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

Thrombocytopenia Clinical Trial

Official title:

A Prospective Observational Descriptive Study and Retrospective Chart Review of Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00454857
• Other study ID #: 20050237
• Status: Completed
• Phase: N/A
• First received: March 29, 2007
• Last updated: October 23, 2013
• Start date: May 2006
• Est. completion date: August 2008

Study information

• Verified date: October 2013
• Source: Amgen
• Contact: n/a
• Is FDA regulated: No
• Health authority: Australia: Department of Health and Ageing Therapeutic Goods AdministrationAustralia: Human Research Ethics CommitteeAustralia: Therapeutic Goods AdministrationCanada: Health CanadaCanada: Health Products and Food BranchEU: CHMPEuropean Union: European Medicines AgencyFrance and Sweden: European Medicines AgencyFrance: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)France: Ministry of HealthGermany: Federal Institute for Drugs and Medical DevicesGermany: Paul_Ehrlich-Institut Bundesamt fur Sera und ImpfstoffeItaly: Ministry of HealthSpain: Spanish Agency of MedicinesSpain: Spanish Drug AgencyUnited Kingdom: Medicines and Healthcare Products Regulatory AgencyUnited States: Food and Drug AdministrationUnited States: Institutional Review Board
• Study type: Observational

Clinical Trial Summary

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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 326
• Est. completion date: August 2008
• Est. primary completion date: June 2008
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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.

Study Design

Observational Model: Cohort

Related Conditions & MeSH terms

• Idiopathic Thrombocytopenic Purpura
• Purpura
• Purpura, Thrombocytopenic
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia
• Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
• Thrombocytopenic Purpura
• Thrombocytosis

Intervention

Other:
• Retrospective Chart Review
Retrospective chart review for up to 36 months of enrollment date.
• 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).
• Physician Survey
Physician characteristics to be collected include demographics, years of practice, medical specialty or primary treating physician, and the referral process.

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Amgen

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants Utilizing Immune (Idiopathic) Thrombocytopenic Purpura (ITP) Therapies for First-line Treatment	The number of participants who received ITP therapies for first-line treatment during either the retrospective or prospective phases of the study.	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).	No
Primary	The Number of Participants Utilizing ITP Therapies for Second-line Treatment.	The number of participants who received ITP therapies for second-line treatment during either the retrospective or prospective phases of the study.	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).	No
Primary	The Number of Participants Utilizing ITP Therapies for Third-line Treatment.	The number of participants who received ITP therapies for third-line treatment during either the retrospective or prospective phases of the study.	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).	No
Primary	The Number of Participants Utilizing ITP Therapies for Fourth-line Treatment.	The number of participants who received ITP therapies for fourth-line treatment during either the retrospective or prospective phases of the study.	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).	No
Primary	The Number of Participants Utilizing ITP Therapies for Fifth-line Treatment	The number of participants who received ITP therapies for fifth-line treatment during either the retrospective or prospective phases of the study.	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).	No
Primary	The Number of Participants Utilizing ITP Therapies for Sixth-line Treatment.	Assessed the number of participants who received ITP therapies for sixth-line treatment during either the retrospective or prospective phases of the study.	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).	No
Primary	The Number of Participants Utilizing ITP Therapies for Seventh or Greater-line Treatment.	The number participants who received ITP therapies as seventh or greater-line treatment during either the retrospective or prospective phases of the study.	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).	No
Primary	The Number of Participants Utilizing ITP Therapies: Treatments With Unknown Starting Date.	The number of participants who received ITP therapies for treatments with unknown starting date during either the retrospective or prospective phases of the study.	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).	No
Secondary	Change From Baseline to Month 12 in Quality of Life Measured by the ITP-Patient Assessment Questionnaire (PAQ)	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).	Baseline to month 12 during prospective data collection phase	No
Secondary	Change From Baseline to Month 12 in Health-related Quality of Life Assessed by EuroQol Visual Analog Scale (EQ-5D VAS)	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).	Baseline to Month 12 during prospective data collection phase	No
Secondary	Change From Baseline to Month 12 in Treatment Satisfaction	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.	Baseline to Month 12 during prospective data collection phase	No
Secondary	Number of Participants Receiving Drug Therapies for Treatment of ITP During the Prospective Phase	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.	12 months (prospective data collection phase)	No
Secondary	Duration of Exposure to ITP Medication	Duration of exposure to each ITP medication measured from enrollment until the end of the 12-month data collection phase.	12 months (prospective data collection phase)	No
Secondary	Number of Participants Requiring Splenectomy	The number of participants who required a splenectomy during the 12-month prospective phase of the study.	12 months	No

External Links

•   AmgenTrials clinical trials website
•   FDA-approved Drug Labeling