Sapablursen for Polycythemia Vera

Recruiting at 46 trial locations

Overseen By Ionis Pharmaceuticals

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Ionis Pharmaceuticals, Inc.

Must be taking: Hydroxyurea, Interferon, Ruxolitinib

Disqualifiers: Myelofibrosis, Splenic pain, Bleeding, Immunodeficiency, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 18

Duration: 37 weeks

50 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial tests sapablursen, an injectable medication, on patients with polycythemia vera to see if it can reduce the need for regular blood removal and improve their quality of life.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop all current medications, but if you are on cytoreductive therapy, you must be on a stable dose of hydroxyurea, recombinant or PEGylated interferon, or ruxolitinib for at least 3 months before joining. If you were previously on cytoreductive therapy, it must have been stopped at least 3 months before the trial.

Eligibility Criteria

This trial is for individuals with polycythemia vera who depend on regular blood removal (phlebotomy) to manage their condition. They must meet specific diagnostic criteria and either have not been on cytoreductive therapy or be on a stable dose for at least 3 months. People can't join if they have severe spleen issues, certain other diseases like HIV or hepatitis, recent major surgery, cancer within the last 5 years (with some exceptions), or bleeding/coagulation disorders.

Inclusion Criteria

I haven't had or don't currently need treatment to reduce my blood cell counts. If I was treated, it ended 3 months ago.

I stopped my cytoreductive therapy 3 months ago or have been on a stable dose for the same duration.

Participant must be phlebotomy dependent

Exclusion Criteria

I have been diagnosed with PPV-MF according to IWG-MRT standards.

I haven't had any serious bleeding or blood clotting issues in the last month.

I haven't had cancer in the last 5 years, except for certain skin cancers, cervical carcinoma in situ, or treated non-metastatic prostate cancer.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

up to 7 weeks

Treatment

Participants receive sapablursen by subcutaneous injection every 4 weeks for 37 weeks

37 weeks

9 visits (in-person)

Treatment Extension

Participants continue to receive sapablursen by subcutaneous injection every 4 weeks for an additional 36 weeks

36 weeks

9 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

13 weeks

Treatment Details

Interventions

• Sapablursen

Trial Overview The study tests sapablursen's effectiveness in reducing the need for phlebotomies and improving life quality in patients with polycythemia vera. It aims to see if this treatment can help manage red blood cell levels better than current methods.

Participant Groups

Group I: Sapablursen Dose Level 2 Experimental Treatment 1 Intervention

Sapablursen will be administered by SC injection every 4 weeks

Group II: Sapablursen Dose Level 1 Experimental Treatment 1 Intervention

Sapablursen will be administered by SC injection every 4 weeks.

Who Is Running the Clinical Trial?

Ionis Pharmaceuticals, Inc.

Lead Sponsor

Trials

151

Recruited

27,800+

Dr. Brett P. Monia

Ionis Pharmaceuticals, Inc.

Chief Executive Officer since 2020

PhD in Pharmacology from the University of Pennsylvania, BSc in Molecular Biology and Analytical Chemistry from Stockton State College

Dr. Eric Bastings

Ionis Pharmaceuticals, Inc.

Chief Medical Officer

MD