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

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

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.

Study Overview

• Status: Completed
• Conditions: Pure Red-cell Aplasia
• Intervention / Treatment: Other: No intervention

Detailed 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.

• Study Type: Observational
• Enrollment (Actual): 52

Contacts and Locations

Study Locations

• Brazil
  • Santos, Brazil
  • Sao Paulo, Brazil
• Canada
  • Saskatchewan
    • Saskatoon, Saskatchewan, Canada
• Germany
  • Darmstadt, Germany
  • Hann. Münden, Germany
• Norway
  • Tvnsberg, Norway
• South Africa
  • Bloemfontein, South Africa
• Sweden
  • Karlshamn, Sweden
  • Linköping, Sweden
  • Stockholm N/A, Sweden
  • Trollhättan, Sweden
• Thailand
  • N/a N/a, Thailand
• United Kingdom
  • Birmingham, United Kingdom
  • Chelmsford, United Kingdom
  • Edinburgh, United Kingdom
  • London, United Kingdom
  • Manchester, United Kingdom
  • Santander N/A, United Kingdom
  • Telford, United Kingdom
  • Valencia, United Kingdom
  • Westcliff-On-Sea, United Kingdom

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Participants diagnosed with Pure Red Cell Aplasia (PRCA)

Description

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 Plan

How is the study designed?

Design Details

• Observational Models: Case-Only
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Pure Red Cell Aplasia (PRCA); This study will examine the relationship of the presence of anti-erythropoietin antibodies to the clinical course and outcome of participants currently or previously treated with recombinant human erythropoietin and who have PRCA identified from all notified reports (spontaneous postmarketing reports or from clinical trials reports).	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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
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
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
Overall clinical outcome of pure red cell aplasia (Extended observation phase)		Up to 2 years after the enrollment in the extended observation phase

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Different treatment modalities with pure red cell aplasia outcome (Initial observation phase)		Up to 24 months after the date of loss of efficacy
Different treatment modalities with pure red cell aplasia outcome (Extended observation phase)		Up to 2 years after the enrollment in the extended observation phase
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

Collaborators and Investigators

• Sponsor: Johnson & Johnson Pharmaceutical Research & Development, L.L.C.

Study record dates

Study Major Dates

• Study Start: February 1, 2004
• Study Completion (Actual): December 1, 2006

Study Registration Dates

• First Submitted: September 13, 2005
• First Submitted That Met QC Criteria: September 13, 2005
• First Posted (Estimate): September 21, 2005

Study Record Updates

• Last Update Posted (Estimate): April 30, 2013
• Last Update Submitted That Met QC Criteria: April 29, 2013
• Last Verified: April 1, 2013

More Information

Terms related to this study

• Keywords: Anemia; Recombinant human erythropoietin; Red blood cells; Pure red-cell aplasia; Epoetin alfa (Eprex); Anti-erythropoietin antibodies
• Additional Relevant MeSH Terms: Hematologic Diseases; Anemia; Red-Cell Aplasia, Pure
• Other Study ID Numbers: CR004393; EPO-IMU-301 (Other Identifier: Johnson & Johnson Pharmaceutical Research and Development, L.L.C.)