Frontline Lenalidomide for AL Amyloidosis Involving Myocardium

NCT04298372 | Completed Phase 3 DMC FDA Drug

• 1 other identifier: 1810-089-981
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 1

Brief Summary

This phase II clinical trial aimed at influencing the improvement of major organ functions, especially the objective response rate, in Amyloid light-chain amyloidosis involving myocardium.

Conditions

Amyloidosis Cardiac

Outcome Measures

Primary Outcomes (1)

• Objective response rate (ORR)

  ORR is defined as the percentage of subjects with evidence of a confirmed CR(complete response), VGPR(very good partial response) or PR(partial response)

  After completion of 12 cycles of treatment(each cycle is 28 days)

Secondary Outcomes (3)

• Change of the cardiac function

  At Baseline and the end of each 1-12 cycles (baseline and 1-12 months)

• Change of the renal function

  At Baseline and the end of each 1-12 cycles (baseline and 1-12 months)

• Change of the hepatic function

  At Baseline and the end of each 1-12 cycles (baseline and 1-12 months)

Interventions

Lenalidomide 25mg DRUG

As single arm study, patients will be treated as follows: * Lenalidomide : D1\~D21 daily oral administration, starting dose 15mg, with an increase of 5mg every cycle, if tolerated, up to 25mg. * Dexamethasone : D1\~D4 daily oral administration or intravenous injection, 40mg(or 20mg for age for age ≥ 65 years old) Patients will be treated every 4 weeks as one cycle, and up to 12 cycles of treatment will be conducted.

Also known as: Revlimid 25mg

Eligibility Criteria

• Age: 19 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age more than 19 years old
• Amyloid light-chain amyloidosis patients who meet both(A and B) of the following criteria
• A. Cardiac involvement: meet one or more of the following criteria
• Echocardiography : mean wall thickness \> 12mm, and no other cardiac cause
• Cardiac MRI : CMR was considered positive for amyloid cardiomyopathy if there were morphologic and structural abnormalities consistent with the diagnosis (wall thickening of the left ventricular \[LV\], right ventricular \[RV\], or interatrial septum and biatrial enlargement) coupled with abnormal myocardial nulling and diffuse or irregular delayed gadolinium enhancement of the LV myocardium.
• N-terminal prohormone of brain natriuretic peptide : \> 332ng/l in the absence pf renal failure
• B. Exposure history to lenalidomide: participants should have never been exposed to lenalidomide within 5 years.
• Eastern Cooperative Oncology Group performance status ≤ 3
• Patients must meet the following clinical laboratory criteria with 2 weeks of starting treatment:
• Serum creatinine ≤ 3.0mg/dl or Creatinine Clearance ≥ 50ml/minute (Cockcroft-Gault)
• Absolute neutrophil count ≥ 1000/ul
• Platelet ≥ 75,000/ul
• Hemoglobin ≥ 8.0mg/dl
• Bilirubin \< 2 times or Alkaline phosphate \< 4 times upper limit of normal
• At least 3 months of existence are expected from the time of enroll this study

You may not qualify if:

• Amyloid light-chain amyloidosis without cardiac involvement
• Patients who are planning to receive autologous stem cell transplantation or received autologous stem cell transplantation, remained in hematologic complete response
• Pregnant, lactating or unwilling to use adequate contraception
• Male patients who unwilling to practice effective barrier contraception during the entire study treatment period and through 30 days after the last dose of study treatment
• Patients with acute infections requiring nonpermanent therapy (antibiotics, antibacterial drugs, or antiviral drugs) within 14 days of the first administration of the lenalidomide or with a history of systemic fungal infections and infections without valid antimicrobial agents
• Any clinically significant history of genetic, kidney, neurological, psychiatric, endocrine, metabolism, immunological, cardiovascular, lung, or liver diseases in the investigator's opinion, may interfere with protocol adherence or a patient's ability to give informed consent.
• Patients with known history of allergic and hypersensitivity for investigational product, their analogous bodies, or the subtypes contained in the various forms of any preparation
• Patients who were given a live vaccine within 8 weeks of the first dose of the drug
• Chemotherapy with approved or investigation anticancer therapeutics within 4 weeks prior to starting this study
• Patients who are unable to voluntarily agree to participate in a study or who are not willing to participate

Sponsors & Collaborators

• Seoul National University Hospital: lead

Study Sites (1)

Seoul National University Hospital

Seoul, 03080, South Korea

Location

MeSH Terms

Conditions

Amyloid Neuropathies, Familial

Interventions

Lenalidomide

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

Intervention Hierarchy (Ancestors)

Phthalimides; Phthalic Acids; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Piperidones; Piperidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Isoindoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Officials

• Youngil Koh, MD

  Seoul National University Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor

Study Record Dates

• First Submitted: December 3, 2019
• First Posted: March 6, 2020
• Study Start: February 20, 2019
• Primary Completion: November 25, 2021
• Study Completion: November 25, 2021
• Last Updated: April 17, 2024

Record last verified: 2024-04

Data Sharing

• IPD Sharing: Will not share

Locations

KR (1)