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Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Healthy Volunteers

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: NCT03165864
Recruitment Status : Completed
First Posted : May 24, 2017
Last Update Posted : January 22, 2019
Sponsor:
Information provided by (Responsible Party):
Ionis Pharmaceuticals, Inc.

Study Description
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Brief Summary:
The purpose is to assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS TMPRSS6-Lrx Administered Subcutaneously for up to 44 Healthy Volunteers

Condition or disease Intervention/treatment Phase
Thalassemia Drug: IONIS TMPRSS6-Lrx Other: Placebo Phase 1

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 36 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Double-Blind, Placebo-Controlled, Dose-Escalation, Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of ISIS 702843 Administered Subcutaneously to Healthy Volunteers
Actual Study Start Date : May 9, 2017
Actual Primary Completion Date : September 25, 2018
Actual Study Completion Date : September 25, 2018

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Thalassemia

Arms and Interventions
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Arm Intervention/treatment
Experimental: IONIS TMPRSS6-Lrx
Ascending single and multiple doses of IONIS TMPRSS6-Lrx administered subcutaneously
 Drug: IONIS TMPRSS6-Lrx
Ascending single and multiple doses of IONIS TMPRSS6-Lrx administered subcutaneously
Placebo Comparator: Placebo
Saline .9%
 Other: Placebo
Saline .9%


Outcome Measures
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Primary Outcome Measures :
  1. Incidence and severity of adverse events that are related to treatment with IONIS TMPRSS6-Lrx [ Time Frame: Up to 148 Days ]
    The safety and tolerability of single and multiple doses of IONIS TMPRSS6-Lrx will be assessed by determining the incidence, severity, and dose relationship of adverse events that are related to treatment with IONIS TMPRSS6-Lrx


Secondary Outcome Measures :
  1. Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (maximum observed drug concentration or Cmax) [ Time Frame: Up to 148 Days ]
    The plasma pharmacokinetics (maximum observed drug concentration or Cmax) of Ionis TMPRSS6-Lrx will be assessed following single and multiple dose SC administration

  2. Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (time taken to reach maximal concentration or Tmax) [ Time Frame: Up to 148 Days ]
    The plasma pharmacokinetics (time taken to reach maximal concentration or Tmax) of Ionis TMPRSS6-Lrx will be assessed following single and multiple dose SC administration

  3. Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (Plasma terminal elimination half-life (t1/2λz) [ Time Frame: Up to 148 Days ]
    The plasma pharmacokinetics (Plasma terminal elimination half-life (t1/2λz) of Ionis TMPRSS6-Lrx will be assessed following single and multiple dose SC administration

  4. Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (Partial areas under the plasma concentration-time curve from zero time (predose) to selected times (t) after the subcutaneous administration (AUCt) [ Time Frame: Up to 148 Days ]
    The plasma pharmacokinetics (Partial areas under the plasma concentration-time curve from zero time (predose) to selected times (t) after the subcutaneous administration (AUCt) of Ionis TMPRSS6-Lrx will be assessed following single and multiple dose SC administration

  5. Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (Percentage of the administered dose excreted in urine (% Dose Excreted) [ Time Frame: Up to 148 Days ]
    The plasma pharmacokinetics (Percentage of the administered dose excreted in urine (% Dose Excreted) of Ionis TMPRSS6-Lrx will be assessed following single and multiple dose SC administration


Other Outcome Measures:
  1. Pharmacodynamics after single and multiple doses of IONIS TMPRSS6-Lrx (Changes in transferrin saturation) [ Time Frame: Up to 148 Days ]
    Effects of IONIS TMPRSS6-Lrx on changes (absolute and percent) in transferrin saturation compared to baseline

  2. Pharmacodynamics after single and multiple doses of IONIS TMPRSS6-Lrx (Changes in serum iron) [ Time Frame: Up to 148 Days ]
    Effects of IONIS TMPRSS6-Lrx on changes (absolute and percent) in serum iron compared to baseline

  3. Pharmacodynamics after single and multiple doses of IONIS TMPRSS6-Lrx (Changes in hepcidin levels) [ Time Frame: Up to 148 Days ]
    Effects of IONIS TMPRSS6-Lrx on changes (absolute and percent) in hepcidin levels compared to baseline


Eligibility Criteria
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Information from the National Library of Medicine

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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:

  • Must have given written informed consent and be able to comply with all study requirements
  • Healthy males or females aged 18-65 inclusive at the time of Informed Consent
  • Females must be non-pregnant and non-lactating, and either surgically sterile or post- menopausal
  • Males must be surgically sterile, abstinent or using an acceptable contraceptive method
  • BMI < 32 kg/m2

Exclusion Criteria:

  • Clinically significant abnormalities in medical history or physical examination
  • Clinically significant lab abnormalities that would render a subject unsuitable for inclusion
  • Known history or positive test for HIV, HCV, or HBV
  • Treatment with another Study Drug, biological agent, or device within one-month of Screening or 5 half-lives of investigational agent, whichever is longer
  • Smoking > 10 cigarettes per day
  • Regular excessive use of alcohol within 6 months of screening
  • Current use of concomitant medications other than occasional acetaminophen (paracetamol) or ibuprofen unless approved by Sponsor Medical Monitor
  • Considered unsuitable for inclusion by the Investigator or Sponsor
Contacts and Locations
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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): NCT03165864


Locations
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Australia, Victoria
Nucleus Network
Melbourne, Victoria, Australia, VIC 3004
Sponsors and Collaborators
Ionis Pharmaceuticals, Inc.
More Information
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Responsible Party: Ionis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT03165864    
Other Study ID Numbers: ISIS 702843-CS1
First Posted: May 24, 2017    Key Record Dates
Last Update Posted: January 22, 2019
Last Verified: January 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Keywords provided by Ionis Pharmaceuticals, Inc.:
Thalassaemia, IONIS TMPRSS6-Lrx
Additional relevant MeSH terms:
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Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
 Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn