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Trial to Evaluate Safety and Tolerability of ALN-TTR02 in Transthyretin (TTR) Amyloidosis

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01617967
First received: June 7, 2012
Last updated: April 22, 2014
Last verified: April 2014

June 7, 2012
April 22, 2014
May 2012
October 2013   (final data collection date for primary outcome measure)
Safety and tolerability in ATTR patients [ Time Frame: Up to 56 Days ] [ Designated as safety issue: Yes ]
The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation
Same as current
Complete list of historical versions of study NCT01617967 on ClinicalTrials.gov Archive Site
  • Pharmacokinetics (PK) of ALN-TTR02 [ Time Frame: Up to 208 days ] [ Designated as safety issue: Yes ]
    Cmax, Area Under Curve, Tmax
  • Serum transthyretin (TTR) protein [ Time Frame: Up to 208 days ] [ Designated as safety issue: No ]
    Determination of % Lowering to pretreatment/Baseline Levels
Same as current
Not Provided
Not Provided
 
Trial to Evaluate Safety and Tolerability of ALN-TTR02 in Transthyretin (TTR) Amyloidosis
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

The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple doses of ALN-TTR02 in patients with transthyretin (TTR) mediated amyloidosis (ATTR).

Not Provided
Interventional
Phase 2
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
TTR-mediated Amyloidosis
Drug: ALN-TTR02
Ascending doses of ALN-TTR02 administered by intravenous (IV) infusion
Experimental: ALN-TTR02
Intervention: Drug: ALN-TTR02
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
29
January 2014
October 2013   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • Body mass index must be between 17 kg/m2 and ≤ 33 kg/m2;
  • 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.
Both
18 Years to 85 Years
No
Contact information is only displayed when the study is recruiting subjects
United States,   Brazil,   France,   Germany,   Portugal,   Spain,   Sweden
 
NCT01617967
ALN-TTR02-002, 2012-000467-24
No
Alnylam Pharmaceuticals
Alnylam Pharmaceuticals
Not Provided
Study Director: Jared Gollob, MD Alnylam Pharmaceuticals
Alnylam Pharmaceuticals
April 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP