Back to resultsA Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALN-TTRSC04 in Healthy Subjects
Primary Purpose
Transthyretin-Mediated Amyloidosis
Status
Enrolling by invitation
Phase
Phase 1
Locations
United Kingdom
Study Type
Interventional
Intervention
ALN-TTRSC04
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Transthyretin-Mediated Amyloidosis focused on measuring ATTR amyloidosis, siRNA
Eligibility Criteria
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).
Sites / Locations
- Clinical Trial Site
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
ALN-TTRSC04
Placebo
Arm Description
Participants will be administered a single dose of ALN-TTRSC04.
Participants will be administered a single dose of placebo.
Outcomes
Primary Outcome Measures
Frequency of Adverse Events
Secondary Outcome Measures
Change from Baseline in Serum Transthyretin (TTR) Levels Over Time
Maximum Observed Plasma Concentration (Cmax) of ALN-TTRSC04
Time to Maximum Observed Plasma Concentration (Tmax) of ALN-TTRSC04
Area Under the Concentration-time Curve (AUC) of ALN-TTRSC04
Fraction of ALN-TTRSC04 Excreted in the Urine (fe)
Full Information
NCT ID
NCT05661916
First Posted
December 15, 2022
Last Updated
June 16, 2023
Sponsor
Alnylam Pharmaceuticals
1. Study Identification
Unique Protocol Identification Number
NCT05661916
Brief Title
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALN-TTRSC04 in Healthy Subjects
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
Study Type
Interventional
2. Study Status
Record Verification Date
June 2023
Overall Recruitment Status
Enrolling by invitation
Study Start Date
January 16, 2023 (Actual)
Primary Completion Date
December 2023 (Anticipated)
Study Completion Date
June 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Alnylam Pharmaceuticals
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
Brief Summary
The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of single ascending doses of ALN-TTRSC04.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Transthyretin-Mediated Amyloidosis
Keywords
ATTR amyloidosis, siRNA
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
132 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
ALN-TTRSC04
Arm Type
Experimental
Arm Description
Participants will be administered a single dose of ALN-TTRSC04.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Participants will be administered a single dose of placebo.
Intervention Type
Drug
Intervention Name(s)
ALN-TTRSC04
Intervention Description
ALN-TTRSC04 will be administered by subcutaneous (SC) injection.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo will be administered by SC injection.
Primary Outcome Measure Information:
Title
Frequency of Adverse Events
Time Frame
Up to 12 months
Secondary Outcome Measure Information:
Title
Change from Baseline in Serum Transthyretin (TTR) Levels Over Time
Time Frame
Up to 12 months
Title
Maximum Observed Plasma Concentration (Cmax) of ALN-TTRSC04
Time Frame
Up to 12 months
Title
Time to Maximum Observed Plasma Concentration (Tmax) of ALN-TTRSC04
Time Frame
Up to 12 months
Title
Area Under the Concentration-time Curve (AUC) of ALN-TTRSC04
Time Frame
Up to 12 months
Title
Fraction of ALN-TTRSC04 Excreted in the Urine (fe)
Time Frame
Up to 12 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility Criteria
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).
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Alnylam Pharmaceuticals Inc
Official's Role
Study Director
Facility Information:
Facility Name
Clinical Trial Site
City
London
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALN-TTRSC04 in Healthy Subjects
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