A Phase 1, Single- and Multi-Dose, Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneously Administered ALN-TTRSC in Healthy Volunteers

This study is currently recruiting participants.
Verified March 2013 by Alnylam Pharmaceuticals
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01814839
First received: March 18, 2013
Last updated: March 20, 2013
Last verified: March 2013
  Purpose

The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-TTRSC in healthy volunteer subjects.


Condition Intervention Phase
TTR-mediated Amyloidosis
Drug: ALN-TTRSC
Drug: Sterile Normal Saline (0.9% NaCl)
Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 1, Randomized, Double-blind, Placebo-controlled, Single- and Multi-Dose, Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneously Administered ALN-TTRSC in Healthy Volunteers

Resource links provided by NLM:


Further study details as provided by Alnylam Pharmaceuticals:

Primary Outcome Measures:
  • The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation. [ Time Frame: Up to 63 days ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Pharmacokinetics (PK) of ALN-TTRSC (Cmax, tmax, t1/2, AUC0-last, CL). [ Time Frame: Up to 90 days ] [ Designated as safety issue: No ]
  • Effect of ALN-TTRSC on transthyretin (TTR) (Determination of % Lowering of TTR to pretreatment/Baseline Levels). [ Time Frame: Up to 90 days ] [ Designated as safety issue: No ]
  • Effect of ALN-TTRSC on vitamin A (Determination of % Lowering of vitamin A to pretreatment/Baseline Levels). [ Time Frame: Up to 90 days ] [ Designated as safety issue: No ]
  • Effect of ALN-TTRSC on retinol binding protein (RBP) (Determination of % Lowering of RBP to pretreatment/Baseline Levels). [ Time Frame: Up to 90 days ] [ Designated as safety issue: No ]

Estimated Enrollment: 40
Study Start Date: March 2013
Estimated Study Completion Date: March 2014
Estimated Primary Completion Date: December 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: ALN-TTRSC Drug: ALN-TTRSC
Ascending doses of ALN-TTRSC by subcutaneous (SC) injection
Placebo Comparator: Sterile Normal Saline (0.9% NaCl) Drug: Sterile Normal Saline (0.9% NaCl)
Calculated volume to match active comparator

  Eligibility

Ages Eligible for Study:   18 Years to 55 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   Yes
Criteria

Inclusion Criteria:

  • Body mass index (BMI) must be ≥ 18.0 kg/m2 and ≤ 30.0 kg/m2;
  • Female subjects must be of non-childbearing potential; e.g., postmenopausal or pre-menopausal with surgical sterilization;
  • Male subjects agree to use appropriate contraception;
  • Adequate blood counts, liver and renal function;
  • Non-smokers for at least 3 months;
  • Willing to give written informed consent and are willing to comply with the study requirements.

Exclusion Criteria:

  • Known human immunodeficiency virus (HIV) positive status or known or suspected systemic bacterial, viral, parasitic, or fungal infection;
  • Subjects with a history of multiple drug allergies or intolerance to SC injection;
  • History of drug abuse and/or alcohol abuse;
  • Receiving an investigational agent within 3 months prior to study drug administration;
  • Considered unfit for the study by the Principal Investigator.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01814839

Contacts
Contact: Alnylam Clinical Trials Hotline. Call for Complete Site List. 617-575-7400 or 1-866-330-0326

Locations
United Kingdom
Clinical Site Recruiting
Leeds, United Kingdom, LS2 9LH
Clinical Site Recruiting
London, United Kingdom, NW10 7EW
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
Study Director: Jared Gollob, MD Alnylam Pharmaceuticals
  More Information

No publications provided

Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01814839     History of Changes
Other Study ID Numbers: ALN-TTRSC-001
Study First Received: March 18, 2013
Last Updated: March 20, 2013
Health Authority: United Kingdom: Medicines and Healthcare Products Regulatory Agency

Additional relevant MeSH terms:
Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases

ClinicalTrials.gov processed this record on April 17, 2014