Transthyretin (TTR)-Mediated AmyloidosisPhase 1NCT02053454
A Study of the Safety, Tolerability and Pharmacokinetics of ALN-TTR02 in Japanese Healthy Volunteers
A Phase 1 study testing patisiran (ALN-TTR02) and Sterile Normal Saline (0.9% NaCl) in people with Transthyretin (TTR)-Mediated Amyloidosis, run by Alnylam Pharmaceuticals at 1 site in United Kingdom.
Trial summary
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics (PK) of patisiran (ALN-TTR02) in Japanese subjects Read more about this study
- Who is it for?
- Ages 20 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Transthyretin (TTR)-Mediated Amyloidosis
- What is being tested?
- patisiran (ALN-TTR02) and Sterile Normal Saline (0.9% NaCl)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- Jan 2014to Jun 2014
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 20 to 65 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Healthy Japanese adult males and females aged 20 to 65 years, inclusive (The subject was born in Japan and has lived outside of Japan for <10 years, and subject's biological parents and grandparents are fully Japanese and were born in Japan);
- Subjects who are healthy as determined by clinical assessments;
- Females subjects must be of non-childbearing potential;
- Males with partners of child-bearing potential, must agree to use appropriate contraception.
Who cannot join
- Subjects with a history of serious mental illness;
- Subjects who have a clinically relevant medical or surgical history;
- Subjects with a positive screen for alcohol or drugs of abuse;
- Subjects with safety laboratory test results deemed clinically significant;
- Subjects with known hepatitis B surface antigen (HBsAg), hepatitis B virus (HBV), hepatitis C virus (HCV) or human immunodeficiency virus (HIV) infection;
- Subjects who have received an investigational agent within the 3 months prior to study entry.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 1, Randomized, Double-blind, Placebo-controlled, Single Ascending Dose, Safety, Tolerability and Pharmacokinetics Study of ALN-TTR02 in Japanese Healthy Volunteers
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics (PK) of patisiran (ALN-TTR02) in Japanese subjects
- Conditions
- Transthyretin (TTR)-Mediated Amyloidosis
- Being tested
- patisiran (ALN-TTR02), Sterile Normal Saline (0.9% NaCl)
- Keywords
- RNAi therapeutic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
patisiran (ALN-TTR02)Drug
Ascending doses administered by intravenous (IV) infusion
Sterile Normal Saline (0.9% NaCl)Drug
Calculated volume to match active comparator
| Study group | Role | What they receive |
|---|---|---|
| patisiran (ALN-TTR02) | Active comparator | Not described Receives: Drug: patisiran (ALN-TTR02) |
| Sterile Normal Saline (0.9% NaCl) | Active comparator | Not described Receives: Drug: Sterile Normal Saline (0.9% NaCl) |
What is being measured
The primary outcome is the main question the study is designed to answer.
The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation
Measured: Up to 28 days
Secondary outcomes (8)
Pharmacodynamics (PD) of ALN-TTR02 (serum concentrations of Transthyretin, Vitamin A, and Retinol Binding Protein)
Measured: Up to 90 days
Observed maximum concentration (Cmax) of ALN-TTR02
Measured: Up to 90 days
Time of observed maximum concentration (tmax) of ALN-TTR02
Measured: Up to 90 days
Area under the plasma concentration versus time curve (AUC) of ALN-TTR02
Measured: Up to 90 days
Terminal elimination half-life (t1/2) of ALN-TTR02
Measured: Up to 90 days
Systemic clearance (CL) of ALN-TTR02
Measured: Up to 90 days
Volume of distribution (V) of ALN-TTR02
Measured: Up to 90 days
Renal clearance (CLR) of ALN-TTR02
Measured: Up to 90 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Feb 2014 (estimated)
- Study startJan 2014
- Primary completionMar 2014
- Study completionJun 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Transthyretin (TTR)-Mediated AmyloidosisStudies of the same condition, open and under way.
- More trials run by Alnylam PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alnylam Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jared Gollob, MD, Study director, Alnylam Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02053454
- Sponsor's protocol ID
- ALN-TTR02-005
- Status last verified
- May 2015
- First submitted
- 24 Jan 2014
- Last updated
- 25 May 2015
- Results posted
- No
- Data monitoring committee
- No