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

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01617967
First Posted: June 13, 2012
Last Update Posted: July 17, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Alnylam Pharmaceuticals
  Purpose
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).

Condition Intervention Phase
TTR-mediated Amyloidosis Drug: ALN-TTR02 Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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

Resource links provided by NLM:


Further study details as provided by Alnylam Pharmaceuticals:

Primary Outcome Measures:
  • Safety and tolerability in ATTR patients [ Time Frame: Up to 56 Days ]
    The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation


Secondary Outcome Measures:
  • Pharmacokinetics (PK) of ALN-TTR02 [ Time Frame: Up to 208 days ]
    Cmax, Area Under Curve, Tmax

  • Serum transthyretin (TTR) protein [ Time Frame: Up to 208 days ]
    Determination of % Lowering to pretreatment/Baseline Levels


Enrollment: 29
Study Start Date: May 2012
Study Completion Date: January 2014
Primary Completion Date: October 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: ALN-TTR02 Drug: ALN-TTR02
Ascending doses of ALN-TTR02 administered by intravenous (IV) infusion

  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   18 Years to 85 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01617967


Locations
United States, Massachusetts
Clinical Trial Site
Boston, Massachusetts, United States
Brazil
Clinical Trial Site
Rio de Janeiro, Brazil
France
Clinical Trial Site
Le Kremlin-bicetre, France
Clinical Trial Site
Marseille Cedex, France
Germany
Clinical Trial Site
Munster, Germany
Portugal
Clinical Trial Site
Lisbon, Portugal
Clinical Trial Site
Porto, Portugal
Spain
Clinical Trial Site
Barcelona, Spain
Clinical Trial Site
Majorca, Spain
Sweden
Clinical Trial Site
Umeå, Sweden
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
Study Director: Jared Gollob, MD Alnylam Pharmaceuticals
  More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01617967     History of Changes
Other Study ID Numbers: ALN-TTR02-002
2012-000467-24 ( EudraCT Number )
First Submitted: June 7, 2012
First Posted: June 13, 2012
Last Update Posted: July 17, 2017
Last Verified: July 2017

Studies a U.S. FDA-regulated Drug Product: Yes

Keywords provided by Alnylam Pharmaceuticals:
RNAi therapeutic

Additional relevant MeSH terms:
Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases


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