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A Study of the Natural History of Leukocyte Chemotactic Factor 2 Amyloidosis (ALECT2) Disease

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ClinicalTrials.gov Identifier: NCT03774784
Recruitment Status : Recruiting
First Posted : December 13, 2018
Last Update Posted : March 12, 2019
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Brief Summary:
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.

Condition or disease
Amyloidosis Leukocyte Chemotactic Factor 2 Amyloidosis

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Study Type : Observational
Estimated Enrollment : 150 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Study of the Natural History of Leukocyte Chemotactic Factor 2 Amyloidosis (ALECT2) Disease
Actual Study Start Date : January 29, 2019
Estimated Primary Completion Date : December 2023
Estimated Study Completion Date : December 2023

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Amyloidosis

Group/Cohort
ALECT2 Disease



Primary Outcome Measures :
  1. Change in Estimated Glomerular Filtration Rate (eGFR) from Baseline Up to End of Study (Month 48) [ Time Frame: Baseline (Day 1), Months 6, 12, 18, 24, 30, 36, 42 and 48 ]
  2. Time to End-Stage Renal Disease (ESRD) [ Time Frame: From baseline to end of study (Month 48) ]

Secondary Outcome Measures :
  1. Level of Leukocyte Chemotactic Factor 2 (LECT2) Messenger Ribonucleic Acid (mRNA) in Blood [ Time Frame: Baseline (Day 1), Months 6, 12, 18, 24, 30, 36, 42 and 48 ]
  2. Level of LECT2 mRNA in Urine [ Time Frame: Baseline (Day 1), Months 12, 24, 36 and 48 ]
  3. Level of LECT2 Protein in Blood [ Time Frame: Baseline (Day 1), Months 6, 12, 18, 24, 30, 36, 42 and 48 ]
  4. Level of LECT2 Protein in Urine [ Time Frame: Baseline (Day 1), Months 12, 24, 36 and 48 ]
  5. Percentage of Participants With Proteinuria [ Time Frame: Baseline (Day 1), Months 12, 24, 36 and 48 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
Participants with biopsy-proven ALECT2.
Criteria

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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03774784


Contacts
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Contact: Alnylam Clinical Trial Information Line 1-877-ALNYLAM clinicaltrials@alnylam.com
Contact: Alnylam Clinical Trial Information Line 1-877-256-9526

Locations
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United States, Colorado
Clinical Trial Site Recruiting
Denver, Colorado, United States, 80218
United States, Texas
Clinical Trial Site Recruiting
Houston, Texas, United States, 77004
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
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Study Director: Nader Najafian, MD Alnylam Pharmaceuticals

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Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03774784     History of Changes
Other Study ID Numbers: ALN-LECT2-NT-001
First Posted: December 13, 2018    Key Record Dates
Last Update Posted: March 12, 2019
Last Verified: March 2019

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases