Amyloidosis Clinical Trial
Official title:
A Study of the Natural History of Leukocyte Chemotactic Factor 2 Amyloidosis (ALECT2) Disease
| Verified date | June 2020 |
| Source | Alnylam Pharmaceuticals |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Observational |
The purpose of this study is to characterize the natural history of leukocyte chemotactic factor 2 amyloidotic (ALECT2) disease. In this observational study participants with ALECT2 disease will be enrolled. Participants, who have already reached end-stage renal disease (ESRD), will provide retrospective chart review data and biological specimens at baseline only. Other participants, in addition to retrospective chart review, will be followed prospectively.
| Status | Terminated |
| Enrollment | 26 |
| Est. completion date | May 18, 2020 |
| Est. primary completion date | May 18, 2020 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 18 Years and older |
| Eligibility |
Inclusion Criteria: - Males and females, age 18 years or older; - Renal biopsy-proven diagnosis of ALECT2; - For patients with kidney disease that could be due to ALECT2, a renal biopsy may be obtained to confirm ALECT2 diagnosis. Exclusion Criteria: - There are no exclusion criteria for this observational study. |
| Country | Name | City | State |
|---|---|---|---|
| Egypt | Clinical Trial Site | Alexandria | |
| Egypt | Clinical Trial Site | Assiut | |
| Egypt | Clinical Trial Site | Cairo | |
| Egypt | Clinical Trial Site | Mansoura City | |
| India | Clinical Trial Site | Chandigarh | |
| India | Clinical Trial Site | Ludhiana | |
| India | Clinical Trial Site | Manipal | |
| India | Clinical Trial Site | Nadiad | |
| India | Clinical Trial Site | Tamil Nadu | |
| Malaysia | Clinical Trial Site | Kuala Lumpur | |
| Mexico | Clinical Trial Site | Culiacán | Sinaloa |
| Mexico | Clinical Trial Site | Mexicali | Baja |
| Mexico | Clinical Trial Site | Mexico | Mexico DF |
| Mexico | Clinical Trial Site | Monterrey | |
| United Kingdom | Clinical Trial Site | London | |
| United States | Clinical Trial Site | Albuquerque | New Mexico |
| United States | Clinical Trial Site | Baltimore | Maryland |
| United States | Clinical Trial Site | Boston | Massachusetts |
| United States | Clinical Trial Site | Chicago | Illinois |
| United States | Clinical Trial Site | Columbus | Ohio |
| United States | Clinical Trial Site | Denver | Colorado |
| United States | Clinical Trial Site | Detroit | Michigan |
| United States | Clinical Trial Site | Houston | Texas |
| United States | Clinical Trial Site | Houston | Texas |
| United States | Clinical Trial Site | Indianapolis | Indiana |
| United States | Clinical Trial Site | Portland | Oregon |
| United States | Clinical Trial Site | Rochester | Minnesota |
| United States | Clinical Trial Site | Saint George | Utah |
| United States | Clinical Trial Site | San Antonio | Texas |
| United States | Clinical Trial Site | Scottsdale | Arizona |
| United States | Clinical Trial Site | Tucson | Arizona |
| Lead Sponsor | Collaborator |
|---|---|
| Alnylam Pharmaceuticals |
United States, Egypt, India, Malaysia, Mexico, United Kingdom,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Change in Estimated Glomerular Filtration Rate (eGFR) from Baseline Up to End of Study (Month 48) | Baseline (Day 1), Months 6, 12, 18, 24, 30, 36, 42 and 48 | ||
| Primary | Time to End-Stage Renal Disease (ESRD) | From baseline to end of study (Month 48) | ||
| Secondary | Level of Leukocyte Chemotactic Factor 2 (LECT2) Messenger Ribonucleic Acid (mRNA) in Blood | Baseline (Day 1), Months 6, 12, 18, 24, 30, 36, 42 and 48 | ||
| Secondary | Level of LECT2 mRNA in Urine | Baseline (Day 1), Months 12, 24, 36 and 48 | ||
| Secondary | Level of LECT2 Protein in Blood | Baseline (Day 1), Months 6, 12, 18, 24, 30, 36, 42 and 48 | ||
| Secondary | Level of LECT2 Protein in Urine | Baseline (Day 1), Months 12, 24, 36 and 48 | ||
| Secondary | Percentage of Participants With Proteinuria | Baseline (Day 1), Months 12, 24, 36 and 48 |
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Recruiting |
NCT04893889 -
Substudy (NCT04456582): Noninvasive Assessment of Myocardial Stiffness by 2D-SWE Ultrasound Technique (Two-dimensional Shear Wave Elastography) in Patients With Amyloidosis and Fabry Disease.
|
N/A | |
| Withdrawn |
NCT04943302 -
Isatuximab and Bendamustine in Systemic Light Chain Amyloidosis
|
Phase 2 | |
| Active, not recruiting |
NCT02909036 -
Study of Captisol Enabled Melphalan and Pharmacokinetics for Patients With Multiple Myeloma or Light Chain Amyloidosis That Are Receiving an Autologous Transplant.
|
Phase 1 | |
| Completed |
NCT02816476 -
Daratumumab Therapy for Patients With Refractory or Relapsed AL Amyloidosis
|
Phase 2 | |
| Completed |
NCT01083316 -
Bortezomib and Dexamethasone Followed by High-Dose Melphalan and Stem Cell Transplantation for Primary (AL) Amyloidosis
|
Phase 2 | |
| Completed |
NCT01527032 -
Risk-adapted Therapy for Primary Systemic (AL) Amyloidosis
|
Phase 2 | |
| Completed |
NCT02545907 -
A Dose Escalation Study of Carfilzomib Taken With Thalidomide and Dexamethasone in Relapsed AL Amyloidosis
|
Phase 1/Phase 2 | |
| Recruiting |
NCT05263817 -
A Clinical Study of CD19/BCMA CAR-T Cells in the Treatment of Refractory POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, and Vasculitis
|
Early Phase 1 | |
| Active, not recruiting |
NCT03201965 -
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
|
Phase 3 | |
| Withdrawn |
NCT02589860 -
Analysis of Oral Mucositis in Patient's Undergoing Melphalan Conditioning and Autologous Stem Cell Transplant
|
||
| Recruiting |
NCT05635266 -
Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives
|
||
| Completed |
NCT02574676 -
Quality of Life (QOL) Registry for Patients With AL Amyloidosis
|
||
| Active, not recruiting |
NCT02260466 -
Prevalence and Post-surgical Outcomes of CARdiac Wild-type TransthyrEtin amyloidoSIs in Elderly Patients With Aortic steNosis Referred for Valvular Replacement.
|
N/A | |
| Withdrawn |
NCT02462213 -
Prospective Identification of Cardiac Amyloidosis by Cardiac Magnetic Resonance Imaging
|
N/A | |
| Recruiting |
NCT05577819 -
Prevalence and Prediction of ATTR in Ambulatory Patients With HFpEF
|
N/A | |
| Completed |
NCT01406314 -
SAP Depleter Dose Assessment Study in Patients
|
Phase 1 | |
| Not yet recruiting |
NCT04985734 -
Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM) in Patients With Idiopathic Peripheral Neuropathy
|
N/A | |
| Active, not recruiting |
NCT03584022 -
Clinical Trial to Assess the Safety of a Novel Scaffold Biomaterial
|
N/A | |
| Active, not recruiting |
NCT05199337 -
Phase 1/2 Study of ZN-d5 for the Treatment of Relapsed or Refractory Light Chain (AL) Amyloidosis
|
Phase 1/Phase 2 | |
| Active, not recruiting |
NCT05235269 -
A Study to Evaluate Organ Level Uptake Repeatability of 124I AT-01 in Subjects With Systemic Amyloidosis
|
Phase 2 |