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IONIS-ENaCRx

Phase 1

Healthy Subjects | Small molecule | Respiratory |Ionis Pharmaceuticals, Inc.|Last Updated: Feb 4, 2021

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment98

FDA Designations

No designations recorded

Clinical trial landscape

IONIS-ENaCRx · 1 trial · 2 indications

Phase 1 1
NCT03647228A Phase 1/2a Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of IONIS-ENaCRx in Healthy Volunteers and Patients With Cystic FibrosisHealthy Subjects
COMPLETED98 Analytics
PHASE1COMPLETED
A Phase 1/2a Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of IONIS-ENaCRx in Healthy Volunteers and Patients With Cystic Fibrosis
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Study Endpoints

Primary Endpoints

Safety and Tolerability as Measured by the Number of Participants with at least one Treatment-Emergent Adverse Event
Up to 113 Days

Secondary Endpoints

Cmax: maximum observed drug concentration in plasma of IONIS-ENaCRx
Up to 113 Days
Tmax: time taken to reach maximal concentration in plasma of IONIS-ENaCRx
Up to 113 Days
AUCt: area under the plasma concentration-time curve from time zero to time t for IONIS-ENaCRx
Up to 113 Days
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Study Design & Arms

AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
IONIS-ENaCRxEXPERIMENTALAscending single and multiple doses of IONIS-ENaCRx inhaled or nebulized.
PlaceboPLACEBO_COMPARATORPlacebo comparator calculated volume to match active comparator inhaled or nebulized.

Interventions

NameTypeDescription
IONIS-ENaCRxDRUGAscending single and multiple doses of IONIS-ENaCRx inhaled or nebulized.
PlaceboDRUGPlacebo comparator calculated volume to match active comparator inhaled or nebulized.
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Eligibility Criteria

Age Range18 Years to 50 Years
SexALL
Healthy VolunteersYes
Study Sites8

Inclusion Criteria (Healthy Volunteers) 1. Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal. 2. Males must be surgically sterile or, abstinent or, if engaged in sexual relations with a woman of child-bearing potential, the subject or the subject's non...

Countries:GermanyUnited Kingdom
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Frequently asked questions about IONIS-ENaCRx

What is IONIS-ENaCRx used for?

IONIS-ENaCRx is an investigational small molecule being studied for use in healthy subjects and patients with cystic fibrosis. It is in Phase 1 clinical development and has not been approved by regulatory authorities. The drug is being evaluated for safety, tolerability, pharmacokinetics, and pharmacodynamics.

Who makes IONIS-ENaCRx?

IONIS-ENaCRx 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 effects in healthy volunteers and patients with cystic fibrosis.

What phase is IONIS-ENaCRx in?

IONIS-ENaCRx is in Phase 1 clinical development. A Phase 1/2a study has been completed, evaluating single and multiple doses in healthy volunteers and patients with cystic fibrosis. The drug remains investigational and is not yet approved for any use.

What clinical trials is IONIS-ENaCRx in?

IONIS-ENaCRx has one completed clinical trial, NCT03647228, a Phase 1/2a study assessing safety, tolerability, pharmacokinetics, and pharmacodynamics of single and multiple doses in healthy volunteers and patients with cystic fibrosis. The trial enrolled 98 participants in Germany and the United Kingdom.

Is IONIS-ENaCRx the same as IONIS-ENaCRx?

IONIS-ENaCRx is the drug name as provided, with no alternative names listed. It is an investigational small molecule developed by Ionis Pharmaceuticals for respiratory conditions, specifically being studied in healthy subjects and patients with cystic fibrosis.