Patient- and care-related benefits of amyloid PET imaging (ENABLE)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Deutsches Zentrum Fuer Neurodegenerative Erkrankungen e.V.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Psychiatry and Psychology [F] - Mental Disorders [F03]

Trial duration

19 Aug 2024 → ongoing

Decision date (initial)

2023-10-24

Transition trial

No

Low-intervention

Yes

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Gemeinsamer Bundesausschuss (G-BA)

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Diagnosis, Efficacy, Others

Demonstrate a patient-relevant benefit of amyloid PET compared to S3 guideline diagnostics without amyloid PET on dementia-relevant morbidity endpoints.

Conditions and MedDRA coding

Dementia in Pick disease

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 11

1. Age ≥ 50 years
2. Mild to moderate dementia syndrome
3. Clinical Dementia Rating Scale (CDR) > 0.5 and < 3.0
4. Mini-Mental-Status Test (MMSE) > 10
5. Unclear diagnosis of dementia or uncertain diagnosis of Alzheimer's disease (diagnostic certainty < 85 %)
6. Diagnosis of Alzheimer's disease with at least 15% probability, i.e. Alzheimer's disease cannot be ruled out with certainty.
7. No diagnosis possible by examination of the CSF because a) the patient has a contraindication for CSF puncture, b) the patient refuses CSF puncture or c) an unclear diagnosis remains after CSF puncture.
8. Patients who would agree in principle to undergo amyloid PET diagnostics and are willing to know its result, if they are randomised into the amyloid PET arm.
9. Written informed consent, either by the patient or the legal representative according to the presumed will of the patient.
10. Accompaniment by an informant authorised/qualified to provide information
11. Patients with valid insurance cover from a German compulsory health insurance

Exclusion criteria 5

1. Severe dementia (CDR score = 3 and/or an MMST score ≤10).
2. Mild cognitive impairment, CDR score less than 0.5, absence of cognitive impairment relevant to daily living
3. Patients in whom radiation exposure must be avoided
4. Pregnancy or breastfeeding at the time of the PET scan (for women who are less than 12 months postmenopausal, a pregnancy test is done before the PET scan is performed).
5. Patients enrolled in another clinical trial with investigational product at the time of inclusion or within 5 half-lives of the investigational product of another clinical trial.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Measured by investigators blinded to the intervention condition: Ability to manage activities of daily living as measured by the Amsterdam Instrumental Activities of Daily Living Questionnaire© (A-IADL-Q) score 78 weeks after randomization.

Secondary endpoints 6

1. Measured by the specialist at the PET centre: occurrence of adverse events in the amyloid PET arm during and immediately after the performance of the amyloid PET examination; measured by the investigator at the trial site in the amyloid PET and control arms over the entire period of the trial: Incidence of adverse and serious adverse events (SAEs) including adverse drug reactions (ARs), mortality (also in the context of the safety assessment)
2. Measured via the investigator at the trial site (26 weeks after randomisation): Change in aetiological diagnosis of dementia (additional 78 weeks after randomization), change in diagnostic certainty, change in diagnostic and therapeutic (especially administration or discontinuation of medication) management.
3. Measured across patients and/or relatives by investigators blinded to examination condition (26, 52, 78 and 104 weeks after randomisation): Cognitive performance (ADAS-cog, MMSE), quality of life, incl. health-related quality of life (QOL-AD scale)
4. Measured across patients and/or relatives by investigators blinded to examination condition (26, 52, 78 and 104 weeks after randomisation): need for full inpatient or institutionalised outpatient care (institutionalisation) or intensification of institutionalised outpatient care as well as total duration and frequency of unplanned inpatient stays within one year (FIMA).
5. Measured across patients and/or relatives by investigators blinded to examination condition (78 weeks after randomization): CDR(clinical severity rating of dementia) and GDS(Geriatric Depression Scale)
6. Measured by an independent doctor after database closure: Use of potentially unsuitable medication (PRISCUS list)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Deutsches Zentrum Fuer Neurodegenerative Erkrankungen e.V.

Sponsor organisation

Deutsches Zentrum Fuer Neurodegenerative Erkrankungen e.V.

Address

Venusberg-Campus 1/99, Venusberg Venusberg

City

Bonn

Postcode

53127

Country

Germany

Scientific contact point

Organisation

Deutsches Zentrum Fuer Neurodegenerative Erkrankungen e.V.

Contact name

Prof. Dr. Stefan Teipel

Public contact point

Organisation

Deutsches Zentrum Fuer Neurodegenerative Erkrankungen e.V.

Contact name

Prof. Dr. Stefan Teipel

Third parties 3

Locations

1 EU/EEA country · 27 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 18 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications