CLINICAL TRIAL

F-18 florbetapir/C-11 acetate PET for Amyloidosis

Stage IV
Recruiting · 18+ · All Sexes · Boston, MA

This study is evaluating whether a new imaging technique can help detect cardiac amyloidosis.

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About the trial for Amyloidosis

Eligible Conditions
Immunoglobulin Light-chain Amyloidosis · Cardiomyopathies · Cardiomyopathy · Amyloidosis · Light-Chain Amyloidosis

Treatment Groups

This trial involves 4 different treatments. F-18 Florbetapir/C-11 Acetate PET is the primary treatment being studied. Participants will be divided into 3 treatment groups. There is no placebo group. The treatments being tested are not being studied for commercial purposes.

Experimental Group 1
F-18 florbetapir/C-11 acetate PET
RADIATION
+
MRI
DEVICE
+
N-13 ammonia PET
RADIATION
Experimental Group 2
F-18 florbetapir/C-11 acetate PET
RADIATION
+
MRI
DEVICE
Experimental Group 3
F-18 florbetapir/C-11 acetate PET
RADIATION
+
MRI
DEVICE
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About The Treatment

Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
MRI
2009
Completed Phase 2
~2270

Eligibility

This trial is for patients born any sex aged 18 and older. There are 10 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
For subjects traveling from out of town referred for systemic AL therapy based on clinical evaluation and laboratory testing, but, pending biopsy results, study enrollment and procedures may begin before official confirmation of biopsy results. If biopsy is negative for AL amyloidosis, subject will be considered a screen failure. There will be no more than 10 subjects who fall under this screen failure for the duration of the study.
Age > 18 years
Diagnosis of light chain amyloidosis by standard criteria (immunofixation of serum and urine, IgG free light chain (FLC) assay, a biopsy of fat pad/ bone marrow, or organ biopsy, followed by typing of the light chain using immunohistochemistry or immunogold assay with confirmation by Mass spectroscopy as needed)
Subjects with localized amyloid deposition and non-systemic AL disease will be eligible for enrollment in group D.
Willing and able to provide consent
Additional inclusion criteria for the Remission AL-CMP: Hematological response defined as complete hematological remission or very good partial response-differential free light chain (dFLC)<40 mg/dL for > 1 year prior to enrollment
Additional inclusion criteria for the Active AL-CMP - exercise: Ability to perform supine bicycle exercise. Enrollment to this arm will stop after 36 subjects complete baseline and 6 months studies.
Additional inclusion criteria for the Active AL Pre-CMP - Normal left ventricular wall thickness (≤ 12 mm) and normal LVEF (≥55%) on echocardiography within 3 months or increased wall thickness with normal cardiac biomarker levels: not meeting above definition.
Additional inclusion criteria for Control Multiple Myeloma subjects: diagnosis of multiple myeloma without concomitant amyloidosis by standard criteria
Additional inclusion criteria for Control Heart Failure subjects: diagnosis of heart failure without amyloidosis by standard criteria
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Odds of Eligibility
Unknown<50%
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
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Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: Baseline, 6 and 12 months
Screening: ~3 weeks
Treatment: Varies
Reporting: Baseline, 6 and 12 months
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: Baseline, 6 and 12 months.
View detailed reporting requirements
Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether F-18 florbetapir/C-11 acetate PET will improve 4 primary outcomes and 3 secondary outcomes in patients with Amyloidosis. Measurement will happen over the course of Baseline.

Understand the role of gut microbiota and heavy metals in the pathogenesis of AL Amyloidosis
BASELINE
This will be tested using machine learning methods with 16S rRNA sequencing of salivary and stool samples in a 40-patient cohort with AL-amyloidosis compared to healthy controls from the NIH funded human microbiome project (HMP).This will also be used to test if the gut microbiome affects amyloid formation using a transgenic mouse model of AL amyloidosis that expresses the human LC in the gut and develops amyloid in the stomach.
BASELINE
Light Chain Toxicity
BASELINE
Study subject urine light chain's will be extracted and infused into zebrafish and isolated cardiomyocytes to study light chain toxicity
BASELINE
Change in Myocardial oxidative metabolism markers from baseline to 6 months
BASELINE AND 6 MONTHS
K mono and coronary flow reserve obtained by C-11 acetate PET/CT at rest and stress
BASELINE AND 6 MONTHS
Change in Myocardial energy efficiency from baseline to 6 months
BASELINE AND 6 MONTHS
Myocardial energy efficiency, Kmono reserve, will be determined by C-11 acetate PET
BASELINE AND 6 MONTHS
Change in Serum oxidative stress markers from baseline to 6 months and 12 months
BASELINE, 6 AND 12 MONTHS
serum F-2 isoprostane and peroxynitrite levels
BASELINE, 6 AND 12 MONTHS
Change in F-18 florbetapir myocardial retention index from baseline to 6 months and 12 months
BASELINE, 6 AND 12 MONTHS
quantitative measure of F-18 florbetapir uptake by the heart muscle
BASELINE, 6 AND 12 MONTHS
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Who is running the study

Principal Investigator
S. D.
Sharmila Dorbala, Director, Nuclear Cardiology
Brigham and Women's Hospital

Patient Q & A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many people get amyloidosis a year in the United States?

While our findings have implications for the number of patients with advanced amyloidosis seen by physicians or nurses, further study and more representative data will be necessary to better understand the pattern of presentation of amyloidosis in the USA.

Anonymous Patient Answer

What are common treatments for amyloidosis?

Amyloidosis is uncommon because the incidence of amyloidosis is 3 to 10 cases per 1,000,000. This would be an unusually high figure if it were not for the very low mortality rate (typically ≤ 10%). Treatments include chemotherapy and plasma exchange. New agents in development use a monoclonal antibody targeting amyloid deposits for treatment.

Anonymous Patient Answer

What causes amyloidosis?

Although some people develop amyloidosis because of a genetic predisposition, in many cases the cause is unknown. Genetic mutation is believed to be responsible for a small subset of cases. There is no evidence that liver disease causes or enhances the formation of amyloid, and blood vessels may play an important role in the accumulation of the amyloid in the tissues.

Anonymous Patient Answer

Can amyloidosis be cured?

The incidence of amyloidosis in various countries is about 1/1,000,000, but it is difficult to find randomized control trials to test the effectiveness of treatments. For example, many patients enrolled in trials have received some sort of treatment before enrollment and the randomized control trials may not have included all patients who should have been enrolled in the trial. Patients should be well- counseled and informed about the risks and benefits of treatment, and must consent to the participation in the trial.

Anonymous Patient Answer

What is amyloidosis?

Patients with systemic amyloidosis are more likely to be younger in age, male, and present with symptoms of systemic organ involvement and systemic inflammation in the medical history.

Anonymous Patient Answer

What are the signs of amyloidosis?

Amyloidosis may present with the signs and symptoms of heart disease such as shortness of breath, fatigue, and loss of appetite. Skin abnormalities, including erythema of palms and soles, may also appear.\n

Anonymous Patient Answer

What is the survival rate for amyloidosis?

Survival in patients with AL amyloidosis can be very good when disease is properly treated with proper management. Lifelong outpatient hemodialysis is the most common cause of death. Survival rate in patients with ATTR amyloidosis has been affected by the development of disease-modifying therapies(DMTs).

Anonymous Patient Answer

Is f-18 florbetapir/c-11 acetate pet typically used in combination with any other treatments?

f-18 florbetapir PET used alone showed a high specificity (94.6%) in the diagnosis of AL amyloidosis irrespective of other treatments. However, its accuracy was lower at the time of clinical diagnosis.

Anonymous Patient Answer

Is f-18 florbetapir/c-11 acetate pet safe for people?

F-18 florbetapir PET is highly reproducible and does not result in significant change in routine clinical practice. Results from a recent clinical trial show excellent potential applicability of PET in evaluating progression and disease management in AL amyloidosis. The potential use as an initial workup in this disorder is discussed.

Anonymous Patient Answer

How quickly does amyloidosis spread?

We found that the rapid progression to dialysis is most common in patients at intermediate risk. Findings from a recent study suggest that the use of a prognostic algorithm tailored to one's risk of amyloidosis-associated death may be useful to identify patients in whom early intervention may be most beneficial.

Anonymous Patient Answer

Does f-18 florbetapir/c-11 acetate pet improve quality of life for those with amyloidosis?

This pilot study of f-18 FLB PET/c-11 acetate PET in patients with amyloidotic polyneuropathy has shown a high correlation of disease severity with HRQOL. Patients with disease in remission had the highest reported scores on HRQOL domains.

Anonymous Patient Answer

Does amyloidosis run in families?

Genetic predisposition may contribute to the development of AA amyloidosis. It appears that AA amyloidosis in this family involves several families and not a single genetic defect. The high concordance between the two siblings in each of the families also suggests the need for the establishment of a family registry and the study of other families to determine the genetic predisposition to AA amyloidosis in this family.

Anonymous Patient Answer
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