Polycythemia VeraPhase 4HU-F-AIMNCT05853458
Evaluation of HU-resistance in Adult Patients With Polycythemia Vera Who Meet PV-AIM Predictors
A Phase 4 study testing Hydroxyurea in people with Polycythemia Vera, run by Novartis Pharmaceuticals at 21 sites in Germany.
Trial summary
The purpose of this study is to confirm the predictive factors for hydroxyurea (HU) failure (hemoglobin (HGB) <15.5 g/dL (9.62 mmol/L) and red blood cell distribution width (RDW) ≥17%) identified by machine learning in the polycythemia vera advanced integrated model (PV-AIM) project in the real-life setting. Read more about this study
- Who is it for?
- Ages 18 to 99 years
- Conditions the study is for
- Polycythemia Vera
- What is being tested?
- Hydroxyurea
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 76 participants
- Where
- 21 sitesGermany
- Study dates
- 28 Jul 2023to 26 Feb 2026
- Phase
- Phase 4
- Are there results?
- None posted in the registry
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 to 99 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Signed informed consent must be obtained prior to participation in the study
- Patients ≥18 years
- Confirmed diagnosis of Polycythemia vera (according to WHO 2008, 2016, or 2022 criteria) (Tefferi and Vardiman 2008, Arber et al 2016, Khoury et al 2022)
- Eastern Cooperative Oncology Group (ECOG) ≤ 2
- No previous pharmacologic cytoreductive therapy (including investigational drugs)
- No phlebotomy in last 14 days
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- HU-eligible
- High-risk: age ≥ 60 years and/or prior history of thrombosis
- Low-risk: showing at least one of the defined criteria
- Signs of disease progression (myeloproliferation):
- Increase in spleen size or symptomatic splenomegaly
- Platelet increase to > 1,000,000/µl
- WBC increase to > 15,000/µl or higher
- Frequent (> 10 per year) or increasing frequency of phlebotomies
- Increasing risk of thromboembolism and bleeding:
- New thromboembolism and/or hemorrhagic complications
- Microcirculation disorders despite acetyl salicylic acid (ASA) 2x 100 mg/day
- Restricted feasibility or intolerance of phlebotomies
- Symptomatic iron deficiency
- Uncontrolled increase in hematocrit
- Severe or distressing disease-related symptoms
- Female participants of childbearing potential should have a negative serum pregnancy test within 72 hours prior to receiving the first dose of study treatment.
Who cannot join
- Patients with post-polycythemia vera myelofibrosis (post-PV MF) or accelerated phase/ blast phase myeloproliferative neoplasm acute myeloid leukemia (AP/BP-MPN AML).
- Patients with a contraindication to HU according to the SmPC (severe bone marrow depression, leukopenia (< 2.5 x 109 leukocytes/l), thrombocytopenia (< 100 x 109 platelets/L), severe anemia (< 10 g/dL HGB).
- Patients with rare hereditary galactose intolerance, total lactase deficiency or glucose-galactose malabsorption in their past medical history.
- Active uncontrolled infection that is considered by the Investigator as a reason for exclusion.
- Active malignancies (except for carcinoma in situ; prostate cancer and breast cancer in remission and - where necessary - ongoing hormonal therapy).
- Inadequate renal function as demonstrated by Modification of Diet in Renal Disease estimate glomerular filtration rate (MDRDeGFR) < 30 mL/min/1.73m2 or on dialysis.
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- Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive human chorionic gonadotrophin (hCG) laboratory test.
- Sexually active males unwilling to use a condom during intercourse while taking study treatment and for at least 3 months after stopping study treatment.
- HIV patients treated with nucleoside reverse transcriptase inhibitors like didanosine and stavudine.
- Other inclusion/exclusion criteria may apply
Trial locations and contacts
21 sites in 1 country.
21 sites in 1 country
Germany21 sites
Novartis Investigative Site
Aachen, North Rhine-Westphalia, 52064
Novartis Investigative Site
Berlin, 10407
Novartis Investigative Site
Cologne, North Rhine-Westphalia, 50674
Novartis Investigative Site
Donauwörth, 86609
Novartis Investigative Site
Dortmund, North Rhine-Westphalia, 44309
Novartis Investigative Site
Dresden, 01307
Novartis Investigative Site
Erding, 85435
Novartis Investigative Site
Gütersloh, 33332
Novartis Investigative Site
Hanover, 30161
Novartis Investigative Site
Heidelberg, Baden-Wurttemberg, 69115
Novartis Investigative Site
Kiel, 24105
Novartis Investigative Site
Kronach, Bavaria, 96317
Novartis Investigative Site
Langen, Hesse, 63225
Novartis Investigative Site
Lingen, Lower Saxony, 49808
Novartis Investigative Site
Merseburg, Saxony-Anhalt, 06217
Novartis Investigative Site
Mutlangen, 73557
Novartis Investigative Site
Naunhof, 04683
Novartis Investigative Site
Saarbrücken, Saarland, 66113
Novartis Investigative Site
Straubing, Bavaria, 94315
Novartis Investigative Site
Velbert, North Rhine-Westphalia, 42551
Novartis Investigative Site
Würselen, 52146
About this study
Official title: HU-F-AIM - A Prospective, Interventional Study to Evaluate HU-resistance in Polycythemia Vera Patients Who Meet Predictive Parameters Identified in the Machine Learning Project PV-AIM
The purpose of this study is to confirm the predictive factors for hydroxyurea (HU) failure (hemoglobin (HGB) <15.5 g/dL (9.62 mmol/L) and red blood cell distribution width (RDW) ≥17%) identified by machine learning in the polycythemia vera advanced integrated model (PV-AIM) project in the real-life setting.
Read the detailed description
The study consists of three periods: Screening period, treatment period (observation for HU-resistance/intolerance) and follow-up (FU) period.
Eligible participants will enter the treatment period (observation period for HU-resistance/intolerance) and start receiving the de novo HU treatment. The maximum treatment duration for each participant in the study will be up to 15 months.
This study will be conducted in a total of 300 adult PV patients and approximately at 30 to 40 sites in Germany. If necessary, the study will be extended to other countries to achieve the target population.
- Conditions
- Polycythemia Vera
- Being tested
- Hydroxyurea
- Keywords
- Polycythemia vera (PV), Hydroxyurea, Resistance, Intolerance, Predictive parameters
- Medical subject headings
- Polycythemia Vera
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HydroxyureaDrug
Hydroxyurea is commercially available in Germany and will be prescribed based on clinical judgment
| Study group | Role | What they receive |
|---|---|---|
| Hydroxyurea (HU) | Experimental | Participants will be treated with HU capsules, orally taken, for a maximum duration of 15 months. Receives: Drug: Hydroxyurea |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of PV patients with HU-resistance/intolerance within 6-9 months after start of de novo HU- treatment in presence of the PV-AIM HU-resistance predictors at the start of HU treatment.
Proportion of PV participants with HU resistance/intolerance within 6-9 months after start of de novo HU treatment in presence of the PV-AIM HU resistance predictors at the start of HU treatment. The proportion will be assessed calculating the rate together with the respective 95% confidence interval (CI).
Measured: From 6 to 9 months after start of de novo HU-treatment
Secondary outcomes (6)
Proportion of PV patients who meet the PV-AIM HU-resistance predictors before start of HU-treatment
Proportion of PV patients who meet the PV-AIM HU-resistance predictors before start of HU-treatment. The proportion will be assessed calculating the rate together with the respective 95% confidence interval (CI).
Measured: Baseline
Proportion of patients developing HU resistance/intolerance at any time within the maximum treatment period of 15 months
Proportion of patients developing HU resistance/intolerance at any time within the maximum treatment period of 15 months. The proportion will be assessed calculating the rate together with the respective 95% confidence interval (CI).
Measured: Up to 15 months
Proportion of "non-switchers"
For all patients who develop HU resistance/intolerance according to modified European LeukemiaNet (ELN) criteria at any time during the maximum treatment period of 15 months, the proportion of "non-switchers" (i.e., patients remaining on HU despite they meet the HU-resistance/intolerance criteria) will be assessed. The rate will be calculated together with the respective 95% confidence interval (CI).
Measured: Up to 15 months
Timepoint of therapy switch (after confirmation of HU resistance/intolerance)
For all patients who develop HU resistance/intolerance according to modified ELN criteria at any time during the maximum treatment period of 15 months, the timepoint of therapy switch (after confirmation of HU resistance/intolerance) will be assessed.
Measured: Up to 15 months
Reasons for therapy switch / non-switch
For all patients who develop HU resistance/intolerance according to modified ELN criteria at any time during the maximum treatment period of 15 months, the reasons for therapy switch or non-switch will be summarized
Measured: Up to 15 months
Therapies applied during follow-up period
For all patients who develop HU resistance/intolerance according to modified ELN criteria at any time during the maximum treatment period of 15 months, the therapies applied during the follow-up period will be summarized
Measured: Up to 3 months after treatment discontinuation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 May 2023
- Study start28 Jul 2023
- Primary completion26 Feb 2026
- Study completion26 Feb 2026
Why it stopped: Sponsor decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Polycythemia VeraStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 76 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05853458
- Sponsor's protocol ID
- CINC424BDE15
- Other IDs
- 2022-502338-20-00
- Acronym
- HU-F-AIM
- Status last verified
- Aug 2026
- First submitted
- 2 May 2023
- Last updated
- 18 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
Study at a glance
From the registry record, last updated by the sponsor on 18 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- HU-F-AIM - A Prospective, Interventional Study to Evaluate HU-resistance in Polycythemia Vera Patients Who Meet Predictive Parameters Identified in the Machine Learning Project PV-AIM
- ClinicalTrials.gov ID
- NCT05853458
- Acronym / sponsor ID
- HU-F-AIM / CINC424BDE15
- Conditions
- Polycythemia Vera
- Keywords
- Polycythemia vera (PV), Hydroxyurea, Resistance, Intolerance, Predictive parameters
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor decision
- Enrolment
- 76 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 28 Jul 2023 (actual)
- Primary completion
- 26 Feb 2026 (actual)
- Study completion
- 26 Feb 2026 (actual)
- First posted
- 10 May 2023 (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.
The purpose of this study is to confirm the predictive factors for hydroxyurea (HU) failure (hemoglobin (HGB) <15.5 g/dL (9.62 mmol/L) and red blood cell distribution width (RDW) ≥17%) identified by machine learning in the polycythemia vera advanced integrated model (PV-AIM) project in the real-life setting.
Detailed description
The study consists of three periods: Screening period, treatment period (observation for HU-resistance/intolerance) and follow-up (FU) period.
Eligible participants will enter the treatment period (observation period for HU-resistance/intolerance) and start receiving the de novo HU treatment. The maximum treatment duration for each participant in the study will be up to 15 months.
This study will be conducted in a total of 300 adult PV patients and approximately at 30 to 40 sites in Germany. If necessary, the study will be extended to other countries to achieve the target population.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Hydroxyurea (HU) | Experimental | Drug: HydroxyureaParticipants will be treated with HU capsules, orally taken, for a maximum duration of 15 months. |
Who may qualify
- Ages
- 18 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (22 listed)
- Signed informed consent must be obtained prior to participation in the study
- Patients ≥18 years
- Confirmed diagnosis of Polycythemia vera (according to WHO 2008, 2016, or 2022 criteria) (Tefferi and Vardiman 2008, Arber et al 2016, Khoury et al 2022)
- Eastern Cooperative Oncology Group (ECOG) ≤ 2
- No previous pharmacologic cytoreductive therapy (including investigational drugs)
Key exclusion criteria (10 listed)
- Patients with post-polycythemia vera myelofibrosis (post-PV MF) or accelerated phase/ blast phase myeloproliferative neoplasm acute myeloid leukemia (AP/BP-MPN AML).
- Patients with a contraindication to HU according to the SmPC (severe bone marrow depression, leukopenia (< 2.5 x 109 leukocytes/l), thrombocytopenia (< 100 x 109 platelets/L), severe anemia (< 10 g/dL HGB).
- Patients with rare hereditary galactose intolerance, total lactase deficiency or glucose-galactose malabsorption in their past medical history.
- Active uncontrolled infection that is considered by the Investigator as a reason for exclusion.
- Active malignancies (except for carcinoma in situ; prostate cancer and breast cancer in remission and - where necessary - ongoing hormonal therapy).
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 |
|---|---|---|
| HydroxyureaHydroxyurea is commercially available in Germany and will be prescribed based on clinical judgment | Drug | Hydroxyurea (HU) |
Primary outcome
Proportion of PV patients with HU-resistance/intolerance within 6-9 months after start of de novo HU- treatment in presence of the PV-AIM HU-resistance predictors at the start of HU treatment.
Time frame: From 6 to 9 months after start of de novo HU-treatment
Proportion of PV participants with HU resistance/intolerance within 6-9 months after start of de novo HU treatment in presence of the PV-AIM HU resistance predictors at the start of HU treatment. The proportion will be assessed calculating the rate together with the respective 95% confidence interval (CI).
6 secondary outcomes
Proportion of PV patients who meet the PV-AIM HU-resistance predictors before start of HU-treatment
Time frame: Baseline
Proportion of patients developing HU resistance/intolerance at any time within the maximum treatment period of 15 months
Time frame: Up to 15 months
Proportion of "non-switchers"
Time frame: Up to 15 months
Timepoint of therapy switch (after confirmation of HU resistance/intolerance)
Time frame: Up to 15 months
Reasons for therapy switch / non-switch
Time frame: Up to 15 months
Therapies applied during follow-up period
Time frame: Up to 3 months after treatment discontinuation
Results and safety
The study was stopped early (reason given: Sponsor decision). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Aug 2026.
- Individual participant dataSharing planned: Yes. Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
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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.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
21 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Hydroxyurea in people with Polycythemia Vera, run by Novartis Pharmaceuticals at 21 sites in Germany.
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 22 inclusion and 10 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: stopped early, as of the sponsor's last update on 18 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 21 sites in Germany. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Sponsor decision). Results are not available in the source record.
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?
Proportion of PV patients with HU-resistance/intolerance within 6-9 months after start of de novo HU- treatment in presence of the PV-AIM HU-resistance predictors at the start of HU treatment. (time frame: From 6 to 9 months after start of de novo HU-treatment). 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 18 Aug 2026.