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Mild to Moderate Alzheimer's DiseasePhase 2ETHERAL-USNCT03867253

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

A Phase 2 study testing ORY-2001 Low dose and ORY-2001 High dose in people with Mild to Moderate Alzheimer's Disease, run by Oryzon Genomics S.A. at 4 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 50 to 85 years
Conditions the study is for
Mild to Moderate Alzheimer's Disease
What is being tested?
ORY-2001 Low dose and ORY-2001 High dose
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
24 participants
Where
4 sitesUnited States
Study dates
16 May 2019to 12 Nov 2020
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 50 to 85 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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;
Show all 19Show fewer
  • 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

Trial locations and contacts

4 sites in 1 country.

United States4 sites

  • Columbus Memory Center

    Columbus, Georgia, 31909

  • Princeton Medical Institute

    Princeton, New Jersey, 08540

  • Alzheimer's Research and Treatment Center

    Wellington, Florida, 33414

  • Abington Neurological Associates Ltd.

    Willow Grove, Pennsylvania, 191090

Other trials in United States

About this study

Official title: A Multicentre,Randomised, Double-blind, Placebo-controlled, 3-arm, 24-week Parallel-group Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of ORY-2001 in Patients With Mild-moderate Alzheimer's Disease

This is a Phase IIa study assessing the safety, tolerability and preliminary efficacy of ORY-2001 in mild to moderate Alzheimer's Disease patients.

Read the 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.

Being tested
ORY-2001 Low dose, ORY-2001 High dose, Placebo
Keywords
Alzheimer's Disease, ORY-2001
Medical subject headings
Alzheimer Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ORY-2001 Low doseDrug

    0.6mg ORY-2001 capsule

  • ORY-2001 High doseDrug

    1.2mg ORY-2001 capsule

  • PlaceboDrug

    Placebo capsule

Study groups in NCT03867253
Study groupRoleWhat they receive
ORY-2001 Low doseActive comparator0.6mg ORY-2001 capsule
Receives: Drug: ORY-2001 Low dose
ORY-2001 High doseActive comparator1.2mg ORY-2001 capsule
Receives: Drug: ORY-2001 High dose
PlaceboPlacebo comparatorPlacebo capsule
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Treatment Emergent Adverse Events

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

    Measured: Week 24

  2. Treatment Emergent Adverse Events

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

    Measured: Week 48

  3. Withdrawn patients due to TEAEs

    Number and percentage of withdrawn patients due to TEAEs

    Measured: Week 24

  4. Withdrawn patients due to TEAEs

    Number and percentage of withdrawn patients due to TEAEs

    Measured: Week 48

Secondary outcomes (7)

  1. Cohen-Mansfield Agitation Inventory (CMAI)

    Change from baseline to week 48 compared to placebo

    Measured: 48 weeks

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

    Change from baseline to week 48 compared to placebo

    Measured: 48 weeks

  3. 14-item Alzheimer's Disease Assessment Scale-Cognitive

    Change from baseline to week 48 compared to placebo

    Measured: 48 weeks

  4. Computerized Cognitive Test battery

    Change from baseline to week 48 compared to placebo

    Measured: 48 weeks

  5. Mini-Mental State Examination (MMSE)

    Change from baseline compared to placebo

    Measured: 48 weeks

  6. Clinical Dementia Rating Scale Sum of Boxes

    Change from baseline to week 48 compared to placebo

    Measured: 48 weeks

  7. Cornell Scale for Depression in Dementia (CSDD)

    Change from baseline to week 48 compared to placebo

    Measured: 48 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted7 Mar 2019
  2. Study start16 May 2019
  3. Primary completion12 Nov 2020
  4. Study completion12 Nov 2020

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
24 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03867253
Sponsor's protocol ID
CL05-ORY-2001US
Acronym
ETHERAL-US
Status last verified
Jul 2020
First submitted
20 Feb 2019
Last updated
5 Mar 2021
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Undecided

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing ORY-2001 Low dose and ORY-2001 High dose in people with Mild to Moderate Alzheimer's Disease, run by Oryzon Genomics S.A. at 4 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 8 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 5 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Treatment Emergent Adverse Events (time frame: Week 24). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03867253.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Mar 2021.

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