testing the safety and preliminary efficacy of the new drug ory-2001 in mild to moderate alzheimer's disease

Conditions: Mild to Moderate Alzheimer's Disease

Summary: this is a phase iia study assessing the safety, tolerability and preliminary efficacy of ory-2001 in mild to moderate alzheimer's disease patients.

this phase iia study is a double-blind, randomized, parallel-group and multicenter study with a placebo-controlled 24-week treatment period followed by a no placebo-controlled 24-week extension period. it is planned to randomise 25 patients. in the double-blind placebo-controlled treatment period, all patients will be randomized between two doses of ory-2001 and placebo. in the double-blind no placebo-controlled extension period, patients in the placebo arm will be re-allocated in one of the two different dose levels of ory-2001. randomization will be stratified by cognitive impairment severity. an independent data monitoring committee (dmc) will review un-blinded safety data throughout the study.

Eligibility criteria:
inclusion criteria: * probable alzheimer's disease (ad) diagnosed according to national institute of neurological and communicative disorders and stroke and the alzheimer's disease and related disorders association (nincds-adrda) criteria * mmse score at screening and baseline visits of at least 16 and not greater than 26 * evidence of the ad pathophysiological process indicated by decreased levels of amyloid antigen binding (ab) and increased levels of total tau protein or phospho-tau protein in cerebrospinal fluid (csf) * outpatient consulting a general practitioner, or a psychiatrist/neurologist/geriatrician * knowledgeable and reliable close relative/caregiver who will accompany the patient to all clinic visits during the study * daily treatment with the same acetylcholinesterase inhibitor on a stable dose * fertile male and female must use highly effective contraception, from the screening visit until 90 days after last dose. * signed informed consent by patient (or legal representative, if applicable) and a close relative/caregiver prior to the initiation of any study specific procedure exclusion criteria: * failure to perform screening or baseline examinations * hospitalization or change of concomitant medication 1 month prior to screening visit or during screening period * clinical, laboratory or neuroimaging findings consistent with: 1. other primary degenerative dementia; 2. other neurodegenerative condition; 3. cerebrovascular disease; 4. other central nervous system diseases; * a current diagnostic and statistical manual-5 (dsm-5) diagnosis of major depression, schizophrenia or bipolar disorder * positive results for tuberculosis, human immunodeficiency virus (hiv), hepatitis c or hepatitis b (hepatitis b surface antigen \[hbsag\]) serology at the screening visit * clinically significant, advanced or unstable disease that may interfere with evaluation. * disability that may prevent the patients from completing all study requirements. * chronic drug intake of forbidden concomitant medication. * treatment with anti-amyloid beta or anti-tau protein monoclonal antibodies or other disease modifying strategies within three months or five half-lives, whichever is longer, prior to the screening visit * treatment with an active vaccine targeting amyloid beta or tau protein * suspected or known drug or alcohol abuse * metallic implants or any other cause precluding the performance of brain mri * enrolment in another investigational study or intake of investigational drug within the previous 3 months since the last dose * suicide attempt within the last year or significant risk of suicide (in the opinion of the investigator, defined as a "yes" to suicidal ideation questions 4 or 5, or answering "yes" to suicidal behavior on the columbia-suicide severity rating scale within the past 12 months) * any condition that in the opinion of the investigator makes the patient unsuitable for inclusion in the study

Keywords: alzheimer's disease, ory-2001