Transthyretin-Mediated Amyloidosis Clinical Trial
Official title:
A Phase 1, Randomized, Double-Blind, Placebo Controlled, Single-Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK), and Pharmacodynamics (PD) of Subcutaneously Administered 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.
Status | Recruiting |
Enrollment | 180 |
Est. completion date | September 2025 |
Est. primary completion date | September 2025 |
Accepts healthy volunteers | Accepts Healthy Volunteers |
Gender | All |
Age group | 18 Years to 65 Years |
Eligibility | Inclusion Criteria: - Body mass index (BMI) =18.0 kg/m^2 and =30 kg/m^2 assessed at Screening. Exclusion Criteria: - Has an estimated glomerular filtration rate (eGFR) <90 mL/min/1.73m^2 at screening. - Has alanine aminotransferase (ALT), aspartate aminotransferase (AST) or direct bilirubin above the upper limit of normal (ULN). |
Country | Name | City | State |
---|---|---|---|
United Kingdom | Clinical Trial Site | London |
Lead Sponsor | Collaborator |
---|---|
Alnylam Pharmaceuticals |
United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Frequency of Adverse Events | Up to 20 months | ||
Secondary | Change from Baseline in Serum Transthyretin (TTR) Levels Over Time | Up to 18 months | ||
Secondary | Maximum Observed Plasma Concentration (Cmax) of ALN-TTRSC04 | Day 1 up to Day 4 | ||
Secondary | Time to Maximum Observed Plasma Concentration (Tmax) of ALN-TTRSC04 | Day 1 up to Day 4 | ||
Secondary | Area Under the Concentration-time Curve (AUC) of ALN-TTRSC04 | Day 1 up to Day 4 | ||
Secondary | Fraction of ALN-TTRSC04 Excreted in the Urine (fe) | Up to Day 2 |
Status | Clinical Trial | Phase | |
---|---|---|---|
Recruiting |
NCT04561518 -
ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)
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