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Trial record 68 of 359 for:    transthyretin

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

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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

Brief Summary:
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-TTRSC02 in healthy subjects.

Condition or disease Intervention/treatment Phase
Transthyretin-mediated Amyloidosis (ATTR Amyloidosis) Drug: ALN-TTRSC02 Drug: Sterile Normal Saline (0.9% NaCl) Phase 1

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 80 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Participant)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : June 7, 2016
Actual Primary Completion Date : January 12, 2018
Actual Study Completion Date : January 12, 2018


Arm Intervention/treatment
Active Comparator: ALN-TTRSC02 Drug: ALN-TTRSC02
Ascending doses of ALN-TTRSC02 by subcutaneous (SC) injection

Placebo Comparator: Sterile normal saline 0.9% for SC administration Drug: Sterile Normal Saline (0.9% NaCl)
Calculated volume to match active comparator




Primary Outcome Measures :
  1. Safety of ALN-TTRSC02, evaluated by the proportion of subjects experiencing adverse events (AEs) [ Time Frame: Day 1 through to Day 314 ]

Secondary Outcome Measures :
  1. Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
    Cmax

  2. Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
    tmax

  3. Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
    AUC

  4. Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]

  5. Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
    CL/F

  6. Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
    Vss/F

  7. Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
    Vz/F

  8. Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 314 ]
    fe

  9. Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 314 ]
    CLR

  10. 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 ]
  11. 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 ]


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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02797847


Locations
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United Kingdom
Clinical Trial Site
London, United Kingdom
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
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Study Director: John Vest, MD, PhD Alnylam Pharmaceuticals

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Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02797847     History of Changes
Other Study ID Numbers: ALN-TTRSC02-001
2015-005803-98 ( EudraCT Number )
First Posted: June 14, 2016    Key Record Dates
Last Update Posted: September 21, 2018
Last Verified: September 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Keywords provided by Alnylam Pharmaceuticals:
TTR-mediated Amyloidosis
Amyloidosis, Hereditary
Amyloid Neuropathies, Familial
Familial Amyloid Polyneuropathies
Amyloid Neuropathies
Amyloidosis, Hereditary, Transthyretin-Related
Familial Amyloidosis
RNAi therapeutic

Additional relevant MeSH terms:
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Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases