Clinical trial · Interventional
A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia Vera
A Phase 2a, Randomized, Open-Label Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered to Patients With Phlebotomy Dependent Polycythemia Vera (PD-PV)
NCT05143957CI-TRIAL-00117365completedPhase 2ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
The main purpose of this study is to evaluate the efficacy of sapablursen in reducing the frequency of phlebotomy and in improving quality of life assessments in participants with polycythemia vera.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Phlebotomy Dependent Polycythemia Vera | — | UNRESOLVED | — |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| sapablursen | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (2)
- type
- EXPERIMENTAL
- label
- Sapablursen Dose Level 1
- description
- Sapablursen will be administered by SC injection every 4 weeks.
- interventionNames
- Drug: sapablursen
- type
- EXPERIMENTAL
- label
- Sapablursen Dose Level 2
- description
- Sapablursen will be administered by SC injection every 4 weeks
- interventionNames
- Drug: sapablursen
Primary outcomes (1)
- measure
- Change in the frequency of phlebotomy comparing Baseline with the last 20 weeks of the 37 week Treatment Period
- timeFrame
- Week 17 to Week 37
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
- Maximum age
- 90 Years
Show eligibility criteria text
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 treated with cytoreductive therapy. If the patient was previously on cytoreductive therapy it must have been discontinued at least 3 months prior to Screening, with all associated AEs resolved. If the patient is currently on cytoreductive therapy they must be on a stable dose of hydroxyurea, recombinant or PEGylated interferon, or ruxolitinib for at least 3 months prior to Screening. Exclusion Criteria 1. Meets criteria for post-polycythemia vera myelofibrosis (PPV-MF) as defined by the International Working Group- Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) 2. Moderate to severe splenic pain or spleen-related organ obstruction 3. Active or chronic bleeding within 1 month of Screening, significant concurrent/recent coagulopathy, history of immune thrombocytopenic purpura (ITP) 4. Known primary or secondary immunodeficiency 5. Active infection with human immunodeficiency virus (HIV), hepatitis C, or hepatitis B. 6. Active infection requiring systemic antiviral or antimicrobial therapy or active novel coronavirus disease (Covid-19) infection 7. Malignancy within 5 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix or non-metastatic prostate cancer that has been successfully treated 8. Surgery requiring general anesthesia within 1 month prior to Screening
References
Publications (1)
- DERIVEDPalmer JM, How J, Bose P, Vachhani P, Mead AJ, Sillar J, Gerds AT, Fleischman AG, O'Neill CM, Baker RI, Hoffman R, Jung S, Ruckle JL, Yousefi K, Bhanot S, Barrett TD, Mesa RA. Sapablursen for Erythrocytosis Control in Patients with Polycythemia Vera. Blood. 2026 Aug 18:blood.2026033792. doi: 10.1182/blood.2026033792. Online ahead of print. PMID 42611791