Clinical trial NCT02681172: Impact of FBB PET Amyloid Imaging in Change of Diagnosis in Patients With AD
Official title: Multicenter Study to Explore the Impact of Florbetaben (FBB) in Change of Diagnosis in Patients Who Are Evaluated for AD and in Whom Lumbar Puncture is Contraindicated or CSF Results Are Ambiguous
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Alzheimer's Disease (AD)
Keywords: CSF
Alzheimer's Disease (AD) and related dementia
HAS recommendations
Lead sponsor: Piramal Imaging Limited
Sponsor class: INDUSTRY
First posted: 2016-02-12
Enrollment: 218
Sex: ALL
Minimum age (years): 18
Maximum age (years): 90
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: HEALTH_SERVICES_RESEARCH

Brief summary: This phase 4 study will explore, in the context of the present French clinical practice, the impact of florbetaben 18F (FBB) in patients evaluated for AD who require a biomarker for etiologic determination of the cognitive and functional impairment, but in whom:

1. lumbar puncture was not feasible for medical conditions
2. results of cerebrospinal fluid (CSF) analysis were considered ambiguous by treating physicians
3. lumbar puncture (LP) was refused by the patient

Detailed description: It will be conducted in an outpatient setting at the tertiary memory clinics (CMRR) in France in patients with a preliminary diagnosis based on the completion of a prior, full diagnostic workup, not more than 12 months previously - which could include, but will not be limited to brain imaging if needed (magnetic resonance imaging (MRI) or computed tomography (CT)), neuropsychological evaluation including a Mini-Mental Status Examination (MMSE), CSF examination and other examinations according to "Haute Autorité de santé" (HAS, National Authority of Health, France) Recommendations - and in whom:

1. lumbar puncture was not feasible for medical conditions
2. results of cerebrospinal fluid (CSF) analysis were considered ambiguous by treating physicians
3. lumbar puncture (LP) was refused by the patient

For all subjects the study will comprise 3 outpatient clinic visits to record information on previous work ups and to perform the FBB Positron Emission Tomography (PET) scan (Visit 1: screening/ baseline, Visit 2: PET scan, Visit 3: subject informed on the FBB PET scan result).

At Visit 1, the initial diagnosis based on previous work up will be collected and rated on a five-point Likert confidence scale by the Physician.

At Visit 2, the FBB PET scan will be performed. Adverse events will be reported during the exam and up to 7 days after the PET procedure.

At Visit 3, based on the amyloid PET scan results, change of diagnosis will be collected, in addition to physician confidence.

Eligibility criteria: Inclusion Criteria:

* Patients being evaluated for Alzheimer disease and related dementias according to the HAS recommendations (atypical dementia, early onset and rapid progressive dementia)
* Patients who have previously been evaluated for AD and related dementias to whom a CSF examination was recommended in the last 12 months, but lumbar puncture was contraindicated or refused or CSF results were ambiguous, or
* Patients currently being evaluated for AD and related dementias to whom a CSF examination is recommended but lumbar puncture is contraindicated or refused
* Patients have a study partner who is willing and able to accompany him/her to all clinic visits for the duration of the protocol
* Patients able to complete all clinical visits according to the protocol
* Patients able to tolerate a 20-minute FBB PET scan
* Patient or legal representative to provide informed consent for study participation, visits and data source verification.

Exclusion Criteria:

* The subject had a previous beta amyloid imaging scan
* Any significant systemic illness or unstable medical condition which could lead to difficulty complying with the protocol
* Is currently receiving any investigational pharmaceutical product or has participated in a clinical trial with an investigational pharmaceutical product within 30 days prior to screening and/or was administered a radiopharmaceutical within 10 radioactive half-lives prior to study drug administration in this study
* For females of childbearing age, a positive pregnancy test

Primary outcomes:
measure: Number of Participants With a Change of Diagnosis Comparing Pre- and Post-scan Outcomes
timeFrame: Visit 1 (baseline evaluation) and Visit 3 (up to 6 months later)
Secondary outcomes:
measure: Number of Subjects With Improved Level of Physician Confidence in Diagnosis at Visit 3
timeFrame: Visit 1 (baseline evaluation) and Visit 3 (up to 6 months later)
measure: Number of Participants With a Change of Management Plan Comparing Pre- and Post-scan Outcomes
timeFrame: Visit 1 (baseline evaluation) and Visit 3 (up to 3 months later)
measure: Number of Subjects With Positive FBB PET Scan
timeFrame: Visit 3 (up to 6 months after baseline evaluation)
measure: Number of Subjects With Negative FBB PET Scans
timeFrame: Visit 3 (up to 6 months after baseline evaluation)
measure: Number of Subjects With Contraindicated or Failed Lumbar Puncture
timeFrame: Visit 1 (baseline evaluation)
measure: Number of Subjects With Available CSF Analysis But Results Considered as Non-contributory by the Clinician
timeFrame: Visit 1 (baseline evaluation)
measure: Number of Subjects Who Refused Lumbar Puncture.
timeFrame: Visit 1 (baseline evaluation)
Other outcomes:
Locations:
city: Marseille
country: France
facility: Coordinating center (Hôpital de La Timone) and 18 associated centers in France
Study officials:
affiliation: Hôpital de La Timone, Marseille, France
name: Mathieu Ceccaldi, Prof. MD.
role: PRINCIPAL_INVESTIGATOR
affiliation: Hôpital de la Timone, Marseille, France
name: Eric Guedj, Prof.
role: PRINCIPAL_INVESTIGATOR
References:
citation: Ceccaldi M, Jonveaux T, Verger A, Krolak-Salmon P, Houzard C, Godefroy O, Shields T, Perrotin A, Gismondi R, Bullich S, Jovalekic A, Raffa N, Pasquier F, Semah F, Dubois B, Habert MO, Wallon D, Chastan M, Payoux P; NEUUS in AD study group; Stephens A, Guedj E. Added value of 18F-florbetaben amyloid PET in the diagnostic workup of most complex patients with dementia in France: A naturalistic study. Alzheimers Dement. 2018 Mar;14(3):293-305. doi: 10.1016/j.jalz.2017.09.009. Epub 2017 Nov 4.