NCT03165864

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

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
36

participants targeted

Target at P50-P75 for phase_1

Timeline
Completed

Started May 2017

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

May 9, 2017

Completed
9 days until next milestone

First Submitted

Initial submission to the registry

May 18, 2017

Completed
6 days until next milestone

First Posted

Study publicly available on registry

May 24, 2017

Completed
1.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 25, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 25, 2018

Completed
Last Updated

January 22, 2019

Status Verified

January 1, 2019

Enrollment Period

1.4 years

First QC Date

May 18, 2017

Last Update Submit

January 17, 2019

Conditions

Thalassemia

Keywords

Thalassaemia, IONIS TMPRSS6-Lrx

Outcome Measures

Primary Outcomes (1)

  • Incidence and severity of adverse events that are related to treatment with IONIS TMPRSS6-Lrx

    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

    Up to 148 Days

Secondary Outcomes (5)

  • Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (maximum observed drug concentration or Cmax)

    Up to 148 Days

  • Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (time taken to reach maximal concentration or Tmax)

    Up to 148 Days

  • Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (Plasma terminal elimination half-life (t1/2λz)

    Up to 148 Days

  • 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)

    Up to 148 Days

  • Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (Percentage of the administered dose excreted in urine (% Dose Excreted)

    Up to 148 Days

Other Outcomes (3)

  • Pharmacodynamics after single and multiple doses of IONIS TMPRSS6-Lrx (Changes in transferrin saturation)

    Up to 148 Days

  • Pharmacodynamics after single and multiple doses of IONIS TMPRSS6-Lrx (Changes in serum iron)

    Up to 148 Days

  • Pharmacodynamics after single and multiple doses of IONIS TMPRSS6-Lrx (Changes in hepcidin levels)

    Up to 148 Days

Study Arms (2)

IONIS TMPRSS6-Lrx

EXPERIMENTAL

Ascending single and multiple doses of IONIS TMPRSS6-Lrx administered subcutaneously

Drug: IONIS TMPRSS6-Lrx

Placebo

PLACEBO COMPARATOR

Saline .9%

Other: Placebo

Interventions

IONIS TMPRSS6-LrxDRUG

Ascending single and multiple doses of IONIS TMPRSS6-Lrx administered subcutaneously

IONIS TMPRSS6-Lrx
PlaceboOTHER

Saline .9%

Placebo

Eligibility Criteria

Age18 Years - 65 Years
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • Clinically significant abnormalities in medical history or physical examination
  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Nucleus Network

Melbourne, Victoria, VIC 3004, Australia

Location

Related Publications (1)

  • Ganz T, Nemeth E, Rivella S, Goldberg P, Dibble AR, McCaleb ML, Guo S, Monia BP, Barrett TD. TMPRSS6 as a Therapeutic Target for Disorders of Erythropoiesis and Iron Homeostasis. Adv Ther. 2023 Apr;40(4):1317-1333. doi: 10.1007/s12325-022-02421-w. Epub 2023 Jan 23.

    PMID: 36690839DERIVED

MeSH Terms

Conditions

Thalassemia

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 18, 2017

First Posted

May 24, 2017

Study Start

May 9, 2017

Primary Completion

September 25, 2018

Study Completion

September 25, 2018

Last Updated

January 22, 2019

Record last verified: 2019-01

Locations

AU(1)