Polycythemia Vera (PV)NCT05421104
Ruxolitinib for Polycythemia Vera in Patients Resistant to or Intolerant of Hydroxyurea.
An observational study of Polycythemia Vera (PV), run by Novartis Pharmaceuticals at 1 site in United States.
Trial summary
This was an analytical and descriptive, non-interventional, retrospective cohort study of PV patients aged ≥ 18 years in the US using a secondary data source, Optum EHR database. Read more about this study
- Who is it for?
- Ages 18 to 99 years
- Conditions the study is for
- Polycythemia Vera (PV)
- What is being tested?
- Ruxolitinib
- How many people
- 1,576 participants
- Where
- 1 siteUnited States
- Study dates
- 27 Nov 2020to 29 Jun 2021
- Phase
- Observational
- 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
Study population: PV patients who were resistant to or intolerant of HU in the Optum EHR database between April 2007 and June 2019
Who may be able to join
- Included patients:
- With at least one International Classification of Diseases, 9th Revision, Clinical Modification/International Classification of Diseases,10th Revision, Clinical Modification code for PV in the identification period (01-Apr-2007 until 30-Jun-2019) that had non-missing sex and year of birth data and who were treated as part of the Integrated Delivery Network
- That were ≥ 18 years old at PV diagnosis
- With ≥ 2 prescriptions of HU
- That were classified as resistant to or intolerant of HU after a minimum of 3 months HU treatment (index date), defined as:
- HCT ≥ 45% with phlebotomy (last phlebotomy within last 3 months) or Platelet count > 400 x 109/L and presence of palpable splenomegaly (palpable spleen up to 3 months after platelet count).
- To identify patients in the RUX group:
- With ≥ 2 prescriptions of RUX in the post-index period.
Who cannot join
- Excluded patients:
- With a MF or AML diagnosis prior to a PV diagnosis.
Trial locations and contacts
1 site in 1 country.
United States1 site
Novartis Investigative Site
East Hanover, New Jersey, 07936-1080
About this study
Official title: Ruxolitinib for the Treatment of Polycythemia Vera in Patients Who Are Resistant to or Intolerant of Hydroxyurea: a Retrospective Non-interventional Study Using the US Optum Electronic Health Record Data Source.
This was an analytical and descriptive, non-interventional, retrospective cohort study of PV patients aged ≥ 18 years in the US using a secondary data source, Optum EHR database.
Read the detailed description
The Optum EHR database was current up to 30-Jun-2020.
Identification period: From 01-Apr-2007 to 30-Jun-2019
Study period: From 01-Jan-2007 to 30-Jun-2020
Index date:
First evidence of resistance to or intolerance of HU treatment in patients with PV according to modified European Leukemia Net (ELN) criteria and defined as:
- HCT ≥ 45% with phlebotomy (last phlebotomy within last 3 months) Or
- Platelet count > 400 x 109/L and presence of palpable splenomegaly (palpable spleen up to 3 months after platelet count)
Pre-index period:
Patients had a minimum of 3 months pre-index data available. Pre-index data availability was determined using the reported 'first month active' field.
Post-index period:
There was no minimum post-index period required. Each patient had a 'first month active' and 'last month active' reported within the database. As the 'last month active' was based on any activity in the database, including encounters such as letters and emails which occurred several months after the 'death_date' of the patient, using the 'last month active' can overestimate the follow-up for a given patient. For this reason, the end of follow-up for each patient was defined as the date of the last activity within the diagnosis, observations, prescriptions, laboratories, procedures tables or discharge date from the last visit within the visit table (whichever of these activities occurs latest). This underestimated the follow-up for some patients where they were not actively using healthcare resources.
- Conditions
- Polycythemia Vera (PV)
- Being tested
- Ruxolitinib
- Keywords
- Polycythemia vera,, Ruxolitinib,, JAK protein inhibitor,, Hydroxyurea,, Thrombotic events
- Medical subject headings
- Polycythemia Vera
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RuxolitinibOther
Also called Jakavi
PV patients who were resistant to or intolerant of HU (as defined on the index date) and switched to RUX in the post-index period.
| Study group | Role | What they receive |
|---|---|---|
| Ruxolitinib (RUX) | PV patients who were resistant to or intolerant of HU (as defined on the index date) and switched to RUX in the post-index period. Receives: Other: Ruxolitinib | |
| Best available therapy (BAT) | PV patients who were resistant to or intolerant of Hydroxyurea (HU) (as defined on the index date) and continued HU treatment or switched to other available therapies other than RUX in the post-index period. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Thromboembolic events between the RUX and BAT group
Thromboembolic events in overall Polycythemia Vera cohort and in the BAT and RUX groups were reported. A TE was defined using International Classification of Diseases 9th Revision (ICD-9- CM) and International Classification of Diseases 10th Revision (ICD-10-CM) codes previously curated as restrictive (RESPONSE RCT) and extensive (GEMFIN) definitions of TE's within the Diagnosis table in Optum EHR database.
Measured: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
Number of Thromboembolic events between the high and low risk subgroups of BAT group
Within the BAT group, high risk (≥ 1 TE on average per year ) and low risk (< 1 TE on average per year) subgroups were identified based on the frequency of TEs and characterized according to patient sociodemographics, comorbidities, symptoms, clinical, and medication variables.
Measured: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
Secondary outcomes (10)
Incidence rate of thromboembolic event
Difference in the incidence rate of TEs in PV patients resistant to or intolerant of HU treated with BAT compared to those treated with RUX were reported. A TE was defined using International Classification of Diseases 9th Revision (ICD-9- CM) and International Classification of Diseases 10th Revision (ICD-10-CM) codes previously curated as restrictive (RESPONSE RCT) and extensive (GEMFIN) definitions of TE's within the Diagnosis table in Optum EHR database.
Measured: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
Time to first thromboembolic event
Time to first TE in PV patients resistant to or intolerant of HU treated with BAT compared to those treated with RUX was reported. A TE was defined using International Classification of Diseases 9th Revision (ICD-9- CM) and International Classification of Diseases 10th Revision (ICD-10-CM) codes previously curated as restrictive (RESPONSE RCT) and extensive (GEMFIN) definitions of TE's within the Diagnosis table in Optum EHR database.
Measured: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
Incidence rate of phlebotomy procedures
Difference in the incidence rate of phlebotomies in PV patients resistant to or intolerant of HU treated with BAT compared to those treated with RUX was reported.
Phlebotomies were defined using Current Procedural Terminology, Fourth Edition (CPT4) codes within the Procedure table in Optum EHR.
Measured: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
Time to first phlebotomy procedure
Time to first phlebotomy in PV patients resistant to or intolerant of HU treated with BAT compared to those treated with RUX was reported. Phlebotomies were defined using Current Procedural Terminology, Fourth Edition (CPT4) codes within the Procedure table in Optum EHR.
Measured: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
Incidence rate of neoplasm transformations
Difference in the incidence rate of neoplasm transformations in PV patients resistant to or intolerant of HU treated with BAT compared to those treated with RUX was reported.
A neoplasm transformation was defined as:
- PV to MF (Myelofibrosis)
- MF to AML (Acute Myeloid Leukemia)
- PV to AML Neoplasm transformations were detected using ICD-9-CM and ICD-10-CM codes within the Diagnosis table in Optum EHR.
Measured: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
Time to first neoplasm transformation
Time to first neoplasm transformation in PV patients resistant to or intolerant of HU treated with BAT compared to those treated with RUX was reported.
A neoplasm transformation was defined as:
- PV to MF
- MF to AML
- PV to AML Neoplasm transformations were detected using ICD-9-CM and ICD-10-CM codes within the Diagnosis table in Optum EHR.
Measured: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
Treatment patterns: Proportion of patients using different PV-related treatments
Differences in treatment patterns in PV patients resistant to or intolerant of HU treated with BAT compared to those treated with RUX was reported.
BAT comprised of multiple therapies for PV including HU, IFN, pegylated IFN (PEG-IFN) and others. These therapies were reported as subcategories under BAT.
Measured: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
Healthcare resource utilization (HCRU): Number of inpatient hospitalizations
Hospitalization was defined as an inpatient stay with a valid visit_ID within the Visit table in Optum EHR. Inpatient hospitalizations were reported as allcause and as PV-specific respectively
Measured: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
Healthcare resource utilization (HCRU): Number of outpatient visits
Visits with the following visit type: "observation patient" with a valid visit_ID was included as an outpatient visit. Outpatient visits that resulted in an inpatient hospitalization were not included. Outpatient visits were reported as all-cause and as PV-specific respectively.
Measured: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
Healthcare resource utilization (HCRU): Number of emergency room visits
Visits with the following visit type: "emergency patient" with a valid visit_ID was included as an emergency room visit. Emergency room visits that resulted in an inpatient hospitalization were not included. Emergency room visits were reported as all-cause and as PV-specific respectively.
Measured: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jun 2022
- Study start27 Nov 2020
- Primary completion29 Jun 2021
- Study completion29 Jun 2021
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.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 1,576 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
- NCT05421104
- Sponsor's protocol ID
- CINC424B3001
- Status last verified
- Jun 2022
- First submitted
- 13 Jun 2022
- Last updated
- 5 Jul 2022
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Ruxolitinib for the Treatment of Polycythemia Vera in Patients Who Are Resistant to or Intolerant of Hydroxyurea: a Retrospective Non-interventional Study Using the US Optum Electronic Health Record Data Source.
- ClinicalTrials.gov ID
- NCT05421104
- Acronym / sponsor ID
- CINC424B3001
- Conditions
- Polycythemia Vera (PV)
- Keywords
- Polycythemia vera,, Ruxolitinib,, JAK protein inhibitor,, Hydroxyurea,, Thrombotic events
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 1,576 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 27 Nov 2020 (actual)
- Primary completion
- 29 Jun 2021 (actual)
- Study completion
- 29 Jun 2021 (actual)
- First posted
- 16 Jun 2022 (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 was an analytical and descriptive, non-interventional, retrospective cohort study of PV patients aged ≥ 18 years in the US using a secondary data source, Optum EHR database.
Detailed description
The Optum EHR database was current up to 30-Jun-2020.
Identification period: From 01-Apr-2007 to 30-Jun-2019
Study period: From 01-Jan-2007 to 30-Jun-2020
Index date:
First evidence of resistance to or intolerance of HU treatment in patients with PV according to modified European Leukemia Net (ELN) criteria and defined as:
- HCT ≥ 45% with phlebotomy (last phlebotomy within last 3 months) Or
- Platelet count > 400 x 109/L and presence of palpable splenomegaly (palpable spleen up to 3 months after platelet count)
Pre-index period:
Patients had a minimum of 3 months pre-index data available. Pre-index data availability was determined using the reported 'first month active' field.
Post-index period:
There was no minimum post-index period required. Each patient had a 'first month active' and 'last month active' reported within the database. As the 'last month active' was based on any activity in the database, including encounters such as letters and emails which occurred several months after the 'death_date' of the patient, using the 'last month active' can overestimate the follow-up for a given patient. For this reason, the end of follow-up for each patient was defined as the date of the last activity within the diagnosis, observations, prescriptions, laboratories, procedures tables or discharge date from the last visit within the visit table (whichever of these activities occurs latest). This underestimated the follow-up for some patients where they were not actively using healthcare resources.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Ruxolitinib (RUX) | – | Other: RuxolitinibPV patients who were resistant to or intolerant of HU (as defined on the index date) and switched to RUX in the post-index period. |
| Best available therapy (BAT) | – | –PV patients who were resistant to or intolerant of Hydroxyurea (HU) (as defined on the index date) and continued HU treatment or switched to other available therapies other than RUX in the post-index period. |
Who may qualify
- Ages
- 18 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- PV patients who were resistant to or intolerant of HU in the Optum EHR database between April 2007 and June 2019
Key inclusion criteria (8 listed)
- With at least one International Classification of Diseases, 9th Revision, Clinical Modification/International Classification of Diseases,10th Revision, Clinical Modification code for PV in the identification period (01-Apr-2007 until 30-Jun-2019) that had non-missing sex and year of birth data and who were treated as part of the Integrated Delivery Network
- That were ≥ 18 years old at PV diagnosis
- With ≥ 2 prescriptions of HU
- That were classified as resistant to or intolerant of HU after a minimum of 3 months HU treatment (index date), defined as:
- HCT ≥ 45% with phlebotomy (last phlebotomy within last 3 months) or Platelet count > 400 x 109/L and presence of palpable splenomegaly (palpable spleen up to 3 months after platelet count).
Key exclusion criteria (2 listed)
- With a MF or AML diagnosis prior to a PV diagnosis.
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 |
|---|---|---|
| RuxolitinibAlso: JakaviPV patients who were resistant to or intolerant of HU (as defined on the index date) and switched to RUX in the post-index period. | Other | Ruxolitinib (RUX) |
Primary outcome
Number of Thromboembolic events between the RUX and BAT group
Time frame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
Thromboembolic events in overall Polycythemia Vera cohort and in the BAT and RUX groups were reported. A TE was defined using International Classification of Diseases 9th Revision (ICD-9- CM) and International Classification of Diseases 10th Revision (ICD-10-CM) codes previously curated as restrictive (RESPONSE RCT) and extensive (GEMFIN) definitions of TE's within the Diagnosis table in Optum EHR database.
Number of Thromboembolic events between the high and low risk subgroups of BAT group
Time frame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
Within the BAT group, high risk (≥ 1 TE on average per year ) and low risk (< 1 TE on average per year) subgroups were identified based on the frequency of TEs and characterized according to patient sociodemographics, comorbidities, symptoms, clinical, and medication variables.
10 secondary outcomes
Incidence rate of thromboembolic event
Time frame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
Time to first thromboembolic event
Time frame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
Incidence rate of phlebotomy procedures
Time frame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
Time to first phlebotomy procedure
Time frame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
Incidence rate of neoplasm transformations
Time frame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
Time to first neoplasm transformation
Time frame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
Treatment patterns: Proportion of patients using different PV-related treatments
Time frame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
Healthcare resource utilization (HCRU): Number of inpatient hospitalizations
Time frame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
Healthcare resource utilization (HCRU): Number of outpatient visits
Time frame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
Healthcare resource utilization (HCRU): Number of emergency room visits
Time frame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jul 2022.
- Results for CINC424B3001 from Novartis Clinical Trials Results DatabaseLink provided by the sponsor.
Related trials for Polycythemia Vera (PV)
RecruitingPhase 2
A Study of Ropeg-Interferon in People With Polycythemia Vera
Polycythemia Vera (PV), JAK2 Mutation
Active, not recruitingPhase 2
A Phase 2, Open-Label Study of DISC-3405 in Participants With Polycythemia Vera (PV)
Polycythemia Vera (PV)
RecruitingPhase 1
A Phase 1 Study of PRT12396 in Participants With Select Myeloproliferative Neoplasms
Polycythemia Vera (PV), Myelofibrosis (MF)
RecruitingPhase 3
INTREPID: A Study of Sapablursen Evaluating the Safety and Efficacy in Participants With Polycythemia Vera (PV)
Polycythemia Vera
Active, not recruitingPhase 2
Extended Access of Momelotinib in Adults With Myelofibrosis
Primary Myelofibrosis
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
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Polycythemia Vera (PV), run by Novartis Pharmaceuticals at 1 site 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 8 inclusion and 2 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: finished, as of the sponsor's last update on 5 Jul 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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 Thromboembolic events between the RUX and BAT group (time frame: throughout the study, approximately 13 years (Study period: From 01-Jan-2007 to 30-Jun-2020)). The study lists 2 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 5 Jul 2022.