Purpose

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.

Condition

Eligibility

Eligible Ages
Between 18 Years and 90 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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

Study Design

Phase
Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Sapablursen Dose Level 1
Sapablursen will be administered by SC injection every 4 weeks.
  • Drug: sapablursen
    Sapablursen will be administered by SC injection.
    Other names:
    • ISIS 702843
    • IONIS-TMPRSS6-LRx
Experimental
Sapablursen Dose Level 2
Sapablursen will be administered by SC injection every 4 weeks
  • Drug: sapablursen
    Sapablursen will be administered by SC injection.
    Other names:
    • ISIS 702843
    • IONIS-TMPRSS6-LRx

Recruiting Locations

Medstar Georgetown University Hospital
Washington, District of Columbia 20007

More Details

Status
Recruiting
Sponsor
Ionis Pharmaceuticals, Inc.

Study Contact

Ionis Pharmaceuticals
(844) 673-0662
IonisPDPVstudy@clinicaltrialmedia.com

Detailed Description

This is a Phase 2a, multi-center, randomized, open-label study of sapablursen in up to 40 participants with PD-PV. The study consists of 4 periods: 1) Screening Period: up to 7 weeks; 2) Treatment Period: 37 weeks 3) Treatment Extension Period: 36 weeks; 4) Post-treatment Period: 13 weeks. In the Treatment Period, study drug is given by subcutaneous (under the skin) injection(s). There will be a total of 10 doses given over about 8 months. In the Treatment Extension Period, there will be a total of 9 doses given over about 8 months. Participants will be assigned to receive one of 2 Dosing Levels - a higher or a lower level, with an equal chance of being assigned to either Dosing Level. All participants will receive study drug; there is no placebo. This study was extended to allow participants to receive sapablursen for an additional 36 weeks following the initial 37-week treatment period.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.