Clinical trial NCT03774784: A Study of the Natural History of Leukocyte Chemotactic Factor 2 Amyloidosis (ALECT2) Disease
Official title: A Study of the Natural History of Leukocyte Chemotactic Factor 2 Amyloidosis (ALECT2) Disease
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Amyloidosis
Leukocyte Chemotactic Factor 2 Amyloidosis
Lead sponsor: Alnylam Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2018-12-13
Enrollment: 26
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Participants with biopsy-proven ALECT2.

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.

Detailed description: Refer to www.studyALECT2.com

Eligibility 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.

Primary outcomes:
measure: Change in Estimated Glomerular Filtration Rate (eGFR) from Baseline Up to End of Study (Month 48)
timeFrame: Baseline (Day 1), Months 6, 12, 18, 24, 30, 36, 42 and 48
measure: Time to End-Stage Renal Disease (ESRD)
timeFrame: From baseline to end of study (Month 48)
Secondary outcomes:
measure: Level of Leukocyte Chemotactic Factor 2 (LECT2) Messenger Ribonucleic Acid (mRNA) in Blood
timeFrame: Baseline (Day 1), Months 6, 12, 18, 24, 30, 36, 42 and 48
measure: Level of LECT2 mRNA in Urine
timeFrame: Baseline (Day 1), Months 12, 24, 36 and 48
measure: Level of LECT2 Protein in Blood
timeFrame: Baseline (Day 1), Months 6, 12, 18, 24, 30, 36, 42 and 48
measure: Level of LECT2 Protein in Urine
timeFrame: Baseline (Day 1), Months 12, 24, 36 and 48
measure: Percentage of Participants With Proteinuria
timeFrame: Baseline (Day 1), Months 12, 24, 36 and 48
Other outcomes:
Locations:
city: Scottsdale
country: United States
facility: Clinical Trial Site
state: Arizona
city: Tucson
country: United States
facility: Clinical Trial Site
state: Arizona
city: Denver
country: United States
facility: Clinical Trial Site
state: Colorado
city: Chicago
country: United States
facility: Clinical Trial Site
state: Illinois
city: Indianapolis
country: United States
facility: Clinical Trial Site
state: Indiana
city: Baltimore
country: United States
facility: Clinical Trial Site
state: Maryland
city: Boston
country: United States
facility: Clinical Trial Site
state: Massachusetts
city: Detroit
country: United States
facility: Clinical Trial Site
state: Michigan
city: Rochester
country: United States
facility: Clinical Trial Site
state: Minnesota
city: Albuquerque
country: United States
facility: Clinical Trial Site
state: New Mexico
city: Columbus
country: United States
facility: Clinical Trial Site
state: Ohio
city: Portland
country: United States
facility: Clinical Trial Site
state: Oregon
city: Houston
country: United States
facility: Clinical Trial Site
state: Texas
city: Houston
country: United States
facility: Clinical Trial Site
state: Texas
city: San Antonio
country: United States
facility: Clinical Trial Site
state: Texas
city: Saint George
country: United States
facility: Clinical Trial Site
state: Utah
city: Alexandria
country: Egypt
facility: Clinical Trial Site
city: Assiut
country: Egypt
facility: Clinical Trial Site
city: Cairo
country: Egypt
facility: Clinical Trial Site
city: Mansoura City
country: Egypt
facility: Clinical Trial Site
city: Chandigarh
country: India
facility: Clinical Trial Site
city: Ludhiāna
country: India
facility: Clinical Trial Site
city: Manipal
country: India
facility: Clinical Trial Site
city: Nadiād
country: India
facility: Clinical Trial Site
city: Tamil Nadu
country: India
facility: Clinical Trial Site
city: Kuala Lumpur
country: Malaysia
facility: Clinical Trial Site
city: Mexicali
country: Mexico
facility: Clinical Trial Site
state: Baja
city: Mexico
country: Mexico
facility: Clinical Trial Site
state: Mexico DF
city: Culiacán
country: Mexico
facility: Clinical Trial Site
state: Sinaloa
city: Monterrey
country: Mexico
facility: Clinical Trial Site
city: London
country: United Kingdom
facility: Clinical Trial Site
Study officials:
affiliation: Alnylam Pharmaceuticals
name: Joshua Friedman, MD
role: STUDY_DIRECTOR
References: