Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis (NCT01617967) | Clinical Trial Compass
CompletedPhase 2
Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis
United States, Brazil, France29 participantsStarted 2012-05
Plain-language 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).
Who can participate
Age range18 Years – 85 Years
SexALL
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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.
What they're measuring
1
Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation