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NCT00013650

Effects of an Anti-Inflammatory Drug in Alzheimer's Disease

Completed Phase 1 Last updated 2 July 2017
What this trial tests

Phase 1 trial testing Cyclophosphamate in Alzheimer's Disease in 60 participants. Completed in 21 April 2008.

Timeline
22 March 2001
21 April 2008

Quick facts

Lead sponsorNational Institute of Mental Health (NIMH)
PhasePhase 1
StatusCompleted
Study typeINTERVENTIONAL
Primary purposetreatment
Enrollment60
Start date22 March 2001
Estimated completion21 April 2008
Sites1 location across United States

Drugs / interventions tested

Conditions studied

Sponsor

National Institute of Mental Health (NIMH)

Who can join

Adults 50 to 95, any sex, with Alzheimer's Disease. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

The purpose of this study is to evaluate the effects of the drug cyclophosphamide (CY) on inflammation and immune responses in individuals with Alzheimer's Disease (AD). Inflammation and immunologic response appear to contribute to neurodegeneration in people with AD. In a process called gliosis, the brain immune cells microglia and astroglia undergo activation and possible proliferation, which promotes neuronal injury and death. Activated microglia and astroglia produce compounds that are cytotoxic to neurons, and they express molecules that greatly amplify immune and inflammatory processes in the brain. Excessive glial activation and proliferation are thought to be pivotal events that hasten the demise of synapses and neurons in AD. Fortunately, increased understanding of immune and inflammatory pathology in AD has provided new opportunities for designing disease-altering treatments for AD. Studies suggest that medications such as nonsteroidal anti-inflammatory drugs (NSAIDs) and immunomodulatory agents may have an important role in altering the course of AD. CY is a potent anti-inflammatory and immunomodulatory drug that inhibits proliferation of immune cells. This study will evaluate the effects of CY on individuals with mild to moderate AD. Participants in this study will be randomly assigned to receive either two different doses of CY or placebo (an inactive pill) for 6 months. Participants who receive placebo during the 6 months will have the option of receiving CY for an additional 6 months. Participants will undergo magnetic resonance imaging (MRI) scans of the brain. Measures of cerebral spinal fluid biomarkers or neurodegeneration, neuroinflammation, and neuroimmune activation will be taken. In addition, peripheral lymphocyte subsets and peripheral markers of inflammation will be assessed.

Publications & conference data

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

  1. Recent advances in molecular pathways and therapeutic implications targeting neuroinflammation for Alzheimer's disease.
    Dhapola R, Hota SS, Sarma P, Bhattacharyya A, et al · · 2021 · cited 254× · PMID 34813026 · DOI 10.1007/s10787-021-00889-6
  2. Targeting Microglia in Neuroinflammation: H3 Receptor Antagonists as a Novel Therapeutic Approach for Alzheimer's Disease, Parkinson's Disease, and Autism Spectrum Disorder.
    Thomas SD, Abdalla S, Eissa N, Akour A, et al · · 2024 · cited 18× · PMID 39065682 · DOI 10.3390/ph17070831
  3. Intranasal MMI-0100 Attenuates Aβ<sub>1-42</sub>- and LPS-Induced Neuroinflammation and Memory Impairments via the MK2 Signaling Pathway.
    Jiang J, Wang Z, Liang X, Nie Y, et al · · 2019 · cited 14× · PMID 31849936 · DOI 10.3389/fimmu.2019.02707
  4. Dual impact of neuroinflammation on cognitive and motor impairments in Alzheimer's disease.
    Fakorede S, Lateef OM, Garuba WA, Akosile PO, et al · · 2025 · cited 9× · PMID 40463291 · DOI 10.1177/25424823251341870
  5. HEDDI-Net: heterogeneous network embedding for drug-disease association prediction and drug repurposing, with application to Alzheimer's disease.
    Su YY, Huang HC, Lin YT, Chuang YF, et al · · 2025 · cited 3× · PMID 39891114 · DOI 10.1186/s12967-024-05938-6
  6. Neuro-Immune Crosstalk: Molecular Mechanisms, Biological Functions, Diseases, and Therapeutic Targets.
    Guo X, Liu H, Song YJ, Wang JH, et al · · 2026 · cited 2× · PMID 41583906 · DOI 10.1002/mco2.70497

Verify or expand the search:

Other trials of Cyclophosphamate

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Other National Institute of Mental Health (NIMH) trials

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