Trial Title:
Evaluation of HU-resistance in Adult Patients With Polycythemia Vera Who Meet PV-AIM Predictors
NCT ID:
NCT05853458
Condition:
Polycythemia Vera
Conditions: Official terms:
Polycythemia Vera
Polycythemia
Hydroxyurea
Conditions: Keywords:
PV
Hydroxyurea
Resistance
Intolerance
Predictive parameters
Study type:
Interventional
Study phase:
Phase 4
Overall status:
Recruiting
Study design:
Allocation:
N/A
Intervention model:
Single Group Assignment
Primary purpose:
Treatment
Masking:
None (Open Label)
Intervention:
Intervention type:
Drug
Intervention name:
Hydroxyurea
Description:
Hydroxyurea is commercially available in Germany and will be prescribed based on clinical
judgment
Arm group label:
Hydroxyurea (HU)
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 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:
This is an open-label, prospective, single arm, Phase IV interventional study to evaluate
the HU-resistance/intolerance in PV patients who meet predictive parameters identified in
the machine learning project PV-AIM.
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 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
sites in Germany.
Criteria for eligibility:
Criteria:
Inclusion Criteria:
1. Signed informed consent must be obtained prior to participation in the study
2. Confirmed diagnosis of Polycythemia vera (according to WHO 2008, 2016, or 2022
criteria)
3. Eastern Cooperative Oncology Group (ECOG) ≤ 2
4. No previous pharmacologic cytoreductive therapy (including investigational drugs)
5. No phlebotomy in last 28 days
6. 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)
- Increasing risk of thromboembolism and bleeding:
7. 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 skin cancer; prostate cancer and breast cancer in
remission and - where necessary - ongoing hormonal therapy)
6. Inadequate liver function as assessed by Investigator
7. 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
8. 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.
9. Sexually active males unwilling to use a condom during intercourse while taking
study treatment and for at least 6 months after stopping study treatment.
10. HIV patients treated with nucleoside reverse transcriptase inhibitors like
didanosine and stavudine
Other inclusion/exclusion criteria may apply
Gender:
All
Minimum age:
18 Years
Maximum age:
99 Years
Healthy volunteers:
No
Locations:
Facility:
Name:
Novartis Investigative Site
Address:
City:
Heidelberg
Zip:
69115
Country:
Germany
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Straubing
Zip:
94315
Country:
Germany
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Langen
Zip:
63225
Country:
Germany
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Lingen
Zip:
49808
Country:
Germany
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Dortmund
Zip:
44309
Country:
Germany
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Velbert
Zip:
42551
Country:
Germany
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Luebeck
Zip:
23563
Country:
Germany
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Augsburg
Zip:
86150
Country:
Germany
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Berlin
Zip:
10407
Country:
Germany
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Bonn
Zip:
53113
Country:
Germany
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Donauwoerth
Zip:
86609
Country:
Germany
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Dresden
Zip:
01307
Country:
Germany
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Erding
Zip:
85435
Country:
Germany
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Erfurt
Zip:
99085
Country:
Germany
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Gera
Zip:
07548
Country:
Germany
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Halle
Zip:
06110
Country:
Germany
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Hannover
Zip:
30161
Country:
Germany
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Kiel
Zip:
24105
Country:
Germany
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Koeln
Zip:
50671
Country:
Germany
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Koln
Zip:
50674
Country:
Germany
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Kronach
Zip:
96317
Country:
Germany
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Merseburg
Zip:
06217
Country:
Germany
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Moers
Zip:
47441
Country:
Germany
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Mutlangen
Zip:
73557
Country:
Germany
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Naunhof
Zip:
04683
Country:
Germany
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Offenburg
Zip:
77654
Country:
Germany
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Saarbruecken
Zip:
66113
Country:
Germany
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Torgau
Zip:
04860
Country:
Germany
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Westerstede
Zip:
26655
Country:
Germany
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Wuerselen
Zip:
52146
Country:
Germany
Status:
Recruiting
Start date:
July 28, 2023
Completion date:
January 27, 2027
Lead sponsor:
Agency:
Novartis Pharmaceuticals
Agency class:
Industry
Source:
Novartis
Record processing date:
ClinicalTrials.gov processed this data on November 12, 2024
Source: ClinicalTrials.gov page:
https://clinicaltrials.gov/ct2/show/NCT05853458
