Polycythemia Vera (PV)Phase 2NCT06985147
A Phase 2, Open-Label Study of DISC-3405 in Participants With Polycythemia Vera (PV)
A Phase 2 study testing DISC-3405 in people with Polycythemia Vera (PV), run by Disc Medicine, Inc at 15 sites in United States.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Polycythemia Vera (PV)
- What is being tested?
- DISC-3405
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 15 sitesUnited States
- Study dates
- 12 Aug 2025to Feb 2029
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Aged 18 years or older at the time of signing the informed consent form (ICF).
- Meet revised 2022 World Health Organization (WHO) criteria for the diagnosis of PV.
- 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).
- 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.
- 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).
- Participants treated with phlebotomy alone must have stopped cytoreductive therapy 6 months before Screening.
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- Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1, or with medical monitor approval, ECOG 2.
- 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:
- Stable hormonal contraceptive (≥3 months; female partner) in conjunction with a barrier method (eg, condom or diaphragm [female partner])
- Intrauterine device in place for at least 3 months (female partner)
- Surgically sterile hysterectomy, bilateral oophorectomy, or bilateral tubal ligation (female partner) in conjunction with a barrier method (eg, condom [male or female] or diaphragm)
- Confirmed successful vasectomy in conjunction with a barrier method (eg, condom [male or female] or diaphragm)
- 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:
- Stable hormonal contraceptive (≥3 months) in conjunction with a barrier method (eg, condom [male or female] or diaphragm)
- Intrauterine device in place for at least 3 months
- Tubal ligation or single male partner with vasectomy in conjunction with a barrier method (eg, condom [male or female] or diaphragm)
- Negative pregnancy test (females of childbearing potential).
- Able to understand the study aims, procedures, and requirements, and provide written informed consent.
- Able to comply with all study procedures.
Who cannot join
- Clinically significant laboratory abnormalities at Screening.
- Participants who require phlebotomy at HCT levels <45%.
- Clinically significant thrombosis (eg, deep vein thrombosis or splenic vein thrombosis) within 2 months prior to study treatment.
- Clinically significant active or chronic bleeding, considered meaningful in consultation with the medical monitor, within 6 months prior to study treatment.
- Significant renal dysfunction, evidenced by estimated glomerular filtration rate of <30 mL/min/1.73 m2 at the Screening visit, as assessed locally.
- 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.
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- 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.
- Received busulfan, pipobroman, or phosphorus-32 within 7 months prior to Screening.
- Major surgery within 8 weeks before Screening or incomplete recovery from any previous surgery.
- A history or known allergic reaction to any investigational product excipients or history of anaphylaxis to any food or drug.
- History of alcohol dependence or excessive alcohol consumption, as assessed by the Investigator.
- 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.
- 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.
- 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.
- If female, pregnant or breastfeeding.
- 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.
Trial locations and contacts
15 sites in 1 country.
15 sites in 1 country
United States15 sites
Atrium Health - Levine Cancer Center
Charlotte, North Carolina, 28204
Cleveland Clinic
Cleveland, Ohio, 44195
Ohio State University
Columbus, Ohio, 43210
Duke University
Durham, North Carolina, 27705
MD Anderson Cancer Center
Houston, Texas, 77030
Mayo Clinic in Florida
Jacksonville, Florida, 32224
USC Norris Comprehensive Cancer Center
Los Angeles, California, 90033
UCLA Health
Los Angeles, California, 90095
Keck Medicine of USC - Cancer Clinic- Newport Beach
Newport Beach, California, 92663
Mayo Clinic in Arizona
Phoenix, Arizona, 85054
Oregon Health & Science University
Portland, Oregon, 97239
Mayo Clinic in Minnesota
Rochester, Minnesota, 55905
University of Washington - Fred Hutchinson Cancer Research Center
Seattle, Washington, 98109
Siteman Cancer Center - Washington University St. Louis
St Louis, Missouri, 63110
Atrium Health Wake Forest Baptist Medical Center
Winston-Salem, North Carolina, 27157
About this study
Official title: 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).
- Conditions
- Polycythemia Vera (PV)
- Being tested
- DISC-3405
- Medical subject headings
- Polycythemia Vera
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DISC-3405Drug
DISC-3405 is administered subcutaneously.
| Study group | Role | What they receive |
|---|---|---|
| Within-participant dose escalation | Experimental | This is an open-label, multicenter, within-participant dose escalation study examining up to 2 dose levels of DISC-3405. Receives: Drug: DISC-3405 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with treatment-related adverse events as assessed by CTCAE
Proportion of participants with treatment-emergent adverse events
Measured: Up to 365 days
Incidence of clinically abnormal vital signs
Proportion of participants with changes in vital signs
Measured: Up to 365 days
Incidence of clinically abnormal physical exam
Proportion of participants with changes in physical examinations
Measured: Up to 365 days
Incidence of clinically abnormal electrocardiograms
Proportion of participants with changes in electrocardiograms (ECGs)
Measured: Up to 365 days
Incidence of abnormal laboratory test results
Proportion of participants with changes in clinical laboratory results
Measured: Up to 365 days
Secondary outcomes (14)
Proportion of participants achieving therapeutic response, defined as absence of phlebotomy eligibility, during the maintenance period
Measured: Up to 365 days
Number of phlebotomies during the maintenance and optimization periods
Measured: Up to 365 days
Proportion of participants achieving therapeutic response, defined as absence of phlebotomy eligibility, during the optimization period
Measured: Up to 365 days
Proportion of participants with HCT values <45% throughout the study
Measured: Up to 365 days
Area under the plasma concentration versus time curve (AUC) following the first dose
Measured: Up to 365 days
Peak plasma concentration (Cmax) following the first dose
Measured: Up to 365 days
Elimination half-life (t½el) following the first dose
Measured: Up to 365 days
Apparent clearance (CL/F) following the first dose
Measured: Up to 365 days
Maximum concentration at steady state (Cmax_ss) after repeating doses
Measured: Up to 365 days
Pre-dose trough concentration (Ctrough) after repeating doses
Measured: Up to 365 days
Change from baseline for HCT
Measured: Up to 365 days
Change from baseline for serum hepcidin-25
Measured: Up to 365 days
Change from baseline for serum iron
Measured: Up to 365 days
Apparent volume of distribution (Vd/F)
Measured: Up to 365 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 May 2025
- Study start12 Aug 2025
- Primary completionFeb 2027 (estimated)
- Study completionFeb 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Polycythemia Vera (PV)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Model description
- Up to 3 cohorts of participants are planned: * Cohort A will include 20 participants. The first 4 participants enrolled will be administered a starting dose level of Dose A DISC-3405 subcutaneous which will be followed by within-participant dose escalation. Following Safety Review Committee review of the data, an additional 16 participants will be enrolled and administered a starting dose level of Dose B DISC-3405 subcutaneous. * Cohort B will enroll up to 20 participants administered a dose level of Dose B DISC-3405 subcutaneous once every 4 weeks. * Cohort C is optional and will enroll up to 20 participants administered a dose level up to that previously studied of DISC-3405 subcutaneous once every 4 weeks.
- Masking
- None (open label)
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Disc Medicine, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Will Savage, MD PhD, Study director, Disc Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06985147
- Sponsor's protocol ID
- DISC-3405-201
- Status last verified
- Sep 2025
- First submitted
- 7 May 2025
- Last updated
- 31 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Open-Label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of DISC-3405 in Participants With Polycythemia Vera (PV)
- ClinicalTrials.gov ID
- NCT06985147
- Acronym / sponsor ID
- DISC-3405-201
- Conditions
- Polycythemia Vera (PV)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Disc Medicine, IncIndustry (pharma, biotech, devices)
- Study start
- 12 Aug 2025 (actual)
- Primary completion
- Feb 2027 (estimated)
- Study completion
- Feb 2029 (estimated)
- First posted
- 22 May 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Up to 3 cohorts of participants are planned: * Cohort A will include 20 participants. The first 4 participants enrolled will be administered a starting dose level of Dose A DISC-3405 subcutaneous which will be followed by within-participant dose escalation. Following Safety Review Committee review of the data, an additional 16 participants will be enrolled and administered a starting dose level of Dose B DISC-3405 subcutaneous. * Cohort B will enroll up to 20 participants administered a dose level of Dose B DISC-3405 subcutaneous once every 4 weeks. * Cohort C is optional and will enroll up to 20 participants administered a dose level up to that previously studied of DISC-3405 subcutaneous once every 4 weeks.
| Arm | Type | Receives |
|---|---|---|
| Within-participant dose escalation | Experimental | Drug: DISC-3405This is an open-label, multicenter, within-participant dose escalation study examining up to 2 dose levels of DISC-3405. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (19 listed)
- Aged 18 years or older at the time of signing the informed consent form (ICF).
- Meet revised 2022 World Health Organization (WHO) criteria for the diagnosis of PV.
- 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).
- 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.
- 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).
Key exclusion criteria (16 listed)
- Clinically significant laboratory abnormalities at Screening.
- Participants who require phlebotomy at HCT levels <45%.
- Clinically significant thrombosis (eg, deep vein thrombosis or splenic vein thrombosis) within 2 months prior to study treatment.
- Clinically significant active or chronic bleeding, considered meaningful in consultation with the medical monitor, within 6 months prior to study treatment.
- Significant renal dysfunction, evidenced by estimated glomerular filtration rate of <30 mL/min/1.73 m2 at the Screening visit, as assessed locally.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DISC-3405DISC-3405 is administered subcutaneously. | Drug | Within-participant dose escalation |
Primary outcome
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
Incidence of clinically abnormal vital signs
Time frame: Up to 365 days
Proportion of participants with changes in vital signs
Incidence of clinically abnormal physical exam
Time frame: Up to 365 days
Proportion of participants with changes in physical examinations
Incidence of clinically abnormal electrocardiograms
Time frame: Up to 365 days
Proportion of participants with changes in electrocardiograms (ECGs)
Incidence of abnormal laboratory test results
Time frame: Up to 365 days
Proportion of participants with changes in clinical laboratory results
14 secondary outcomes
Proportion of participants achieving therapeutic response, defined as absence of phlebotomy eligibility, during the maintenance period
Time frame: Up to 365 days
Number of phlebotomies during the maintenance and optimization periods
Time frame: 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
Proportion of participants with HCT values <45% throughout the study
Time frame: Up to 365 days
Area under the plasma concentration versus time curve (AUC) following the first dose
Time frame: Up to 365 days
Peak plasma concentration (Cmax) following the first dose
Time frame: Up to 365 days
Elimination half-life (t½el) following the first dose
Time frame: Up to 365 days
Apparent clearance (CL/F) following the first dose
Time frame: Up to 365 days
Maximum concentration at steady state (Cmax_ss) after repeating doses
Time frame: Up to 365 days
Pre-dose trough concentration (Ctrough) after repeating doses
Time frame: Up to 365 days
Change from baseline for HCT
Time frame: Up to 365 days
Change from baseline for serum hepcidin-25
Time frame: Up to 365 days
Change from baseline for serum iron
Time frame: Up to 365 days
Apparent volume of distribution (Vd/F)
Time frame: Up to 365 days
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Jul 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Will Savage, MD PhD study director
Disc Medicine
15 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing DISC-3405 in people with Polycythemia Vera (PV), run by Disc Medicine, Inc at 15 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 19 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 31 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with treatment-related adverse events as assessed by CTCAE (time frame: Up to 365 days). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Jul 2026.