EUCTR2012-000467-24-DEActive, not recruitingNot Applicable
A Phase 2, Open-Label, Multi-Dose, Dose Escalation Trial to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intravenous Infusions of ALN-TTR02 in Patients with TTR Amyloidosis
Conditions
Trial Snapshot
- Phase
- Not Applicable
- Status
- Active, not recruiting
- Enrollment
- 27
Study Overview
Brief Summary
No summary available.
Study Design
- Study Type
- Interventional clinical trial of medicinal product
Eligibility Criteria
- Sex
- All
Inclusion Criteria
- •1. Male or female aged 18 years or older.
- •2. Patients has a biopsy-proven diagnosis of TTR amyloidosis with documented signs/symptoms of the disease (e.g., sensory, motor, or autonomic neuropathy) that are at least mild to moderate in severity.
- •3. Body mass index (BMI) of 17–33 kg/m2.
- •4. Karnofsky performance status of 60% or greater.
- •5. Absolute neutrophil count (ANC) =1500 cells/mm³,
- •platelet count =100,000 cells/mm³, and hemoglobin =10 g/dL.
- •6. Adequate liver function, demonstrated by an aspartate transaminase (AST) and alanine transaminase (ALT) =2.5 x the upper limit of normal (ULN), total bilirubin within normal limits, albumin >3 g/dL, international normalized ratio (INR) =1.2.
- •7. Adequate renal function: serum creatinine =1.5 x ULN.
- •8. Seronegative for hepatitis B virus (HBV) and hepatitis C virus (HCV).
- •9. Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must be using two highly effective methods of contraception ( hormonal - oral, implantable, injectable, or transdermal contraceptives in conjunction with spermicide, condom, or diaphragm; mechanical - spermicide in conjunction with a barrier such as a condom or diaphragm; intrauterine device (IUD) in conjunction with spermicide or condom; or surgical sterilization of partner in conjunction with spermicide, condom, or diaphragm) prior to screening, throughout study participation, and for 1 month after ending study participation.
- •10. Males agree to use appropriate contraception throughout study participation and for 1 month after ending study participation.
- •11. Patient is willing and able to comply with protocol required visit schedule and visit requirements and provide written informed consent.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 24
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 3
Exclusion Criteria
- •1. Pregnant or nursing.
- •2. Has had a liver transplant.
- •3. Has a known surgery planned during any point of the study period.
- •4. Has known human immunodeficiency virus (HIV) positive status.
- •5. Has a known or suspected systemic bacterial, viral, parasitic, or fungal infection.
- •6. Received an investigational agent, other than tafamidis or diflunisal, within 30 days prior to study drug administration.
- •7. Has a New York Heart Association heart failure classification >2.
- •8. Has unstable angina.
- •9. Has uncontrolled clinically significant cardiac arrhythmia.
- •10. Is considered unfit for the study by the Principal Investigator.
- •11. Had a prior severe reaction to a liposomal product.
- •12. Has known hypersensitivity to oligonucleotides.
- •13. Is an employee or family member of Alnylam, the CRO, or the clinical study site personnel.
Investigators
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