Idiopathic Thrombocytopenic PurpuraNCT00454857

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

An observational study of Idiopathic Thrombocytopenic Purpura and Thrombocytopenia, run by Amgen.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Who is it for?: Ages 18 years and older
Conditions the study is for: Idiopathic Thrombocytopenic Purpura, Thrombocytopenia, Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
What is being tested?: Retrospective Chart Review, Patient-reported Outcome Questionnaires and Physician Survey
How many people: 326 participants
Where: No sites listed yet
Study dates: May 2006to Aug 2008
Phase: Observational
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

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.

Who may be able to join

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.

Who cannot join

Participated in clinical trial(s) during the past 36 months.
Is considering participation in a clinical trial within the next 12 months.

About this study

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

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.

Read the 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.

Conditions: Idiopathic Thrombocytopenic Purpura, Thrombocytopenia, Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP), Thrombocytopenic Purpura
Being tested: Retrospective Chart Review, Patient-reported Outcome Questionnaires, Physician Survey
Keywords: 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
Medical subject headings: Purpura, Thrombocytopenic, Idiopathic, Thrombocytopenia, Purpura, Thrombocytopenic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Retrospective Chart ReviewOther
Retrospective chart review for up to 36 months of enrollment date.
Patient-reported Outcome QuestionnairesOther
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 SurveyOther
Physician characteristics to be collected include demographics, years of practice, medical specialty or primary treating physician, and the referral process.

Study groups in NCT00454857
Study group	Role	What they receive
Patients with ITP		Participants with ITP and currently treated for ITP were followed prospectively for a period of 12 months. Receives: Other: Retrospective Chart Review; Other: Patient-reported Outcome Questionnaires; Other: Physician Survey

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: 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).
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.
Measured: 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).
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.
Measured: 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).
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.
Measured: 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).
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.
Measured: 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).
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.
Measured: 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).
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.
Measured: 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).
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.
Measured: 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).

Secondary outcomes (6)

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).
Measured: Baseline to month 12 during prospective data collection phase
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).
Measured: Baseline to Month 12 during prospective data collection phase
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.
Measured: Baseline to Month 12 during prospective data collection phase
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.
Measured: 12 months (prospective data collection phase)
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.
Measured: 12 months (prospective data collection phase)
Number of Participants Requiring Splenectomy
The number of participants who required a splenectomy during the 12-month prospective phase of the study.
Measured: 12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

May 2006Aug 2008

First posted2 Apr 2007 (estimated)
Study startMay 2006
Primary completionJun 2008
Study completionAug 2008

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Idiopathic Thrombocytopenic PurpuraStudies of the same condition, open and under way.
More trials run by AmgenThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Observational
Observational model: Cohort
Enrolment: 326 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00454857
Sponsor's protocol ID: 20050237
Status last verified: Oct 2013
First submitted: 29 Mar 2007
Last updated: 26 Nov 2013
Results posted: Yes, on ClinicalTrials.gov

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

Official record

RecruitingPhase 2
A Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP)
Primary Immune Thrombocytopenia (ITP), Primary Evans Syndrome (ES)
RecruitingPhase 1/2
A Study of Pirtobrutinib in Participants With Immune Thrombocytopenia
Immune Thrombocytopenia (ITP)
RecruitingPhase 3
A Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)
RecruitingPhase 2
Study to Assess the Safety and Tolerability of Tafasitamab in Adult Participants With Primary Autoimmune Blood Cell Disorders
Immune Thrombocytopenia
RecruitingPhase 3
A Follow-up Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)

All Idiopathic Thrombocytopenic Purpura trials

Study at a glance

From the registry record, last updated by the sponsor on 26 Nov 2013. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Prospective Observational Descriptive Study and Retrospective Chart Review of Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
ClinicalTrials.gov ID: NCT00454857
Acronym / sponsor ID: 20050237
Conditions: Idiopathic Thrombocytopenic Purpura, Thrombocytopenia, Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP), Thrombocytopenic Purpura
Keywords: Thrombocytopenia, Chart Review, Platelet, Retrospective, Prospective, Non-interventional, Health Resource Utilization (HRU), Quality of Life (QOL)
Study type: Observational
Recruitment status: Completed
Enrolment: 326 participants (actual)
Lead sponsor: AmgenIndustry (pharma, biotech, devices)
Study start: May 2006
Primary completion: Jun 2008 (actual)
Study completion: Aug 2008 (actual)
First posted: 2 Apr 2007 (estimated)
Results: Posted 12 May 2010

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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.

Study design

Observational model: Cohort
Number of arms: 1

Arm	Type	Receives
Patients with ITP	–	Other: Retrospective Chart Review, Other: Patient-reported Outcome Questionnaires, Other: Physician SurveyParticipants with ITP and currently treated for ITP were followed prospectively for a period of 12 months.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult
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.

Key inclusion criteria (4 listed)

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.

Key exclusion criteria (2 listed)

Participated in clinical trial(s) during the past 36 months.
Is considering participation in a clinical trial within the next 12 months.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Retrospective Chart ReviewRetrospective chart review for up to 36 months of enrollment date.	Other	Patients with ITP
Patient-reported Outcome QuestionnairesQuality 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	Patients with ITP
Physician SurveyPhysician characteristics to be collected include demographics, years of practice, medical specialty or primary treating physician, and the referral process.	Other	Patients with ITP

Primary outcome

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).
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).
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).
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).
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).
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).
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).
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).
The number of participants who received ITP therapies for treatments with unknown starting date during either the retrospective or prospective phases of the study.

6 secondary outcomes

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
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
Change From Baseline to Month 12 in Treatment Satisfaction
Time frame: Baseline to Month 12 during prospective data collection phase
Number of Participants Receiving Drug Therapies for Treatment of ITP During the Prospective Phase
Time frame: 12 months (prospective data collection phase)
Duration of Exposure to ITP Medication
Time frame: 12 months (prospective data collection phase)
Number of Participants Requiring Splenectomy
Time frame: 12 months

Results and safety

Results posted to the registry on 12 May 2010, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Patients With ITP	326	272

Primary outcome: 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).. Number. Unit: Participants

Measure	Patients With ITP n = 326
Corticosteroids	128
IVIG (Intravenous immunoglobulin)	37
Rituximab	3
Anti-D immunoglobulin	11
Splenectomy	74
Other	36

Population analysed: All enrolled participants

Primary outcome: The Number of Participants Utilizing ITP Therapies for Second-line Treatment.

Measure	Patients With ITP n = 326
Corticosteroids	98
IVIG	54
Rituximab	16
Anti-D immunoglobulin	16
Splenectomy	0
Other	48

Population analysed: All enrolled participants

Primary outcome: The Number of Participants Utilizing ITP Therapies for Third-line Treatment.

Measure	Patients With ITP n = 326
Corticosteroids	47
IVIG	50
Rituximab	15
Anti-D immunoglobulin	14
Splenectomy	0
Other	50

Population analysed: All enrolled participants

Primary outcome: The Number of Participants Utilizing ITP Therapies for Fourth-line Treatment.

Measure	Patients With ITP n = 326
Corticosteroids	26
IVIG	28
Rituximab	14
Anti-D immunoglobulin	14
Splenectomy	0
Other	56

Population analysed: All enrolled participants

6 secondary outcomes are reported in the full results.

Adverse events

Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Overall Study	0 of 0	0 of 0	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 26 Nov 2013.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
FDA-approved Drug LabelingLink provided by the sponsor.
AmgenTrials clinical trials websiteLink provided by the sponsor.

Related trials for Idiopathic Thrombocytopenic Purpura

RecruitingPhase 2
A Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP)
Primary Immune Thrombocytopenia (ITP), Primary Evans Syndrome (ES)
RecruitingPhase 1/2
A Study of Pirtobrutinib in Participants With Immune Thrombocytopenia
Immune Thrombocytopenia (ITP)
RecruitingPhase 3
A Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)
RecruitingPhase 2
Study to Assess the Safety and Tolerability of Tafasitamab in Adult Participants With Primary Autoimmune Blood Cell Disorders
Immune Thrombocytopenia
RecruitingPhase 3
A Follow-up Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

MD study director

Amgen

Questions about this trial

What is this clinical trial studying?

An observational study of Idiopathic Thrombocytopenic Purpura and Thrombocytopenia, run by Amgen.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 26 Nov 2013. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

Yes. Results are posted in the registry (first posted 12 May 2010). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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).). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00454857.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Nov 2013.