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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.

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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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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. Pick your insurance to see what fits.

Your insurance
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 · The Assistance Fund
    Amyloidosis fund
    Open

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

    The foundation says: “OPEN — Accepting New Patients. TAF is currently accepting new patient enrollments for this program.”
  • 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.

Status as each foundation showed it on September 28, 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 18 studies recruiting patients and 2 opening soon in the United States and worldwide, with eligibility criteria in plain English. 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.

TrialsSite mapPipeline timeline

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.

70 active trials worldwide
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RECRUITINGPHASE1Recently 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 yesterdayEst. Apr 2027 (~6 months)
RECRUITINGPHASE1Recently 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 4 days agoEst. Jan 2029 (~2y 3m)
NOT YET RECRUITINGEARLY_PHASE1Recently updatedNCT07615270

A Pilot Study of Anselamimab in Patients With AL Amyloidoma and Measurable Tissue Involvement

Intervention: Anselamimab

Sponsor: Stanford University

This is an exploratory study to assess the binding of CAEL-101/anselamimab to amyloid in vivo, recruitment of inflammatory cells and reduction of the amyloid mass.

Ages 18 Years+1 location
Started Sep 2026Updated 1 month agoEst. Jul 2028 (~1y 10m)
RECRUITINGPHASE1, PHASE2Recently updatedNCT05145816

Phase 1/2a Study of Belantamab Mafodotin in Relapsed or Refractory AL Amyloidosis

Intervention: Belantamab mafodotin 2.5 mg/kg (8 weeks), Belantamab mafodotin 1.9 mg/kg (8 weeks), Belantamab mafodotin 1.4 mg/kg (12 weeks), Belantamab mafodotin 1.9 mg/kg (12 weeks), Belantamab mafodotin every 8 weeks or 12 weeks as determined by Part 1 recommended dosages, Belantamab mafodotin 1.0 mg/kg (12 weeks)

Sponsor: University of Texas Southwestern Medical Center · GlaxoSmithKline

The goal of this study is to test the safety of drug, Belantamab Mafodotin, and see what effects (good and bad) it has on people who take it and have amyloidosis, and to determine the most effective dose of the drug.

Ages 18 Years+3 locations
Started Feb 2024Updated 1 month agoEst. Mar 2027 (~5 months)
RECRUITINGPHASE1, PHASE2Recently updatedNCT06569147

Elranatamab in Patients With Relapsed or Refractory AL Amyloidosis

Intervention: Elranatamab

Sponsor: Dana-Farber Cancer Institute

This study will evaluate the safety, tolerability and efficacy of elranatamab in patients with relapsed or refractory AL amyloidosis.

Ages 18 Years+5 locations
Started Nov 2024Updated 1 month agoEst. Jan 2029 (~2y 3m)
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Active trial locations114 cities in the US
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Trial Pipeline

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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

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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