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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05661916
Recruitment Status : Recruiting
First Posted : December 22, 2022
Last Update Posted : May 10, 2024
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Study Description
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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.

Condition or disease Intervention/treatment Phase
Transthyretin-Mediated Amyloidosis Drug: ALN-TTRSC04 Drug: Placebo Phase 1

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 180 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
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
Actual Study Start Date : January 16, 2023
Estimated Primary Completion Date : September 2025
Estimated Study Completion Date : September 2025

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Amyloidosis

Arms and Interventions
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Arm Intervention/treatment
Experimental: ALN-TTRSC04
Participants will be administered a single dose of ALN-TTRSC04.
 Drug: ALN-TTRSC04
ALN-TTRSC04 will be administered by subcutaneous (SC) injection.
Placebo Comparator: Placebo
Participants will be administered a single dose of placebo.
 Drug: Placebo
Placebo will be administered by SC injection.


Outcome Measures
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Primary Outcome Measures :
  1. Frequency of Adverse Events [ Time Frame: Up to 20 months ]

Secondary Outcome Measures :
  1. Change from Baseline in Serum Transthyretin (TTR) Levels Over Time [ Time Frame: Up to 18 months ]
  2. Maximum Observed Plasma Concentration (Cmax) of ALN-TTRSC04 [ Time Frame: Day 1 up to Day 4 ]
  3. Time to Maximum Observed Plasma Concentration (Tmax) of ALN-TTRSC04 [ Time Frame: Day 1 up to Day 4 ]
  4. Area Under the Concentration-time Curve (AUC) of ALN-TTRSC04 [ Time Frame: Day 1 up to Day 4 ]
  5. Fraction of ALN-TTRSC04 Excreted in the Urine (fe) [ Time Frame: Up to Day 2 ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
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).
Contacts and Locations
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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): NCT05661916


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 clinicaltrials@alnylam.com

Locations
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United Kingdom
Clinical Trial Site Recruiting
London, United Kingdom
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
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Study Director: Medical Director Alnylam Pharmaceuticals Inc
More Information
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Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT05661916    
Other Study ID Numbers: ALN-TTRSC04-001
First Posted: December 22, 2022    Key Record Dates
Last Update Posted: May 10, 2024
Last Verified: May 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Alnylam Pharmaceuticals:
ATTR amyloidosis
siRNA
Additional relevant MeSH terms:
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Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases