SAPABLURSEN for Polycythemia vera|Phlebotomy-dependent polycythemia vera

Inclusion criteria

• {"criterion_text":"- Meet modified World Health Organization (WHO) 2016 diagnostic criteria for polycythemia vera (PV) at the time of clinical diagnosis\n- Participant must be phlebotomy dependent\n- Patients 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 treated with the cytoreductive therapy it must have been discontinued at least 3 months prior to Screening. If patients are currently on cytoreductive therapy they must be on a stable dose for at least 3 months prior to Screening."}

Exclusion criteria

• {"criterion_text":"- Meets criteria for post-polycythemia vera myelofibrosis (PPV-MF) as defined by the International Working Group- Myeloproliferative Neoplasms Research and Treatment (IWG-MRT)\n- Moderate to severe splenic pain or spleen-related organ obstruction\n- Active or chronic bleeding within 1 month of Screening, significant concurrent/recent coagulopathy, history of immune thrombocytopenic purpura (ITP)\n- Known primary or secondary immunodeficiency\n- Active infection with human immunodeficiency virus (HIV), hepatitis C, or hepatitis B.\n- Active infection requiring systemic antiviral or antimicrobial therapy or active novel coronavirus disease (Covid-19) infection\n- Malignancy within 5 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix or nonmetastatic prostate cancer that has been successfully treated\n- Surgery requiring general anesthesia within 1 month prior to Screening"}

Primary endpoints

• {"endpoint_text":"- Reduction in the frequency of phlebotomy comparing Baseline with the last 20 weeks of the 37-week Treatment Period","definition_or_measurement_approach":"Compare frequency of phlebotomy at Baseline with frequency during the last 20 weeks of the 37-week Treatment Period."}

Secondary endpoints

• {"endpoint_text":"- 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 [ Time Frame: Week 17 to Week 37 ]","definition_or_measurement_approach":"Proportion of patients meeting threshold reductions (≥30%, ≥50%, ≥75%, ≥90%) comparing Baseline to last 20 weeks of Treatment Period; time frame Week 17 to Week 37."}
• {"endpoint_text":"- Change in the Myeloproliferative Neoplasm Symptom Assessment Form-Total Symptom Score (MPN-SAF-TSS) From Baseline to Week 37 [ Time Frame: Baseline up to Week 37 ]","definition_or_measurement_approach":"Change from Baseline in MPN-SAF Total Symptom Score measured through Week 37."}
• {"endpoint_text":"- Safety Endpoint: AEs, vital signs, clinical laboratory tests (serum chemistry, hematology, urinalysis, coagulation panel, thyroid panel [TSH, T3, T4], electrocardiogram [ECG]","definition_or_measurement_approach":"Safety assessments including adverse events, vital signs, specified laboratory tests and ECGs collected per study schedule."}
• {"endpoint_text":"- Exploratory Endpoints: Proportion of patients achieving Hct control (i.e. Hct<45%) without receiving phlebotomy throughout the last 20 weeks of the 37-week Treatment Period","definition_or_measurement_approach":"Proportion of patients with hematocrit <45% without phlebotomy during the last 20 weeks of the 37-week Treatment Period."}
• {"endpoint_text":"- Exploratory Endpoints: Reduction in the frequency of phlebotomy comparing Baseline to the last 36 Weeks of the Treatment Extension Period","definition_or_measurement_approach":"Compare frequency of phlebotomy at Baseline to the last 36 weeks of the Treatment Extension Period."}
• {"endpoint_text":"- Exploratory Endpoints: Proportion of patients achieving Hct control (i.e., Hct<45%) without receiving phlebotomy from Week 37 to 73 of the Treatment Extension Period","definition_or_measurement_approach":"Proportion of patients with hematocrit <45% without phlebotomy between Week 37 and Week 73 of the Treatment Extension Period."}
• {"endpoint_text":"- PK Endpoint: A PK profile including pre-dose, 1,2,4, and optional 6-hour samples will be collected at Day 1. A PK profile will also be collected at Week 25 and will include: pre-dose, 1-, 2-, and 3-hour samples. Trough levels will be evaluated at all clinic visits at will not be required for hom health care visits.","definition_or_measurement_approach":"Pharmacokinetic sampling at specified timepoints: Day 1 (pre-dose, 1, 2, 4, optional 6-hour), Week 25 (pre-dose, 1, 2, 3-hour), and trough levels at clinic visits."}

Poland

Earliest CTIS Part Ii Submission Date

22-05-2024

Latest Decision Or Authorization Date

07-04-2026

Processing Time Days

685

Number Of Sites

4

Number Of Participants

10

Primary sponsor

Full Name

Ionis Pharmaceuticals Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Icon Clinical Research Limited

Responsibilities

Responsibilities include SUSAR reporting and various clinical trial operational roles (multiple sponsorDuties codes present in CTIS record).

Third parties

• {"country":"United States","full_name":"Yprime LLC","duties_or_roles":"Electronic clinical outcome assessment (eCOA) platform","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"Patient Reimbursement","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Ireland","full_name":"Accellacare Limited","duties_or_roles":"Home healthcare services","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Icon Laboratory Services Inc.","duties_or_roles":"HIV test","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Sitero LLC","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"Ireland","full_name":"Millmount Healthcare Limited","duties_or_roles":"QP Declaration","organisation_type":"Pharmaceutical company"}
• {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"Multiple operational CRO roles including SUSAR reporting and other clinical trial operational functions (codes and roles listed in CTIS record).","organisation_type":"Pharmaceutical company"}