A Phase 1 interventional study of Protollin in Alzheimer Disease (AD), sponsored by Brigham and Women's Hospital. Completed at 1 site in United States. Open to participants aged 60 Years to 85 Years. Per ClinicalTrials.gov, last updated 2025-09-22.
Sponsored by Brigham and Women's Hospital · Phase 1, Interventional, and Treatment
In this research study investigators aim to learn more about a new drug called Protollin as a possible new treatment for Alzheimer's Disease (AD). The primary goal is to assess the safety and tolerability.
In this research study investigators want to learn more about a new drug called nasal Protollin as a possible new treatment for Alzheimer's Disease (AD). In AD, there is accumulation in the brain of a toxic substance called amyloid. It is believed that this toxic substance causes brain cells to progressively waste away (degenerate) and die, causing a continuous decline in thinking, behavioral and social skills. Why amyloid accumulates is not completely understood.
The aim of this treatment is to remove toxic amyloid from the brain and prevent further degeneration. Although nasal Protollin has been given as part of vaccination programs, this is the first time Protollin will be given nasally (through the nose) in AD patients. Investigators aim to see if this way of giving the Protollin is safe and whether it stimulates the body's white blood cells to remove toxic amyloid from the brain and ultimately improve cognition. This will be a dose escalating (gradually increasing the dose in different subjects) study, which means we want to find the highest dose of Protollin that is safe to take. Protollin is not approved by the U.S. Food and Drug Administration (FDA). This means that Protollin can only be used in research studies.
This research study will compare Protollin to placebo. The placebo looks exactly like Protollin, but it does not contain any Protollin. During this study participants may get a placebo instead of Protollin. Placebos are used in research studies to see if the results are due to the study drug or due to other reasons.
3,675 studies on the registry are indexed under Alzheimer Disease; 869 are open to participants now.
This study's enrollment of 16 is below the median of 70 across 2,805 interventional studies indexed under Alzheimer Disease.
Browse Alzheimer Disease studies →Brigham and Women's Hospital is the lead sponsor of 1,236 studies on the registry; 224 are open to participants now.
Of its 116 completed or terminated interventional studies of FDA-regulated products, 64 (55%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Protollin 0.1 mg or placebo
Drug: Protollin
Protollin 0.5 mg or placebo
Drug: Protollin
Protollin 1.0 mg or placebo
Drug: Protollin
Protollin 1.5 mg or placebo
Drug: Protollin
For the 0.1, 0.5, and 1.0 mg dose groups, Protollin (450 μL per vial) in an aqueous buffer will be administered in two, 0.1 μL sprays, one per nostril. For the 1.5 mg dose group, Protollin (450 muL per vial) in an aqueous buffer will be administered in three, 0.1 μL sprays, two in one nostril and one in the other nostril.
Safety and tolerability of ascending doses of nasal Protollin
Safety and tolerability of ascending doses of nasal Protollin following administration of two doses one week apart in subjects, 60-85 years inclusive with Early Symptomatic Alzheimer's Disease (29-20 MMSE classification). Safety will be assessed by physical examination (including evaluation of the nose and oropharynx), laboratory studies, EKG, and elicited and spontaneously reported signs and symptoms, using the FDA Guidance for Industry: Toxicity Grading Scale for Healthy Adult and Adolescent Volunteers Enrolled in Preventive Vaccine Clinical Trials (September 2007) for the evaluation of toxicities and reactogenicity. Tolerability will be assessed by the degree of severity of both spontaneously reported and elicited symptoms and associated signs, systemically and at the site of administration following administration of nasal Protollin, if thought by the investigator to be related or possibly related to its administration.
Time frame: Day 1 (enrollment) to 180 days (end of study)
Plan to share: No
This study is completed, as verified in Sep 2025. You cannot join it, but the record below documents what was studied.
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Brigham and Women's Hospital