Now Available for Public Comment: Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

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
  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: Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: 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 ] [ Designated as safety issue: Yes ]
    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 ] [ 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


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

Ages Eligible for Study:   18 Years to 85 Years
Genders Eligible for Study:   Both
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
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: 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

No publications provided

Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01617967     History of Changes
Other Study ID Numbers: ALN-TTR02-002, 2012-000467-24
Study First Received: June 7, 2012
Last Updated: April 22, 2014
Health Authority: Portugal: National Pharmacy and Medicines Institute
Sweden: Medical Products Agency
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Spain: Agencia Española de Medicamentos y Productos Sanitarios
Germany: Federal Institute for Drugs and Medical Devices
United States: Food and Drug Administration
Brazil: National Health Surveillance Agency

Keywords provided by Alnylam Pharmaceuticals:
RNAi therapeutic

Additional relevant MeSH terms:
Amyloidosis
Metabolic Diseases
Proteostasis Deficiencies

ClinicalTrials.gov processed this record on November 24, 2014