Rare Dermatology News
Spotlight On
AL amyloidosis
AL Amyloidosis is caused by the accumulation of an immunoglobulin light chain protein
Prevelance
30 / 100,000
Age of Onset
ICD-10
E85.9
Inheritance
Autosomal dominant
Autosomal recessive
Mitochondrial/Multigenic
X-linked dominant
X-linked recessive
5 Facts you should know
FACT
1
In AL amyloidosis, light chains come together to form amyloid deposits, which can cause serious damage to different organs
FACT
2
The kidneys are the most commonly affected organ in AL amyloidosis
FACT
3
Heart complications, which affect more than a third of AL patients, include heart failure and irregular heart beat
FACT
4
The most effective treatment is autologous bone marrow transplants with stem cell rescues
FACT
5
Two thirds of patients with AL amyloidosis are male and less than 5% of patients are under 40 years of age
Interest over time
Google searches
Common signs & symptoms
At the time this analysis was conducted, there were no common signs or symptoms for this disease.
Current treatments
The resources below provide information about treatment options for this condition. If you have questions about which treatment is right for you, talk to your healthcare professional.
Management Guidelines
- The Amyloid Treatment and Research Program at Boston University School of Medicine provides information on the treatment of different forms of amyloidosis.
Top Clinical Trials
| Title | Description | Phases | Status | Interventions | More Information |
|---|---|---|---|---|---|
| Propylene Glycol-Free Melphalan Hydrochloride (Evomela) in AL Amyloidosis Patients | This is a single arm, open label study designed to evaluate the safety and efficacy of propylene glycol-free melphalan hydrochloride in patients with AL amyloidosis. Treatment will be comprised of propylene glycol-free melphalan hydrochloride administered intravenously at a dose of 70-100 mg/m2/day on Days -3 and -2 as conditioning prior to autologous stem cell transplantation. | Phase 2 | Recruiting | Drug: Propylene Glycol-Free Melphalan Hydrochloride | Click here for more information |
| Phase 1/2 Study of ZN-d5 for the Treatment of Relapsed or Refractory Light Chain (AL) Amyloidosis | This is a single arm, Open-Label, Phase 1/2 Study of ZN-d5 for the Treatment of Relapsed or Refractory Light Chain (AL) Amyloidosis. | Phase 1/2 | Recruiting | Drug: ZN-d5 | Click here for more information |
| COVID-19 VAX Booster Dosing in Patients With Hematologic Malignancies | To determine whether protective antibody levels increase after booster dosing with the Moderna COVID-19 vaccine in patients diagnosed with Hematologic Malignancies who have low antibody levels after a prior first vaccination with any of the SARS-CoV2 vaccines that were authorized for use in the USA. | Phase 2 | Recruiting | Drug: A single "booster" dose of the Moderna mRNA COVID-19 vaccine | Click here for more information |
| Daratumumab, Pomalidomide, and Dexamethasone (DPd) in Relapsed/Refractory Light Chain Amyloidosis Patients Previously Exposed to Daratumumab | This study will test the hypothesis that in patients with previous daratumumab exposure, combination therapy of daratumumab, pomalidomide, and dexamethasone (DPd) will yield higher complete remission (CR) rates in relapsed/refractory amyloidosis than historical pomalidomide/dexamethasone treatment. | Phase 2 | Recruiting | Drug: Daratumumab|Drug: Pomalidomide|Drug: Dexamethasone | Click here for more information |
| Ixazomib In Combination With Cyclophosphamide And Dexamethasone for Newly Diagnosed AL Amyloidosis | The primary purpose of this study is to determine the safest dose of the medications and how well the combination is, or what is the "maximum tolerated dose" (MTD). | Phase 1|Phase 2 | Recruiting | Drug: Ixazomib|Drug: Cyclophosphamide|Drug: Dexamethasone | Click here for more information |
| A Study to Evaluate the Effectiveness and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis | The primary purpose of this study is to determine if CAEL-101 improves the overall survival in Patients with cardiac AL Amyloidosis. | Phase 3 | Recruiting | Drug: CAEL-101|Other: Placebo|Drug: cyclophosphamide, bortezomib, and dexamethasone (CyBorD) regimen | Click here for more information |
| A Study to Evaluate the Effectiveness and Safety of CAEL-101 in Patients With Mayo Stage IIIb AL Amyloidosis | The primary purpose of this study is to determine if CAEL-101 improves the overall survival in Patients with cardiac AL Amyloidosis. | Phase 3 | Recruiting | Drug: CAEL-101|Other: Placebo|Drug: cyclophosphamide, bortezomib, and Dexamethasone (CyBorD) regimen | Click here for more information |
| Ixazomib Maintenance Study in Patients With AL Amyloidosis | The purpose of this study is to learn if Ixazomib maintenance treatment (chemotherapy) works to control the disease. | Phase 2 | Recruiting | Drug: Ixazomib|Drug: Dexamethasone | Click here for more information |
| APG-2575 in Combination With Novel Therapeutic Regimens in Subjects With Relapsed or Refractory Multiple Myeloma | This is a Phase Ib/II, open-label, multi-center study evaluating the safety, tolerability, efficacy, and PK/ Pharmacodynamics of APG-2575 in combination with Pd/DRd in patients with relapsed/refractory (RR) multiple myeloma (MM) | Phase 1/2 | Recruiting | Drug: APG-2575+ Pd|Drug: APG-2575 + DRd | Click here for more information |
| A Study to Evaluate the Efficacy and Safety of Birtamimab in Mayo Stage IV Patients With AL Amyloidosis | A Phase 3 study to evaluate the efficacy and safety of birtamimab plus standard of care compared to placebo plus standard of care in patients with AL amyloidosis in Mayo Stage IV. | Phase 3 | Recruiting | Drug: Birtamimab|Other: Placebo|Drug: Standard of Care Chemotherapy | Click here for more information |
Top Treatments in Research
| Agent | Class/Mechanism of Action | Development Status | Company | Clinical Studies | More Information |
|---|---|---|---|---|---|
| propylene glycol-free melphalan hydrochloride | Propylene Glycol-Free melphalan HCL for Injection (PGF-Mel) is a new formulation that incorporates Captisol, a specially modified cyclodextrin, to improve melphalan stability. | Phase 2 | Spectrum Pharamceuticals | Click here for more information | Click here for more information |
| ZN-d5 | A potent, selective, and orally bioavailable BCL-2 inhibitor | Phase 1/2 | K-Group Alpha, Inc. | Click here for more information | Click here for more information |
| Moderna COVID-19 vaccine | mRNA vaccine | Phase 2 | Moderna | Click here for more information | Click here for more information |
| Daratumumab/Pomalidomide/Dexamethasone | Daratumumab is a human mab that binds to a unique cd38 epitope on cd38-expressing cells. Pomalidomide is an immunomodulatory and antineoplastic agent. | Phase 2 | Weill Medical College of Cornell University | Click here for more information | Click here for more information |
| Ixazomib/Cyclophosphamide/Dexamethasone | Ixazomib is a second-generation proteasome inhibitor | Phase 1/2 | Icahn School of Medicine at Mount Sinai Collaborator: Millennium Pharmaceuticals, Inc. | Click here for more information | Click here for more information |
| CAEL-101 | CAEL-101 is a monoclonal antibody that acts by binding to amyloid light chain fibrils and promoting their clearance from affected tissues | Phase 3 | Caelum Biosciences, Inc. | Click here for more information | Click here for more information |
| CAEL-101 | CAEL-101 is a monoclonal antibody that acts by binding to amyloid light chain fibrils and promoting their clearance from affected tissues | Phase 3 | Caelum Biosciences, Inc. | Click here for more information | Click here for more information |
| Ixazomib | Ixazomib is a second-generation proteasome inhibitor | Phase 2 | Memorial Sloan Kettering Cancer Center | Click here for more information | Click here for more information |
| APG-2575+ Pd | A novel, orally administered Bcl-2‒selective inhibitor | Phase 1/2 | Ascentage Pharma Group | Click here for more information | Click here for more information |
| Birtamimab | Birtamimab (NEOD001), is an investigational monoclonal antibody designed to specifically and selectively target and clear the amyloid that accumulates and causes organ dysfunction and failure in patients with AL amyloidosis | Phase 3 | Prothena Biosciences | Click here for more information | Click here for more information |