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Evinacumab

Phase 3

Homozygous Familial Hypercholesterolemia | Small molecule | Metabolic |Ultragenyx Pharmaceutical Inc.|Last Updated: Mar 25, 2025

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment10

FDA Designations

No designations recorded

Clinical trial landscape

Evinacumab · 1 trial · 1 indication

Phase 3 1
NCT05611528Safety and Effectiveness of Evinacumab for the Treatment of Homozygous Familial HypercholesterolemiaHomozygous Familial Hypercholesterolemia
COMPLETED10 Analytics
PHASE3COMPLETED
Safety and Effectiveness of Evinacumab for the Treatment of Homozygous Familial Hypercholesterolemia
Homozygous Familial HypercholesterolemiaUnlock trial analytics

Study Endpoints

Primary Endpoints

Change in lipid profile
Every 4 weeks up to 2 years

Change in total cholesterol, LDL-Cholesterol, HDL-Cholesterol, triglyceride, non-HDL-Cholesterol and apolipoprotein B plasma concentration

Change in aspartate transaminase (AST) plasma concentration
Every 4 weeks up to 2 years
change in alanine aminotransferase (ALT) plasma concentration
Every 4 weeks up to 2 years

Secondary Endpoints

Lipoprotein (a)
Yearly up to 2 years
Complete blood count
Every 12 weeks up to 2 years
Total bilirubin
Every 12 weeks up to 2 years
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Evinacumab-treated patientsEXPERIMENTAL -

Interventions

NameTypeDescription
EvinacumabDRUGEvinacumab 15 mg/kg administered intravenously every 4 weeks
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * HoFH patients having been previously or being currently treated with evinacumab or clinical diagnosis of HoFH requiring additional lipid lowering therapy. Exclusion Criteria: * Any new condition or worsening of an existing condition, which in the opinion of the investigator ...

Countries:Canada
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Frequently asked questions about Evinacumab

What is Evinacumab used for?

Evinacumab is used for the treatment of homozygous familial hypercholesterolemia, a genetic condition characterized by extremely high levels of low-density lipoprotein cholesterol. It is being developed by Ultragenyx Pharmaceutical Inc. (ticker: RARE) and is currently in Phase 3 clinical development.

Who makes Evinacumab?

Evinacumab is being developed by Ultragenyx Pharmaceutical Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol RARE. The company is conducting clinical trials to evaluate the drug's safety and effectiveness for the treatment of homozygous familial hypercholesterolemia.

What phase is Evinacumab in?

Evinacumab is in Phase 3 clinical development. It is an investigational drug, meaning it has not yet been approved by regulatory authorities. A Phase 3 clinical trial has been completed to assess its safety and effectiveness in patients with homozygous familial hypercholesterolemia.

What clinical trials is Evinacumab in?

Evinacumab has been studied in a completed Phase 3 clinical trial with the identifier NCT05611528. This trial, titled 'Safety and Effectiveness of Evinacumab for the Treatment of Homozygous Familial Hypercholesterolemia,' enrolled 10 participants in Canada and was not randomized or double-blinded.

Is Evinacumab FDA approved?

Evinacumab is not FDA approved. It is an investigational drug currently in Phase 3 clinical development for the treatment of homozygous familial hypercholesterolemia. The completed Phase 3 trial, NCT05611528, has finished, but the drug remains under investigation and has not received regulatory approval.