Clinical trials: Amyloidosis
Light Chain (AL) 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
The purpose of this study is to evaluate the efficacy and safety of NXC-201 compared with Dara-CyBorD (daratumumab-cyclophosphamide-bortezomib-dexamethasone) in the treatment of newly diagnosed amyloid light chain (AL)…
- Trial name
- NEXICART-3 (NCT07709715)
- Testing
- NXC-201, Dara-CyBorD
- Volunteers
- 260 people
- Sponsor
- Nexcella Inc.
Light Chain (AL) Amyloidosis
Study of NXC-201 CAR-T in Patients With Light Chain (AL) Amyloidosis
Open-label Phase 1b Dose Escalation/Dose Expansion study exploring the safety and efficacy of NXC-201 in patients with relapsed or refractory light chain amyloidosis (AL).
- Trial name
- NEXICART-2 (NCT06097832)
- Testing
- NXC-201 CAR-T
- Where
- 18 sites in United States
- Volunteers
- 45 people
- Sponsor
- Nexcella Inc.
AL Amyloidosis
A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIb AL Amyloidosis (CARES)
AL (or light chain) amyloidosis begins in the bone marrow where abnormal proteins misfold and create free light chains that cannot be broken down.
- Trial name
- NCT04504825
- Testing
- CAEL-101, cyclophosphamide, bortezomib, and Dexamethasone (CyBorD) regimen
- Where
- 90 sites in United States, Australia and 17 more
- Volunteers
- 125 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Relapsed AL Amyloidosis, Refractory AL Amyloidosis
A Phase 1b/2 Study of CAR T Cell Therapy Targeting CD19 and BCMA in Participants With Relapsed or Refractory AL Amyloidosis.
Open-label Phase 1b/2 study with primary objective of this study is to evaluate the safety, tolerability and efficacy of AZD0120 in participants with light chain (AL) amyloidosis.
- Conditions
- Relapsed AL Amyloidosis, Refractory AL Amyloidosis, Light Chain Amyloidosis, Amyloidosis
- Trial name
- ALACRITY (NCT07081646)
- Testing
- AZD0120
- Where
- 18 sites in United States, Canada and 1 more
- Volunteers
- 91 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
AL Amyloidosis
A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis (CARES)
AL (or light chain) amyloidosis begins in the bone marrow where abnormal proteins misfold and create free light chains that cannot be broken down.
- Trial name
- NCT04512235
- Testing
- CAEL-101, cyclophosphamide, bortezomib, and dexamethasone (CyBorD) regimen
- Where
- 110 sites in United States, Australia and 17 more
- Volunteers
- 281 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Cardiac Amyloidosis
PATHWAY-RCT: Preventing Admission To Hospital With Attr cardiomyopathY
Cardiac amyloidosis is a condition where the heart muscle, amongst other tissues, is infiltrated by the abnormal build-up of proteins called amyloid.
- Trial name
- PATHWAY-RCT (NCT05098665)
- Testing
- Telemonitoring service
- Where
- United Kingdom
- Volunteers
- 320 people
- Sponsor
- Richmond Research Institute
Hereditary Transthyretin Amyloidosis, Carpal Tunnel Syndrome
Hereditary Transthyretin Amyloidosis Polyneuropathy in Patients With Carpal Tunnel Syndrome in Russia
This is a multicenter observational study consisting of retrospective and prospective phases. The retrospective phase will entail secondary data collection from electronic or paper medical records of patients who underwent…
- Conditions
- Hereditary Transthyretin Amyloidosis, Carpal Tunnel Syndrome, Polyneuropathy, Amyloidosis
- Trial name
- LOCUS (NCT06414746)
- Where
- 13 sites in Russia
- Volunteers
- 721 people
- Sponsor
- AstraZeneca
Relapsed/Refractory Multiple Myeloma, Amyloid Light-chain Amyloidosis
A Study to Investigate Safety and Efficacy With SAR445514 in Participants With Relapsed/Refractory Multiple Myeloma (RRMM) and Relapsed/Refractory Light-chain Amyloidosis (RRLCA)
This is a first-in-human (FIH) Phase 1/Phase 2 study for evaluating SAR445514 in monotherapy in participants with relapsed/refractory multiple myeloma (RRMM) and relapsed/refractory light chain amyloidosis (RRLCA).
- Trial name
- NCT05839626
- Testing
- SAR445514
- Where
- 16 sites in Australia, Belgium and 5 more
- Volunteers
- 32 people
- Sponsor
- Sanofi
Immunoglobulin Light Chain (AL) Amyloidosis
A Study to Assess Change in Disease Activity and Adverse Events (AE)s in Adult Participants With Immunoglobulin Light Chain (AL) Amyloidosis Receiving Etentamig (ABBV-383) as an Intravenous (IV) Infusion
Immunoglobulin light chain (AL) amyloidosis is the most common form of systemic amyloidosis. AL amyloidosis has many root causes and is characterized by the overproduction of AL that are secreted by clonal bone marrow plasma…
- Trial name
- NCT06158854
- Testing
- ABBV-383 (Etentamig)
- Where
- 23 sites in United States, Australia and 4 more
- Volunteers
- 76 people
- Sponsor
- AbbVie
Systemic Light Chain Amyloidosis
Assessment of the Efficacy and Safety of Injectable TQB2934 (Subcutaneous Injection) in Systemic Light Chain Amyloidosis Patients
This study is a clinical trial aimed at the marketing of TQB2934 for injection. The project plans to enroll 70 subjects, including 13-21 subjects in Phase Ib, to evaluate the safety and preliminary efficacy, pharmacokinetic (PK)…
- Trial name
- NCT07266116
- Testing
- TQB2934 injection
- Where
- 27 sites in China
- Volunteers
- 70 people
- Sponsor
- Shanghai Chia Tai Tianqing Pharmaceutical Technology Development Co., Ltd.
Amyloidosis; Systemic
A Study of AT-02 in Subjects With Systemic Amyloidosis.
This is a Phase 2 open-label extension study to evaluate the long-term safety, tolerability, and clinical activity of AT-02. AT-02 is an investigational medicinal product being developed to treat systemic amyloidosis.
- Trial name
- NCT05951049
- Testing
- AT02
- Where
- 4 sites in United States
- Volunteers
- 120 people
- Sponsor
- Attralus, Inc.
Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)
Real-World Effectiveness of High-Dose Tafamidis on Neurologic Disease Progression in Mixed-Phenotype Transthyretin Amyloid Cardiomyopathy
This study will examine the clinical effectiveness of Tafamidis in patients with Mixed Phenotype Transthyretin Amyloidosis using data that already exist in patients' medical records
- Trial name
- NCT06393465
- Testing
- Tafamidis
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Pfizer
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)
The purpose of this study is to evaluate the efficacy and safety of patisiran in participants with ATTR amyloidosis with cardiomyopathy.
- Trial name
- NCT03997383
- Testing
- Patisiran
- Where
- 90 sites in United States, Argentina and 20 more
- Volunteers
- 360 people
- Sponsor
- Alnylam Pharmaceuticals
Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
The purpose of this study is to evaluate the safety and tolerability of extended dosing with eplontersen in participants with ATTR-CM.
- Trial name
- NCT05667493
- Testing
- Eplontersen
- Where
- 134 sites in United States, Argentina and 18 more
- Volunteers
- 1,400 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Transthyretin-Mediated Amyloidosis
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALN-TTRSC04 in Healthy Subjects
The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of single ascending doses of ALN-TTRSC04.
- Trial name
- NCT05661916
- Testing
- ALN-TTRSC04
- Where
- United Kingdom
- Volunteers
- 96 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
Amyloidosis
A Study to Evaluate the Safety and Efficacy of SCTC21C in Combination With Bortezomib, Cyclophosphamide, and Dexamethasone in Patients With Newly Diagnosed Systemic Light-Chain Amyloidosis (NDSLCA)
The purpose of this study is to evaluate the efficacy and safety of SCTC21C plus cyclophosphamide, bortezomib and dexamethasone (VCd) compared with VCd alone in treatment of newly diagnosed amyloid light chain (AL) amyloidosis…
- Trial name
- NCT07388602
- Testing
- SCTC21C, Bortezomib, Dexamethasone
- Where
- China
- Volunteers
- 90 people
- Sponsor
- Sinocelltech Ltd.
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy
Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)
This study will be conducted to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NTLA-2001 in participants with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) and…
- Conditions
- Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Wild-Type Transthyretin Cardiac Amyloidosis
- Trial name
- NCT04601051
- Testing
- NTLA-2001
- Where
- 4 sites in France, New Zealand and 2 more
- Volunteers
- 72 people
- Sponsor
- Intellia Therapeutics
Amyloidosis, Hereditary, Transthyretin Amyloidosis
HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
The purpose of this study is to evaluate the efficacy and safety of vutrisiran (ALN-TTRSC02) in participants with hereditary transthyretin amyloidosis (hATTR amyloidosis).
- Trial name
- NCT03759379
- Testing
- Patisiran, Vutrisiran
- Where
- 64 sites in United States, Argentina and 20 more
- Volunteers
- 164 people
- Sponsor
- Alnylam Pharmaceuticals
Transthyretin-type Cardiac Amyloidosis
Real-Life Clinical Efficacy of Acoramidis in Participants With ATTR-CM and Association With Cardiac Biomarkers
The purpose of this study is to confirm that the treatment with acoramidis prevents the deterioration of the ATTR-CM disease progression index and that these indexes are surrogate markers of disease progression.
- Trial name
- ATTR-CM (NCT07306949)
- Testing
- Acoramidis
- Where
- 16 sites in Japan
- Volunteers
- 200 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Light Chain (AL) Amyloidosis
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 Mayo Stage IV patients with AL amyloidosis.
- Trial name
- AFFIRM-AL (NCT04973137)
- Testing
- Birtamimab, Standard of Care Chemotherapy
- Where
- 110 sites in United States, Australia and 21 more
- Volunteers
- 208 people
- Sponsor
- Prothena Biosciences Ltd.