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

Primary Purpose

Transthyretin Mediated Amyloidosis (ATTR)

Status
Completed
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
ALN-TTR01
Sterile Normal Saline (0.9% NaCl)
Sponsored by
Alnylam Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Transthyretin Mediated Amyloidosis (ATTR)

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Clinical Site
  • Clinical Site
  • Clinical Site
  • Clinical Site

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Placebo Comparator

Arm Label

ALN-TTR01

Sterile Normal Saline (0.9% NaCl)

Arm Description

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Pharmacokinetics (PK) of ALN-TTR01 (Cmax, tmax, t1/2, AUC0-last, CL)
Effect of ALN-TTR01 on Circulating TTR Levels (Determination of % Lowering of TTR to Pretreatment/Baseline TTR Level)

Full Information

First Posted
June 21, 2010
Last Updated
May 23, 2012
Sponsor
Alnylam Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT01148953
Brief Title
Trial to Evaluate Safety and Tolerability of ALN-TTR01 in Transthyretin (TTR) Amyloidosis
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 Type
Interventional

2. Study Status

Record Verification Date
May 2012
Overall Recruitment Status
Completed
Study Start Date
June 2010 (undefined)
Primary Completion Date
January 2012 (Actual)
Study Completion Date
February 2012 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Alnylam Pharmaceuticals

4. Oversight

Data Monitoring Committee
No

5. 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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Transthyretin Mediated Amyloidosis (ATTR)

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Parallel Assignment
Masking
Participant
Allocation
Randomized
Enrollment
32 (Actual)

8. Arms, Groups, and Interventions

Arm Title
ALN-TTR01
Arm Type
Active Comparator
Arm Title
Sterile Normal Saline (0.9% NaCl)
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
ALN-TTR01
Intervention Description
Dose levels between 0.01 and 1.0 mg/kg by intravenous (IV) infusion
Intervention Type
Drug
Intervention Name(s)
Sterile Normal Saline (0.9% NaCl)
Intervention Description
Calculated volume to match active comparator
Primary Outcome Measure Information:
Title
The proportion of patients experiencing adverse events (AEs), serious adverse events (SAEs), and study drug discontinuation.
Time Frame
Up to 28 Days
Secondary Outcome Measure Information:
Title
Pharmacokinetics (PK) of ALN-TTR01 (Cmax, tmax, t1/2, AUC0-last, CL)
Time Frame
Up to 70 days
Title
Effect of ALN-TTR01 on Circulating TTR Levels (Determination of % Lowering of TTR to Pretreatment/Baseline TTR Level)
Time Frame
up to 70 days

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jared Gollob, MD
Organizational Affiliation
Alnylam Pharmaceuticals Inc
Official's Role
Study Director
Facility Information:
Facility Name
Clinical Site
City
Le Kremlin Bicêtre
ZIP/Postal Code
94275
Country
France
Facility Name
Clinical Site
City
Porto
ZIP/Postal Code
4099-001
Country
Portugal
Facility Name
Clinical Site
City
Umeå
ZIP/Postal Code
SE 901 85
Country
Sweden
Facility Name
Clinical Site
City
London
ZIP/Postal Code
SE1 1YR
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
23984729
Citation
Coelho T, Adams D, Silva A, Lozeron P, Hawkins PN, Mant T, Perez J, Chiesa J, Warrington S, Tranter E, Munisamy M, Falzone R, Harrop J, Cehelsky J, Bettencourt BR, Geissler M, Butler JS, Sehgal A, Meyers RE, Chen Q, Borland T, Hutabarat RM, Clausen VA, Alvarez R, Fitzgerald K, Gamba-Vitalo C, Nochur SV, Vaishnaw AK, Sah DW, Gollob JA, Suhr OB. Safety and efficacy of RNAi therapy for transthyretin amyloidosis. N Engl J Med. 2013 Aug 29;369(9):819-29. doi: 10.1056/NEJMoa1208760.
Results Reference
derived

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

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