- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01559077
Trial to Evaluate Safety, Tolerability, and Parmacokinetics of ALN-TTR02 in Healthy Volunteer Subjects
January 14, 2013 updated by: Alnylam Pharmaceuticals
A Phase 1, Randomized, Single-blind, Placebo-Controlled, Single Ascending Dose, Safety, Tolerability and Pharmacokinetics Study of ALN-TTR02 in Healthy Volunteers
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of ALN-TTR02 in healthy volunteer subjects.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
17
Phase
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
-
Leeds, United Kingdom, LS2 9LH
- Clinical Site
-
London, United Kingdom, NW10 7EW
- Clinical Site
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years to 45 years (ADULT)
Accepts Healthy Volunteers
Yes
Genders Eligible for Study
All
Description
Inclusion Criteria:
- Body mass index must be between 18.0 kg/m2 and ≤ 31.5 kg/m2;
- Non-smokers for at least 3 months preceding screening;
- Females subjects must be of non-childbearing potential e.g., post-menopausal or pre-menopausal with surgical sterilization;
- Males agree to use appropriate contraception;
- Medical history must be verified by either a personal physician or medical practitioner as appropriate;
- Willing to give written informed consent and are willing to comply with the study requirements.
Exclusion Criteria:
- Known hepatitis B surface antigen (HBsAg), hepatitis B virus (HBV), hepatitis C virus (HCV) or human immunodeficiency virus (HIV) infection
- Multiple drug allergies or know sensitivity to oligonucleotide
- History of drug abuse and/or alcohol abuse
- Receiving an investigational agent within 3 months prior to study drug administration
- Subjects with safety laboratory test results deemed clinical significant by the Investigator;
- Received prescription drugs within 4 weeks of first dosing
- Subjects who have donated more than 500 mL of blood within the 3 months prior to ALN-PCS02 or placebo administration;
- Subjects who have used prescription drugs within 4 weeks of first dosing
- Considered unfit for the study by the Principal Investigator
- Employee or family member of the sponsor or the clinical study site personnel
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: RANDOMIZED
- Interventional Model: PARALLEL
- Masking: SINGLE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
ACTIVE_COMPARATOR: ALN-TTR02
|
Dose levels between 0.01 and 0.5 mg/kg by intravenous (IV) infusion
|
PLACEBO_COMPARATOR: Sterile Normal Saline (0.9% NaCl)
|
Calculated volume to match active comparator
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation.
Time Frame: Up to 28 days
|
Up to 28 days
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Pharmacokinetics (PK) of ALN-TTR02 (Cmax, tmax, t1/2, AUC0-last, CL).
Time Frame: Up to 180 days
|
Up to 180 days
|
Effect of ALN-TTR02 on transthyretin (TTR), vitamin A, and retinol binding protein (RBP) (Determination of % Lowering of TTR, vitamin A and RBP to pretreatment/Baseline Levels)
Time Frame: Up to 56 days
|
Up to 56 days
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
March 1, 2012
Primary Completion (ACTUAL)
June 1, 2012
Study Completion (ACTUAL)
November 1, 2012
Study Registration Dates
First Submitted
March 19, 2012
First Submitted That Met QC Criteria
March 20, 2012
First Posted (ESTIMATE)
March 21, 2012
Study Record Updates
Last Update Posted (ESTIMATE)
January 15, 2013
Last Update Submitted That Met QC Criteria
January 14, 2013
Last Verified
January 1, 2013
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- ALN-TTR02-001
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on TTR-mediated Amyloidosis
-
Alnylam PharmaceuticalsCompletedTTR-mediated AmyloidosisUnited States, United Kingdom
-
Alnylam PharmaceuticalsCompletedA Study of the Safety, Tolerability and Pharmacokinetics of ALN-TTR02 in Japanese Healthy VolunteersTransthyretin (TTR)-Mediated AmyloidosisUnited Kingdom
-
Alnylam PharmaceuticalsCompletedTTR-mediated AmyloidosisPortugal, Spain, Sweden, Brazil, United States, France, Germany
-
Alnylam PharmaceuticalsCompleted
-
Alnylam PharmaceuticalsCompletedTTR-mediated AmyloidosisPortugal, Sweden, Brazil, United States, France, Spain, Germany
-
Alnylam PharmaceuticalsCompletedTTR-mediated AmyloidosisUnited States, Canada, United Kingdom
-
Mayo ClinicCompletedTTR Cardiac AmyloidosisUnited States
-
IRCCS Policlinico S. MatteoCompleted
-
Alnylam PharmaceuticalsNo longer availableAmyloid Neuropathies | Amyloid Neuropathies, Familial | TTR-mediated Amyloidosis | Amyloidosis, Hereditary | Amyloidosis, Hereditary, Transthyretin-Related | Familial Amyloid Polyneuropathies
-
Alnylam PharmaceuticalsCompletedAmyloid Neuropathies | Amyloid Neuropathies, Familial | TTR-mediated Amyloidosis | Amyloidosis, Hereditary | Amyloidosis, Hereditary, Transthyretin-Related | Familial Amyloid PolyneuropathiesUnited States, Canada, Argentina, Australia, Bulgaria, Cyprus, Italy, Japan, Malaysia, Mexico, Netherlands, Portugal, Spain, Sweden, Germany, France, Brazil, Turkey, Korea, Republic of, Taiwan, United Kingdom
Clinical Trials on Sterile Normal Saline (0.9% NaCl)
-
Arrowhead PharmaceuticalsCompletedDyslipidemias | Hypertriglyceridemia | Familial HypercholesterolemiaAustralia, New Zealand
-
Arrowhead PharmaceuticalsCompletedHypertriglyceridemia | Familial ChylomicronemiaAustralia, Canada, New Zealand
-
Alnylam PharmaceuticalsCompletedParoxysmal Nocturnal Hemoglobinuria (PNH)Spain, United Kingdom
-
Alnylam PharmaceuticalsCompletedTransthyretin-mediated Amyloidosis (ATTR Amyloidosis)United Kingdom
-
Alnylam PharmaceuticalsThe Medicines CompanyCompletedHypercholesterolemiaUnited Kingdom
-
Alnylam PharmaceuticalsCompletedElevated LDL-Cholesterol (LDL-C)United Kingdom
-
Alnylam PharmaceuticalsCompletedTransthyretin Mediated Amyloidosis (ATTR)United Kingdom, France, Portugal, Sweden
-
Alnylam PharmaceuticalsCompletedAmyloid Neuropathies | Amyloid Neuropathies, Familial | Amyloidosis, Hereditary | Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC) | Amyloidosis, Hereditary, Transthyretin-Related | Familial Transthyretin Cardiac AmyloidosisUnited States, France, Italy, Spain, Canada, Germany, Belgium, Sweden, United Kingdom
-
Alnylam PharmaceuticalsCompletedAcute Intermittent PorphyriaUnited States, United Kingdom, Sweden
-
Alnylam PharmaceuticalsActive, not recruitingTransthyretin Amyloidosis (ATTR) With CardiomyopathyUnited States, Poland, Argentina, Australia, Belgium, France, Germany, Japan, Netherlands, Portugal, Spain, Sweden, United Kingdom, Canada, Czechia, Denmark, Hungary, Ireland, Norway, Korea, Republic of, Austria, Croatia, Israel, Latvia, Lithuan... and more