Clinical trials on Light Chain Amyloidosis

Light chain amyloidosis, also known as AL amyloidosis, is a rare disease that occurs when abnormal proteins, called light chains, build up in various tissues and organs in the body. These proteins are produced by plasma cells, a type of white blood cell. In healthy individuals, light chains function normally and do not accumulate. However, in patients with AL amyloidosis, the light chains misfold and form amyloid deposits, which can disrupt the normal function of organs. This condition can affect different parts of the body, including the heart, kidneys, liver, and nerves.


The complications of light chain amyloidosis are serious and can affect multiple body systems. Key complications include:
  • Heart problems: Amyloid deposits in the heart can lead to cardiomyopathy, which impairs the heart’s ability to pump blood and can cause heart failure.
  • Kidney damage: The kidneys can be affected, leading to protein in the urine and progressive kidney failure.
  • Nerve damage: Peripheral neuropathy might occur, causing numbness, tingling, and pain in the hands and feet.
  • Gastrointestinal disturbances: Amyloidosis can cause issues like gastrointestinal bleeding, malabsorption, and altered bowel habits.
  • Liver enlargement: Involvement of the liver can lead to hepatomegaly (an enlarged liver), which may impair liver function.

Treatment Methods

The treatment of light chain amyloidosis focuses on reducing the production of abnormal light chains and managing symptoms. Current, scientifically-backed treatment methods include:
  • Chemotherapy: Drugs used to kill plasma cells that produce the abnormal proteins.
  • Stem cell transplantation: High-dose chemotherapy followed by stem cell transplantation to help rebuild a healthy bone marrow.
  • Targeted therapy: Drugs like bortezomib that specifically target and kill plasma cells.
  • Immunotherapy: Treatments that help the immune system recognize and destroy plasma cells producing abnormal light chains.


The prognosis for patients with light chain amyloidosis varies based on the extent of organ involvement and response to treatment. Without treatment, the disease typically progresses rapidly, leading to significant organ failure and reduced survival. With appropriate treatment, patients may experience stabilization of the disease or even improvement, particularly if the treatment is started early before extensive organ damage occurs. Early diagnosis and effective management are crucial for improving the outlook and quality of life for patients with this condition.
  • CT-EU-00117859

    Isatuximab Plus Pomalidomide and Dexamethasone for AL Amyloidosis

    This clinical trial is focused on patients with AL Amyloidosis who have not achieved a very good partial response or better following previous therapies. The trial investigates the efficacy of combining Isatuximab, Pomalidomide, and Dexamethasone. The aim is to see if this drug combination can significantly reduce the levels of misfolded proteins in the blood, which are characteristic of this disease, thereby improving the condition and potentially prolonging life. This study seeks to offer a new potential treatment option for those who have limited responses to existing therapies.

    • Isatuximab
  • Study of JNJ-79635322 for Multiple Myeloma and Amyloidosis

    This study is investigating a new drug called JNJ-79635322, which is a trispecific antibody. The main goal of this study is to establish a recommended dose and schedule of drug administration that will be safe for participants. This medicine is intended for people with relapsed or refractory multiple myeloma or previously treated light chain (AL) amyloidosis.

    In the first part of the study, called dose escalation, doctors will gradually increase the dose of the medicine to find the highest dose that is well tolerated. In the second part, called dose expansion, the medicine will be administered at a previously established dose and doctors will monitor the safety and tolerability of the medicine in different groups of patients.

    JNJ-79635322 will be administered as a subcutaneous injection. During the study, doctors will carefully monitor any side effects and abnormal laboratory test results. The most important thing is to ensure the safety of participants and find the optimal dose of the drug.

    • JNJ-79635322
  • Study of SAR445514 for patients with relapsed/refractory multiple myeloma and light-chain amyloidosis.

    This comprehensive study delves into SAR445514’s potential to treat two serious disorders: relapsed/refractory multiple myeloma (RRMM) and relapsed/refractory light-chain amyloidosis (RRLCA). The trial is structured into three detailed phases. Initially, the dose-escalation phase aims to identify the safest dose level. Following this, the dose-optimization phase seeks to determine the most effective dose. Finally, the expansion phase rigorously evaluates the therapeutic impact of SAR445514 at the optimized dose. Throughout these phases, the study meticulously monitors participant responses, side effects, and overall health changes. The ultimate goal is to establish a new, effective treatment option, enhancing the quality of life and survival prospects for individuals battling these complex and challenging conditions.

    • SAR445514- new potential medication for advanced blood cancers
  • Testing birtamimab for safety and effectiveness in patients with severe AL Amyloidosis

    This medical study aims to investigate a drug called birtamimab to understand if it effectiveness and is safe for people in the advanced stage (Mayo Stage IV) of a disease called AL Amyloidosis. In this study, scientists are comparing the outcomes of two groups of people: one group receiving birtamimab in addition to standard-of-care chemotherapy, and another group receiving a neutral drug (placebo) along with standard-of-care chemotherapy. The goal is to assess whether the group receiving birtamimab has better results or outcomes compared to the group receiving the placebo. Patients will be chosen randomly to either receive birtamimab or placebo. The study includes an Open-label Extension (OLE) Phase, which allows participants who complete the initial trial phase to continue receiving birtamimab.

    • Birtamimab
  • Treatment study of AL amyloidosis with belantamab mafodotin

    This study examines belantamab mafodotin in patients with relapsed or refractory light chain (AL) amyloidosis, a rare disease caused by an abnormal protein. Approximately 35 participants will receive this drug therapy. The study consists of a screening phase, treatment phase, post-treatment observation, and long-term follow-up, with a safety run-in for the first 6 subjects. The primary goal is to assess treatment responses, while also monitoring for adverse events. This research is crucial for advancing treatment options for patients with this challenging condition.

    • Belantamab mafodotin