The Study of an Investigational Drug, ALN-TTR02, for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis

• Conditions: Transthyretin mediated amyloidosis (ATTR); MedDRA version: 15.1Level: PTClassification code 10007509Term: Cardiac amyloidosisSystem Organ Class: 10007541 - Cardiac disorders; MedDRA version: 15.1Level: PTClassification code 10019889Term: Hereditary neuropathic amyloidosisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2012-000467-24-SE
• Lead Sponsor: Alnylam Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2012-02-21
• Last Update Posted: 10 July 2015

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 27

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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified