A Phase 2, Open-Label Study of DISC-3405 in Participants With Polycythemia Vera (PV)

May 26, 2026 updated by: Disc Medicine, Inc

A Phase 2, Open-Label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of DISC-3405 in Participants With Polycythemia Vera (PV)

This open-label, multicenter, within-participant dose escalation study examining up to 2 dose levels of DISC-3405 will assess the safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of DISC-3405 in participants with polycythemia vera (PV).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
60

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85054
        • Recruiting
        • Mayo Clinic in Arizona
        • Principal Investigator:
          • Jeanne Palmer, M.D.
        • Contact:
    • California
      • Los Angeles, California, United States, 90033
        • Recruiting
        • Usc Norris Comprehensive Cancer Center
        • Contact:
        • Contact:
        • Principal Investigator:
          • Caitlin O'Neill
      • Los Angeles, California, United States, 90095
      • Newport Beach, California, United States, 92663
        • Recruiting
        • Keck Medicine of USC - Cancer Clinic- Newport Beach
        • Principal Investigator:
          • Caitlin O'Neill
        • Contact:
    • Florida
      • Jacksonville, Florida, United States, 32224
        • Recruiting
        • Mayo Clinic in Florida
        • Principal Investigator:
          • Candido Rivera, M.D.
        • Contact:
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Recruiting
        • Mayo Clinic in Minnesota
        • Principal Investigator:
          • Naseema Gangat, M.B.B.S.
        • Contact:
    • Missouri
      • St Louis, Missouri, United States, 63110
        • Recruiting
        • Siteman Cancer Center - Washington University St. Louis
        • Contact:
        • Principal Investigator:
          • Stephen Oh
    • North Carolina
      • Charlotte, North Carolina, United States, 28204
        • Recruiting
        • Atrium Health - Levine Cancer Center
        • Contact:
        • Principal Investigator:
          • Anne Wofford
        • Sub-Investigator:
          • Michael Grunwald
      • Durham, North Carolina, United States, 27705
        • Recruiting
        • Duke University
        • Contact:
        • Contact:
        • Principal Investigator:
          • Lindsay Rein, MD
      • Winston-Salem, North Carolina, United States, 27157
        • Recruiting
        • Atrium Health Wake Forest Baptist Medical Center
        • Principal Investigator:
          • Anne Wofford
        • Contact:
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Recruiting
        • Cleveland Clinic
        • Principal Investigator:
          • Aaron Gerds, MD
        • Contact:
        • Contact:
      • Columbus, Ohio, United States, 43210
        • Recruiting
        • Ohio State University
        • Contact:
        • Principal Investigator:
          • Jennifer E Vaughn, MD
    • Oregon
      • Portland, Oregon, United States, 97239
        • Recruiting
        • Oregon Health & Science University
        • Principal Investigator:
          • Joseph Shatzel
        • Contact:
        • Contact:
    • Texas
      • Houston, Texas, United States, 77030
        • Recruiting
        • MD Anderson Cancer Center
        • Principal Investigator:
          • Naveen Pemmaraju, M.D.
        • Contact:
        • Contact:
    • Washington
      • Seattle, Washington, United States, 98109
        • Recruiting
        • University of Washington - Fred Hutchinson Cancer Research Center
        • Principal Investigator:
          • Anna Halpern
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Aged 18 years or older at the time of signing the informed consent form (ICF).
  2. Meet revised 2022 World Health Organization (WHO) criteria for the diagnosis of PV.
  3. Complete blood count values at Screening of HCT <45% or HCT <48% if followed by a phlebotomy within 2 weeks, white blood cells 4000/μL to 20,000/μL (inclusive), and platelets 100,000/μL to 1,000,000/μL (inclusive).
  4. At least 3 phlebotomies in 26 weeks before Screening or at least 5 phlebotomies in 52 weeks before Screening. At least 1 phlebotomy must be within the 12 weeks prior to Screening.
  5. Participants receiving cytoreductive therapy must have been taking for at least 6 months and be on a stable PV therapy regimen for at least 2 months for hydroxyurea, interferon or ruxolitinib with no anticipated need for dose adjustments during the study, or have decreasing dose (with medical monitor approval).
  6. Participants treated with phlebotomy alone must have stopped cytoreductive therapy 6 months before Screening.
  7. Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1, or with medical monitor approval, ECOG 2.

  8. If male with female sexual partner(s) of childbearing potential, agrees to use one of the following acceptable methods of contraception during the study and for at least 120 days after the last study drug dose:

    1. Stable hormonal contraceptive (≥3 months; female partner) in conjunction with a barrier method (eg, condom or diaphragm [female partner])
    2. Intrauterine device in place for at least 3 months (female partner)
    3. Surgically sterile hysterectomy, bilateral oophorectomy, or bilateral tubal ligation (female partner) in conjunction with a barrier method (eg, condom [male or female] or diaphragm)
    4. Confirmed successful vasectomy in conjunction with a barrier method (eg, condom [male or female] or diaphragm)

  9. If female, then EITHER postmenopausal, defined as at least 12 months of natural, spontaneous amenorrhea, 6 months of spontaneous amenorrhea with serum follicle-stimulating hormone >40 mIU/mL at Screening, or at least 6 weeks following surgical menopause (bilateral oophorectomy with or without hysterectomy); surgically sterile, OR agreeable to use of highly effective contraception (listed below) on Day 1 (or earlier) and for at least 120 days after the last dose of study drug:

    1. Stable hormonal contraceptive (≥3 months) in conjunction with a barrier method (eg, condom [male or female] or diaphragm)
    2. Intrauterine device in place for at least 3 months
    3. Tubal ligation or single male partner with vasectomy in conjunction with a barrier method (eg, condom [male or female] or diaphragm)
  10. Negative pregnancy test (females of childbearing potential).
  11. Able to understand the study aims, procedures, and requirements, and provide written informed consent.
  12. Able to comply with all study procedures.

Exclusion Criteria:

  1. Clinically significant laboratory abnormalities at Screening.
  2. Participants who require phlebotomy at HCT levels <45%.
  3. Clinically significant thrombosis (eg, deep vein thrombosis or splenic vein thrombosis) within 2 months prior to study treatment.
  4. Clinically significant active or chronic bleeding, considered meaningful in consultation with the medical monitor, within 6 months prior to study treatment.
  5. Significant renal dysfunction, evidenced by estimated glomerular filtration rate of <30 mL/min/1.73 m2 at the Screening visit, as assessed locally.
  6. History of invasive malignancies within the last 5 years, except localized cured prostate cancer and cervical cancer, or other malignancies deemed acceptable by the Sponsor.
  7. Participants with in situ or stage 1 squamous cell carcinoma of the skin, in situ or stage 1 basal cell carcinoma of the skin, or in situ melanoma of the skin identified during Screening unless the cancer is adequately treated before study entry.
  8. Received busulfan, pipobroman, or phosphorus-32 within 7 months prior to Screening.
  9. Major surgery within 8 weeks before Screening or incomplete recovery from any previous surgery.
  10. A history or known allergic reaction to any investigational product excipients or history of anaphylaxis to any food or drug.
  11. History of alcohol dependence or excessive alcohol consumption, as assessed by the Investigator.
  12. Active human immunodeficiency virus (HIV), hepatitis B or C. A positive hepatitis or HIV result should be discussed between the Investigator and Sponsor prior to enrollment.
  13. Other medical or psychiatric condition or laboratory finding not specifically noted above that, in the judgment of the Investigator or Sponsor, would put the participant at unacceptable risk or otherwise preclude the participant from participating in the study.
  14. Condition or concomitant medication that would confound the ability to interpret clinical data, including a major psychiatric condition that has had an exacerbation or required hospitalization in the last 6 months.
  15. If female, pregnant or breastfeeding.
  16. Participation in any other clinical protocol or investigational study that involves administration of experimental therapy and/or therapeutic devices within 30 days (or 5 half-lives for drugs, whichever is longer) of Screening. Previous use of other hepcidin inducing agents that may impact TMPRSS6 expression are not allowed. Previous use of hepcidin mimetics may be allowed in discussion with the Sponsor.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Within-participant dose escalation
This is an open-label, multicenter, within-participant dose escalation study examining up to 2 dose levels of DISC-3405.
Drug: DISC-3405
DISC-3405 is administered subcutaneously.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Number of participants with treatment-related adverse events as assessed by CTCAE
Time Frame: Up to 365 days
Proportion of participants with treatment-emergent adverse events
Up to 365 days
Incidence of clinically abnormal vital signs
Time Frame: Up to 365 days
Proportion of participants with changes in vital signs
Up to 365 days
Incidence of clinically abnormal physical exam
Time Frame: Up to 365 days
Proportion of participants with changes in physical examinations
Up to 365 days
Incidence of clinically abnormal electrocardiograms
Time Frame: Up to 365 days
Proportion of participants with changes in electrocardiograms (ECGs)
Up to 365 days
Incidence of abnormal laboratory test results
Time Frame: Up to 365 days
Proportion of participants with changes in clinical laboratory results
Up to 365 days

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Proportion of participants achieving therapeutic response, defined as absence of phlebotomy eligibility, during the maintenance period
Time Frame: Up to 365 days
Up to 365 days
Number of phlebotomies during the maintenance and optimization periods
Time Frame: Up to 365 days
Up to 365 days
Proportion of participants achieving therapeutic response, defined as absence of phlebotomy eligibility, during the optimization period
Time Frame: Up to 365 days
Up to 365 days
Proportion of participants with HCT values <45% throughout the study
Time Frame: Up to 365 days
Up to 365 days
Area under the plasma concentration versus time curve (AUC) following the first dose
Time Frame: Up to 365 days
Up to 365 days
Peak plasma concentration (Cmax) following the first dose
Time Frame: Up to 365 days
Up to 365 days
Elimination half-life (t½el) following the first dose
Time Frame: Up to 365 days
Up to 365 days
Apparent clearance (CL/F) following the first dose
Time Frame: Up to 365 days
Up to 365 days
Maximum concentration at steady state (Cmax_ss) after repeating doses
Time Frame: Up to 365 days
Up to 365 days
Pre-dose trough concentration (Ctrough) after repeating doses
Time Frame: Up to 365 days
Up to 365 days
Change from baseline for HCT
Time Frame: Up to 365 days
Up to 365 days
Change from baseline for serum hepcidin-25
Time Frame: Up to 365 days
Up to 365 days
Change from baseline for serum iron
Time Frame: Up to 365 days
Up to 365 days
Apparent volume of distribution (Vd/F)
Time Frame: Up to 365 days
Up to 365 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Disc Medicine, Inc

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Will Savage, MD PhD, Disc Medicine

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
August 12, 2025

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
February 1, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
February 1, 2029

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
May 7, 2025

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 14, 2025

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
May 22, 2025

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 28, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 26, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
September 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • DISC-3405-201

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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