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Completed

NCT Number: NCT01148953

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

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).

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Key information

Conditions

Transthyretin Mediated Amyloidosis (ATTR)

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Clinical Site, Le Kremlin-Bicêtre, France

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

ALN-TTR01

Drug

Dose levels between 0.01 and 1.0 mg/kg by intravenous (IV) infusion

Sterile Normal Saline (0.9% NaCl)

Drug

Calculated volume to match active comparator

Primary outcomes

  1. The proportion of patients experiencing adverse events (AEs), serious adverse events (SAEs), and study drug discontinuation.

    Time frame: Up to 28 Days

Secondary outcomes

  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

Sponsors and collaborators

Lead sponsor

Alnylam Pharmaceuticals

Industry

Registry information

Official study 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

Important dates

Study start
2010
Primary completion
2012
Study completion
2012
First posted
Jun 23, 2010
Registry last updated
May 24, 2012

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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