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A Study to Evaluate IONIS-TMPRSS6-LRx (ISIS 702843) in Patients With Polycythemia Vera

Sponsor
Ionis Pharmaceuticals, Inc. (Industry)
Overall Status
Recruiting
CT.gov ID
NCT05143957
Collaborator
(none)
40
Enrollment
9
Locations
2
Arms
29
Anticipated Duration (Months)
4.4
Patients Per Site
0.2
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The main purpose of this study is to evaluate the efficacy of IONIS-TMPRSS6-LRx in reducing the frequency of phlebotomy and in improving quality of life assessments in participants with polycythemia vera.

Condition or Disease Intervention/Treatment Phase
Phase 2

Detailed Description

This is a Phase 2a, multi-center, randomized, open-label study of IONIS-TMPRSS6-LRx in up to 40 participants with PD-PV. The study consists of 3 periods: 1) Screening Period: up to 7 weeks; 2) Treatment Period: 33 weeks + a 4-week post-last dose visit; 3) 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.

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.

Study Design

Study Type:
Interventional
Anticipated Enrollment :
40 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
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)
Actual Study Start Date :
Dec 30, 2021
Anticipated Primary Completion Date :
Mar 1, 2024
Anticipated Study Completion Date :
Jun 1, 2024

Arms and Interventions

Arm Intervention/Treatment
Experimental: IONIS-TMPRSS6-LRx Dose Level 1

IONIS-TMPRSS6-LRx will be administered by SC injection every 4 weeks.

Drug: IONIS-TMPRSS6-LRx
IONIS-TMPRSS6-LRx will be administered by SC injection.
Other Names:
  • ISIS 702843

    		•
    Experimental: IONIS-TMPRSS6-LRx Dose Level 2

    IONIS-TMPRSS6-LRx will be administered by SC injection every 4 weeks

    Drug: IONIS-TMPRSS6-LRx
    IONIS-TMPRSS6-LRx will be administered by SC injection.
    Other Names:
  • ISIS 702843

    		•

    Outcome Measures

    Primary Outcome Measures

    1. 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 Outcome Measures

    1. 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]

    2. Change in the Myeloproliferative Neoplasm Symptom Assessment Form-Total Symptom Score (MPN-SAF-TSS) From Baseline to Week 37 [Baseline up to Week 37]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years to 80 Years
    Sexes Eligible for Study:
    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. If the participant is currently on cytoreductive therapy or has been previously treated with cytoreductive therapy, the participant's cytoreductive therapy must either be discontinued at least 3 months prior to Screening, OR participant must be on a stable dose 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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 City of Hope National Medical Center Duarte California United States 91010
    2 University of California Irvine Orange California United States 92868
    3 Innovative Clinical Research Institute Whittier California United States 90603
    4 Washington University School of Medicine Saint Louis Missouri United States 63110
    5 Herbert Irving Comprehensive Cancer Center New York New York United States 10032
    6 University of Texas MD Anderson Cancer Center Houston Texas United States 77030
    7 Mays Cancer Center San Antonio Texas United States 78229
    8 McGill University Health Centre Montreal Quebec Canada H4A 3J1
    9 Sandwell and West Birmingham Hospitals NHS Trust West Bromwich United Kingdom B71 4HJ

    Sponsors and Collaborators

    • Ionis Pharmaceuticals, Inc.

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Ionis Pharmaceuticals, Inc.
    ClinicalTrials.gov Identifier:
    NCT05143957
    Other Study ID Numbers:
    • ISIS 702843-CS4
    • 2021-003704-40
    First Posted:
    Dec 3, 2021
    Last Update Posted:
    Aug 25, 2022
    Last Verified:
    Aug 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Ionis Pharmaceuticals, Inc.
    Polycythemia Vera
    Additional relevant MeSH terms:
    Polycythemia Vera
    Polycythemia

    Study Results

    No Results Posted as of Aug 25, 2022