Phase 2
N=29
Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis
TTR-mediated Amyloidosis
Bottom Line
View on ClinicalTrials.gov: NCT01617967 ↗Enrolled (actual)
29
Serious AEs
6.9%
Results posted
Nov 2018
Primary outcome: Primary: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation — 1; 3; 2; 7 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Patisiran (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Alnylam Pharmaceuticals
- Primary completion
- Oct 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation |
1; 3; 2; 7; 3; 7 | — |
| SECONDARY Percentage Change From Baseline in Serum Transthyretin (TTR) Protein |
-8.9; -21.7; -51.6; -73.9; -78.0; -80.7 | — |
| SECONDARY Pharmacokinetic Parameters of Patisiran - Area Under the Concentration Curve From Time 0 to Last Measurable Time Point (AUC0-last) |
2738; 9604; 18998; 53724; 39741; 2799 | — |
| SECONDARY Pharmacokinetic Parameters of Patisiran - Maximum Observed Plasma Concentration (Cmax) |
145; 672; 2560; 6053; 4539; 106 | — |
| SECONDARY Pharmacokinetic Parameters of Patisiran - Beta Elimination Half-life (t1/2 Beta) |
59.5; 39.6; 52.2; 48; 59.3; 88.1 | — |
| SECONDARY Pharmacokinetic Parameters of Patisiran - Systemic Clearance (CL) |
0.0163; 0.123; 0.00883; 0.00851; 0.0130; 0.0211 | — |
| SECONDARY Pharmacokinetic Parameters of Patisiran - Apparent Volume of Distribution at Steady State (Vss) |
0.169; 0.279; 0.428; 0.360; 0.588; 0.264 | — |
| SECONDARY Pharmacokinetic Parameters of Patisiran - Renal Clearance (CLR) |
0.0153; 0.0268; 0.0129; 0.0329; 0.0334; 0.0320 | — |
Summary
This was a multiple dose, dose escalation study designed to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of patisiran (ALN-TTR02) in participants with transthyretin (TTR) mediated amyloidosis (ATTR).
Eligibility Criteria
Inclusion Criteria
- Body mass index must be between 17 kg/m^2 and ≤ 33 kg/m^2;
- Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must use appropriate contraception;
- Males agree to use appropriate contraception;
- Diagnosis of TTR amyloidosis;
- Adequate blood counts, liver and renal function;
- Willing to give written informed consent and are willing to comply with the study requirements.
Exclusion Criteria
- Known human immunodeficiency virus (HIV) positive status or known or suspected systemic bacterial, viral, parasitic, or fungal infection;
- Received an investigational agent, other than tafamidis or diflunisal, within 30 days prior to first dose study drug administration;
- Prior liver transplant;
- Poor cardiac function;
- Considered unfit for the study by the Principal Investigator;
- Employee or family member of the sponsor or the clinical study site personnel.
Data sourced from ClinicalTrials.gov (NCT01617967). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.