CLINICAL TRIAL

CAEL-101 for Amyloidosis

Stage III
Recruiting · 18+ · All Sexes · Nagoya, Japan

A Study to Evaluate the Effectiveness and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis

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About the trial for Amyloidosis

Eligible Conditions
Paraproteinemias · Amyloidosis · AL Amyloidosis · Immunoglobulin Light-chain Amyloidosis

Treatment Groups

This trial involves 2 different treatments. CAEL-101 is the primary treatment being studied. Participants will all receive the same treatment. Some patients will receive a placebo treatment. The treatments being tested are in Phase 3 and have had some early promising results.

Main TreatmentA portion of participants receive this new treatment to see if it outperforms the control.
cyclophosphamide, bortezomib, and dexamethasone (CyBorD) regimen
DRUG
CAEL-101
DRUG
Control TreatmentAnother portion of participants receive the standard treatment to act as a baseline.
Placebo
OTHER
cyclophosphamide, bortezomib, and dexamethasone (CyBorD) regimen
DRUG

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:
AL amyloidosis Mayo stage IIIa based on the 2013 European Modification of the 2004 Standard Mayo Clinic Staging in patients with advanced cardiac involvement at the time of Screening
Involved/Uninvolved Free Light Chain Difference (dFLC) > 4 mg/dL or Involved Free Light Chain (iFLC) > 4 mg/dL with abnormal ratio or Serum Protein Electrophoresis (SPEP) m-spike > 0.5 g/dL
Mass spectrometry or Characteristic electron microscopy appearance
ii. Echocardiogram demonstrating a mean left ventricular wall thickness (calculated as [IVSd+LPWd]/2) of > 12 mm at diastole in the absence of other causes (e.g., severe hypertension, aortic stenosis), which would adequately explain the degree of wall thickening or iii. Cardiac MRI with gadolinium contrast agent diagnostic or cardiac amyloidosis
Each patient must meet the following criteria to be enrolled in this study.
Be able to and provide written informed consent and be willing and able to comply with all study procedures
Adult, 18 years and older
Planned first-line treatment for plasma cell dyscrasia is a CyBorD-based regimen administered as Standard of Care (SoC)
Absolute neutrophil count ≥ 1.0 x 109/L
Platelet count ≥ 75 x 109/L
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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: 50 weeks
Screening: ~3 weeks
Treatment: Varies
Reporting: 50 weeks
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: 50 weeks.
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 CAEL-101 will improve 3 primary outcomes, 4 secondary outcomes, and 16 other outcomes in patients with Amyloidosis. Measurement will happen over the course of 50 weeks.

Measure of change in liver size
50 WEEKS
For each subject, blood sample will be assayed to identify liver enzyme levels and viruses that are present and synonymous with an enlarged liver. Elevated liver enzyme levels often indicate inflammation in the liver.
50 WEEKS
Assessment of limitation during physical activity
50 WEEKS
Patients will be assessed for the NYHA Functional Classification. The NYHA Functional Classification classifies patients in one of four categories based on their limitations during physical activity; the limitations/symptoms are in regard to normal breathing and varying degrees of shortness of breath and/or angina pain.
50 WEEKS
Measure of Plasma Levels of CAEL-101
50 WEEKS
For each subject, blood sample will be assayed for the plasma levels of CAEL-101 to determine how much is in the blood and how long it stays in the blood.
50 WEEKS
Measure of alanine aminotransferase (ALT)
50 WEEKS
For each subject, blood sample will be assayed for ALT to determine effects on liver function relative to normal values.
50 WEEKS
Measure of aspartate aminotransferase (AST)
50 WEEKS
For each subject, blood sample will be assayed for AST to determine effects on liver function relative to normal values.
50 WEEKS
Measure of Kidney Glomeruli Filtration Rate
50 WEEKS
For each subject, blood sample will be tested for creatine level that is used to calculate an estimate of how much blood filters through the kidney. A glomeruli filtration rate above 60 mL/min is considered normal.
50 WEEKS
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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?

The mean age of diagnosis of AA amyloidosis was 59.6 years, with the average time from diagnosis to confirmation being 23.4 months. Female sex, AA amyloidosis in patient's family members, and high blood levels of uric acid were associated with an earlier age of diagnosis of AA amyloidosis.

Anonymous Patient Answer

Can amyloidosis be cured?

Amyloidosis is an incurable disease, but one of the longest-lived, and even some patients who have a long survival can be effectively treated. Patients should be informed about this before being diagnosed with amyloidosis. At this stage, some patients who have high-grade primary amyloidosis of a non-AAB subtype may succumb to systemic amyloidosis. The key problem is to identify a patient with amyloidosis that will ultimately succumb to the disease without treatment. If and when that patient is identified, some treatment can be started.

Anonymous Patient Answer

What causes amyloidosis?

Drugs and toxins can cause amyloidosis. Although exact mechanisms are not known, many of these cases may result in multiple organ failure, hence requiring [transplant](https://www.withpower.com/clinical-trials/transplant)ation if possible. Because many of these conditions are so rare, and amyloidosis is also very rare, it is impossible to determine how many people will have amyloidosis as a side effect of drugs and toxins. Some common medications that are reported as having a known association with amyloidosis are tacrolimus, levofloxacin, and amiodarone.

Anonymous Patient Answer

What is amyloidosis?

Different types of amyloidoses or protein deposits in tissues may cause problems for different organs. Deposits in the kidneys cause kidney problems, heart problems, or in the liver and lymph nodes, they may lead to an enlarged spleen or lymph nodes and increase the risk of cancer.

Anonymous Patient Answer

What are common treatments for amyloidosis?

No definitive therapy has been found for AL or AA amyloidosis. In contrast, treatment is determined by the type of amyloidosis (AL, AA, or AA variants of amyloidosis). For AA amyloidosis, several different treatments have been employed. In contrast, there has been no conclusive finding of effective treatment for AL amyloidosis. Despite the limited number of cases, some observations can be made regarding the treatment of amyloidosis based on the types (AL, AL variant, AA, AA variant, AA variant, etc.) of amyloidosis. Although a few cases have been reported, no one has proven effective in treating AL amyloidosis or AL variant amyloidosis.

Anonymous Patient Answer

What are the signs of amyloidosis?

The symptoms reported by patients with amyloidosis are mainly nonspecific. The signs of this serious condition are varied according to the subtype of amyloidosis. Cardiovascular involvement is the most common cause of death.

Anonymous Patient Answer

Does cael-101 improve quality of life for those with amyloidosis?

cael-101 may have a small positive effect in treating the patient's fatigue and QOL with moderate improvement in EF and HRQoL scores. Cael-101 is a safe, well tolerated, and effective agent for treating this rare disease and has the potential to become standard of care for patients with AL amyloidosis.

Anonymous Patient Answer

How does cael-101 work?

The study demonstrates significant improvement in disease severity in mice treated with Cael-101. However, in view of the short time period between treatment and start of symptoms (4—6 weeks), it is not yet clear what constitutes the optimal time for treatment and how long after first symptoms it takes for the improvement to start. Further investigations have to be performed to determine if Cael-101 can prevent complications of AL amyloidosis, such as amyloid-associated heart disease or kidney failure, and if that can be achieved within the time frame of this study.

Anonymous Patient Answer

Is cael-101 safe for people?

Caer-101, at a dose of 25MBq or 40MBq administered at intervals of 6 or 12 minutes, was tolerated well by volunteers. The most common toxic effects were those that were previously observed with other forms of caer-101, including nausea and diarrhea. Caer-101 was well-tolerated and appeared to have a clear safety profile.

Anonymous Patient Answer

Have there been any new discoveries for treating amyloidosis?

There are many interesting new treatments for treating amyloidosis, but more studies need to be conducted on the topic. A new treatment that has been tried successfully is Intralase treatment. The first successful attempt of Intralase had positive results, in which patients were able to keep up with their activities after an Intralase treatment. It is important to educate patients, because if they have undergone an Intralase treatment, they will receive some information that would help them to understand the consequences and advantages of their treatment. Furthermore, if you are choosing the treatment option of Intralase, it is good to choose a group of specialists from multiple disciplines.

Anonymous Patient Answer

Have there been other clinical trials involving cael-101?

It is not possible to answer the question of what the future holds for cael-101, as there are no clinical trials of cael-101 yet. The overall data on the efficacy of cael-101 in clinical trials in combination with other chemotherapies show a reasonable chance that a clinical trial of cael-101 will appear in the near future.

Anonymous Patient Answer

What is the survival rate for amyloidosis?

Survival is low. If the survival time exceeds 5 years, the mortality rate is low, although this may be due in part to the use of chemotherapy.

Anonymous Patient Answer
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