Testing the Safety and Preliminary Efficacy of the New Drug ORY-2001 in Mild to Moderate Alzheimer's Disease

NCT ID: NCT03867253

Last Updated: 2021-03-05

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 24 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-05-16
• Study Completion Date: 2020-11-12

Brief 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.

Detailed Description

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.

Conditions

Mild to Moderate Alzheimer's Disease

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

ORY-2001 Low dose

0.6mg ORY-2001 capsule

Group Type: ACTIVE_COMPARATOR

ORY-2001 Low dose

Intervention Type: DRUG

0.6mg ORY-2001 capsule

ORY-2001 High dose

1.2mg ORY-2001 capsule

Group Type: ACTIVE_COMPARATOR

ORY-2001 High dose

Intervention Type: DRUG

1.2mg ORY-2001 capsule

Placebo

Placebo capsule

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Placebo capsule

Interventions

ORY-2001 Low dose

0.6mg ORY-2001 capsule

Intervention Type: DRUG

ORY-2001 High dose

1.2mg ORY-2001 capsule

Intervention Type: DRUG

Placebo

Placebo capsule

Intervention Type: DRUG

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

• Minimum Eligible Age: 50 Years
• Maximum Eligible Age: 85 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Alzheimer's Drug Discovery Foundation

OTHER

Sponsor Role: collaborator

Oryzon Genomics S.A.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Michael Ropacki, MD

Role: STUDY_DIRECTOR

Oryzon Genomics S.A.

Locations

Alzheimer's Research and Treatment Center

Wellington, Florida, United States

Columbus Memory Center

Columbus, Georgia, United States

Princeton Medical Institute

Princeton, New Jersey, United States

Abington Neurological Associates Ltd.

Willow Grove, Pennsylvania, United States

Countries

United States

Other Identifiers

CL05-ORY-2001US

Identifier Type: -

Identifier Source: org_study_id