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

Transthyretin Mediated Amyloidosis (ATTR) Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01148953
• Other study ID #: ALN-TTR01-001
• Secondary ID: 2009-017383-16
• Status: Completed
• Phase: Phase 1
• First received: June 21, 2010
• Last updated: May 23, 2012
• Start date: June 2010
• Est. completion date: February 2012

Study information

• Verified date: May 2012
• Source: Alnylam Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Health authority: Portugal: National Pharmacy and Medicines InstituteUnited Kingdom: Medicines and Healthcare Products Regulatory AgencySweden: Medical Products AgencyFrance: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 32
• Est. completion date: February 2012
• Est. primary completion date: January 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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.

Study Design

Allocation: Randomized, Endpoint Classification: Safety Study, Intervention Model: Parallel Assignment, Masking: Single Blind (Subject), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Amyloidosis
• Transthyretin Mediated Amyloidosis (ATTR)

Intervention

Drug:
• ALN-TTR01
Dose levels between 0.01 and 1.0 mg/kg by intravenous (IV) infusion
• Sterile Normal Saline (0.9% NaCl)
Calculated volume to match active comparator

Locations

Country	Name	City	State
France	Clinical Site	Le Kremlin Bicêtre
Portugal	Clinical Site	Porto
Sweden	Clinical Site	Umeå
United Kingdom	Clinical Site	London

Sponsors (1)

Lead Sponsor	Collaborator
Alnylam Pharmaceuticals

Countries where clinical trial is conducted

France,  Portugal,  Sweden,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The proportion of patients experiencing adverse events (AEs), serious adverse events (SAEs), and study drug discontinuation.		Up to 28 Days	Yes
Secondary	Pharmacokinetics (PK) of ALN-TTR01 (Cmax, tmax, t1/2, AUC0-last, CL)		Up to 70 days	Yes
Secondary	Effect of ALN-TTR01 on Circulating TTR Levels (Determination of % Lowering of TTR to Pretreatment/Baseline TTR Level)		up to 70 days	No