ClinicalTrials.gov record NCT07524634
Dose Schedule Study of BCMA Bispecific Antibody, Elranatamab, for Newly Diagnosed Immunoglobulin Light Chain (AL) Amyloidosis
Dose Schedule Investigation of B-Cell Maturation Antigen (BCMA) Bispecific Antibody, Elranatamab, for Treatment of Newly Diagnosed Light Chain Amyloidosis
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This Phase 2 trial enrolls adults newly diagnosed with immunoglobulin light‑chain (AL) amyloidosis who have not yet received treatment. Participants get inpatient step‑up doses, then subcutaneous elranatamab in one of two randomized 6‑month dosing schedules. Visits include blood tests, bone marrow biopsies, heart assessments, and follow‑up for up to 2 years (option to extend to 5 years).
What it is studying. The study tests elranatamab given for a fixed 6‑month course after initial step‑up dosing, and compares two dosing schedules to see effects on hematologic complete responses, organ (cardiac and renal) responses, disease measures, and safety.
What the researchers hope to learn. Measure hematologic complete response (hCR) at end of therapy; compare time to hCR and overall response rates between arms; assess duration of hematologic response; track progression‑free and overall survival; evaluate organ (cardiac and renal) response rates and time to organ response; record major organ deterioration‑free survival, hospitalization rates, cardiac events and infections; assess MRD negativity at end of therapy and at 24 months; measure quality of life changes and rates of hypogammaglobulinemia.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
This research study is for people who have newly diagnosed with AL (light chain) amyloidosis and have not yet received any treatment for this condition. The purpose of this study is to evaluate whether elranatamab, a type of immunotherapy drug, can produce deep remissions and organ recovery in people with newly diagnosed AL amyloidosis, and to compare two different dosing schedules. Elranatamab (brand name ELREXFIO™) is an investigational (experimental) drug in the setting of AL amyloidosis. It works by connecting immune cells (T-cells) directly to the abnormal plasma cells that are causing amyloidosis, triggering the immune system to destroy those cells. It is not approved by the Food and Drug Administration (FDA) for use in AL amyloidosis. Participants in this study will receive elranatamab as a series of injections under the skin (subcutaneously) over 6 treatment cycles (approximately 6 months). Treatment begins with inpatient "step-up" doses designed to reduce side effects, followed by two different dosing schedules based on which study arm participants are randomly assigned to. Participants will have regular blood tests, physical exams, bone marrow biopsies, and heart assessments throughout the study, and follow-up visits for up to 2 years after treatment ends. This study is randomized, meaning that participants will be assigned by chance (similar to a coin flip) to one of two treatment arms. Participants cannot choose their arm. Participation in this research will last approximately 6 months of active treatment, followed by follow-up visits for up to 2 years (with an option to extend to 5 years).
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- BCMA bispecific
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- Newly diagnosed Immunoglobulin Light Chain (AL) amyloidosis who have not received any prior therapy.
- Participants must have a tissue biopsy demonstrating Congo red positivity with characteristic birefringence on polarized microscopy and immunohistochemistry or mass spectrometry confirming light chain type.
- Participants must not have any evidence of myeloma defining events based on the International Myeloma Working Group (IMWG) myeloma diagnostic criteria (SLIM-CRAB). This excludes the light chain ratio criteria of involved versus uninvolved free light chains (FLC) over 100 in the absence of CRAB criteria.
- Age ≥ 18 years
- Eastern Cooperative Oncology Group (ECOG) performance status ≤2
- Participants must meet the following organ and marrow function as defined below: Absolute neutrophil count ≥1,000/mcL, Absolute platelet count ≥50,000/mcL, Direct bilirubin ≤1.5 × institutional upper limit of normal (ULN) AST(SGOT)/ALT(SGPT) ≤3 × institutional ULN.
- Participants must have a clonal plasma cell burden of less than 40%.
- For participants with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated.
- Participants with a history of hepatitis C virus (HCV) infection must have been treated and cured. For participants with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load.
- AL Amyloidosis Cardiac stage I, II or IIIa disease based on the 2015 European Modification of the 2004 Standard Mayo Clinic Staging in participants with advanced cardiac involvement (Dispenzieri et al., 2004; Wechalekar et al., 2013) (NT-proBNP <8500 ng/L and troponin criteria per staging system).
- The effects of Elranatamab on the developing human fetus are unknown. Based on the mechanism of action, Elranatamab may cause fetal harm when administered to a pregnant woman and therefore should not be used during pregnancy. For this reason, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation and until 90 days since the last dose of Elranatamab. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 90 days after completion of Elranatamab administration.
- Ability to understand and the willingness to sign a written informed consent document.
- Willingness to undergo study procedures, including bone marrow biopsies as detailed in the schedule of events.
Exclusion criteria
- Participants who are receiving any other investigational agents for this condition.
- Participants with Stage IIIB Amyloidosis as defined by the Europeans Revised 2004 Mayo Clinic Criteria.
- Participants with an active malignancy (including lymphoma) with the following exceptions: adequately treated basal cell carcinoma, squamous cell carcinoma, or in situ cervical cancer; adequately treated stage I cancer from which the participant is currently in remission and has been for over 2 years; low-risk prostate cancer with a Gleason score < 7 and prostate specific antigen < 10ng/mL; other localized, indolent and/or low risk cancer may be permitted.
- Women who are pregnant, breastfeeding, or planning to become pregnant while enrolled in this study or 4 months following discontinuation of Elranatamab, whichever is longer. Pregnant women are excluded from this study because Elranatamab is an agent with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with Elranatamab, breastfeeding should be discontinued if the mother is treated with Elranatamab.
- Have any other medical, social or psychological factors that could affect the participant's safety or ability to consent personally or comply with study procedures.
- Participants with active clinically significant autoimmune diseases.
- Participants seropositive for the human immunodeficiency virus (HIV).
- Severe, uncontrolled orthostatic hypotension resulting in syncopal/pre-syncopal events despite optimized medical management (e.g., midodrine, pyridostigmine) and in the absence of volume depletion.
- Plan for autologous stem cell transplant or solid organ transplant during the first 6 months of protocol therapy.
- History of acute coronary syndrome or uncontrolled ventricular arrhythmias within 3 months prior to screening.
- Evidence of Left Ventricular (LV) systolic dysfunction as defined by Left Ventricular Ejection Fraction (LVEF) is < 30% by echocardiogram at Screening per site cardiology interpretation.
- Have history of sustained ventricular tachycardia or aborted ventricular fibrillation or a history of atrioventricular nodal or sinoatrial nodal dysfunction if a permanent pacemaker (PPM) or implantable cardioverter-defibrillator (ICD) is not placed.
- QT corrected by Fridericia (QTcF) is > 550 msec on Screening ECG unless they have a PPM/ICD implanted.
- Screening EKG showing acute myocardial ischemia or active conduction system abnormalities with the exception of any of the following: First degree atrioventricular block; Second degree atrioventricular block Type 1 (Mobitz Type 1/Wenckebach type); Right or left bundle branch block (e.g., Left Bundle Branch Block, Right Bundle Branch Block, Left Anterior Fascicular Block, or Left Posterior Fascicular Block); Atrial fibrillation with a controlled ventricular rate; Bifascicular block assessed as benign by the Investigator
- Major surgery that required general anesthesia within 4 weeks of randomization or is planning major surgery during the study.
- NYHA class IV symptoms
- Participants with a Glomerular Filtration Rate (GFR) <20, unless stable on dialysis for at least 3 months
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Show the detailed description from the registry
Immunoglobulin light chain (AL) amyloidosis is a disorder of a certain type of blood cell (plasma cells). With this disorder, some plasma cells make abnormal, misfolded light chain proteins that can build up in tissues and organs, damaging them over time. In the United States, about 5 in every 100,000 people have this disorder. Even though it is rare, it can be very serious. Many people who have this disease are very sick upon diagnosis and require care from many different types of doctors. Cardiac (heart) involvement is the most common reason why people may get very sick and die. Contemporary plasma cell directed therapy has helped to improve outcomes, but many people still do not respond quickly or strongly enough. Some people still have worsening organ damage or cannot handle the strong treatments due to organ damage. In fact, early mortality is common, especially in people with cardiac involvement. There is a need for treatments that can quickly and safely lower harmful light chain levels, especially for people whose organs are already affected by the disease. Elranatamab is a type of drug called a bispecific antibody that connects a B cell maturation antigen (BCMA), a protein on plasma cells, with CD3, a protein on T cells. It can redirect T cells to kill plasma cells that are expressing BCMA. Treatments that use elranatamab have led to positive responses in people with multiple myeloma, a type of blood disease. There is early evidence to show that this drug may also help people with AL amyloidosis. This study will test elranatamab given for a set period of time in people with newly diagnosed AL amyloidosis. It will use two dosing schedules designed to balance efficacy with infection and tolerability risk in a population that is medically fragile.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants receive subcutaneous elranatamab with an initial inpatient “step‑up” dosing period, then continue for five additional 28‑day cycles (total ~6 months) on one of two randomized dosing schedules. They will have regular blood tests, physical exams, heart assessments, and bone marrow biopsies as scheduled. The study team will monitor for side effects and responses during treatment and at follow‑up visits for up to 2 years after treatment ends (with an option to extend follow‑up to 5 years). Participants cannot choose which dosing arm they are assigned to.
How the study is structured. Phase 2, open‑label, randomized trial with two parallel treatment arms. All participants receive inpatient "step‑up" subcutaneous doses of elranatamab, then continue for five additional 28‑day cycles on one of two assigned schedules (total ~6 months of active treatment). Participants are followed with clinical assessments, blood tests, bone marrow biopsies, and heart evaluations; some outcomes are followed for up to 60 months.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The registry says recruitment has not started. When is it expected to open, and can I register interest now?
- The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on AL amyloidosis
Important dates
- Study start
- August 2026 (estimated)
- Primary completion
- June 2031 (estimated)
- Study completion
- June 2031 (estimated)
- Target enrollment
- 64 participants (estimated)
- Last updated on registry
- Aug 5, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Case Comprehensive Cancer Center
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Sandra Mazzoni, DO 📞 216-444-8111 ✉ mazzons@ccf.org
Participating sites (2)
- Mayo Clinic
Rochester, Minnesota, 55902 · United States
- Taxiarchis Kourelis, MD · 507-284-2511 · Kourelis.taxiarchis@mayo.edu
- Taxiarchis Kourelis, MD
- Case Comprehensive Cancer Center, Cleveland Clinic Foundation Taussig Cancer Institute
Cleveland, Ohio, 44195 · United States
- Sandra Mazzoni, DO · 216-444-8111 · mazzons@ccf.org
- Sandra Mazzoni, DO
Terms used on this page · explanations by MedicalUpdates.ai
- investigational
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- study arm
- One of the groups in a study. Each arm receives a particular intervention, comparator or no intervention, so the groups can be compared.
- randomly assigned
- Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
- follow-up
- The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
- prior therapy
- Treatment a person has already had for the condition. A 'line of therapy' is one course of treatment; studies often require, or limit, the number of prior lines.
- biopsy
- Removal of a small piece of tissue so it can be examined under a microscope. 'Biopsy-proven' or 'histologically confirmed' means the diagnosis was confirmed by examining tissue under a microscope.
- free light chains
- Small pieces of antibody protein made by plasma cells and measured in a blood test. In some conditions, such as AL amyloidosis, abnormal free light chains are the source of the disease.
- ECOG
- A scale from 0 to 5 that describes how a person's condition affects daily activity. 0 means fully active; 1 means restricted in strenuous activity; 2 means able to self-care but unable to work; higher numbers mean more limitation.
- Absolute neutrophil count
- The number of neutrophils, a type of white blood cell that fights infection, in a blood sample. Studies often require it to be above a set level.
- upper limit of normal
- The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
- written informed consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- adverse events
- Any unwanted medical problem that happens during a study, whether or not it was caused by the treatment. A 'serious' adverse event is one that meets specific criteria, such as causing death, being life-threatening, requiring or prolonging hospital admission, causing significant disability, or another medically important outcome.
- autologous stem cell transplant
- A procedure that replaces blood-forming cells after high-dose treatment. 'Autologous' uses the person's own cells collected beforehand; 'allogeneic' uses cells from a donor.
- prior to screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- echocardiogram
- An ultrasound scan of the heart. A transesophageal echocardiogram is done through a probe passed into the food pipe for a closer view.
- QTcF
- A recording of the heart's electrical activity. QTc is one measurement from it; some drugs are avoided if it is prolonged.
- Glomerular Filtration Rate
- A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- bispecific antibody
- An engineered antibody that attaches to two targets at once, often linking an immune cell to a diseased cell so the immune cell can attack it.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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