Using PET imaging to diagnose cardiac amyloidosis

An Open-label, Multi-center, Non-randomized Pivotal Phase 3 Study to Evaluate the Efficacy and Safety of [18F]Florbetaben Positron Emission Tomography (PET) Imaging to Diagnose Cardiac AL Amyloidosis

This study is testing if a special PET scan can help doctors better diagnose heart problems caused by amyloidosis in patients who might have it.

Quick facts

• Phase: PHASE3
• Study type: Interventional
• Enrollment: 200 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Life Molecular Imaging SA (industry)
• Locations: 14 sites (Kansas City, Kansas and 13 other locations)
• Trial ID: NCT05184088 on ClinicalTrials.gov

What this trial studies

This open-label, multi-center Phase 3 study aims to evaluate the effectiveness of [18F]florbetaben PET imaging in diagnosing cardiac AL amyloidosis. Participants will receive a single dose of 300 MBq [18F]florbetaben followed by PET scanning to assess the presence of cardiac amyloidosis. The diagnostic efficacy will be compared to standard clinical diagnostic methods to determine the accuracy of this imaging technique in patients with suspected or uncertain cardiac involvement due to amyloidosis.

Who should consider this trial

Good fit: Ideal candidates include adults aged 18 and older with suspected cardiac amyloidosis or those with a known diagnosis of amyloidosis but uncertain cardiac involvement.

Not a fit: Patients without any suspicion of cardiac amyloidosis or those with confirmed cardiac involvement from other causes may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a more accurate and non-invasive method for diagnosing cardiac amyloidosis, leading to timely and appropriate treatment for patients.

How similar studies have performed: Other studies using PET imaging for amyloidosis diagnosis have shown promising results, indicating potential success for this approach.

Eligibility criteria

Inclusion Criteria:

* 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
* 1\. 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
* 2\. 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)
* Male subjects must commit to not donate sperm for a minimum of 90 days after the PET scan

Exclusion Criteria:

* 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

Where this trial is running

Kansas City, Kansas and 13 other locations

• St Luke's Hospital — Kansas City, Kansas, United States (RECRUITING)
• University of Pennsylvania — Philadelphia, Pennsylvania, United States (RECRUITING)
• University of Pittsburgh — Pittsburgh, Pennsylvania, United States (RECRUITING)
• University of Augsburg — Augsburg, Germany (RECRUITING)
• Charite Berlin — Berlin, Germany (RECRUITING)
• University of Essen — Essen, Germany (RECRUITING)
• HOPA Hamburg — Hamburg, Germany (RECRUITING)
• University of Heidelberg — Heidelberg, Germany (RECRUITING)
• University of Würzburg — Würzburg, Germany (RECRUITING)
• Hospital University Bellvitge — Barcelona, Spain (RECRUITING)
• Hospital University Puerta de Hierro — Madrid, Spain (RECRUITING)
• Clínica Universidad de Navarra — Pamplona, Spain (RECRUITING)
• University of Salamanca — Salamanca, Spain (RECRUITING)
• Royal Free Hospital — London, United Kingdom (RECRUITING)

Study contacts

• Study coordinator: Iris Hardewig, PhD

Conditions: Cardiac Amyloidosis, AL Amyloidosis, ATTR Amyloidosis