Efficacy of [18F]Florbetaben PET for Diagnosis of Cardiac AL Amyloidosis

NCT05184088 | Recruiting Phase 3 FDA Drug

• 1 other identifier: FBB-02-01-21
• Study Type: interventional
• Target: 200
• Locations: 4 countries
• Sites: 14

Brief Summary

This is an open-label, multi-center pivotal Phase 3 study to visually and quantitatively assess PET images obtained after single application of 300 MBq \[18F\]florbetaben and PET scanning of patients with suspected cardiac amyloidosis.

Conditions

Cardiac Amyloidosis; AL Amyloidosis; ATTR Amyloidosis

Keywords

AL Amyloidosis; ATTR Amyloidosis; Cardiac Amyloidosis

Outcome Measures

Primary Outcomes (1)

• Sensitivity and specificity of the visual assessment of [18F]florbetaben PET images for the diagnosis of cardiac AL amyloidosis.

  The results from the visual assessment of \[18F\]florbetaben PET images are compared to the clinical diagnosis established through histological verification of the presence or absence of AL amyloidosis with cardiac involvement determined either through endomyocardial biopsy or through extracardiac biopsy in conjunction with typical CMR or echocardiography imaging features as the standard of truth.

  Up to 12 weeks

Secondary Outcomes (5)

• Sensitivity and specificity of [18F]florbetaben PET for the diagnosis of cardiac AL amyloidosis using quantification.

  Up to 12 weeks

• Correlation of quantitative [18F]florbetaben PET results with left ventricular ejection fraction (LV EF) and left ventricular mass (LV mass).

  Up to 12 weeks

• Correlation of quantitative [18F]florbetaben PET results with AL CA stage I - IV based on FLC-diff, cTnT and NT-proBNP levels.

  Up to 12 weeks

• Impact of PET imaging (AL-CA/non AL-CA) on diagnostic thinking and patient management will be assessed with physician's questionnaires before and after the diagnostic work-up, and after receipt of the PET results.

  Up to 14 weeks

• Number of adverse events

  Up to 17 days after imaging visit

Other Outcomes (1)

• Sensitivity and specificity of [18F]florbetaben PET images for a differential diagnosis between AL CA, ATTR CA and non CA will be assessed.

  Up to 12 weeks

Study Arms (1)

Patients with suspected cardiac amyloidosis

EXPERIMENTAL

After enrolment, patients will be subjected to diagnostic procedures according to standard of care to resolve diagnostic uncertainties and to clarify possible cardiac involvement. The results of the clinical work-up will be used a standard of truth, i.e. patients with initially suspected cardiac amyloidosis that where subsequently clinically diagnosed with cardiac AL Amyloidosis, cardiac ATTR Amyloidosis, other cardiac Amyloidosis or non cardiac amyloidosis.

Drug: [18F]florbetaben

Interventions

[18F]florbetaben DRUG

All enrolled patients will undergo \[18F\]florbetaben PET imaging.

Also known as: Neuraceq

Patients with suspected cardiac amyloidosis

Eligibility Criteria

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

You may qualify if:

• Males and females age ≥18 years
• Able to understand, sign and date written informed consent
• Written informed consent must be obtained before any study procedures are performed
• Subjects being considered for a possible diagnosis of cardiac amyloidosis by
• \. One of the following conditions:
• Established systemic amyloidosis without proven cardiac involvement,
• Known plasma cell dyscrasia (MGUS, multiple myeloma),
• Pathological free light chain levels in urine or serum,
• Presence of heart failure with preserved ejection fraction
• \. AND one of the following parameters, indicative of cardiac manifestation:
• Mean (left ventricular (LV) wall + septum) thickness \>12mm as measured by echocardiography or CMR in absence of other known cause of left ventricular hypertrophy (LVH),
• NT-proBNP \>335 ng/L (in case a value for NT-proBNP is not available, BNP \> 81ng/L may be used instead)
• Planned diagnostic procedure to establish diagnosis and cardiac involvement (e.g., endomyocardial biopsy or extracardiac biopsy in conjunction with cardiac magnetic resonance imaging/echocardiography or bone scintigraphy)
• Female subjects must be documented by medical records or physician's note to be either surgically sterile (by means of hysterectomy, bilateral salpingectomy, or bilateral oophorectomy) or post-menopausal for at least 1 year (no menses for 12 months without an alternative medical cause). If they are of child-bearing potential, they must commit to use of a highly effective contraceptive measure for at least one week following the PET scan (including combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal or transdermal), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable or implantable), intrauterine device, intrauterine hormone-releasing system, bilaterial tubal occlusion, vasectomised parner or secual abstinence).
• Male subjects and their partners of childbearing potential must commit to the use of a highly effective method of contraception for a minimum of 90 days following the PET scan (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal or transdermal), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable or implantable), intrauterine device, intrauterine hormone-releasing system, bilaterial tubal occlusion, male subjects with vasectomy or sexual abstinence)

You may not qualify if:

• Any known allergic reactions or hypersensitivity towards any compound of the study drug
• Severe hepatic impairment (AST/ALT \>5 x ULN; bilirubin \>3 x ULN)
• Inability to lay flat for up to 60 min
• Pregnant, lactating or breastfeeding
• Unwilling and/or unable to cooperate with study procedures
• Having been administered a radiopharmaceutical within 10 radioactive half-lives prior to study drug administration in this study

Sponsors & Collaborators

• Life Molecular Imaging GmbH: lead
• pharmtrace klinische Entwicklung GmbH: collaborator

Study Sites (14)

St Luke's Hospital

Kansas City, Kansas, 64111, United States

RECRUITING

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

University of Pittsburgh

Pittsburgh, Pennsylvania, 15213, United States

RECRUITING

University of Augsburg

Augsburg, 86156, Germany

RECRUITING

Charite Berlin

Berlin, 13353, Germany

RECRUITING

University of Essen

Essen, 45147, Germany

RECRUITING

HOPA Hamburg

Hamburg, 22767, Germany

RECRUITING

University of Heidelberg

Heidelberg, 69120, Germany

RECRUITING

University of Würzburg

Würzburg, 97080, Germany

RECRUITING

Hospital University Bellvitge

Barcelona, 08907, Spain

RECRUITING

Hospital University Puerta de Hierro

Madrid, 28222, Spain

RECRUITING

Clínica Universidad de Navarra

Pamplona, 31008, Spain

RECRUITING

University of Salamanca

Salamanca, 37007, Spain

RECRUITING

Royal Free Hospital

London, NW3 2PF, United Kingdom

RECRUITING

MeSH Terms

Conditions

Amyloid Neuropathies, Familial; Immunoglobulin Light-chain Amyloidosis

Interventions

4-(N-methylamino)-4'-(2-(2-(2-fluoroethoxy)ethoxy)ethoxy)stilbene

Condition Hierarchy (Ancestors)

Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Amyloid Neuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Amyloidosis, Familial; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases; Amyloidosis; Proteostasis Deficiencies; Neoplasms, Plasma Cell; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases; Paraproteinemias

Study Officials

• Andrew Stephens, MD, PhD

  Life Molecular Imaging

  STUDY DIRECTOR

Central Study Contacts

Iris Hardewig, PhD

CONTACT

+49 (0)30 461 1246 03; clinicaltrials@life-mi.com

Aleksandar Jovalekic, PhD

CONTACT

+49 (0)30 461 1246 03; clinicaltrials@life-mi.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: All eligible patients will receive a single administration of the imaging agent \[18F\]florbetaben at a radioactive dose of 300 MBq.

Study Record Dates

• First Submitted: January 6, 2022
• First Posted: January 11, 2022
• Study Start: January 13, 2023
• Primary Completion: March 1, 2026
• Study Completion: March 1, 2026
• Last Updated: November 25, 2025

Record last verified: 2025-11

Locations

DE (6); ES (4); GB (1); US (3)