Randomized, Controlled Study Evaluating CERE-110 in Subjects With Mild to Moderate Alzheimer's Disease

NCT00876863 | Completed Phase 2 DMC FDA Drug

• 2 other identifiers: CERE-110-03NIH Grant 1R01AG030048-01A1
• Study Type: interventional
• Target: 49
• Locations: 1 country
• Sites: 10

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.

Conditions

Alzheimer's Disease

Keywords

Alzheimer's disease; Dementia; Memory loss; Cognitive impairment; Nerve growth factor; NGF; Neurotrophic factors; Gene therapy

Outcome Measures

Primary Outcomes (1)

• Alzheimer's Disease Assessment Scale - Cognitive Subscale (ADAS-Cog)

  24 Months

Secondary Outcomes (4)

• Neuropsychological Test Battery

  24 Months

• Mini-Mental State Examination (MMSE)

  24 Months

• Neuropsychiatric Inventory (NPI)

  24 Months

• Alzheimer's Disease Cooperative Study - Activities of Daily Living (ADCS-ADL)

  24 Months

Study Arms (2)

CERE-110

EXPERIMENTAL

CERE-110: Adeno-Associated Virus Delivery of NGF

Drug: CERE-110: Adeno-Associated Virus injection Surgery

Placebo

SHAM COMPARATOR

Placebo Surgery

Procedure: Placebo Surgery

Interventions

CERE-110: Adeno-Associated Virus injection Surgery DRUG

Injecting CERE-110 2.0 X 10\^11 vg into brain during surgical procedure.

CERE-110

Placebo Surgery PROCEDURE

Placebo Surgery

Placebo

Eligibility Criteria

• Age: 55 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

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

Sponsors & Collaborators

• Sangamo Therapeutics: lead
• Alzheimer's Disease Cooperative Study (ADCS): collaborator

Study Sites (10)

University of Alabama

Birmingham, Alabama, 35294, United States

Location

University of California, Los Angeles

Los Angeles, California, 90095, United States

Location

University of California, San Diego

San Diego, California, 92037, United States

Location

Georgetown University

Washington D.C., District of Columbia, 20007, United States

Location

Emory University

Atlanta, Georgia, 30325, United States

Location

Mount Sinai School of Medicine

New York, New York, 10029, United States

Location

Duke University

Durham, North Carolina, 27710, United States

Location

Case Western Reserve University

Cleveland, Ohio, 44120, United States

Location

Medical University of South Carolina

North Charleston, South Carolina, 29406, United States

Location

University of Utah

Salt Lake City, Utah, 84108, United States

Location

Related Publications (1)

• 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.

  PMID: 29582053 DERIVED

MeSH Terms

Conditions

Alzheimer Disease; Dementia; Memory Disorders; Cognitive Dysfunction; Hereditary Sensory and Autonomic Neuropathies

Condition Hierarchy (Ancestors)

Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Tauopathies; Neurodegenerative Diseases; Neurocognitive Disorders; Mental Disorders; Neurobehavioral Manifestations; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Cognition Disorders; Nervous System Malformations; Heredodegenerative Disorders, Nervous System; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Study Officials

• Paul S. Aisen, M.D.

  Alzheimer's Disease Cooperative Study (ADCS)

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 3, 2009
• First Posted: April 7, 2009
• Study Start: November 1, 2008
• Primary Completion: August 13, 2015
• Study Completion: August 13, 2015
• Last Updated: December 21, 2020

Record last verified: 2017-01

Locations

US (10)