Evaluation of HU-resistance in Adult Patients With Polycythemia Vera Who Meet PV-AIM Predictors

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE4

Total Enrollment

76 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-07-28

Study Completion Date

2027-01-27

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Assessment of Algorithm-Based Hydroxyurea Dosing on Fetal Hemoglobin Response, Acute Complications, and Organ Function in People With Sickle Cell Disease

NCT02225132

Sickle Cell Disease

COMPLETED PHASE1/PHASE2

A Low-Interventional Study of an Electronic Sickle Cell Disease Patient Reported Outcomes in Sickle Cell Participants

NCT06503458

Sickle Cell Disease Sickle Cell Anemia

TERMINATED

Prospective Observational Study Of Patients With Polycythemia Vera In US Clinical Practices (REVEAL)

NCT02252159

MPN (Myeloproliferative Neoplasms)

COMPLETED

Identification of Factors Associated With Treatment Response in Patients With Polycythemia Vera, Essential Thrombocythemia, and Pre-myelofibrosis.

NCT05440838

Myeloproliferative Neoplasm

NOT_YET_RECRUITING

Predisposing Factors for Liver Diseases in Patients With Chronic Hemolytic Anemia

NCT06743854

Hemolytic Anemia, Chronic Liver Diseases

NOT_YET_RECRUITING

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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

See the medical conditions and disease areas that this research is targeting or investigating.

Polycythemia Vera

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Hydroxyurea (HU)

Participants will be treated with HU capsules, orally taken, for a maximum duration of 15 months.

Group Type EXPERIMENTAL

Hydroxyurea

Intervention Type DRUG

Hydroxyurea is commercially available in Germany and will be prescribed based on clinical judgment

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Hydroxyurea

Hydroxyurea is commercially available in Germany and will be prescribed based on clinical judgment

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

1. Signed informed consent must be obtained prior to participation in the study
2. Patients ≥18 years
3. 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)
4. Eastern Cooperative Oncology Group (ECOG) ≤ 2
5. No previous pharmacologic cytoreductive therapy (including investigational drugs)
6. No phlebotomy in last 14 days
7. 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
8. Female participants of childbearing potential should have a negative serum pregnancy test within 72 hours prior to receiving the first dose of study treatment.

Exclusion Criteria

1. Patients with post-polycythemia vera myelofibrosis (post-PV MF) or accelerated phase/ blast phase myeloproliferative neoplasm acute myeloid leukemia (AP/BP-MPN AML).
2. 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).
3. Patients with rare hereditary galactose intolerance, total lactase deficiency or glucose-galactose malabsorption in their past medical history.
4. Active uncontrolled infection that is considered by the Investigator as a reason for exclusion.
5. Active malignancies (except for carcinoma in situ; prostate cancer and breast cancer in remission and - where necessary - ongoing hormonal therapy).
6. 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.
7. 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.
8. Sexually active males unwilling to use a condom during intercourse while taking study treatment and for at least 3 months after stopping study treatment.
9. HIV patients treated with nucleoside reverse transcriptase inhibitors like didanosine and stavudine.

Minimum Eligible Age

18 Years

Maximum Eligible Age

99 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No