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A Safety and Tolerability Study of an Investigational Drug, ALN-TTRSC02, in Healthy Subjects

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02797847
Recruitment Status : Completed
First Posted : June 14, 2016
Last Update Posted : September 21, 2018
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Tracking Information
First Submitted Date  ICMJE May 18, 2016
First Posted Date  ICMJE June 14, 2016
Last Update Posted Date September 21, 2018
Actual Study Start Date  ICMJE June 7, 2016
Actual Primary Completion Date January 12, 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 26, 2018)
Safety of ALN-TTRSC02, evaluated by the proportion of subjects experiencing adverse events (AEs) [ Time Frame: Day 1 through to Day 314 ]
Original Primary Outcome Measures  ICMJE
 (submitted: June 8, 2016)
Safety of ALN-TTRSC02, evaluated by the proportion of subjects experiencing adverse events (AEs) [ Time Frame: Day 1 through to Day 118 ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: February 26, 2018)
  • Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
    Cmax
  • Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
    tmax
  • Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
    AUC
  • Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
  • Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
    CL/F
  • Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
    Vss/F
  • Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
    Vz/F
  • Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 314 ]
    fe
  • Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 314 ]
    CLR
  • Effect of ALN-TTRSC02 on serum TTR levels as measured by reduction from baseline in serum TTR [ Time Frame: Day 1 through to Day 314 ]
  • Effect of ALN-TTRSC02 on serum Vitamin A levels as measured by reduction from baseline in serum Vitamin A [ Time Frame: Screening through to Day 314 ]
Original Secondary Outcome Measures  ICMJE
 (submitted: June 8, 2016)
  • Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
    Cmax
  • Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
    tmax
  • Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
    AUC
  • Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
  • Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
    CL/F
  • Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
    Vss/F
  • Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
    Vz/F
  • Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
    fe
  • Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
    CLR
  • Effect of ALN-TTRSC02 on serum TTR levels as measured by reduction from baseline in serum TTR [ Time Frame: Day 1 through to Day 118 ]
  • Effect of ALN-TTRSC02 on serum Vitamin A levels as measured by reduction from baseline in serum Vitamin A [ Time Frame: Screening through to Day 118 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Safety and Tolerability Study of an Investigational Drug, ALN-TTRSC02, in Healthy Subjects
Official Title  ICMJE A Phase 1, Randomized, Single-Blind, Placebo Controlled, Single-Ascending Dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN-TTRSC02 in Healthy Subjects
Brief Summary The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-TTRSC02 in healthy subjects.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Participant)
Primary Purpose: Treatment
Condition  ICMJE Transthyretin-mediated Amyloidosis (ATTR Amyloidosis)
Intervention  ICMJE
  • Drug: ALN-TTRSC02
    Ascending doses of ALN-TTRSC02 by subcutaneous (SC) injection
  • Drug: Sterile Normal Saline (0.9% NaCl)
    Calculated volume to match active comparator
Study Arms  ICMJE
  • Active Comparator: ALN-TTRSC02
    Intervention: Drug: ALN-TTRSC02
  • Placebo Comparator: Sterile normal saline 0.9% for SC administration
    Intervention: Drug: Sterile Normal Saline (0.9% NaCl)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: February 26, 2018)
80
Original Estimated Enrollment  ICMJE
 (submitted: June 8, 2016)
110
Actual Study Completion Date  ICMJE January 12, 2018
Actual Primary Completion Date January 12, 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Male and female subjects, age 18 to 65 years, inclusive.
  • Body mass index (BMI) ≥18.0 kg/m2 and ≤30 kg/m2 assessed at Screening.
  • No clinically significant health concerns, as determined by medical history and physical examination, in the opinion of the Investigator.
  • Women of child bearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception, willing and able to comply with the study requirements and to provide written informed consent.
  • For Japanese cohorts, subjects of Japanese descent are defined as people carrying a Japanese passport, descendants of 4 Japanese grandparents, and have not been outside Japan for more than 5 years.

Exclusion Criteria:

  • Clinically relevant history or presence of respiratory, gastrointestinal, renal, cardiovascular, hepatic, hematological, lymphatic, neurological, psychiatric, musculoskeletal, genitourinary, immunological, and other inflammatory diseases, or dermatological or connective tissue diseases or disorders.
  • Active serious mental illness or psychiatric disorder.
  • Clinically significant ECG abnormalities. Abnormal for AST/ALT and any other clinical safety laboratory result considered clinically significant.
  • Known history of allergic reaction to an oligonucleotide or GalNAc.
  • History of intolerance to subcutaneous injection.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 65 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE Yes
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02797847
Other Study ID Numbers  ICMJE ALN-TTRSC02-001
2015-005803-98 ( EudraCT Number )
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party Alnylam Pharmaceuticals
Study Sponsor  ICMJE Alnylam Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: John Vest, MD, PhD Alnylam Pharmaceuticals
PRS Account Alnylam Pharmaceuticals
Verification Date September 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP