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

NCT00211042 | Completed DMC

• 2 other identifiers: CR004393EPO-IMU-301
• Study Type: observational
• Target: 52
• Locations: 8 countries
• Sites: 21

Brief 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.

Conditions

Pure Red-cell Aplasia

Keywords

Pure red-cell aplasia; Anemia; Red blood cells; Epoetin alfa (Eprex); Anti-erythropoietin antibodies; Recombinant human erythropoietin

Outcome Measures

Primary Outcomes (4)

• 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 Outcomes (3)

• 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

  Period between LOE date and date of enrollment in the study

Study Arms (1)

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

Interventions

No intervention OTHER

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

Pure Red Cell Aplasia (PRCA)

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Participants diagnosed with Pure Red Cell Aplasia (PRCA)

You may qualify if:

• 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

Sponsors & Collaborators

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

Study Sites (21)

Unknown Facility

Santos, Brazil

Location

Unknown Facility

São Paulo, Brazil

Location

Unknown Facility

Saskatoon, Saskatchewan, Canada

Location

Unknown Facility

Darmstadt, Germany

Location

Unknown Facility

Hannoversch Münden, Germany

Location

Unknown Facility

Tvnsberg, Norway

Location

Unknown Facility

Bloemfontein, South Africa

Location

Unknown Facility

Karlshamn, Sweden

Location

Unknown Facility

Linköping, Sweden

Location

Unknown Facility

Stockholm, Sweden

Location

Unknown Facility

Trollhättan, Sweden

Location

Unknown Facility

N/a N/a, Thailand

Location

Unknown Facility

Birmingham, United Kingdom

Location

Unknown Facility

Chelmsford, United Kingdom

Location

Unknown Facility

Edinburgh, United Kingdom

Location

Unknown Facility

London, United Kingdom

Location

Unknown Facility

Manchester, United Kingdom

Location

Unknown Facility

Santander N/A, United Kingdom

Location

Unknown Facility

Telford, United Kingdom

Location

Unknown Facility

Valencia, United Kingdom

Location

Unknown Facility

Westcliff-on-Sea, United Kingdom

Location

MeSH Terms

Conditions

Red-Cell Aplasia, Pure; Anemia

Condition Hierarchy (Ancestors)

Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Johnson & Johnson Pharmaceutical Research and Development, L. L. C. Clinical trial

  Johnson & Johnson Pharmaceutical Research & Development, L.L.C.

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 13, 2005
• First Posted: September 21, 2005
• Study Start: February 1, 2004
• Study Completion: December 1, 2006
• Last Updated: April 30, 2013

Record last verified: 2013-04

Locations

GB (9); BR (2); ZA (1); SE (4); CA (1); DE (2); NO (1); TH (1)