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Trial to Evaluate Safety and Tolerability of ALN-TTR01 in Transthyretin (TTR) Amyloidosis

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01148953
First received: June 21, 2010
Last updated: May 23, 2012
Last verified: May 2012
  Purpose
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of ALN-TTR01 in patients with transthyretin (TTR) mediated amyloidosis (ATTR).

Condition Intervention Phase
Transthyretin Mediated Amyloidosis (ATTR) Drug: ALN-TTR01 Drug: Sterile Normal Saline (0.9% NaCl) Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single Blind (Participant)
Primary Purpose: Treatment
Official Title: A Phase 1, Randomized, Single-Blind, Placebo-Controlled, Dose Escalation Trial to Evaluate the Safety and Tolerability of a Single Dose of Intravenous ALN-TTR01 in Patients With TTR Amyloidosis

Resource links provided by NLM:


Further study details as provided by Alnylam Pharmaceuticals:

Primary Outcome Measures:
  • The proportion of patients experiencing adverse events (AEs), serious adverse events (SAEs), and study drug discontinuation. [ Time Frame: Up to 28 Days ]

Secondary Outcome Measures:
  • Pharmacokinetics (PK) of ALN-TTR01 (Cmax, tmax, t1/2, AUC0-last, CL) [ Time Frame: Up to 70 days ]
  • Effect of ALN-TTR01 on Circulating TTR Levels (Determination of % Lowering of TTR to Pretreatment/Baseline TTR Level) [ Time Frame: up to 70 days ]

Enrollment: 32
Study Start Date: June 2010
Study Completion Date: February 2012
Primary Completion Date: January 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: ALN-TTR01 Drug: ALN-TTR01
Dose levels between 0.01 and 1.0 mg/kg by intravenous (IV) infusion
Placebo Comparator: Sterile Normal Saline (0.9% NaCl) Drug: Sterile Normal Saline (0.9% NaCl)
Calculated volume to match active comparator

  Eligibility

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

Inclusion Criteria:

  • Diagnosis of TTR amyloidosis
  • Adequate blood counts, liver and renal function
  • Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must use an adequate method of birth control
  • Males agree to use appropriate contraception
  • Willing and able to comply with protocol-required visit schedule and visit requirements and provide written informed consent.

Exclusion Criteria:

  • Known human immunodeficiency virus (HIV) positive status
  • Receiving antibiotics for bacterial infection within 7 days of screening
  • Known or suspected systemic viral, parasitic or fungal infection
  • Receiving an investigational agent within 30 days prior to study drug administration
  • Poor cardiac function
  • Considered unfit for the study by the Principal Investigator
  • Known sensitivity to oligonucleotides
  • 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: NCT01148953

Locations
France
Clinical Site
Le Kremlin Bicêtre, France, 94275
Portugal
Clinical Site
Porto, Portugal, 4099-001
Sweden
Clinical Site
Umeå, Sweden, SE 901 85
United Kingdom
Clinical Site
London, United Kingdom, SE1 1YR
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
Study Director: Jared Gollob, MD Alnylam Pharmaceuticals, Inc.
  More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01148953     History of Changes
Other Study ID Numbers: ALN-TTR01-001
2009-017383-16 ( EudraCT Number )
Study First Received: June 21, 2010
Last Updated: May 23, 2012

Additional relevant MeSH terms:
Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases

ClinicalTrials.gov processed this record on July 21, 2017