NCT01148953

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).

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
32

participants targeted

Target at P50-P75 for phase_1

Timeline
Completed

Started Jun 2010

Geographic Reach
4 countries

4 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 1, 2010

Completed
20 days until next milestone

First Submitted

Initial submission to the registry

June 21, 2010

Completed
2 days until next milestone

First Posted

Study publicly available on registry

June 23, 2010

Completed
1.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2012

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2012

Completed
Last Updated

May 24, 2012

Status Verified

May 1, 2012

Enrollment Period

1.6 years

First QC Date

June 21, 2010

Last Update Submit

May 23, 2012

Conditions

Transthyretin Mediated Amyloidosis (ATTR)

Outcome Measures

Primary Outcomes (1)

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

    Up to 28 Days

Secondary Outcomes (2)

  • Pharmacokinetics (PK) of ALN-TTR01 (Cmax, tmax, t1/2, AUC0-last, CL)

    Up to 70 days

  • Effect of ALN-TTR01 on Circulating TTR Levels (Determination of % Lowering of TTR to Pretreatment/Baseline TTR Level)

    up to 70 days

Study Arms (2)

ALN-TTR01

ACTIVE COMPARATOR
Drug: ALN-TTR01

Sterile Normal Saline (0.9% NaCl)

PLACEBO COMPARATOR
Drug: Sterile Normal Saline (0.9% NaCl)

Interventions

ALN-TTR01DRUG

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

ALN-TTR01
Sterile Normal Saline (0.9% NaCl)DRUG

Calculated volume to match active comparator

Sterile Normal Saline (0.9% NaCl)

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

Clinical Site

Le Kremlin-Bicêtre, 94275, France

Location

Clinical Site

Porto, 4099-001, Portugal

Location

Clinical Site

Umeå, SE 901 85, Sweden

Location

Clinical Site

London, SE1 1YR, United Kingdom

Location

Related Publications (1)

  • 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.

    PMID: 23984729DERIVED

Study Officials

  • Jared Gollob, MD

    Alnylam Pharmaceuticals Inc

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
SINGLE
Who Masked
PARTICIPANT
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 21, 2010

First Posted

June 23, 2010

Study Start

June 1, 2010

Primary Completion

January 1, 2012

Study Completion

February 1, 2012

Last Updated

May 24, 2012

Record last verified: 2012-05

Locations

FR(1)GB(1)PT(1)SE(1)