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Home › Conditions › Amyloidosis

Amyloidosis

0 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for Amyloidosis

15 trials tracked for Amyloidosis: 5 in phase 3 or 4 and 5 with published results. The most-cited published study has 1451 citations.

15Trials tracked
5Phase 3 & 4
0Recruiting
5With published results
Phase distribution
Phase 3 5 Phase 2 7 Other / NA 3
  1. Phase 3 Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy Completed · 1,451 cited
  2. Phase 3 A Study to Evaluate the Efficacy and Safety of Daratumumab in Combination With Cyclophosphamide, Bortezomib and Dexamethasone (CyBorD) Compared to CyBorD Alone in Newly Diagnosed Systemic Amyloid Light-chain (AL) Amyloidosis Completed · 702 cited
  3. Phase 3 The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Participants Who Have Already Been Treated With ALN-TTR02 (Patisiran) Completed · 159 cited
  4. Phase 3 Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP) Completed · 72 cited
  5. Phase 3 Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy Completed · 11 cited
  6. Phase 2 Ixazomib With Cyclophosphamide and Dexamethasone in Patients With Previously Untreated Symptomatic Multiple Myeloma or Light Chain Amyloidosis Completed
Show 9 more trials
  1. Phase 2 A Dose Escalation Study of Carfilzomib Taken With Thalidomide and Dexamethasone in Relapsed AL Amyloidosis Completed
  2. Phase 2 Ixazomib Rollover Study Completed
  3. Phase 2 A Study to Evaluate Organ Level Uptake Repeatability of 124I AT-01 in Subjects With Systemic Amyloidosis Completed
  4. Phase 2 Bortezomib and Dexamethasone Followed by High-Dose Melphalan and Stem Cell Transplantation for Primary (AL) Amyloidosis Completed
  5. Phase 2 Open-Label Phase 1/2 Study of VELCADE for Injection in Patients With Light-chain (AL)-Amyloidosis Completed
  6. Phase 2 Safety and Effect of Doxycycline in Patients With Amyloidosis Completed
  7. N/A Combined Bone Marrow and Renal Transplantation for Hematologic Disorders With End Stage Renal Disease Completed
  8. N/A Bortezomib/Dexamethasone (BD), Followed By Autologous Stem Cell Transplantation and Maintenance Bortezomib/Dexamethasone For the Initial Treatment of Monoclonal Immunoglobulin Deposition Disease (MIDD) Associated With Multiple Myeloma and AL Amyloidosis Completed
  9. N/A A Pilot Study of Lenalidomide, Melphalan and Dexamethasone in AL Amyloidosis Completed

Showing the 15 most-cited and recently-updated of 15 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found For clinicians

Amyloidosis: what the trials found

Several pharmacological interventions have demonstrated efficacy in treating various manifestations of amyloidosis. Inotersen showed statistically significant improvements in the Modified Neuropathy Impairment Score (mNIS) +7 at week 66 1 and improvements in the Norfolk Quality of Life Diabetic Neuropathy (QoL-DN) questionnaire 1. However, Inotersen was also associated with high rates of treatment-emergent adverse events (TEAEs) and serious TEAEs 4.

Cyclophosphamide demonstrated a significant overall complete hematologic response (CHR) 2 and showed measurable organ response rates for both cardiac (22.2% to 41.5%) and renal involvement (27.4% to 53.8%) at 6 months 2. In a separate Phase 2 study, Cyclophosphamide was associated with a 35% complete or very good partial response rate 8.

Targeted therapies like Patisiran showed significant changes in the Total Neuropathy Impairment Score (NIS) at year 5 3 and the mNIS +7 score at year 3 3. Acoramidis demonstrated a significant hierarchical combination of all-cause mortality, CV-related hospitalization, NT-proBNP changes, and 6MWT improvements 5. Specifically, patients treated with acoramidis showed significant improvements in 6MWT distance and KCCQ-OS scores at month 30 5.

Recent results — preliminary, needs further review

  • * 124I-AT-01 showed changes in clinical laboratory chemistry values (not yet corroborated) 6. * Ixazomib was evaluated for safety, with a low number of patients experiencing Grade 3 adverse events or Grade 2 peripheral neuropathy 7. * Carfilzomib was evaluated for clonal response and amyloidotic organ response rates 9. * Doxycycline showed a significant reduction in BNP in patients with amyloid cardiomyopathy 10. * Bortezomib was evaluated in transplant contexts 11. * Bortezomib/Dexamethasone was evaluated for cardiac and renal organ response 14.

For the clinician treating this condition

  • Inotersen is associated with significant improvements in neuropathy scores and quality of life, but is linked to high rates of serious treatment-emergent adverse events.
  • Cyclophosphamide shows significant hematologic response and measurable organ response rates in cardiac and renal involvement.
  • Acoramidis demonstrated significant improvements in functional outcomes (6MWT) and quality of life (KCCQ-OS) in patients with amyloid cardiomyopathy.

AI synthesis of 12 cited trials, updated Sep 12, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
No published articles yet for this condition. Check back soon.