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

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ClinicalTrials.gov Identifier: NCT01148953
Recruitment Status : Completed
First Posted : June 23, 2010
Last Update Posted : May 24, 2012
Sponsor:
Information provided by (Responsible Party):

Study Description
Brief Summary:
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 or disease Intervention/treatment Phase
Transthyretin Mediated Amyloidosis (ATTR) Drug: ALN-TTR01 Drug: Sterile Normal Saline (0.9% NaCl) Phase 1

Study Design

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 32 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (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
Study Start Date : June 2010
Primary Completion Date : January 2012
Study Completion Date : February 2012

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Amyloidosis
U.S. FDA Resources

Arms and Interventions

Arm Intervention/treatment
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


Outcome Measures

Primary Outcome Measures :
  1. 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 :
  1. Pharmacokinetics (PK) of ALN-TTR01 (Cmax, tmax, t1/2, AUC0-last, CL) [ Time Frame: Up to 70 days ]
  2. Effect of ALN-TTR01 on Circulating TTR Levels (Determination of % Lowering of TTR to Pretreatment/Baseline TTR Level) [ Time Frame: up to 70 days ]

Eligibility Criteria

Information from the National Library of Medicine

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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

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): 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 )
First Posted: June 23, 2010    Key Record Dates
Last Update Posted: May 24, 2012
Last Verified: May 2012

Additional relevant MeSH terms:
Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases