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ISIS-APORx

Phase 2

Elevated Lipoprotein(a) | Small molecule | Cardiovascular |Ionis Pharmaceuticals, Inc.|Last Updated: Dec 20, 2019

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment64

FDA Designations

No designations recorded

Clinical trial landscape

ISIS-APORx · 1 trial · 1 indication

Phase 2 1
NCT02160899Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS-APO(a)Rx in Participants With High Lipoprotein(a)Elevated Lipoprotein(a)
COMPLETED64 Analytics
PHASE2COMPLETED
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS-APO(a)Rx in Participants With High Lipoprotein(a)
Elevated Lipoprotein(a)Unlock trial analytics

Study Endpoints

Primary Endpoints

Percent Change From Baseline in Lipoprotein Lp(a) Plasma Concentration at Day 85/Day 99
Day 85/Day 99

Data are reported for evaluable participants.

Number of Participants With at Least One Treatment-emergent Adverse Event (TEAE)
Up to approximately 32 weeks

An adverse event is any unfavorable and unintended sign (including a clinically significant abnormal laboratory finding, for example), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE is considered related to the investigational drug product.

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Study Design & Arms

AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Cohort A: PlaceboPLACEBO_COMPARATORParticipants will receive placebo (normal saline) subcutaneously on Days 1, 8, 15, 22, 29, 36, 43, 50, 57, 64, 71, and 78.
Cohort A: ISIS-APO(a)Rx < 2000 mgEXPERIMENTALParticipants will receive ISIS-APO(a)Rx subcutaneously: 100 mg on Days 1, 8, 15, and 22; 200 mg on Days 29, 36, 43, and 50 unless down-titrated; and 300 mg on Days 57, 64, 71, and 78 unless down-titrated.
Cohort A: ISIS-APO(a)Rx >= 2000 mgEXPERIMENTALParticipants will receive ISIS-APO(a)Rx subcutaneously: 100 mg on Days 1, 8, 15, and 22; 200 mg on Days 29, 36, 43, and 50 unless down-titrated; and 300 mg on Days 57, 64, 71, and 78 unless down-titrated.
Cohort B: PlaceboPLACEBO_COMPARATORParticipants will receive placebo (normal saline) subcutaneously on Days 1, 8, 15, 22, 29, 36, 43, 50, 57, 64, 71, and 78.
Cohort B: ISIS-APO(a)Rx < 2000 mgEXPERIMENTALParticipants will receive ISIS-APO(a)Rx subcutaneously: 100 mg on Days 1, 8, 15, and 22; 200 mg on Days 29, 36, 43, and 50 unless down-titrated; and 300 mg on Days 57, 64, 71, and 78 unless down-titrated.
Cohort B: ISIS-APO(a)Rx >= 2000 mgEXPERIMENTALParticipants will receive ISIS-APO(a)Rx subcutaneously: 100 mg on Days 1, 8, 15, and 22; 200 mg on Days 29, 36, 43, and 50 unless down-titrated; and 300 mg on Days 57, 64, 71, and 78 unless down-titrated.

Interventions

NameTypeDescription
ISIS-APO(a)RxDRUGISIS-APO(a)Rx subcutaneously: 100 mg on Days 1, 8, 15, and 22; 200 mg on Days 29, 36, 43, and 50 unless down-titrated; and 300 mg on Days 57, 64, 71, and 78 unless down-titrated.
PlaceboDRUGNormal saline as Placebo, subcutaneously on Days 1, 8, 15, 22, 29, 36, 43, 50, 57, 64, 71, and 78.
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Eligibility Criteria

Age Range18 Years to 65 Years
SexALL
Healthy VolunteersNo
Study Sites13

Inclusion Criteria: * Males or females aged 18-65 inclusive * Females must be non-pregnant and non-lactating, and either surgically sterile (e.g., tubal occlusion, hysterectomy, bilateral salpingectomy, bilateral oophorectomy) or post-menopausal (defined as 12 months of spontaneous amenorrhea witho...

Countries:CanadaDenmarkGermanyNetherlandsUnited Kingdom
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Frequently asked questions about ISIS-APORx

What is ISIS-APORx used for?

ISIS-APORx is an investigational drug being developed for elevated lipoprotein(a), a condition associated with increased cardiovascular risk. It is intended to lower elevated levels of lipoprotein(a) in patients with high levels of this lipid. The drug is currently in clinical development and has not been approved by regulatory authorities.

What does ISIS-APORx target?

ISIS-APORx is designed to target apolipoprotein(a), the protein component of lipoprotein(a). By targeting this protein, the drug aims to reduce the production of lipoprotein(a) particles in the body, thereby lowering elevated lipoprotein(a) levels. This mechanism is being evaluated in clinical trials for its potential to address cardiovascular risk associated with high lipoprotein(a).

Who makes ISIS-APORx?

ISIS-APORx is being developed by Ionis Pharmaceuticals, Inc., a biopharmaceutical company focused on RNA-targeted therapies. Ionis Pharmaceuticals is publicly traded under the ticker symbol IONS on the NASDAQ stock exchange. The company is responsible for the clinical development and potential commercialization of ISIS-APORx.

What phase is ISIS-APORx in?

ISIS-APORx is in Phase 2 of clinical development. It is an investigational drug, meaning it has not been approved by the FDA or other regulatory agencies. The Phase 2 trial has been completed, and the drug is no longer in active clinical trials. Further development status beyond Phase 2 has not been disclosed.

What clinical trials is ISIS-APORx in?

ISIS-APORx has been studied in one clinical trial with the identifier NCT02160899. This Phase 2 trial, titled 'Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS-APO(a)Rx in Participants With High Lipoprotein(a),' enrolled 64 participants with elevated lipoprotein(a). The study was completed and was conducted in Canada, Denmark, Germany, Netherlands, and the United Kingdom.

Is ISIS-APORx the same as ISIS-APO(a)Rx?

Yes, ISIS-APORx is also known as ISIS-APO(a)Rx. The clinical trial NCT02160899 uses the name ISIS-APO(a)Rx to refer to the same investigational drug. Both names refer to the same compound being developed by Ionis Pharmaceuticals for elevated lipoprotein(a).