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sapablursen

Phase 2

Phlebotomy Dependent Polycythemia Vera | Small molecule | Hematology |Ionis Pharmaceuticals, Inc.|Last Updated: Jul 9, 2026

Success Probability

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Trial Design

RandomizedCONTROLLED
Total Trials1
Total Enrollment50

FDA Designations

No designations recorded

Clinical trial landscape

sapablursen · 1 trial · 1 indication

Phase 2 1
NCT05143957A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia VeraPhlebotomy Dependent Polycythemia Vera
COMPLETED50 Analytics
PHASE2COMPLETED
A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia Vera
Phlebotomy Dependent Polycythemia Vera
NCT0514395750
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Study Endpoints

Primary Endpoints

Change in the frequency of phlebotomy comparing Baseline with the last 20 weeks of the 37 week Treatment Period
Week 17 to Week 37

Secondary Endpoints

Proportion of patients achieving a reduction in the frequency of phlebotomy by ≥ 30%, ≥ 50%, ≥ 75% and ≥ 90% comparing Baseline with the last 20 weeks of the 37-week Treatment Period
Week 17 to Week 37
Change in the Myeloproliferative Neoplasm Symptom Assessment Form-Total Symptom Score (MPN-SAF-TSS) From Baseline to Week 37
Baseline up to Week 37
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Study Design & Arms

AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Sapablursen Dose Level 1EXPERIMENTALSapablursen will be administered by SC injection every 4 weeks.
Sapablursen Dose Level 2EXPERIMENTALSapablursen will be administered by SC injection every 4 weeks

Interventions

NameTypeDescription
sapablursenDRUGSapablursen will be administered by SC injection.
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Eligibility Criteria

Age Range18 Years to 90 Years
SexALL
Healthy VolunteersNo
Study Sites13

Inclusion Criteria 1. Meet modified World Health Organization (WHO) 2016 diagnostic criteria for polycythemia vera (PV) at the time of clinical diagnosis 2. Participant must be phlebotomy dependent. 3. Participants do not need to be on cytoreductive therapy and do not need to have been previously t...

Countries:United StatesAustraliaCanadaPolandUnited Kingdom
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Recent Changes (Last 90 Days)

MEDIUMAug 9, 2026NCT05143957TRIAL_REMOVED: changed
MEDIUMAug 9, 2026NCT05143957TRIAL_REMOVED: changed
MEDIUMAug 9, 2026NCT05143957TRIAL_REMOVED: changed
HIGHJul 9, 2026NCT05143957Status: ACTIVE_NOT_RECRUITING → COMPLETED
HIGHJul 9, 2026NCT05143957Status: ACTIVE_NOT_RECRUITING → COMPLETED

Frequently asked questions about sapablursen

What is sapablursen used for?

Sapablursen is an investigational small molecule being developed for the treatment of phlebotomy dependent polycythemia vera, a hematologic condition. It is currently in Phase 2 clinical development and has not been approved by regulatory authorities.

Who makes sapablursen?

Sapablursen 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 efficacy in patients with phlebotomy dependent polycythemia vera.

What phase is sapablursen in?

Sapablursen is in Phase 2 clinical development. It is an investigational drug and has not been approved by the FDA or other regulatory agencies. A Phase 2 clinical trial has been completed to evaluate its effects in patients with phlebotomy dependent polycythemia vera.

What clinical trials is sapablursen in?

Sapablursen has been studied in a Phase 2 clinical trial with the identifier NCT05143957. This trial, titled 'A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia Vera,' enrolled 50 participants and has been completed. The study was conducted in the United States, Australia, Canada, Poland, and the United Kingdom.

Is sapablursen the same as ISIS 702843 or IONIS-TMPRSS6-LRx?

Yes, sapablursen was formerly known as ISIS 702843 and IONIS-TMPRSS6-LRx. These names refer to the same investigational drug being developed by Ionis Pharmaceuticals for the treatment of phlebotomy dependent polycythemia vera.