Clinical trial NCT00876863: Randomized, Controlled Study Evaluating CERE-110 in Subjects With Mild to Moderate Alzheimer's Disease
Official title: A Double-Blind, Placebo-Controlled (Sham Surgery), Randomized, Multicenter Study Evaluating CERE-110 Gene Delivery in Subjects With Mild to Moderate Alzheimer's Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Alzheimer's Disease
Keywords: Alzheimer's disease
Dementia
Memory loss
Cognitive impairment
Nerve growth factor
NGF
Neurotrophic factors
Gene therapy
Lead sponsor: Sangamo Therapeutics
Sponsor class: INDUSTRY
Collaborators: name: Alzheimer's Disease Cooperative Study (ADCS)
First posted: 2009-04-07
Enrollment: 49
Sex: ALL
Minimum age (years): 55
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the potential benefits of CERE-110 in the treatment of Alzheimer's disease. CERE-110 is an experimental drug that is designed to help nerve cells in the brain function better. CERE-110 uses a virus to transfer a gene that makes Nerve Growth Factor (NGF), a protein that may make nerve cells in the brain healthier and protect them from dying. The virus used in CERE-110 does not cause disease in people. CERE-110 has been carefully studied in laboratory animals and is in the early stages of being tested in people.

Fifty patients with mild to moderate Alzheimer's disease will participate in this study. Half of the study subjects will have CERE-110 injected into the brain during a surgical procedure, while the other half will undergo a "placebo" surgery where no medication will be injected. All study participants will be followed for at least two years after surgery.


Eligibility criteria: Inclusion Criteria:

* Diagnosis of mild to moderate Alzheimer's disease
* Approved medications for Alzheimer's disease may be taken if the dose has been stable for 3 months
* A study partner who can attend all study visits
* Good general health
* Medically able to undergo neurosurgery

Exclusion Criteria:

* Significant neurological disease other than Alzheimer's disease
* Significant depression or other psychiatric disorder
* Unstable medical conditions

Primary outcomes:
measure: Alzheimer's Disease Assessment Scale - Cognitive Subscale (ADAS-Cog)
timeFrame: 24 Months
Secondary outcomes:
measure: Neuropsychological Test Battery
timeFrame: 24 Months
measure: Mini-Mental State Examination (MMSE)
timeFrame: 24 Months
measure: Neuropsychiatric Inventory (NPI)
timeFrame: 24 Months
measure: Alzheimer's Disease Cooperative Study - Activities of Daily Living (ADCS-ADL)
timeFrame: 24 Months
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama
state: Alabama
city: Los Angeles
country: United States
facility: University of California, Los Angeles
state: California
city: San Diego
country: United States
facility: University of California, San Diego
state: California
city: Washington
country: United States
facility: Georgetown University
state: District of Columbia
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: New York
country: United States
facility: Mount Sinai School of Medicine
state: New York
city: Durham
country: United States
facility: Duke University
state: North Carolina
city: Cleveland
country: United States
facility: Case Western Reserve University
state: Ohio
city: North Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
Study officials:
affiliation: Alzheimer's Disease Cooperative Study (ADCS)
name: Paul S. Aisen, M.D.
role: STUDY_DIRECTOR
References:
citation: Rafii MS, Tuszynski MH, Thomas RG, Barba D, Brewer JB, Rissman RA, Siffert J, Aisen PS; AAV2-NGF Study Team. Adeno-Associated Viral Vector (Serotype 2)-Nerve Growth Factor for Patients With Alzheimer Disease: A Randomized Clinical Trial. JAMA Neurol. 2018 Jul 1;75(7):834-841. doi: 10.1001/jamaneurol.2018.0233.