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 |
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