NPC1 for Alzheimer's Disease
(NPC1-AD Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to assess how the treatment NPC1 affects Alzheimer's Disease (AD) over six months. Researchers seek to determine if NPC1, a combination of parthenolide and ipriflavone, can benefit the condition by examining specific markers in the body linked to Alzheimer's. The trial is open-label, meaning all participants know the treatment being administered. Individuals diagnosed with Alzheimer's, Mild Cognitive Impairment, or those experiencing memory and thinking issues might be suitable candidates for this trial. As a Phase 1, Phase 2 trial, this research focuses on understanding how NPC1 works in people and measuring its effectiveness in an initial group, offering participants a chance to contribute to early-stage treatment development.
Do I need to stop taking my current medications for the trial?
You may need to stop certain medications or supplements before joining the trial. If you're taking dietary supplements with parthenolide or ipriflavone, you'll need a 1-month break before starting. Some medications must be stable for at least 2-3 months, and others may need to be stopped if they were used recently.
Is there any evidence suggesting that NPC1 is likely to be safe for humans?
Research has shown that the treatment NPC1, which includes parthenolide and ipriflavone, is generally well-tolerated. In earlier studies, most reported side effects were mild, with 80% being mild and related to the condition under study. This suggests the treatment is relatively safe, though some moderate side effects were also noted.
As this trial is in the early stages, it primarily focuses on understanding safety and tolerability. While initial data appears promising, researchers are still learning important details about how the treatment works in humans.12345Why do researchers think this study treatment might be promising?
Researchers are excited about NPC1 for Alzheimer's disease because it offers a novel approach to treatment. Unlike standard options like cholinesterase inhibitors and memantine, NPC1 combines the active ingredients Ipriflavone and Parthenolide. These components are thought to potentially influence inflammation and neuroprotection, offering a fresh angle on tackling Alzheimer's. Additionally, the specific dosing regimen—taking capsules both in the morning and evening—could provide more consistent therapeutic effects throughout the day. This new strategy might bring hope for improving symptoms or slowing disease progression in ways current treatments do not.
What evidence suggests that NPC1 might be an effective treatment for Alzheimer's Disease?
Research suggests that NPC1, which includes substances like parthenolide and ipriflavone, might help protect the brain. In this trial, participants will receive NPC1 as an open-label intervention. Studies have shown that compounds similar to estrogen, such as ipriflavone, can protect brain cells and may slow down Alzheimer's disease. These compounds might work by reducing oxidative stress, a harmful process linked to Alzheimer's. Early findings indicate that NPC1 could affect Alzheimer's markers, which are signs of the disease's presence and progression. Although direct evidence in humans remains limited, initial research provides a hopeful basis for NPC1's potential in addressing Alzheimer's disease.16789
Who Is on the Research Team?
Gene L. Bowman, ND, MPH
Principal Investigator
Massachusetts General Hospital
Are You a Good Fit for This Trial?
Adults aged 55+ with early signs of Alzheimer's, such as memory loss or mild cognitive impairment, who have specific biomarkers indicating Alzheimer's pathology. Participants need a study partner and stable health without major psychiatric disorders, uncontrolled hypertension, recent cancer (except certain types), or significant cardiovascular disease.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Lead-in observational period
Serial blood draws to characterize pre-treatment biomarker status
Treatment
Participants receive NPC1 (parthenolide and ipriflavone) for 6 months
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- NPC1
Trial Overview
The trial is testing NPC1—a combination of parthenolide and ipriflavone—on adults showing early biological signs of Alzheimer's. Over six months, researchers will monitor how this treatment affects Alzheimer's biomarkers compared to a placebo/control group.
How Is the Trial Designed?
2
Treatment groups
Active Control
Placebo Group
Three capsules of NPC1 taken daily. One 300 mg cap of Ipriflavone and 1 cap of 2.5 mg Parthenolide in the morning; One 300 mg cap of Ipriflavone taken in the evening)
Serial blood draws to characterize pre-treatment biomarker status
Find a Clinic Near You
Who Is Running the Clinical Trial?
Massachusetts General Hospital
Lead Sponsor
Citations
Biomarker-based Trial of NPC-1 for Alzheimer's Pathology
The goal of this early phase, open-label, single arm clinical trial is to determine the 6-month effects and tolerability of NPC1 ...
The neuroprotective effects of ipriflavone against H ₂O ₂ ...
Estrogenic compounds have been shown to have great potential for the treatment of Alzheimer's disease as demonstrated by its ability to ...
Antineoplastics for treating Alzheimer's disease and ...
Recent clinical trials demonstrated that lecanemab, aducanumab, and donenemab remove amyloid plaques and reduce cognitive deterioration in AD ...
Exploring the Impact of Bioactive Compounds Found in ...
Current treatments provide symptomatic relief, but do not halt the disease's progression. OS plays a crucial role in AD pathogenesis by promoting Aβ ...
5.
news-medical.net
news-medical.net/news/20250204/International-trial-aims-to-prevent-Alzheimere28099s-in-high-risk-young-adults.aspxInternational trial aims to prevent Alzheimer's in high-risk ...
Two amyloid-targeting drugs were shown to slow symptoms of the disease and have now been approved by the Food and Drug Administration (FDA) as ...
6.
cyclotherapeutics.com
cyclotherapeutics.com/cyclo-therapeutics-presents-encouraging-preliminary-safety-data-from-ongoing-pivotal-phase-3-study-and-substudy-for-the-treatment-of-niemann-pick-disease-type-c1-npc1/News
A total of 625 Adverse Events (AEs) were reported, 80% were Grade 1 (mild) in severity most related to underlying NPC disease; Grade 2 were 16.7 ...
Miglustat in Alzheimer's Disease Associated With ...
Several studies have previously demonstrated an increased risk of dementia and brain amyloid deposition in individuals with heterozygous NPC1 ...
Familial Alzheimer's disease associated with heterozygous ...
Our data support the hypothesis that heterozygous NPC1 mutations leading to altered lipid metabolism are associated with late-onset neurodegeneration and could ...
9.
rafaelholdings.irpass.com
rafaelholdings.irpass.com/Rafael-Holdings-Announces-Continuation-of-its-Phase-3-Study-for-the-Treatment-of-Niemann-Pick-Disease-Type-C1--NPC1--Following-Independent-Data-Monitoring-Committee--DMC--Review-of-Prespecified-48-WeeRafael Holdings Announces Continuation of its Phase 3 ...
NPC1 is characterized by an inability for cells to transport and process cholesterol, resulting in excessive amounts of cholesterol accumulating ...
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