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IONIS TMPRSS6-Lrx

Phase 1

Thalassemia | Small molecule | Hematology |Ionis Pharmaceuticals, Inc.|Last Updated: Jan 22, 2019

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment36

FDA Designations

No designations recorded

Clinical trial landscape

IONIS TMPRSS6-Lrx · 1 trial · 1 indication

Phase 1 1
NCT03165864Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Healthy VolunteersThalassemia
COMPLETED36 Analytics
PHASE1COMPLETED
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Healthy Volunteers
ThalassemiaUnlock trial analytics

Study Endpoints

Primary Endpoints

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

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
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Study Design & Arms

AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
IONIS TMPRSS6-LrxEXPERIMENTALAscending single and multiple doses of IONIS TMPRSS6-Lrx administered subcutaneously
PlaceboPLACEBO_COMPARATORSaline .9%

Interventions

NameTypeDescription
IONIS TMPRSS6-LrxDRUGAscending single and multiple doses of IONIS TMPRSS6-Lrx administered subcutaneously
PlaceboOTHERSaline .9%
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Eligibility Criteria

Age Range18 Years to 65 Years
SexALL
Healthy VolunteersYes
Study Sites1

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 * Male...

Countries:Australia
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Frequently asked questions about IONIS TMPRSS6-Lrx

What is IONIS TMPRSS6-Lrx used for?

IONIS TMPRSS6-Lrx is an investigational small molecule being developed for thalassemia, a blood disorder. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities. The drug is being studied for its potential role in managing this condition, though its efficacy and safety are still being evaluated.

Who makes IONIS TMPRSS6-Lrx?

IONIS TMPRSS6-Lrx is being developed by Ionis Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol IONS. The company is conducting clinical trials to evaluate the drug's safety and tolerability in healthy volunteers as part of early-stage research.

What phase is IONIS TMPRSS6-Lrx in?

IONIS TMPRSS6-Lrx is in Phase 1 clinical development. A Phase 1 trial has been completed, which focused on assessing the drug's safety, tolerability, pharmacokinetics, and pharmacodynamics in healthy volunteers. The drug remains investigational and has not received regulatory approval for any indication.

What clinical trials is IONIS TMPRSS6-Lrx in?

IONIS TMPRSS6-Lrx has one completed clinical trial, registered under NCT03165864. This Phase 1 study evaluated the safety, tolerability, pharmacokinetics, and pharmacodynamics of the drug when administered subcutaneously to healthy volunteers. The trial was randomized, double-blind, and placebo-controlled, enrolling 36 participants in Australia.

Is IONIS TMPRSS6-Lrx the same as ISIS 702843?

Yes, IONIS TMPRSS6-Lrx is also known as ISIS 702843. The completed clinical trial NCT03165864 used the name ISIS 702843 to refer to the drug. Both names refer to the same investigational compound being developed by Ionis Pharmaceuticals for thalassemia.