NCT00001933

Nefiracetam Therapy of Alzheimer's Type Dementia

Completed Phase 2 Last updated 3 March 2008

What this trial tests

Phase 2 trial testing Nefiracetam in Alzheimer's Disease in 50 participants. Completed in 1 January 2002.

Timeline

1 July 1999

1 January 2002

Quick facts

Lead sponsor	National Institute of Neurological Disorders and Stroke (NINDS)
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Primary purpose	treatment
Enrollment	50
Start date	1 July 1999
Estimated completion	1 January 2002
Sites	1 location across United States

Drugs / interventions tested

• Nefiracetam (NEFIRACETAM) — full drug profile →

Conditions studied

• Alzheimer's Disease — all drugs for Alzheimer's Disease →

Sponsor

National Institute of Neurological Disorders and Stroke (NINDS)

Who can join

Eligibility, any sex, with Alzheimer's Disease. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Some of the thinking difficulties of Alzheimer's patients may be due to a deficiency in a brain chemical called acetylcholine, which helps transmit messages between nerve cells. Nefiracetam is a new drug that stimulates acetylcholine. This study will test whether Nefiracetam can safely improve memory, thinking and activities of daily living in patients with mild to moderate intellectual impairment due to Alzheimer's disease.

Patients in the study must have a caregiver and designated representative. Candidates will be given a medical history and physical examination that includes a complete neurologic and neuropsychologic evaluation, blood tests, and an electrocardiogram. A chest X ray and magnetic resonance imaging (MRI) test will be done on patients who have not had these tests within the previous two years. During the 20-week study, each patient will take three pills twice a day for twenty weeks of either Nefiracetame or placebo (sugar pill). Neither the patients nor the doctors will know which patients are getting the drug and which are getting the placebo. Blood and urine tests will be done frequently throughout the study. Patients will be asked to have a spinal tap (on a voluntary basis) to measure the levels of drug in the spinal fluid, and a PET scan (a brain imaging test).

At the end of the study, patients who feel they are doing well with no side effects from the drug (or placebo) may be given the option of continuing treatment for another seven months.

Animal studies showed that Nefiracetam improved learning impairment and memory in rats with dementia. In a small study of humans, about one-fourth of patients who were given a low dose of the drug had improved intellectual function, and about one-half who received a higher dose improved.

Publications & conference data

5 peer-reviewed publications reference this trial (live from Europe PMC):

1. NMDA receptors in clinical neurology: excitatory times ahead.
   Kalia LV, Kalia SK, Salter MW. · · 2008 · cited 307× · PMID 18635022 · DOI 10.1016/s1474-4422(08)70165-0
2. Therapeutics of Neurotransmitters in Alzheimer's Disease.
   Kandimalla R, Reddy PH. · · 2017 · cited 161× · PMID 28211810 · DOI 10.3233/jad-161118
3. Allosteric Modulation of Ionotropic Glutamate Receptors: An Outlook on New Therapeutic Approaches To Treat Central Nervous System Disorders.
   Brogi S, Campiani G, Brindisi M, Butini S. · · 2019 · cited 31× · PMID 30891118 · DOI 10.1021/acsmedchemlett.8b00450
4. A Systematic Review on Drugs Acting as Nicotinic Acetylcholine Receptor Agonists in the Treatment of Dementia.
   Crestini A, Carbone E, Rivabene R, Ancidoni A, et al · · 2024 · cited 11× · PMID 38334629 · DOI 10.3390/cells13030237
5. Neurotransmitters and Immunity: Molecular Mechanisms, Biological Functions, Diseases, and Potential Therapeutic Targets.
   Li G, Li F, Tang Y, Guo S, et al · · 2025 · cited 1× · PMID 41415714 · DOI 10.1002/mco2.70556