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Home/Rare Diseases/Light Chain Amyloidosis

Blood & Immune

Light Chain Amyloidosis Clinical Trials and Treatments

Also called AL Amyloidosis, Primary Amyloidosis, Systemic Light Chain Amyloidosis

Light Chain Amyloidosis results from clonal plasma cell proliferation producing excessive amounts of misfolded immunoglobulin light chains (kappa or lambda) that cannot be properly degraded. These misfolded proteins aggregate to form amyloid fibrils that deposit in various tissues, causing progressive organ dysfunction.

FDA-approved medicines

1

Darzalex Faspro

Active trials

48

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Next FDA decision

None set

FDA calendar

Medicines are those whose current FDA label names Light Chain Amyloidosis, checked October 14, 2026. Corticosteroids and immune globulins count once each. Download the data

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About Light Chain Amyloidosis

Light Chain Amyloidosis results from clonal plasma cell proliferation producing excessive amounts of misfolded immunoglobulin light chains (kappa or lambda) that cannot be properly degraded. These misfolded proteins aggregate to form amyloid fibrils that deposit in various tissues, causing progressive organ dysfunction.

The heart is affected in approximately 50-70% of patients, leading to restrictive cardiomyopathy with diastolic dysfunction, arrhythmias, and sudden cardiac death. Renal involvement occurs in 40-50% of patients, presenting as nephrotic syndrome with heavy proteinuria. Peripheral neuropathy develops in approximately 10-15% of patients, ranging from small fiber neuropathy to autonomic dysfunction. Other affected organs include liver, eyes, gastrointestinal tract, and soft tissues.

The underlying plasma cell clone is usually modest in size and may not meet criteria for multiple myeloma. Early diagnosis is critical, as untreated disease rapidly progresses to multi-organ failure. Median survival without treatment is approximately 2-4 years when cardiac involvement is present.

Common Symptoms of Light Chain Amyloidosis

Recognizing the signs of Light Chain Amyloidosis early can lead to faster diagnosis and better outcomes. Symptoms may vary in severity from person to person. If you or a loved one are experiencing any of the following, consider speaking with a specialist.

  • Fatigue and weakness
  • Shortness of breath and heart failure symptoms
  • Swelling in ankles, feet, legs, and abdomen (edema)
  • Numbness, tingling, or pain in hands and feet (peripheral neuropathy)
  • Proteinuria (protein in urine) and kidney dysfunction
  • Unexplained weight loss

Who Light Chain Amyloidosis Affects

Light Chain Amyloidosis typically affects older adults, with median age of diagnosis around 63 years, though younger patients can be affected. It affects males more frequently than females. The disease occurs across all racial and ethnic groups. It predominantly affects individuals of European ancestry, though increasing recognition in other populations suggests possible underdiagnosis.

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Find trials I might fit Answer a few questions and see which open Light Chain Amyloidosis trials fit your age, location and treatment history.

FDA-Approved Treatments for Light Chain Amyloidosis

There is currently 1 FDA-approved medication for Light Chain Amyloidosis. These therapies represent the current standard of care and may be used alongside or compared against investigational treatments in active clinical trials.

daratumumab and hyaluronidase-fihj
Janssen Biotech (Johnson & Johnson)
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Source: openFDA drug labeling data. This list may not include all treatments. Always consult your doctor.

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Help Paying for Light Chain Amyloidosis Treatment

Charity funds and drugmaker programs for Light Chain Amyloidosis, checked at the source.

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Charity funds
  • From a charity · HealthWell Foundation
    Amyloidosis fund
    Open

    Pays for: Copays, premiums or other treatment costs.

  • From a charity · NORD RareCare
    Amyloidosis Medical Assistance fund
    Open

    Pays for: Medical and medication costs.

    The foundation says: “Accepting Applications”
  • From a charity · NORD RareCare
    Amyloidosis Premium Copay Assistance fund
    Open

    Pays for: Insurance premiums and copays.

    The foundation says: “Accepting Applications”
  • From a charity · TotalAssist (formerly PAN Foundation)
    Amyloidosis fund
    Open

    Pays for: Out-of-pocket costs for approved medications, up to $5,500 per year. Requires Medicare, Medicaid or TRICARE.

  • From a charity · The Assistance Fund
    Amyloidosis fund
    Waitlist

    Pays for: Copays, coinsurance, deductibles and other health-related expenses.

    The foundation says: “WAITLIST — Accepting Waitlist Patients. TAF is currently accepting requests to join the enrollment waitlist for this program. Waitlists a…”
Status as each foundation showed it on October 12, 2026.
Drugmaker programs
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Side Effect Explorer

Real-world side effect reports from the FDA Adverse Event Reporting System (FAERS). Includes both FDA-approved drugs and investigational therapies from active clinical trials. Click any drug to see what patients reported.

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Genetic Testing

Genetic testing can confirm a diagnosis, guide treatment decisions, and identify family members who may be at risk.

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Trusted Light Chain Amyloidosis Resources

Reputable organizations and medical references for learning more about Light Chain Amyloidosis, including disease registries, foundation resources, and clinical guidelines.

Active Clinical Trials for Light Chain Amyloidosis

Use this Light Chain Amyloidosis clinical trial finder to see the 16 studies recruiting patients and 4 opening soon in the United States and worldwide, with eligibility criteria in plain English. Of these, 17 test a treatment and 3 are observational studies or registries; each card says which. These studies play a critical role in advancing care for blood & immune conditions and may offer access to treatments not yet widely available. Each trial below is sourced directly from ClinicalTrials.gov, with eligibility criteria translated into plain English to help patients and caregivers evaluate whether a study may be a fit.

New to trials? What Placebos and Randomization Actually Mean for You (In Plain English) · 4 min readWorried about cost? The Hidden Costs of Joining a Clinical Trial (And How to Get Help Paying for Them) · 4 min read
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Note: Trial recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator before making plans.

48 active trials worldwide
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NOT YET RECRUITINGTreatment trialPhase 1Recently updatedNCT07874334

Phase 1a/b Study of GPRC5D CART Therapy in Relapsed/Refractory Multiple Myeloma and Light-chain Amyloidosis

Intervention: GPRC5D CART

Sponsor: Hadassah Medical Organization

A Phase 1a/b Open-Label Study to Evaluate Safety and Preliminary Efficacy of GPRC5D CART Therapy for the Treatment of Relapsed/Refractory Multiple Myeloma and Light-chain Amyloidosis.

Ages 18 Years+1 location
Expected to start Sep 2026Updated yesterdayEst. Dec 2032 (~6y 2m)
RECRUITINGTreatment trialPhase 2Recently updatedNCT05898646

Daratumumab Maintenance Therapy for Improving Survival in Patients With Light Chain Amyloidosis, EMILIA Trial

Intervention: Bone Marrow Aspiration, Daratumumab, Echocardiography Test, Questionnaire Administration, X-Ray Imaging, Biospecimen Collection

Sponsor: Mayo Clinic

This phase II trial compares shorter-duration versus longer-duration maintenance therapy with daratumumab for improving survival in patients who have received initial treatment with daratumumab for light chain (AL) amyloidosis. Maintenance therapy is treatment that is given to he...

Ages 18 Years+2 locations
Started Sep 2023Updated 2 weeks agoEst. Nov 2029 (~3y 1m)
RECRUITINGObservational study, no treatment givenRecently updatedNCT07850284

SMS-Based Remote Symptom Monitoring for Patients With Plasma Cell Disorders

Intervention: Non-Interventional Study

Sponsor: Mayo Clinic

This study evaluates whether daily SMS-based (text messages) symptom monitoring is feasible and acceptable in patients who are undergoing treatment for multiple myeloma (MM) or systemic light chain amyloidosis (AL), and whether it can help in early identification of symptoms that may require clinical attention.

Ages 18 Years+1 location
Started Aug 2026Updated 2 weeks agoEst. Aug 2028 (~1y 10m)
RECRUITINGTreatment trialPhase 1Recently updatedNCT05652335

A Study of JNJ-79635322 in Participants With Relapsed or Refractory Multiple Myeloma or Previously Treated Amyloid Light-chain (AL) Amyloidosis

Intervention: JNJ-79635322

Sponsor: Janssen Research & Development, LLC

The primary purpose of this study is to identify the recommended phase 2 dose (RP2D[s]) and schedule(s) to be safe for JNJ-79635322 in Part 1 (dose escalation), and to characterize the safety and tolerability of JNJ-79635322 at the RP2D(s) selected and in disease subgroups in Part 2 (dose expansion).

Ages 18 Years+29 locations
Started Nov 2022Updated 2 weeks agoEst. Apr 2027 (~6 months)
RECRUITINGTreatment trialPhase 1Recently updatedNCT06768489

A Study of JNJ-79635322 in Combination With Daratumumab With or Without Lenalidomide for Multiple Myeloma, Newly Diagnosed AL Amyloidosis, and High-risk Smoldering Multiple Myeloma or JNJ-79635322 in Combination With Pomalidomide for Multiple Myeloma

Intervention: JNJ-79635322, Daratumumab, Pomalidomide, Lenalidomide

Sponsor: Janssen Research & Development, LLC

The primary purpose of this study for Part 1 (Dose Escalation) is to identify the safe effective dose (recommended Phase 2 doses [RP2Ds]) and schedule for JNJ-79635322 treatment regimen in combination with daratumumab with or without lenalidomide or with pomalidomide; and for Par...

Ages 18 Years+16 locations
Started Dec 2024Updated 2 weeks agoEst. Jan 2029 (~2y 2m)
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Find Light Chain Amyloidosis trials near you, ranked by distance →
Active trial locations106 cities in the US
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Trial Pipeline

Jan 2021 to Oct 2031
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Data from ClinicalTrials.gov, U.S. National Library of Medicine.
Always talk to your doctor before considering a clinical trial.

Patient Communities

Connect with other Light Chain Amyloidosis patients, caregivers, and advocacy groups across Facebook groups, Reddit communities, and YouTube channels. These patient communities offer peer support, shared experiences, caregiver resources, and real-time discussion about Light Chain Amyloidosis treatments, clinical trial participation, and day-to-day disease management.

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Related Blood & Immune Conditions

Other rare diseases in the blood & immune category. Patients with Light Chain Amyloidosis may find relevant research, shared treatment pathways, or overlapping clinical trials among these related conditions.

Companies Developing Light Chain Amyloidosis Treatments

12 pharmaceutical companies have Light Chain Amyloidosis in their rare disease portfolio

Frequently Asked Questions About Light Chain Amyloidosis