NCT01831635

Brief Summary

There is a paucity of data on the aetiology of myeloproliferative neoplasms (MPNs). The investigators conducted a systematic review of the literature which identified several cohort and case-control studies that have investigated a wide range of potential medical, environmental and occupational risk factors. However, these studies have been limited by a wide variation in case definition and small sample sizes limiting the potential to detect modest risk differences between cases and controls. The research group propose an exploratory case-control study of 100 patients with classic MPNs and 200 controls to determine the optimal methods for roll out of this study to a multi-centred UK-based case-control study that will investigate the aetiology of MPN subtypes. The objectives of the study are to evaluate recruitment procedures, response rates and the development of a telephone administered questionnaire. The findings of this exploratory study will form the basis of a protocol for a large United Kingdom (UK)-wide case-control study of MPNs.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
234

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Apr 2013

Typical duration for all trials

Geographic Reach
1 country

3 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 27, 2013

Completed
5 days until next milestone

Study Start

First participant enrolled

April 1, 2013

Completed
14 days until next milestone

First Posted

Study publicly available on registry

April 15, 2013

Completed
1.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2014

Completed
11 months until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2015

Completed
8.6 years until next milestone

Results Posted

Study results publicly available

January 8, 2024

Completed
Last Updated

January 8, 2024

Status Verified

March 1, 2023

Enrollment Period

1.2 years

First QC Date

March 27, 2013

Results QC Date

July 29, 2015

Last Update Submit

March 30, 2023

Conditions

Keywords

case-controlrecruitmentresponse ratesincentives

Outcome Measures

Primary Outcomes (1)

  • Influence of Pre- and Post- Incentives on Participation Rates.

    * Difference in participation rates in those receiving: * vs. not receiving a pen at initial contact. * vs. not receiving a trolley token at initial contact. * vs. not receiving a monetary incentive at initial contact. * a MOSAICC pen vs. a non-branded pen following second contact. * vs. not receiving a trolley token following second contact. * a MOSAICC branded Pen +/or trolley token alone vs no non-monetary incentive following a second invite. * a MOSAICC branded Pen +/or trolley token alone vs no incentive following a second invite. * a MOSAICC branded Pen +/or trolley token alone vs pen/no pen \&/or money following a second invite (branded vs unbranded incentive) * 0/1 of these incentives vs. receiving 2/3 incentives following a second invite. * Difference in proportion of patients completing study elements comparing: * 0/1 vs 2/3 incentives. * those receiving pre vs post invitation incentives.

    average 2 weeks

Study Arms (3)

Myeloproliferative neoplasm case

Patients with Polycythemia vera (PV), essential thrombocythemia (ET) and primary myelofibrosis (PMF) will be recruited based on the WHO diagnostic criteria. Exclusion Criteria * younger than 18 years old. * where the clinician/General Practitioner (GP) does not provide consent. * incapable of giving informed consent. * physically or cognitively incapable of completing the questionnaire. * too ill to participate. We will assess whether a pen (branded, non-branded or none), a £10 cheque and/or a charity branded trolley token will affect uptake rates in the study.

Other: Methodological pensOther: Methodological compensationOther: Methodological trolley tokens

General Practice Control

GP controls will be randomly selected and frequency matched to the distribution of cases by 5-year age band, geographic location (Belfast and Southampton) and gender. The following patient groups will be excluded. Those: * younger than 18 years old. * where the clinician/GP does not provide consent. * incapable of giving informed consent. * physically or cognitively incapable of completing the questionnaire. * too ill to participate. We will assess whether a pen (branded, non-branded or none), a £10 cheque (at study completion) and/or a charity branded trolley token will affect uptake rates in the study.

Other: Methodological pensOther: Methodological compensationOther: Methodological trolley tokens

Non-blood relative or family Control

Recruitment of 100 non-blood relative/friend controls will be undertaken by asking cases to pass on a flyer to up to 2 or 3 non-blood relatives/friends aged 18 years or older. We will assess whether a pen (branded, non-branded or none) and/or a £10 cheque (at study completion) will affect uptake rates in the study. Only a limited number of trolley tokens were available so not assessed in this group.

Other: Methodological pensOther: Methodological compensation

Interventions

Randomisation of branded pen, non-branded pen and no pen

General Practice ControlMyeloproliferative neoplasm caseNon-blood relative or family Control

Randomisation of a £10 monetary reimbursement for expenses on completion of the study

General Practice ControlMyeloproliferative neoplasm caseNon-blood relative or family Control

Trolley tokens to be randomly assigned to half of cases and General Practice controls.

General Practice ControlMyeloproliferative neoplasm case

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodProbability Sample
Study Population

Hematology clinic Primary Care clinic Non-blood relatives/ friends of cases

You may qualify if:

  • Clinical diagnosis of polycythemia vera, essential thrombocythaemia or primary myelofibrosis based on the WHO diagnostic criteria.
  • Aged 18 years or older.

You may not qualify if:

  • younger than 18 years old.
  • where the clinician/General Practitioner (GP) does not provide consent.
  • incapable of giving informed consent.
  • physically or cognitively incapable of completing the questionnaire.
  • too ill to participate
  • General Practice Controls
  • Randomly selected, frequency matched to the distribution of cases by 5-year age band, geographic location (Belfast and Southampton) and gender.
  • Aged 18 years or older.
  • younger than 18 years old.
  • where the clinician/GP does not provide consent.
  • incapable of giving informed consent.
  • physically or cognitively incapable of completing the questionnaire.
  • too ill to participate.
  • Relative/Friend Controls
  • Non-blood relative/friend of a case participating in the study.
  • +6 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

University Hospitals Southampton NHS Foundation Trust

Southampton, England, SO16 6YD, United Kingdom

Location

Belfast Health And Social Care Trust

Belfast, Northern Ireland, BT12 6BJ, United Kingdom

Location

Queen's University Belfast

Belfast, Northern Ireland, BT389EX, United Kingdom

Location

Related Publications (3)

  • Anderson LA, Duncombe AS, Hughes M, Mills ME, Wilson JC, McMullin MF. Environmental, lifestyle, and familial/ethnic factors associated with myeloproliferative neoplasms. Am J Hematol. 2012 Feb;87(2):175-82. doi: 10.1002/ajh.22212. Epub 2011 Nov 11.

    PMID: 22076943BACKGROUND
  • James G, McMullin MF, Duncombe AS, Clarke M, Anderson LA. The MOSAICC study: Assessing feasibility for biological sample collection in epidemiology studies and comparison of DNA yields from saliva and whole blood samples. Ann Hum Genet. 2018 Mar;82(2):114-118. doi: 10.1111/ahg.12227. Epub 2017 Oct 27.

    PMID: 29076129BACKGROUND
  • Anderson LA, James G, Duncombe AS, Mesa R, Scherber R, Dueck AC, de Vocht F, Clarke M, McMullin MF. Myeloproliferative neoplasm patient symptom burden and quality of life: evidence of significant impairment compared to controls. Am J Hematol. 2015 Oct;90(10):864-70. doi: 10.1002/ajh.24098. Epub 2015 Sep 10.

Biospecimen

Retention: SAMPLES WITH DNA

Venous blood sample (up to 12 ml) - whole blood, DNA Saliva (2ml) Dried Blood spots (500 µl) Toe-nails (2 great toe-nail clippings)

MeSH Terms

Conditions

Polycythemia VeraThrombocythemia, EssentialPrimary Myelofibrosis

Condition Hierarchy (Ancestors)

Bone Marrow NeoplasmsHematologic NeoplasmsNeoplasms by SiteNeoplasmsBone Marrow DiseasesHematologic DiseasesHemic and Lymphatic DiseasesMyeloproliferative DisordersBlood Coagulation DisordersThrombocytosisBlood Platelet DisordersHemorrhagic Disorders

Results Point of Contact

Title
Dr Lesley Anderson
Organization
Queen's University Belfast

Study Officials

  • Lesley A Anderson, PhD

    Queen's University, Belfast

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
observational
Observational Model
CASE CONTROL
Time Perspective
RETROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Chief Investigator

Study Record Dates

First Submitted

March 27, 2013

First Posted

April 15, 2013

Study Start

April 1, 2013

Primary Completion

July 1, 2014

Study Completion

June 1, 2015

Last Updated

January 8, 2024

Results First Posted

January 8, 2024

Record last verified: 2023-03

Data Sharing

IPD Sharing
Will share

Researchers can contact the study lead (Dr Anderson) to request a data access form. Once a data access form has been completed it will be reviewed by the study team and a decision made. If sharing of data has been agreed by the study team the contracts department and legal teams will review the proposed sharing of data to ensure that it meets with international standards.

Shared Documents
STUDY PROTOCOL, SAP
Time Frame
Data is available from 01/09/2018 and will be available for a period of 5 years in the first instance.
Access Criteria
* Non commercial * Contracts/legal team to review data transfer agreements * Data transfer and use policy document * Completion of a data access form (obtainable from Dr Anderson l.anderson@qub.ac.uk)

Locations