A Study of Patients With Pure Red Cell Aplasia Associated With Recombinant Human Erythropoietin Treatment

Pure Red-cell Aplasia Clinical Trial

Official title:

Observational, Multicenter Study of Subjects With Pure Red Cell Aplasia Associated With r-HuEPO Treatment

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00211042
• Other study ID #: CR004393
• Secondary ID: EPO-IMU-301
• Status: Completed
• Phase: Phase 4
• First received: September 13, 2005
• Last updated: April 29, 2013
• Start date: February 2004
• Est. completion date: December 2006

Study information

• Verified date: April 2013
• Source: Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
• Contact: n/a
• Is FDA regulated: No
• Health authority: Thailand: Food and Drug Administration
• Study type: Observational

Clinical Trial Summary

The purpose of this study is to investigate the relationship of anti-erythropoietin antibodies to the clinical course and outcome of pure red cell aplasia (PRCA) in participants currently or previously treated with recombinant human erythropoietin.

Description:

This is a multicenter (study conducted at multiple sites), observational (study in which the investigators/physicians observe the participant's data and measure their outcomes) study. Approximately 150 participants will be enrolled in this study. The study consists of an initial observation phase and extended observation period. An initial observation phase starting at enrollment and ending when 24 months have elapsed since the date of loss of efficacy (LOE), supplemented with retrospective data collection for the period between LOE date and date of enrollment in the study. Participants remaining epoetin alfa (EPO-Ab) positive 24 months after LOE will enter a 2-year extended observation period. Study visits will take place every month during the initial observation phase and data will be collected every 6 months during the extended observation phase. Safety evaluations will include assessment of adverse events, clinical laboratory tests, vital signs, and physical examination which will be monitored throughout the study. The total study duration for each participant will be approximately for 4 years.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 52
• Est. completion date: December 2006
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• Pure red cell aplasia (PRCA) associated with recombinant human erythropoietin (r-HuEPO) treatment

• Anemia unresponsive to r-HuEPO treatment

• PRCA associated with erythropoietin treatment followed by a sudden decrease (more than or equal to 2 gram per deciliter within 30 days) in a previously stable hemoglobin level

Exclusion criteria:

• Participants who are not fulfilling the inclusion criteria

Study Design

Observational Model: Case-Only, Time Perspective: Prospective

Related Conditions & MeSH terms

• Pure Red-cell Aplasia
• Red-Cell Aplasia, Pure

Intervention

Other:
• No intervention
This is an observational study. No medication will be given to the participants. Participants will receive standard-of-care treatment from their individual physicians.

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Johnson & Johnson Pharmaceutical Research & Development, L.L.C.

Countries where clinical trial is conducted

Brazil,  Canada,  Germany,  Norway,  South Africa,  Sweden,  Thailand,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of participants with Pure Red Cell Aplasia (PRCA) outcome (Initial observation phase)	The PRCA outcome is measured by anti-epoetin alfa qualitative test. Persistence of PRCA is defined as: 1) absolute reticulocyte count less than 30,000 per cubic millimeter; and/or 2) no reversal of erythroblastopenia on repeated bone marrow testing. Resolution of PRCA is defined as: 1) absolute reticulocyte count greater than or equal to 30,000 per cubic millimeter; and/or 2) reversal of erythroblastopenia on repeated bone marrow testing.	Up to 24 months after the date of loss of efficacy	No
Primary	Number of participants with pure red cell aplasia outcome (Extended observation phase)	Participants remaining anti-epoetin alfa positive 24 months after loss of efficacy will enter in the extended observation period.	Up to 2 years after the enrollment in the extended observation phase	No
Primary	Overall clinical outcome of pure red cell aplasia (Initial observation phase)	The overall clinical outcome is evaluated by anti-epoetin alfa qualitative test. Overall clinical status will be recorded at each visit in the initial and extended observation phases using a categorical scale (improved, same, worsened, death). In case of death, the date and cause of death along with the the date and cause of death will be recorded.	Up to 24 months after the date of loss of efficacy	No
Primary	Overall clinical outcome of pure red cell aplasia (Extended observation phase)		Up to 2 years after the enrollment in the extended observation phase	No
Secondary	Different treatment modalities with pure red cell aplasia outcome (Initial observation phase)		Up to 24 months after the date of loss of efficacy	No
Secondary	Different treatment modalities with pure red cell aplasia outcome (Extended observation phase)		Up to 2 years after the enrollment in the extended observation phase	No
Secondary	Risk factors for Loss of Efficacy (LOE) and pure red cell aplasia (PRCA) outcome	This data will be collected retrospectively and the date of LOE will be determined by the sponsor based upon reported data. PRCA duration groups will be summarized by potential risk factors to evaluate the relationship of risk factors to the duration of PRCA.	Period between LOE date and date of enrollment in the study	No