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

NCT03867253 | Completed Phase 2 DMC FDA Drug

• 1 other identifier: CL05-ORY-2001US
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 4

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.

Conditions

Mild to Moderate Alzheimer's Disease

Keywords

Alzheimer's Disease, ORY-2001

Outcome Measures

Primary Outcomes (4)

• Treatment Emergent Adverse Events

  Number, frequency and severity of Treatment Emergent Adverse Events (TEAEs) including serious TEAEs.

  Week 24

• Treatment Emergent Adverse Events

  Number, frequency and severity of Treatment Emergent Adverse Events (TEAEs) including serious TEAEs.

  Week 48

• Withdrawn patients due to TEAEs

  Number and percentage of withdrawn patients due to TEAEs

  Week 24

• Withdrawn patients due to TEAEs

  Number and percentage of withdrawn patients due to TEAEs

  Week 48

Secondary Outcomes (7)

• Cohen-Mansfield Agitation Inventory (CMAI)

  48 weeks

• Clinician version of the Apathy Evaluation Scale (AES-C)

  48 weeks

• 14-item Alzheimer's Disease Assessment Scale-Cognitive

  48 weeks

• Computerized Cognitive Test battery

  48 weeks

• Mini-Mental State Examination (MMSE)

  48 weeks

Study Arms (3)

ORY-2001 Low dose

ACTIVE COMPARATOR

0.6mg ORY-2001 capsule

Drug: ORY-2001 Low dose

ORY-2001 High dose

ACTIVE COMPARATOR

1.2mg ORY-2001 capsule

Drug: ORY-2001 High dose

Placebo

PLACEBO COMPARATOR

Placebo capsule

Drug: Placebo

Interventions

ORY-2001 Low dose DRUG

0.6mg ORY-2001 capsule

ORY-2001 Low dose

ORY-2001 High dose DRUG

1.2mg ORY-2001 capsule

ORY-2001 High dose

Placebo DRUG

Placebo capsule

Placebo

Eligibility Criteria

• Age: 50 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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:
• Other primary degenerative dementia;
• Other neurodegenerative condition;
• Cerebrovascular disease;
• 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

Sponsors & Collaborators

• Oryzon Genomics S.A.: lead
• Alzheimer's Drug Discovery Foundation: collaborator

Study Sites (4)

Alzheimer's Research and Treatment Center

Wellington, Florida, 33414, United States

Location

Columbus Memory Center

Columbus, Georgia, 31909, United States

Location

Princeton Medical Institute

Princeton, New Jersey, 08540, United States

Location

Abington Neurological Associates Ltd.

Willow Grove, Pennsylvania, 191090, United States

Location

MeSH Terms

Conditions

Alzheimer Disease

Interventions

vafidemstat

Condition Hierarchy (Ancestors)

Dementia; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Tauopathies; Neurodegenerative Diseases; Neurocognitive Disorders; Mental Disorders

Study Officials

• Michael Ropacki, MD

  Oryzon Genomics S.A.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 20, 2019
• First Posted: March 7, 2019
• Study Start: May 16, 2019
• Primary Completion: November 12, 2020
• Study Completion: November 12, 2020
• Last Updated: March 5, 2021

Record last verified: 2020-07

Locations

US (4)