Evaluating a new treatment for transthyretin-mediated amyloidosis in healthy individuals
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
This study is testing a new drug called ALN-TTRSC04 in healthy people to see if it's safe and how it works in the body.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 180 (estimated) |
| Ages | 18 Years to 65 Years |
| Sex | All |
| Sponsor | Alnylam Pharmaceuticals Industry-sponsored |
| Locations | 1 site (London) |
| Trial ID | NCT05661916 on ClinicalTrials.gov |
What this trial studies
This study aims to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of a new drug called ALN-TTRSC04 in healthy subjects. Participants will receive single ascending doses of the drug or a placebo to determine how well it is tolerated and how it behaves in the body. The study will monitor various health parameters to ensure participant safety and gather data on the drug's effects. This is a Phase 1 interventional trial, focusing on initial evaluations of the treatment.
Who should consider this trial
Good fit: Ideal candidates for this study are healthy adults with a body mass index (BMI) between 18.0 and 30.0 kg/m².
Not a fit: Patients with significant kidney impairment or elevated liver enzymes may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option for patients suffering from transthyretin-mediated amyloidosis.
How similar studies have performed: Other studies involving similar siRNA approaches have shown promise, indicating potential for success in this area.
Eligibility criteria
Show full inclusion / exclusion 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).
Where this trial is running
London
- Clinical Trial Site — London, United Kingdom (Recruiting)
Study contacts
- Study coordinator: Alnylam Clinical Trial Information Line
- Email: clinicaltrials@alnylam.com
- Phone: 1-877-ALNYLAM
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.