A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALN-TTRSC04 in Healthy Subjects

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05661916
• Other study ID #: ALN-TTRSC04-001
• Status: Recruiting
• Phase: Phase 1
• Start date: January 16, 2023
• Est. completion date: September 2025

Study information

• Verified date: May 2024
• Source: Alnylam Pharmaceuticals
• Contact: Alnylam Clinical Trial Information Line
• Phone: 1-877-ALNYLAM
• Email: clinicaltrials[@]alnylam.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of single ascending doses of ALN-TTRSC04.

Recruitment information / eligibility

• 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).

Related Conditions & MeSH terms

• Amyloidosis
• Transthyretin-Mediated Amyloidosis

Intervention

Drug:
• ALN-TTRSC04
ALN-TTRSC04 will be administered by subcutaneous (SC) injection.
• Placebo
Placebo will be administered by SC injection.

Locations

Country	Name	City	State
United Kingdom	Clinical Trial Site	London

Sponsors (1)

Lead Sponsor	Collaborator
Alnylam Pharmaceuticals

Country where clinical trial is conducted

United Kingdom, 

Outcome

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