Retrospective & Prospective Observational Study of Patients With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Amgen
ClinicalTrials.gov Identifier:
NCT00454857
First received: March 29, 2007
Last updated: October 23, 2013
Last verified: October 2013
  Purpose

This is a multi-center prospective observational descriptive study complemented by a retrospective chart review. Patients diagnosed with ITP and currently treated for ITP by a hematologist or hematologist-oncologist will be recruited from community-based clinics and academic/referral centers. They will be followed prospectively for a period of 12 months. At inception, participants' charts will also be reviewed from the date of enrollment retrospectively to the date of diagnosis or the previous 36 months, whichever is less.


Condition Intervention
Idiopathic Thrombocytopenic Purpura
Thrombocytopenia
Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
Thrombocytopenic Purpura
Other: Retrospective Chart Review
Other: Patient-reported Outcome Questionnaires
Other: Physician Survey

Study Type: Observational
Study Design: Observational Model: Cohort
Official Title: A Prospective Observational Descriptive Study and Retrospective Chart Review of Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)

Resource links provided by NLM:


Further study details as provided by Amgen:

Primary Outcome Measures:
  • Number of Participants Utilizing Immune (Idiopathic) Thrombocytopenic Purpura (ITP) Therapies for First-line Treatment [ Time Frame: Includes the retrospective chart review (from the date of enrollment retrospectively to the date of diagnosis or the previous 36 months, whichever was less) and the prospective portion of the study (enrollment through Month 12). ] [ Designated as safety issue: No ]
    The number of participants who received ITP therapies for first-line treatment during either the retrospective or prospective phases of the study.

  • The Number of Participants Utilizing ITP Therapies for Second-line Treatment. [ Time Frame: Includes the retrospective chart review (from the date of enrollment retrospectively to the date of diagnosis or the previous 36 months, whichever was less) and the prospective portion of the study (enrollment through Month 12). ] [ Designated as safety issue: No ]
    The number of participants who received ITP therapies for second-line treatment during either the retrospective or prospective phases of the study.

  • The Number of Participants Utilizing ITP Therapies for Third-line Treatment. [ Time Frame: Includes the retrospective chart review (from the date of enrollment retrospectively to the date of diagnosis or the previous 36 months, whichever was less) and the prospective portion of the study (enrollment through Month 12). ] [ Designated as safety issue: No ]
    The number of participants who received ITP therapies for third-line treatment during either the retrospective or prospective phases of the study.

  • The Number of Participants Utilizing ITP Therapies for Fourth-line Treatment. [ Time Frame: Includes the retrospective chart review (from the date of enrollment retrospectively to the date of diagnosis or the previous 36 months, whichever was less) and the prospective portion of the study (enrollment through Month 12). ] [ Designated as safety issue: No ]
    The number of participants who received ITP therapies for fourth-line treatment during either the retrospective or prospective phases of the study.

  • The Number of Participants Utilizing ITP Therapies for Fifth-line Treatment [ Time Frame: Includes the retrospective chart review (from the date of enrollment retrospectively to the date of diagnosis or the previous 36 months, whichever was less) and the prospective portion of the study (enrollment through Month 12). ] [ Designated as safety issue: No ]
    The number of participants who received ITP therapies for fifth-line treatment during either the retrospective or prospective phases of the study.

  • The Number of Participants Utilizing ITP Therapies for Sixth-line Treatment. [ Time Frame: Includes the retrospective chart review (from the date of enrollment retrospectively to the date of diagnosis or the previous 36 months, whichever was less) and the prospective portion of the study (enrollment through Month 12). ] [ Designated as safety issue: No ]
    Assessed the number of participants who received ITP therapies for sixth-line treatment during either the retrospective or prospective phases of the study.

  • The Number of Participants Utilizing ITP Therapies for Seventh or Greater-line Treatment. [ Time Frame: Includes the retrospective chart review (from the date of enrollment retrospectively to the date of diagnosis or the previous 36 months, whichever was less) and the prospective portion of the study (enrollment through Month 12). ] [ Designated as safety issue: No ]
    The number participants who received ITP therapies as seventh or greater-line treatment during either the retrospective or prospective phases of the study.

  • The Number of Participants Utilizing ITP Therapies: Treatments With Unknown Starting Date. [ Time Frame: Includes the retrospective chart review (from the date of enrollment retrospectively to the date of diagnosis or the previous 36 months, whichever was less) and the prospective portion of the study (enrollment through Month 12). ] [ Designated as safety issue: No ]
    The number of participants who received ITP therapies for treatments with unknown starting date during either the retrospective or prospective phases of the study.


Secondary Outcome Measures:
  • Change From Baseline to Month 12 in Quality of Life Measured by the ITP-Patient Assessment Questionnaire (PAQ) [ Time Frame: Baseline to month 12 during prospective data collection phase ] [ Designated as safety issue: No ]
    The Immune Thrombocytopenic Purpura Patient Assessment Questionnaire (ITP-PAQ) was developed to assess disease-specific quality of life (QoL) in adults with ITP. It is a 44-item questionnaire that includes scales for physical health (symptoms, fatigue/sleep, bother, and activity), emotional health (psychological and fear), overall QoL, social activity, women's reproductive health, and work. Scores for each scale range from 0 (worst) to 100 (best).

  • Change From Baseline to Month 12 in Health-related Quality of Life Assessed by EuroQol Visual Analog Scale (EQ-5D VAS) [ Time Frame: Baseline to Month 12 during prospective data collection phase ] [ Designated as safety issue: No ]
    The EQ VAS records the respondent's self-rated health on a vertical, visual analogue scale where the endpoints are labelled 'Best imaginable health state' (score = 100) and 'Worst imaginable health state' (score = 0).

  • Change From Baseline to Month 12 in Treatment Satisfaction [ Time Frame: Baseline to Month 12 during prospective data collection phase ] [ Designated as safety issue: No ]
    Participant satisfaction with treatment was measured using the Treatment Satisfaction Questionnaire for Medication (TSQM), an 11-item questionnaire providing scores on four scales - side effects, effectiveness, convenience and global satisfaction. TSQM Scale scores range from 0 to 100 with higher scores indicating more satisfaction with treatment.

  • Number of Participants Receiving Drug Therapies for Treatment of ITP During the Prospective Phase [ Time Frame: 12 months (prospective data collection phase) ] [ Designated as safety issue: No ]
    The number of participants who received drug therapies for the treatment of ITP during the prospective phase of the study. Use of multiple medications by the same participants is possible.

  • Duration of Exposure to ITP Medication [ Time Frame: 12 months (prospective data collection phase) ] [ Designated as safety issue: No ]
    Duration of exposure to each ITP medication measured from enrollment until the end of the 12-month data collection phase.

  • Number of Participants Requiring Splenectomy [ Time Frame: 12 months ] [ Designated as safety issue: No ]
    The number of participants who required a splenectomy during the 12-month prospective phase of the study.


Biospecimen Retention:   None Retained

None retained.


Enrollment: 326
Study Start Date: May 2006
Study Completion Date: August 2008
Primary Completion Date: June 2008 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
Patients with ITP
Participants with ITP and currently treated for ITP were followed prospectively for a period of 12 months.
Other: Retrospective Chart Review
Retrospective chart review for up to 36 months of enrollment date.
Other: Patient-reported Outcome Questionnaires
Quality of lige questionnaires as well as treatment satisfaction questionnaires are required monthly. Includes QOL, ITP-PAQ (immune thrombocytopenic purpura - patient assessment questionnaire), EQ-5D, and TSQM (Treatment Satisfaction Questionnaire for Medication).
Other: Physician Survey
Physician characteristics to be collected include demographics, years of practice, medical specialty or primary treating physician, and the referral process.

Detailed Description:

Time Perspective: 12 months prospective chart review study with a retrospective chart review for a period up to 36 months from the start of the study, since the date of diagnosis for ITP.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population

Patients s diagnosed with ITP and currently treated for ITP by a hematologist or hematologist-oncologist recruited from community based clinics and adademic/referral centers.

Criteria

Inclusion Criteria:

  • Has a diagnosis of ITP according to the American Society of Hematology guidelines (George et al., 1996).
  • Is equal to or greater than 18 years of age.
  • Is willing and able to complete a series of questionnaires.
  • Before any study-specific procedure, the appropriate written informed consent must be obtained.

Exclusion Criteria:

  • Participated in clinical trial(s) during the past 36 months.
  • Is considering participation in a clinical trial within the next 12 months.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00454857

Sponsors and Collaborators
Amgen
Investigators
Study Director: MD Amgen
  More Information

Additional Information:
No publications provided

Responsible Party: Amgen
ClinicalTrials.gov Identifier: NCT00454857     History of Changes
Other Study ID Numbers: 20050237
Study First Received: March 29, 2007
Results First Received: October 16, 2009
Last Updated: October 23, 2013
Health Authority: Australia: Department of Health and Ageing Therapeutic Goods Administration
Australia: Human Research Ethics Committee
Australia: Therapeutic Goods Administration
Canada: Health Canada
Canada: Health Products and Food Branch
EU: CHMP
European Union: European Medicines Agency
France and Sweden: European Medicines Agency
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
France: Ministry of Health
Germany: Federal Institute for Drugs and Medical Devices
Germany: Paul_Ehrlich-Institut Bundesamt fur Sera und Impfstoffe
Italy: Ministry of Health
Spain: Spanish Agency of Medicines
Spain: Spanish Drug Agency
United Kingdom: Medicines and Healthcare Products Regulatory Agency
United States: Food and Drug Administration
United States: Institutional Review Board

Keywords provided by Amgen:
Thrombocytopenia
Chart Review
Platelet
Retrospective
Prospective
Non-interventional
Health Resource Utilization (HRU)
Quality of Life (QOL)
Immune Thrombocytopenic Purpura (ITP)
Idiopathic Thrombocytopenic Purpura (ITP)
Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
Observational

Additional relevant MeSH terms:
Purpura
Purpura, Thrombocytopenic
Thrombocytopenia
Purpura, Thrombocytopenic, Idiopathic
Blood Coagulation Disorders
Hematologic Diseases
Hemorrhage
Pathologic Processes
Skin Manifestations
Signs and Symptoms
Thrombotic Microangiopathies
Blood Platelet Disorders
Immune System Diseases
Hemorrhagic Disorders
Autoimmune Diseases

ClinicalTrials.gov processed this record on July 20, 2014