Recent Updates
Recently added Catalysts

Pelacarsen

Phase 1

Healthy Participants | Small molecule | Other |Ionis Pharmaceuticals, Inc.|Last Updated: Apr 20, 2022

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment29

FDA Designations

No designations recorded

Clinical trial landscape

Pelacarsen · 1 trial · 1 indication

Phase 1 1
NCT05337878A Study to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pelacarsen (ISIS 681257) in Healthy Japanese ParticipantsHealthy Participants
COMPLETED29 Analytics
PHASE1COMPLETED
A Study to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pelacarsen (ISIS 681257) in Healthy Japanese Participants
Healthy ParticipantsUnlock trial analytics

Study Endpoints

Primary Endpoints

SAD: Percentage of Participants With Clinically Significant Changes From Baseline in Clinical Laboratory Evaluation Parameters
Up to Day 90
SAD: Percentage of Participants With Adverse Events
Up to Day 90
SAD: Percentage of Participants With Serious Adverse Events
Up to Day 90
MAD: Percentage of Participants With Clinically Significant Changes From Baseline in Clinical Laboratory Evaluation Parameters
Up to Day 204
MAD: Percentage of Participants With Adverse Events
Up to Day 204
MAD: Percentage of Participants With Serious Adverse Events
Up to Day 204

Secondary Endpoints

Maximum Observed Drug Concentration (Cmax) in Plasma After Single Ascending Dose of Pelacarsen
Day 1: Pre-dose; 0.5, 1, 1.5, 2, 3, 4, 6, 8, 12 up to 168 hours post-dose
Area Under Curve (AUC) From Time Zero to the Last Quantifiable Concentration (AUClast) in Plasma After Single Ascending Dose of Pelacarsen
Day 1: Pre-dose; 0.5, 1, 1.5, 2, 3, 4, 6, 8, 12 up to 168 hours post-dose
Maximum Observed Drug Concentration (Cmax) in Plasma After Multiple Doses of Pelacarsen
Days 1 and 85: Pre-dose; 1, 2, 4, 8, 24 up to 168 hours post-dose
Unlock Study Endpoints

Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelSEQUENTIAL
PurposeOTHER

Treatment Arms

ArmTypeDescription
SAD: PlaceboPLACEBO_COMPARATORSingle dose of Pelacarsen-matching placebo administered by SC injection on Day 1 of single-dose treatment period.
SAD: Pelacarsen 20 milligrams (mg)EXPERIMENTALSingle dose of Pelacarsen, 20 mg, administered by SC injection on Day 1 of single-dose treatment period.
SAD: Pelacarsen 40 mgEXPERIMENTALSingle dose of Pelacarsen, 40 mg, administered by SC injection on Day 1 of single-dose treatment period.
SAD: Pelacarsen 80 mgEXPERIMENTALSingle dose of Pelacarsen, 80 mg, administered by SC injection on Day 1 of single-dose treatment period.
MD: PlaceboPLACEBO_COMPARATORMultiple doses of Pelacarsen-matching placebo administered by SC injection every 4 weeks, on Days 1, 29, 57 and 85 of multiple-dose treatment period.
MD: Pelacarsen 80 mgEXPERIMENTALMultiple doses of Pelacarsen, 80 mg, administered by SC injection every 4 weeks, on Days 1, 29, 57 and 85 of multiple-dose treatment period.

Interventions

NameTypeDescription
PlaceboDRUGPelacarsen-matching placebo administered by SC injection.
PelacarsenDRUGPelacarsen administered by SC injection.
Unlock Study Design Details

Eligibility Criteria

Age Range18 Years to 65 Years
SexALL
Healthy VolunteersYes
Study Sites1

Inclusion Criteria: 1. Provided written informed consent (signed and dated) and any authorizations required by local law and was able to comply with all study requirements. 2. Male and female of first-, second- or third-generation Japanese participants. 3. Japanese healthy or obese male and female ...

Countries:United States
Unlock Eligibility Criteria

Frequently asked questions about Pelacarsen

What is Pelacarsen used for?

Pelacarsen is an investigational small molecule being studied in healthy participants. It is being evaluated in a Phase 1 clinical trial to assess its safety, tolerability, pharmacokinetics, and pharmacodynamics. The drug is not approved for any indication and remains in clinical development.

Who makes Pelacarsen?

Pelacarsen is being developed by Ionis Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol IONS. The company is conducting clinical research on this investigational drug.

What phase is Pelacarsen in?

Pelacarsen is in Phase 1 clinical development. It is an investigational drug and has not been approved by regulatory authorities. A Phase 1 trial has been completed, and the drug is still in the early stages of clinical research.

What clinical trials is Pelacarsen in?

Pelacarsen has been studied in one completed Phase 1 clinical trial, identified as NCT05337878. This trial assessed the safety, tolerability, pharmacokinetics, and pharmacodynamics of Pelacarsen in healthy Japanese participants. The study enrolled 29 participants and was conducted in the United States.

Is Pelacarsen FDA approved?

Pelacarsen is not FDA approved. It is an investigational drug currently in Phase 1 clinical development. The completed Phase 1 trial focused on healthy participants, and the drug has not yet progressed to later-stage trials or regulatory approval.