 of the following PD features: resting tremor or rigidity
- A Hoehn and Yahr score of no greater than 3 in the "off" condition at Screening
- A robust response to dopaminergic therapy as judged by the investigator based on the UPDRS Part III: Motor Examination
- Experiencing motor complications despite adequate antiparkinsonian therapy
Key exclusion criteria (15 listed)
- Atypical or secondary parkinsonism, including, but not limited to, multiple system atrophy (MSA) or progressive supranuclear palsy
- Any subject for whom participation in the study would pose a substantial safety risk
- Any condition that would compromise the ability of the subject to undergo study procedures, including allergy to gadolinium
- Presence of any known brain abnormality that could interfere with the assessment of safety or efficacy or represents a surgical risk to the subject
- Evidence of significant brain atrophy on the Baseline MRI
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 Delivery of NeurturinAlso: AAV2-Neurturin | Biological | Phase 1: Cohort 1, Phase 1: Cohort 2, Phase 2: CERE-120 |
| Sham Surgery | Procedure or surgery | Phase 2: Sham Surgery |
Primary outcome
Number of Participants Who Received CERE-120 Treatment
Time frame: 5 years
Number of Participants who received CERE-120 Treatment
Phase 1 and Phase 2: Change From Baseline in Motor Examination Part III Total Score in the "Off" Condition for the CERE-120 Group as Compared to the Sham Surgery Control Group at the Last Double-blind Assessment.
Time frame: Baseline, Months 15, 18, 21 and 24
UPDRS is a tool to evaluate the impact of symptomatic and potential disease modifying treatments. Part III focuses on 14 motor functions, assessing each on a scale from 0 (normal) to 4 (severe) with total score 0 - 56. The 5 Feb 2003 version of the UPDRS Rating was used in this trial. The total score is the sum of 14 motor functions. Change from baseline in total score is reported. The last double blind assessment for each subject is defined as the most recent assessment at the time when the final randomized subject completes the Month 15 Visit. The length of the double-blind follow-up period for each subject ranged from 15 to 24 months, depending on the enrollment rate.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Apr 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 23576625Bartus RT, Baumann TL, Siffert J, Herzog CD, Alterman R, Boulis N, Turner DA, Stacy M, Lang AE, Lozano AM, Olanow CW. Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients. Neurology. 2013 Apr 30;80(18):1698-701. doi: 10.1212/WNL.0b013e3182904faa. Epub 2013 Apr 10.
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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.
The record lists no contact. Use the official record to reach the sponsor.
11 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing CERE-120: Adeno-Associated Virus Delivery of Neurturin in people with Idiopathic Parkinson's Disease, run by Sangamo Therapeutics at 11 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 13 inclusion and 15 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 16 Apr 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 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 combined 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.
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 26 Mar 2020). 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?
Number of Participants Who Received CERE-120 Treatment (time frame: 5 years). The study lists 2 primary outcomes in all. 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 NCT00985517, 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 16 Apr 2020.