Ongoing Clinical Trials for Amyloidosis
Currently, there are 3 ongoing clinical trials investigating new treatment approaches for amyloidosis. These studies are being conducted across several European countries including Belgium, France, Germany, Greece, Italy, and the Netherlands. The trials focus on different forms of the disease, including AL amyloidosis and transthyretin amyloid cardiomyopathy, testing various medications such as Isatuximab, Belantamab Mafodotin, and Tafamidis.
Clinical trial locations
- Belgium
- France
- Germany
- Greece
- Italy
- Netherlands
Study on the Effectiveness of Isatuximab, Pomalidomide, and Dexamethasone for Patients with AL Amyloidosis Not Responding Well to Previous Treatments
This trial focuses on patients with AL amyloidosis, a rare condition where abnormal proteins called amyloids accumulate in organs and tissues, disrupting their normal function. The study evaluates a combination treatment of three medications: Isatuximab, Pomalidomide, and Dexamethasone.
Main inclusion criteria: Participants must be at least 18 years old with a confirmed diagnosis of AL amyloidosis. They should have received at least one previous treatment with specific medications such as an alkylating agent, PI, or anti-CD38, at least one year before joining the study, without achieving a very good partial response. Participants must have measurable disease with specific blood protein differences greater than 50 mg/L and symptoms affecting organs like the heart, kidneys, liver, digestive system, or nerves. They must have adequate bone marrow and organ function, and an ECOG status of 2 or less, which measures their ability to perform daily activities. Women of childbearing age must use reliable birth control methods and have negative pregnancy tests throughout the study.
Main exclusion criteria: Patients with other serious health conditions that could interfere with the study, those who are pregnant or breastfeeding, recent participants in other clinical trials, those with allergies to the study medication, patients unable to follow study procedures, those with uncontrolled infections, and patients with severe kidney or liver problems are excluded.
Focus and goals: The study aims to assess how well this combination of medications works in patients who have not achieved a very good response from previous treatments. The primary goal is to evaluate the hematologic response after 6 cycles of treatment. Participants will receive treatment over a period of up to 12 months, with regular monitoring and assessments to track their health and treatment effectiveness.
Investigational drugs: Isatuximab is a monoclonal antibody administered intravenously that targets specific cells to help the immune system attack abnormal cells. Pomalidomide is taken orally in capsule form and helps the immune system fight the disease by affecting cell growth and reducing inflammation. Dexamethasone is a steroid taken orally as tablets that reduces inflammation and enhances the effects of the other medications.
Study of Belantamab Mafodotin for Patients with Relapsed or Refractory AL Amyloidosis
This trial is designed for patients with AL amyloidosis whose disease has returned after treatment or has not responded to previous therapies. The condition involves the buildup of amyloid proteins in organs and tissues, leading to organ dysfunction.
Main inclusion criteria: Participants must be at least 18 years old with a confirmed diagnosis of AL amyloidosis. They must have received at least two cycles of plasma cell-directed treatment or high-dose therapy with melphalan. Participants must have an ECOG performance status of 0, 1, or 2, and be in Mayo stage 1, 2, or 3A1-3. They must have a supine systolic blood pressure of at least 90 mmHg and measurable disease with specific protein levels in the blood. Symptoms must affect organs such as the heart, kidneys, liver, or nervous system, and participants must have adequate organ function. Both male and female participants must agree to use effective birth control methods during the study and for a specified period afterward.
Main exclusion criteria: Patients without relapsed or refractory AL amyloidosis, those outside the specified age range, patients not in the designated clinical trial groups, and members of vulnerable populations not selected for the study cannot participate.
Focus and goals: The study evaluates the effectiveness of Belantamab Mafodotin in treating relapsed or refractory cases. The primary objective is to assess the response rate at 6 months or after 4 cycles of therapy. Participants will be closely monitored through regular follow-up visits to evaluate treatment effectiveness and observe any side effects.
Investigational drugs: Belantamab Mafodotin is administered intravenously as a powder mixed into a solution for injection. It is an antibody-drug conjugate that targets and binds to a specific protein on the surface of certain cells, delivering a toxic agent to destroy these cells and potentially reduce symptoms.
Study on How Tafamidis is Absorbed in the Blood of Healthy Adults with Transthyretin Amyloid Cardiomyopathy
This trial studies how different forms of Tafamidis are absorbed into the blood in healthy adults. Tafamidis is used to treat transthyretin amyloid cardiomyopathy, a heart condition where abnormal protein deposits accumulate in heart tissue, causing the heart walls to become stiff and affecting its ability to pump blood effectively.
Main inclusion criteria: Participants must be 18 years or older and determined to be healthy through medical evaluation, including medical history review, physical examination, and laboratory tests. They should be healthy females who cannot have children or healthy males. Participants must have a body mass index between 16 and 32 kg/m² and a total body weight of more than 45 kg.
Main exclusion criteria: Participants with any medical condition that might interfere with study results, those outside the specified age range, individuals who are not healthy, and members of vulnerable populations are excluded.
Focus and goals: The study compares the absorption of two different tablet forms of Tafamidis (61 mg and 70 mg) to a capsule form (61 mg) when taken by healthy participants who have not eaten. Each participant will receive a single dose of each form in a randomized order across three periods. The goal is to estimate the relative bioavailability and understand how much of the drug enters the bloodstream and is available to have an effect.
Investigational drugs: Tafamidis is administered orally in tablet or capsule form. It works by stabilizing a protein called transthyretin, preventing it from breaking apart and forming harmful deposits in the body. This medication is classified as a transthyretin stabilizer.
Summary
The three ongoing clinical trials for amyloidosis reflect diverse research approaches targeting different forms of the disease. Two trials focus specifically on AL amyloidosis, particularly in patients whose disease has relapsed or not responded well to previous treatments, while one trial addresses transthyretin amyloid cardiomyopathy.
Geographically, the trials show concentration in France, which hosts two studies, while Belgium hosts one focused trial on medication absorption in healthy volunteers. The multi-country trial involving Belantamab Mafodotin spans five European nations, demonstrating broad international collaboration.
The investigational approaches vary considerably, from combination therapies using Isatuximab, Pomalidomide, and Dexamethasone to single-agent studies with Belantamab Mafodotin, and bioavailability research with different Tafamidis formulations. These trials collectively represent important efforts to develop new treatment options for patients affected by these challenging conditions.
