Primary MyelofibrosisNCT02760238
Myeloproliferative Neoplasms (MPNs) Patient Registry
An observational study of Primary Myelofibrosis and Polycythemia Vera, run by University Health Network, Toronto at 1 site in Canada.
Trial summary
The mandate of this MPN registry is to collect clinical information, including molecular results, from consenting patients with a variety of MPNs at different time points during the course of their disease. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Primary Myelofibrosis, Polycythemia Vera, Essential Thrombocythemia
- What is being tested?
- Observational
- How many people
- 5,000 participants
- Where
- 1 siteCanada
- Study dates
- Apr 2015to 31 Oct 2027
- How long will I take part?
- 10 Years
- Phase
- Observational
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients at Princess Margaret Cancer Centre with an MPN diagnosis who consent to inclusion in this registry.
Who may be able to join
- Diagnosis of one of the following myeloproliferative neoplasms (MPNs):
- Atypical CML (aCML)
- Chronic eosinophilic leukemia-not otherwise specified (CEL, NOS),
- Chronic myelomonocytic leukemia (CMML)
- Chronic neutrophilic leukemia (CNL),
- Essential thrombocythemia (ET),
Show all 13Show fewer
- Juvenile myelomonocytic leukemia (JMML),
- Mastocytosis, MPN unclassifiable
- MPN/MDS unclassifiable,
- Primary myelofibrosis (PMF),
- Post-essential thrombocythemia myelofibrosis (post-ET MF),
- Post-polycythemia vera MF (post-PV MF)
- Refractory anemia with ringed sideroblasts associated with marked thrombocytosis (RARS-T)
Who cannot join
- None
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Canada1 site, 1 recruiting
- Recruiting
Princess Margaret Cancer Centre
Toronto, Ontario, M5G 1Z5
Vikas Gupta, MD, 416-946-2885, vikas.gupta@uhn.ca
About this study
Official title: Clinical and Molecular Epidemiology of Myeloproliferative Neoplasms (MPNs)
The mandate of this MPN registry is to collect clinical information, including molecular results, from consenting patients with a variety of MPNs at different time points during the course of their disease.
Read the detailed description
The myeloproliferative neoplasms (MPNs) are a group of rare hematological malignancies in which the bone marrow cells that produce the body's blood cells develop and function abnormally.
Despite the gains that have already been made in understanding and treatment of MPNs there is much that can still be learned. This registry will establish a clinical annotation database would help to better understand this group of diseases and to more effectively assign individual patients to the optimal therapy and so, improve their outcomes. This project will provide new insights on the molecular profiling of patients with MPN. It will be used as future resource for observational studies related to MPN.
The registry involves the collection of clinical information from patients with diagnosis of MPN at different time points during the course of their disease. The clinical data is collected following written informed consent from the Hematologic Malignancy tissue bank (UHN REB 01-0573C).
Data collected includes: a range of clinical measures, disease-associated factors, details of treatment and its results, complications during treatment, molecular and cytogenetic data, symptom assessment and survival outcome (up to 10 years).
Data will be collected prospectively and retrospectively, in both cases after obtaining written informed consent as per the study standard operating procedure (SOP).
- Conditions
- Primary Myelofibrosis, Polycythemia Vera, Essential Thrombocythemia, Mastocytosis, Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative, Leukemia, Myelomonocytic, Juvenile
- Being tested
- Observational
- Keywords
- myeloproliferative neoplasm, myelodysplastic syndrome, registry, leukemia, Bone Marrow Diseases, Hematologic Diseases, Neoplasms
- Medical subject headings
- Primary Myelofibrosis, Polycythemia Vera, Thrombocythemia, Essential, Mastocytosis, Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative, Leukemia, Myelomonocytic, Juvenile, Hypereosinophilic Syndrome, Myelodysplastic-Myeloproliferative Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ObservationalOther
| Study group | Role | What they receive |
|---|---|---|
| Patients with a diagnosis of MPN | Patients with a myeloproliferative neoplasm (MPN) diagnosis:
Atypical chronic myeloid leukemia (aCML), chronic eosinophilic leukemia-not otherwise specified (CEL NOS), chronic myelomonocytic leukemia (CMML), chronic neutrophilic leukemia (CNL), polycythemia vera (PV), essential thrombocythemia (ET), JMML, mastocytosis, MPN unclassifiable, myeloproliferative neoplasm/myelodysplastic syndrome unclassifiable (MPN/MDS unclassifiable), primary myelofibrosis (PMF), post-ET MF, post-PV MF, or (refractory anemia with ringed sideroblasts associated with marked thrombocytosis) RARS-T Receives: Other: Observational |
What is being measured
The primary outcome is the main question the study is designed to answer.
Survival
Survival of patients with MPN
Measured: Annually or at the time of transformation of disease, up to 10 years
Secondary outcomes (16)
General patient characteristics will be captured from the Hematologic Malignancy tissue bank
Type and phase of MPN, previous cancer history, age, sex
Measured: Annually or at the time of transformation of disease, up to 10 years
Disease risk score
Risk stratification (IPSS, DIPSS and DIPSS)
- Details of transformation to accelerated/phase phase disease
Measured: Annually or at the time of transformation of disease, up to 10 years
Quality of life - Neoplasm Symptom
MPN-SAF TSS questionnaire
Measured: Annually or at the time of transformation of disease, up to 10 years
Co-morbidities
HCT-CI
Measured: Annually or at the time of transformation of disease, up to 10 years
Physical symptoms of MPN
Physical examination: Splenomegaly and hepatomegaly, ascites, EMS, ECOG
Measured: Annually or at the time of transformation of disease, up to 10 years
MPN treatment type received
Medical therapies received
Measured: Annually or at the time of transformation of disease, up to 10 years
Transfusion dependence status
Transfusion status
Measured: Annually or at the time of transformation of disease, up to 10 years
Current Blood Work
CBC, INR, PT, APTT, fibrinogen, creatinine, ALP, ALT, AST, GGT, total bilirubin, LDH, urate, CRP, erythropoietin, hepatitis B and HIV
Measured: Annually or at the time of transformation of disease, up to 10 years
Identifying MPN driver mutations by using next generation sequencing.
Next generation sequencing gene panel
Measured: Annually or at the time of transformation of disease, up to 10 years
Bone marrow transplant details (if received)
Details of recipient (CMV status, ABO blood group)
- Details of donor (gender, CMV status, ABO blood group)
- Disease status at time of transplant (blood work disease status)
- Transplant details (stem cell source, HLA matching, conditioning intensity \& regimen, serotherapy, GVHD prophylaxis)
Measured: Annually or at the time of transformation of disease, up to 10 years
Bone marrow transplant complications (if received)
Toxicities, engraftment and chimerism, GVHD, significant infections in the first 100 days
Measured: Annually or at the time of transformation of disease, up to 10 years
Portal hypertension
Presence and details of ascites, GIT bleeding, esophageal \& gastric varices, cirrhosis and portal hypertensive gastropathy
- Endoscopy results
Measured: Annually or at the time of transformation of disease, up to 10 years
Pulmonary hypertension
WHO classification, echocardiogram results, CNP, troponin, pulmonary function tests, 6 minute walk test distance, blood gas, treatment, complications
Measured: Annually or at the time of transformation of disease, up to 10 years
Thrombosis
Details of thrombosis (type, site)
- Treatment of thrombosis (type, duration)
Measured: Annually or at the time of transformation of disease, up to 10 years
Family history of MPN will be obtained from the patient record.
Relative affected (e.g. daughter, uncle, mother), details of MPN (type, phase, treatment received)
Measured: Annually or at the time of transformation of disease, up to 10 years
Disease progression
Risk stratification (IPSS, DIPSS and DIPSS)
Measured: Annually or at the time of transformation of disease, up to 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 May 2016 (estimated)
- Study startApr 2015
- Primary completion31 Oct 2027 (estimated)
- Study completion31 Oct 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary MyelofibrosisStudies of the same condition, open and under way.
- More trials run by University Health Network, TorontoThe 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
- Other
- Enrolment
- 5,000 participants (estimated)
- Target duration
- 10 Years
Sponsor and investigators
- Lead sponsor
- University Health Network, Toronto
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Vikas Gupta, MD, Principal investigator, University Health Network, Toronto
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02760238
- Sponsor's protocol ID
- UHN REB 15-9814 CE
- Status last verified
- Aug 2024
- First submitted
- 24 Mar 2016
- Last updated
- 27 Aug 2024
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Yes: De-identified participant data may be shared for REB approved research studies. Only the data necessary to achieve the study's aims will be shared and all data will be de-identified prior to sharing.
Study at a glance
From the registry record, last updated by the sponsor on 27 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical and Molecular Epidemiology of Myeloproliferative Neoplasms (MPNs)
- ClinicalTrials.gov ID
- NCT02760238
- Acronym / sponsor ID
- UHN REB 15-9814 CE
- Conditions
- Primary Myelofibrosis, Polycythemia Vera, Essential Thrombocythemia, Mastocytosis, Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative, Leukemia, Myelomonocytic, Juvenile
- Keywords
- myeloproliferative neoplasm, myelodysplastic syndrome, registry, leukemia, Bone Marrow Diseases, Hematologic Diseases, Neoplasms
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 5,000 participants (estimated)
- Lead sponsor
- University Health Network, TorontoUniversity, hospital or other
- Study start
- Apr 2015
- Primary completion
- 31 Oct 2027 (estimated)
- Study completion
- 31 Oct 2027 (estimated)
- First posted
- 3 May 2016 (estimated)
- 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 mandate of this MPN registry is to collect clinical information, including molecular results, from consenting patients with a variety of MPNs at different time points during the course of their disease.
Detailed description
The myeloproliferative neoplasms (MPNs) are a group of rare hematological malignancies in which the bone marrow cells that produce the body's blood cells develop and function abnormally.
Despite the gains that have already been made in understanding and treatment of MPNs there is much that can still be learned. This registry will establish a clinical annotation database would help to better understand this group of diseases and to more effectively assign individual patients to the optimal therapy and so, improve their outcomes. This project will provide new insights on the molecular profiling of patients with MPN. It will be used as future resource for observational studies related to MPN.
The registry involves the collection of clinical information from patients with diagnosis of MPN at different time points during the course of their disease. The clinical data is collected following written informed consent from the Hematologic Malignancy tissue bank (UHN REB 01-0573C).
Data collected includes: a range of clinical measures, disease-associated factors, details of treatment and its results, complications during treatment, molecular and cytogenetic data, symptom assessment and survival outcome (up to 10 years).
Data will be collected prospectively and retrospectively, in both cases after obtaining written informed consent as per the study standard operating procedure (SOP).
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Target follow-up
- 10 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients with a diagnosis of MPN | – | Other: ObservationalPatients with a myeloproliferative neoplasm (MPN) diagnosis: Atypical chronic myeloid leukemia (aCML), chronic eosinophilic leukemia-not otherwise specified (CEL NOS), chronic myelomonocytic leukemia (CMML), chronic neutrophilic leukemia (CNL), polycythemia vera (PV), essential thrombocythemia (ET), JMML, mastocytosis, MPN unclassifiable, myeloproliferative neoplasm/myelodysplastic syndrome unclassifiable (MPN/MDS unclassifiable), primary myelofibrosis (PMF), post-ET MF, post-PV MF, or (refractory anemia with ringed sideroblasts associated with marked thrombocytosis) RARS-T |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients at Princess Margaret Cancer Centre with an MPN diagnosis who consent to inclusion in this registry.
Key inclusion criteria (13 listed)
- Atypical CML (aCML)
- Chronic eosinophilic leukemia-not otherwise specified (CEL, NOS),
- Chronic myelomonocytic leukemia (CMML)
- Chronic neutrophilic leukemia (CNL),
- Essential thrombocythemia (ET),
Key exclusion criteria (1 listed)
- None
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 |
|---|---|---|
| Observational | Other | Patients with a diagnosis of MPN |
Primary outcome
Survival
Time frame: Annually or at the time of transformation of disease, up to 10 years
Survival of patients with MPN
16 secondary outcomes
General patient characteristics will be captured from the Hematologic Malignancy tissue bank
Time frame: Annually or at the time of transformation of disease, up to 10 years
Disease risk score
Time frame: Annually or at the time of transformation of disease, up to 10 years
Quality of life - Neoplasm Symptom
Time frame: Annually or at the time of transformation of disease, up to 10 years
Co-morbidities
Time frame: Annually or at the time of transformation of disease, up to 10 years
Physical symptoms of MPN
Time frame: Annually or at the time of transformation of disease, up to 10 years
MPN treatment type received
Time frame: Annually or at the time of transformation of disease, up to 10 years
Transfusion dependence status
Time frame: Annually or at the time of transformation of disease, up to 10 years
Current Blood Work
Time frame: Annually or at the time of transformation of disease, up to 10 years
Identifying MPN driver mutations by using next generation sequencing.
Time frame: Annually or at the time of transformation of disease, up to 10 years
Bone marrow transplant details (if received)
Time frame: Annually or at the time of transformation of disease, up to 10 years
Bone marrow transplant complications (if received)
Time frame: Annually or at the time of transformation of disease, up to 10 years
Portal hypertension
Time frame: Annually or at the time of transformation of disease, up to 10 years
Pulmonary hypertension
Time frame: Annually or at the time of transformation of disease, up to 10 years
Thrombosis
Time frame: Annually or at the time of transformation of disease, up to 10 years
Family history of MPN will be obtained from the patient record.
Time frame: Annually or at the time of transformation of disease, up to 10 years
Disease progression
Time frame: Annually or at the time of transformation of disease, up to 10 years
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 27 Aug 2024.
- Individual participant dataSharing planned: Yes. De-identified participant data may be shared for REB approved research studies. Only the data necessary to achieve the study's aims will be shared and all data will be de-identified prior to sharing.
Related trials for Primary Myelofibrosis
RecruitingPhase 1/2
Treating Anemia in Myelofibrosis With Repurposed Drugs (Nelfinavir) That Restore Iron Delivery to the Bone Marrow
Myelofibrosis, Myelofibrosis; Anemia
RecruitingPhase 3
A Phase 3 Study of Pelabresib (DAK539) and Ruxolitinib in Myelofibrosis (MF)
Primary Myelofibrosis (PMF), Post-polycythemia Vera Myelofibrosis (PPV-MF)
RecruitingPhase 1
A Vaccine (CMV-MVA Triplex Vaccine) for the Enhancement of CMV-Specific Immunity and the Prevention of CMV Viremia in Patients Undergoing Haploidentical Hematopoietic Stem Cell Transplant
Accelerated Phase Chronic Myeloid Leukemia, BCR-ABL1 Positive, Acute Lymphoblastic Leukemia
RecruitingPhase 2
CMV-MVA Triplex Vaccination in HLA-Matched Related Stem Cell Donors for the Prevention of CMV Infection in Patients Undergoing Hematopoietic Stem Cell Transplant
Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
Active, not recruitingObservational study
Infection Prophylaxis and Management in Treating Cytomegalovirus (CMV) Infection in Patients With Hematologic Malignancies Previously Treated With Donor Stem Cell Transplant
Hematopoietic/Lymphoid Cancer, Accelerated Phase Chronic Myelogenous Leukemia
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.
Vikas Gupta, MD principal investigator
University Health Network, Toronto
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 Primary Myelofibrosis and Polycythemia Vera, run by University Health Network, Toronto at 1 site in Canada.
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 13 inclusion and 1 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: looking for participants now, as of the sponsor's last update on 27 Aug 2024. 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 Canada. 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?
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?
Contact Vikas Gupta, MD (416-946-4521 ext. 4521, vikas.gupta@uhn.ca), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Survival (time frame: Annually or at the time of transformation of disease, up to 10 years). 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 27 Aug 2024.