Polycythemia Vera, Myelofibrosis and Essential Thrombocyt... | NCT00715247

Studies
Polycythemia Vera, Myelofibrosis and Essential Thrombocythemia: Identification of PV, MF & ET Genes

NCT00715247

Sponsor

University of Utah

Status

Completed

Last Update Posted

Oct 15, 2019Actual

Enrollment

726Actual

Phase

Not provided

Conditions

Polycythemia Vera
Essential Thrombocythemia
Myelofibrosis

Interventions

Not provided

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

ID	Type	Description	Link
1P01CA10867101A2	Other Grant/Funding Number	NCI

Status Module

Record Verification Date

Oct 2019

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jul 2006

Primary Completion Date

Dec 2018Actual

Completion Date

Dec 2018Actual

First Submitted Date

Jul 11, 2008

First Submission Date that Met QC Criteria

Jul 14, 2008

First Posted Date

Jul 15, 2008Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Oct 14, 2019

Last Update Posted Date

Oct 15, 2019Actual

Name	Class
Myeloproliferative Disorders-Research Consortium	NETWORK
National Cancer Institute (NCI)	NIH

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

The purpose of this project is to find genes whose mutations cause Polycythemia Vera, Essential Thrombocythemia and Primary Myelofibrosis.

Detailed Description

Polycythemia Vera (PV), Essential Thrombocythemia (ET) and Primary Myelofibrosis (PMF), also known as the Philadelphia Chromosome negative myeloproliferative disorders (MPDs), are not congenital, but acquired. The purpose of this project is to find genes whose mutations cause these disorders, as well as improve diagnostic measures for these diseases. When this is accomplished new therapies to control and eventually cure the disease can be designed.

All subjects will be asked to donate 4-6 teaspoons of blood. On occasion, if the blood cells from a particular sample do not grow well and the DNA from that sample is used up or other tests are needed, we may ask to collect additional samples. In patients who have undergone a bone marrow biopsy as part of their clinical evaluation, we will test the reproducibility between pathologists for the revised 2008 WHO diagnostic criteria to diagnose myeloproliferative disorders (MPD).

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

Retention

Enrollment

726Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Affected Population		Patients suspected to have one of the following blood disorders: polycythemia vera, myelofibrosis or essential thrombocythemia.
Healthy Female Controls		Healthy females who do not have the blood disorders; Polycythemia Vera, Essential Thrombocythemia and/or Myelofibrosis.

Interventions

Not provided

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Identify genes whose mutations cause Polycythemia Vera, Essential Thrombocythemia and Primary Myelofibrosis.		Weekly

Secondary Outcomes

Measure	Description	Time Frame
To determine if there are proteins expressed by cells from patients that might be targets for the immune response.		Weekly

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Patients with an elevated hemoglobin concentration (>18 in males and >16 in females) and who are suspected to have congenital or acquired primary polycythemia
Patients with a persistent thrombocytosis (>400,000) that does not have an obvious secondary cause
Patients with a bone marrow biopsy that shows increased cellularity and fibrosis
Patients where there is clinical concern for primary myelofibrosis, such as anemia in combination with leukocytosis, thrombocytosis, splenomegaly and/or a leukoerythroblastic blood smear
Patients with thrombosis at unusual sites, such as Budd-Chiari syndrome, can have early PV before hemoglobin is elevated, these patients will also be included.

Exclusion Criteria:

Subjects who have a known acquired cause of polycythemia (increased hemoglobin/hematocrit) such as people living in high altitudes (in excess of 14,000 feet), subjects with heart disease, left to right heart shunt, severe hypoxia or severe pulmonary disease will be excluded from this study.
Subjects with a known acquired cause of thrombocytosis.
Subjects will be excluded if they cannot demonstrate decision making capacity sufficient to agree or decline the blood drawing or use of their blood for the study.

Accepts Healthy Volunteers

Yes

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Patients suspected to have one of the following blood disorders: polycythemia vera, myelofibrosis or essential thrombocythemia.

Sampling Method

Non-Probability Sample

Name	Affiliation	Role
Josef T Prchal, MD	University of Utah	Principal Investigator

Facility	Status	City	State	ZIP	Country	Contacts
University of Utah		Salt Lake City	Utah	84132	United States

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
17976520	Background	Jelinek J, Li J, Mnjoyan Z, Issa JP, Prchal JT, Afshar-Kharghan V. Epigenetic control of PRV-1 expression on neutrophils. Exp Hematol. 2007 Nov;35(11):1677-83. doi: 10.1016/j.exphem.2007.09.008.
17952198	Background	Agarwal N, Mojica-Henshaw MP, Simmons ED, Hussey D, Ou CN, Prchal JT. Familial polycythemia caused by a novel mutation in the beta globin gene: essential role of P50 in evaluation of familial polycythemia. Int J Med Sci. 2007 Oct 4;4(4):232-6. doi: 10.7150/ijms.4.232.

ID	Term
D011087	Polycythemia Vera
D013920	Thrombocythemia, Essential
D055728	Primary Myelofibrosis
D009196	Myeloproliferative Disorders
D019337	Hematologic Neoplasms

ID	Term
D019046	Bone Marrow Neoplasms
D009371	Neoplasms by Site
D009369	Neoplasms
D001855	Bone Marrow Diseases