Biomarker-based Trial of NPC-1 for Alzheimer's Pathology
Early-phase Biomarker-based Trial of NPC-1 for Alzheimer's Disease Pathology
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 55 Years and older
- Sex
- All sexes
- Healthy volunteers
- Not accepted
- Compensation
- Not stated
Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.
What to expect
No one can promise a trial will help you personally — that is what the research is trying to find out.
What participation involves
Not stated in the study description — ask the study team about visits and procedures.
- Compensation
- Not stated
- Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
- Participants are monitored closely by the research team throughout the study.
- Some participants may receive access to an investigational treatment, which is still being studied.
- Your participation helps researchers learn whether this approach could help future patients.
About this study
In plain language
This early study tests the safety and side effects of NPC1, a mix of parthenolide and ipriflavone, and its effects on blood signs of Alzheimer's disease. It is for adults age 55 or older who have memory or thinking concerns, mild thinking problems, or Alzheimer's dementia, plus test signs of Alzheimer's disease.
This early phase, open label, single arm clinical trial will determine the intraindividual safety, tolerability and effects of NPC1 (parthenolide and ipriflavone) on blood-based biomarkers of Alzheimer's disease (AD) pathology among adults with subjective cognitive decline, mild cognitive impairment, or Alzheimer's disease and objective indicators of seeding AD pathology
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- Age 55 or older, with memory or thinking concerns, mild thinking problems, or Alzheimer's dementia.
- Meet study limits on memory and daily function tests: Clinical Dementia Rating of 2 or less and Mini Mental Status Exam score of 16 or more.
- Have a modified Hachinski score, which checks for blood vessel causes of thinking problems, of 4 or less. The exclusion list also rules out scores of 4 or more; ask the study team about this overlap.
- Score below 6 on the 15-question Geriatric Depression Scale, showing no major signs of depression.
- Other medicines, including medicines for Alzheimer's symptoms, must be stable for at least 1 month before screening.
- Have signs of Alzheimer's on blood tests: an abeta42/40 ratio of 0.12 or less AND p-tau217 of at least 2.1 pg/mL; OR a positive amyloid PET brain scan with a centiloid score of at least 20 from routine care.
- Have enough sight and hearing to finish testing, and health that allows you to finish the study.
- Have a study partner in contact at least 1 hour a day or 1 day a week to report changes and side effects and help with taking the study drug.
Who can't join
- Memory or daily function scores outside the study limits; or a serious brain or nervous system disease in the past 2 years that would make it hard to finish the study.
- Alcohol or drug abuse in the past 2 years; major depression or anxiety in the past year; or schizophrenia, bipolar disorder, or another major mental health disorder.
- Abnormal tests suggesting a treatable cause of memory loss, such as low vitamin B12, thyroid disease, or a urinary tract infection; an INR blood-clotting score above 1.2; or platelets below 100 K/uL.
- Unstable or severe heart disease; uncontrolled blood pressure above 160/100; symptoms from blood pressure dropping when standing; or diabetes that needs insulin shots.
- Cancer in the past 5 years, with listed exceptions for certain prostate and skin cancers; or an illness needing more than 1 clinician visit each month.
- Use of certain Alzheimer's antibody drugs, supplements with parthenolide or ipriflavone, warfarin, or certain medicines for mental health, Parkinson's disease, inflammation, or pain. Some may be allowed after a waiting period. Medicines that affect the brain must have stable doses for at least 1 month.
- Taking part in another Alzheimer's treatment trial. The study doctor will review participation in trials for other conditions.
- Currently pregnant. Women who could become pregnant will have pregnancy blood tests at screening and at months 2, 5, and 8.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
Inclusion Criteria: 1. Age 55 and older, male and female; 2. Subjective Cognitive Impairment or MCI or AD dementia per the Revised criteria for diagnosis and staging of Alzheimer's disease criteria; 3. Clinical Dementia Rating \< or = to 2 and Mini Mental Status Exam \> or = to 16; 4. Modified Hachinski Ischemic Score \< or = to 4 5. Geriatric Depression Scale - 15 \< 6 documenting absence from significant depressive syndromes 6. Other medications including non-disease modifying for MCI and AD (e.g., acetylcholine esterase inhibitor, N-methyl D-aspartate receptor antagonist) stable \> or = to 1-month prior to screening visit; 7. Biomarker evidence of AD pathology: Plasma abeta42/40 ratio \< or = to 0.12 AND Plasma p-tau217 \> or = to 2.1 pg/mL OR Amyloid PET positive (centiloid \> or = to 20) as part of routine clinical care. 8. Sufficient vision and hearing to complete all tests 9. Study partner available with frequent (at least 1 hour/day or 1 day/week) contact with participant to provide collateral information about cognition, daily functioning, adverse events reporting, and support for study drug intake 10. General health status that will not interfere with the ability to complete the prospective study (these conditions are listed below in the study exclusion list) Exclusion Criteria: 1. CDR \> 2 MMSE \< 16; 2. Other significant CNS disease within the last 2 years (i.e., brain tumor, seizure disorder, subdural hematoma, cranial arteritis, cortical stroke) That would impact the participant's ability to complete the study; 3. Alcohol or substance abuse according to DSM-IV criteria within the last 2 years 4. Major depressive disorder or anxiety within the last year; Schizophrenia, bipolar disorder or other major psychiatric disorder defined by DSM-IV criteria 5. Abnormal labs indicating potential reversible causes of dementing illness such as vitamin B12 deficiency, thyroid disease, or UTI (documented bacterial colonization is acceptable) or INR \> 1.2 (1.3 or higher) or platelets \< 100 K/uL at screening 6. Unstable or significantly symptomatic CVD (e.g. CAD with frequent angina, CHF with dyspnea at rest) 7. Hypertension: defined as uncontrolled BP \> 160/100 8. Clinical symptomatic orthostatic hypotension 9. Diabetes mellitus that requires insulin injections 10. Hachinski ischemic score \> or = to 4 11. Cancer within the last 5 years, apart from localized prostate cancer (Gleason Grade \< 3) and non-metastatic skin cancers (melanoma). 12. Illness that requires \>1 visit /month to a clinician 13. Medications and dietary supplements: * a. AD disease modifying monoclonal antibody treatment e.g., donanemab (Kisunla) or lecanemab (Leqembi). Wash out period of five half-lives prior to screening visit is permitted e.g., \> or = 60 days since last dose of donanemab and \> or = 35 days since last dose of lecanemab) * b. Dietary supplements containing parthenolide or ipriflavone (1-month wash out period prior to screening is permitted) * c. CNS active meds that have not been on stable doses for at least 1 month prior to screening visit e.g., cimetidine, beta-blockers, and SSRIs * d. Neuroleptics, antiparkinsonian agents, systemic corticosteroids, and narcotic analgesics; in the case where these were used for a self-limited time they must have been discontinued for a period of five half-lives prior to screening visit * e. Over the counter supplements are not by themselves exclusionary, however, participants are asked not to change the dosing regimen over the course of the trial unless medically indicated; the presence and dose of these product are recorded * f. Anticoagulation therapy: Vitamin K antagonist: warfarin (Coumadin, jantoven) 14. Participation in any Alzheimer's Disease interventional trial. Participation in other non-AD related trials will be evaluated at the discretion of the investigator 15. Currently pregnant. Positive pregnancy tests during the course of the trial will be evaluated at the discretion of the investigator. Women of Child Bearing Potential (WOCBP) For the purposes of this study, women of childbearing potential are defined as all women who are capable of becoming pregnant, unless they meet one of the following criteria: 1. 12-months post-menopausal 2. Post-hysterectomy/surgically sterile If a female Participant does not meet either of these criteria they will be considered of childbearing potential and will have a serum pregnancy test performed at Screening, Visit 3 (2 months), Visit 6 (5 months), and Visit 10 (8 months).
Study sites (1)
MA
- Massachusetts General Hospital · Boston, MA 02129Brianna Wang, MS · Study contact · 857-282-1520 · npc1clinicaltrial@mgb.orgMore trials in Boston