> Trialight public data. Index of pages: https://trialight.com/llms.txt
> Web page: https://trialight.com/data/condition/amyloidosis-b8b4cb9d-0eae-5851-8654-8d69fd195e67
> Record changed: 2026-10-13

# Amyloidosis

Therapeutic area

Metabolic Disease

Rare disease

Yes

## Definition and classification

**Definition**

A disorder characterized by the localized or diffuse accumulation of amyloid protein in various anatomic sites. It may be primary, due to clonal plasma cell proliferations; secondary, due to long standing infections, chronic inflammatory disorders, or malignancies; or familial. It may affect the nerves, skin, tongue, joints, heart, liver, spleen, kidneys and adrenal glands.

**Genetic basis**

No

**Non-human condition**

No

**Rare disease**

Yes

**Listing at source**

Listed

**Condition name**

Amyloidosis

**Therapeutic area**

Metabolic Disease

### Alternative names (6)

- amyloidosis
- amyloid
- amyloid disease
- amyloidoses
- amyloidosis
- amyloidosis (disease)

## Clinical profile

**All therapeutic areas**

- Metabolic Disease

**Severity**

Severe

## Identifiers

**MONDO ID**

MONDO:0019065

Source: [monarchinitiative.org](https://monarchinitiative.org/MONDO:0019065)

**Parent condition**

MONDO:0021179

## Related records

### Broader conditions (6)

- Disease
- Disease by developmental or physiological process
- Disease by molecular mechanism
- Human disease
- Metabolic disease
- Proteostasis deficiencies

### Narrower conditions (52)

- AA amyloidosis
- AApoAI amyloidosis
- AApoAIV amyloidosis
- ABeta amyloidosis, Arctic type
- ABeta amyloidosis, dutch type
- ABeta amyloidosis, Iowa type
- ABeta amyloidosis, Italian type
- ABeta2M amyloidosis
- ABetaA21G amyloidosis
- ABetaL34V amyloidosis
- ABri amyloidosis
- ACys amyloidosis
- ADan amyloidosis
- AFib amyloidosis
- AH amyloidosis
- AL amyloidosis
- ALECT2 amyloidosis
- ALys amyloidosis
- Alzheimer disease type 1
- Amyloidosis bronchopulmonary

### Trials (302)

- \[18F\]FT8 PET Imaging in Immunoglobulin Light Chain Amyloidosis
- \[64Cu\]FBP8 PET for Early Detection of Intracardiac Thrombus in Amyloid Cardiomyopathy
- 124I-Evuzamitide PET/CT Imaging In Patients With Lumbar Spinal Stenosis and Carpal Tunnel Syndrome
- 124I-p5+14 Injection Safety in Subjects With Systemic Amyloidosis
- 24 Month Open Label Study of the Tolerability and Efficacy of Inotersen in TTR Amyloid Cardiomyopathy Patients
- A Clinical Study of CD19/BCMA CAR-T Cells in the Treatment of Refractory POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, and Vasculitis
- A Clinical Study on Minimal Residual Disease in Patients With Systemic Light Chain Amyloidosis
- A Cohort Study of Plasma Cell Disorders (PCDs) in PKUFH
- A Dose Escalation Study of Carfilzomib Taken With Thalidomide and Dexamethasone in Relapsed AL Amyloidosis
- A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation
- A Non-interventional Cohort Safety Study of Patients With hATTR-PN
- A Phase 1 Study of 99mTc-p5+14 in Healthy Volunteers and Patients With AL or ATTR Systemic Amyloidosis
- A Phase 1b/2 Study of CAR T Cell Therapy Targeting CD19 and BCMA in Participants With Relapsed or Refractory AL Amyloidosis.
- A Phase 3 Study of NTLA-2001 in ATTRv-PN
- A Phase I/II Open-label, Single-arm, Multicenter Clinical Study to Evaluate the Safety and Efficacy of FKC289 in Subjects With Relapsed/Refractory Primary AL Amyloidosis
- A Pilot Study of Lenalidomide, Melphalan and Dexamethasone in AL Amyloidosis
- A PILOT Study on LSGB vs EMB in the Diagnosis of Cardiac Transthyretin Amyloidosis
- A Randomized Phase 3 Study to Evaluate the Efficacy and Safety of NXC-201 Compared With Daratumumab With Cyclophosphamide, Bortezomib and Dexamethasone (CyBorD) in Newly Diagnosed Systemic AL Amyloidosis
- A Registry of AL Amyloidosis (ReAL)
- A Safety and Tolerability Study of an Investigational Drug, ALN-TTRSC02, in Healthy Subjects

### Approved drugs (5)

- Acoramidis hydrochloride
- Inotersen sodium
- Patisiran sodium
- Tafamidis
- Tafamidis meglumine

### Drugs in development (31)

- Acoramidis Phase 1
- Anselamimab Phase 1
- APG-2575 Phase 1
- Belantamab mafodotin Phase 1
- Birtamimab Phase 2
- [Carfilzomib](https://trialight.com/data/asset/carfilzomib-42f49602-1f34-4e14-b345-79d6fd69324f.md) Phase 1
- [Cyclophosphamide](https://trialight.com/data/asset/cyclophosphamide-25b75968-e3cd-410a-9b24-0c349d8f7663.md) Phase 3
- [Daratumumab](https://trialight.com/data/asset/daratumumab-28c327e5-1741-4dd5-89d4-6562e941ab50.md) Phase 3
- [Dexamethasone](https://trialight.com/data/asset/dexamethasone-b923ddae-f095-408c-96e9-975238a429ad.md) Phase 3
- Dezamizumab Phase 2
- [Doxycycline](https://trialight.com/data/asset/doxycycline-9994da3d-bd1e-4dba-9a9e-392765b5aa58.md) Phase 2
- [Eplontersen](https://trialight.com/data/asset/eplontersen-9180ea26-6471-4bf8-b8bc-f14ee872ae88.md) Phase 1
- Eplontersen sodium Phase 1
- Eprodisate disodium Phase 3
- GSK-294 Phase 1
- [Ibrutinib](https://trialight.com/data/asset/ibrutinib-0b3ac4fd-6e98-4847-a711-2f599e8bb93d.md) Phase 2
- Idelalisib Phase 2
- Inotersen Phase 3
- Iodine I124 evuzamitide Phase 2
- [Lenalidomide](https://trialight.com/data/asset/lenalidomide-1e614dfb-efd0-4006-a121-30a97656c7a9.md) Phase 3

### Other linked drugs (3)

- [Bortezomib](https://trialight.com/data/asset/bortezomib-35a853cf-26ab-4a4c-a405-ae671d364aad.md)
- Diflunisal
- Ixazomib citrate

---

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