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Myeloproliferative Neoplasms (MPNs) Patient Registry

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ClinicalTrials.gov Identifier: NCT02760238
Recruitment Status : Recruiting
First Posted : May 3, 2016
Last Update Posted : April 2, 2021
Sponsor:
Information provided by (Responsible Party):
University Health Network, Toronto

Tracking Information
First Submitted Date March 24, 2016
First Posted Date May 3, 2016
Last Update Posted Date April 2, 2021
Study Start Date April 2016
Estimated Primary Completion Date October 2025   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: October 27, 2017)
Survival [ Time Frame: Annually or at the time of transformation of disease, up to 10 years ]
Survival of patients with MPN
Original Primary Outcome Measures
 (submitted: April 28, 2016)
Survival [ Time Frame: Bi-annually or at the time of transformation of disease, up to 10 years ]
Change History
Current Secondary Outcome Measures
 (submitted: November 22, 2019)
  • 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 ]
    Type and phase of MPN, previous cancer history, age, sex
  • Disease risk score [ Time Frame: Annually or at the time of transformation of disease, up to 10 years ]
    Risk stratification (IPSS, DIPSS and DIPSS) o Details of transformation to accelerated/phase phase disease
  • Quality of life - Neoplasm Symptom [ Time Frame: Annually or at the time of transformation of disease, up to 10 years ]
    MPN-SAF TSS questionnaire
  • Co-morbidities [ Time Frame: Annually or at the time of transformation of disease, up to 10 years ]
    HCT-CI
  • Physical symptoms of MPN [ Time Frame: Annually or at the time of transformation of disease, up to 10 years ]
    Physical examination: Splenomegaly and hepatomegaly, ascites, EMS, ECOG
  • MPN treatment type received [ Time Frame: Annually or at the time of transformation of disease, up to 10 years ]
    Medical therapies received
  • Transfusion dependence status [ Time Frame: Annually or at the time of transformation of disease, up to 10 years ]
    Transfusion status
  • Current Blood Work [ Time Frame: Annually or at the time of transformation of disease, up to 10 years ]
    CBC, INR, PT, APTT, fibrinogen, creatinine, ALP, ALT, AST, GGT, total bilirubin, LDH, urate, CRP, erythropoietin, hepatitis B and HIV
  • Identifying MPN driver mutations by using next generation sequencing. [ Time Frame: Annually or at the time of transformation of disease, up to 10 years ]
    Next generation sequencing gene panel
  • Bone marrow transplant details (if received) [ Time Frame: Annually or at the time of transformation of disease, up to 10 years ]
    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)
  • Bone marrow transplant complications (if received) [ Time Frame: Annually or at the time of transformation of disease, up to 10 years ]
    Toxicities, engraftment and chimerism, GVHD, significant infections in the first 100 days
  • Portal hypertension [ Time Frame: Annually or at the time of transformation of disease, up to 10 years ]
    Presence and details of ascites, GIT bleeding, esophageal & gastric varices, cirrhosis and portal hypertensive gastropathy o Endoscopy results
  • Pulmonary hypertension [ Time Frame: Annually or at the time of transformation of disease, up to 10 years ]
    WHO classification, echocardiogram results, CNP, troponin, pulmonary function tests, 6 minute walk test distance, blood gas, treatment, complications
  • Thrombosis [ Time Frame: Annually or at the time of transformation of disease, up to 10 years ]
    Details of thrombosis (type, site) o Treatment of thrombosis (type, duration)
  • 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 ]
    Relative affected (e.g. daughter, uncle, mother), details of MPN (type, phase, treatment received)
  • Disease progression [ Time Frame: Annually or at the time of transformation of disease, up to 10 years ]
    Risk stratification (IPSS, DIPSS and DIPSS)
Original Secondary Outcome Measures
 (submitted: April 28, 2016)
  • General patient characteristics will be captured from the Hematologic Malignacy tissue bank [ Time Frame: Bi-annually or at the time of transformation of disease, up to 10 years ]
    Type and phase of MPN, previous cancer history, age, sex
  • Disease risk score [ Time Frame: Bi-annually or at the time of transformation of disease, up to 10 years ]
    Risk stratification (IPSS, DIPSS and DIPSS) o Details of transformation to accelerated/phase phase disease
  • Quality of life [ Time Frame: Bi-annually or at the time of transformation of disease, up to 10 years ]
    MPN-SAF TSS questionnaire
  • Co-morbidities [ Time Frame: Bi-annually or at the time of transformation of disease, up to 10 years ]
    HCT-CI
  • Physical symptoms of MPN [ Time Frame: Bi-annually or at the time of transformation of disease, up to 10 years ]
    Physical examination: Splenomegaly and hepatomegaly, ascites, EMS, ECOG
  • MPN treatment type [ Time Frame: Bi-annually or at the time of transformation of disease, up to 10 years ]
  • Transfusion dependence [ Time Frame: Bi-annually or at the time of transformation of disease, up to 10 years ]
  • Current Blood Work [ Time Frame: Bi-annually or at the time of transformation of disease, up to 10 years ]
    CBC, INR, PT, APTT, fibrinogen, creatinine, ALP, ALT, AST, GGT, total bilirubin, LDH, urate, CRP, erythropoietin, hepatitis B and HIV
  • Identifying MPN driver mutations by using next generation sequencing. [ Time Frame: Bi-annually or at the time of transformation of disease, up to 10 years ]
  • Bone marrow transplant details (if received) [ Time Frame: Bi-annually or at the time of transformation of disease, up to 10 years ]
    • 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)
  • Bone marrow transplant complications (if received) [ Time Frame: Bi-annually or at the time of transformation of disease, up to 10 years ]
    Toxicities, engraftment and chimerism, GVHD, significant infections in the first 100 days
  • Portal hypertension [ Time Frame: Bi-annually or at the time of transformation of disease, up to 10 years ]
    • Presence and details of ascites, GIT bleeding, esophageal & gastric varices, cirrhosis and portal hypertensive gastropathy
    • Endoscopy results
  • Pulmonary hypertension [ Time Frame: Bi-annually or at the time of transformation of disease, up to 10 years ]
    WHO classification, echocardiogram results, CNP, troponin, pulmonary function tests, 6 minute walk test distance, blood gas, treatment, complications
  • Thrombosis [ Time Frame: Bi-annually or at the time of transformation of disease, up to 10 years ]
    • Details of thrombosis (type, site)
    • Treatment of thrombosis (type, duration)
  • Family history of MPN will be obtained from the patient record. [ Time Frame: Bi-annually or at the time of transformation of disease, up to 10 years ]
    Relative affected (e.g. daughter, uncle, mother), details of MPN (type, phase, treatment received)
  • Disease progression [ Time Frame: Bi-annually or at the time of transformation of disease, up to 10 years ]
    Risk stratification (IPSS, DIPSS and DIPSS)
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Myeloproliferative Neoplasms (MPNs) Patient Registry
Official Title Clinical and Molecular Epidemiology of Myeloproliferative Neoplasms (MPNs)
Brief 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.
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 Type Observational [Patient Registry]
Study Design Observational Model: Cohort
Time Perspective: Other
Target Follow-Up Duration 10 Years
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Patients at Princess Margaret Cancer Centre with an MPN diagnosis who consent to inclusion in this registry.
Condition
  • Primary Myelofibrosis
  • Polycythemia Vera
  • Essential Thrombocythemia
  • Mastocytosis
  • Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative
  • Leukemia, Myelomonocytic, Juvenile
  • Chronic Eosinophilic Leukemia-not Otherwise Specified
  • Myelodysplastic-Myeloproliferative Diseases
  • Neoplasms
  • Leukemia, Myelomonocytic, Chronic
Intervention Other: Observational
Study Groups/Cohorts 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

Intervention: Other: Observational
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: April 28, 2016)
5000
Original Estimated Enrollment Same as current
Estimated Study Completion Date October 2025
Estimated Primary Completion Date October 2025   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

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),
  • 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)

Exclusion Criteria:

  • None
Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts
Contact: Vikas Gupta, MD 416-946-4521 ext 4521 vikas.gupta@uhn.ca
Contact: Jaime O. Claudio, PhD 416-946-4501 ext 2648 jclaudio@uhnresearch.ca
Listed Location Countries Canada
Removed Location Countries  
 
Administrative Information
NCT Number NCT02760238
Other Study ID Numbers UHN REB 15-9814 CE
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement
Plan to Share IPD: Yes
Plan Description: 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.
Time Frame: Continuing
Access Criteria: De-identified participant data may only be shared for REB approved research studies.
Responsible Party University Health Network, Toronto
Study Sponsor University Health Network, Toronto
Collaborators Not Provided
Investigators
Principal Investigator: Vikas Gupta, MD University Health Network, Toronto
PRS Account University Health Network, Toronto
Verification Date April 2021