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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
ClinicalTrials.gov Identifier:
NCT00211042
First received: September 13, 2005
Last updated: April 29, 2013
Last verified: April 2013
  Purpose

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.


Condition Intervention Phase
Pure Red-cell Aplasia
Other: No intervention
Phase 4

Study Type: Observational
Study Design: Observational Model: Case-Only
Time Perspective: Prospective
Official Title: Observational, Multicenter Study of Subjects With Pure Red Cell Aplasia Associated With r-HuEPO Treatment

Resource links provided by NLM:


Further study details as provided by Johnson & Johnson Pharmaceutical Research & Development, L.L.C.:

Primary Outcome Measures:
  • Number of participants with Pure Red Cell Aplasia (PRCA) outcome (Initial observation phase) [ Time Frame: Up to 24 months after the date of loss of efficacy ] [ Designated as safety issue: No ]
    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.

  • Number of participants with pure red cell aplasia outcome (Extended observation phase) [ Time Frame: Up to 2 years after the enrollment in the extended observation phase ] [ Designated as safety issue: No ]
    Participants remaining anti-epoetin alfa positive 24 months after loss of efficacy will enter in the extended observation period.

  • Overall clinical outcome of pure red cell aplasia (Initial observation phase) [ Time Frame: Up to 24 months after the date of loss of efficacy ] [ Designated as safety issue: No ]
    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.

  • Overall clinical outcome of pure red cell aplasia (Extended observation phase) [ Time Frame: Up to 2 years after the enrollment in the extended observation phase ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Different treatment modalities with pure red cell aplasia outcome (Initial observation phase) [ Time Frame: Up to 24 months after the date of loss of efficacy ] [ Designated as safety issue: No ]
  • Different treatment modalities with pure red cell aplasia outcome (Extended observation phase) [ Time Frame: Up to 2 years after the enrollment in the extended observation phase ] [ Designated as safety issue: No ]
  • Risk factors for Loss of Efficacy (LOE) and pure red cell aplasia (PRCA) outcome [ Time Frame: Period between LOE date and date of enrollment in the study ] [ Designated as safety issue: No ]
    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.


Enrollment: 52
Study Start Date: February 2004
Study Completion Date: December 2006
Groups/Cohorts Assigned Interventions
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.

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.

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Participants diagnosed with Pure Red Cell Aplasia (PRCA)

Criteria

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

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00211042

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
Sponsors and Collaborators
Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
Investigators
Study Director: Johnson & Johnson Pharmaceutical Research and Development, L. L. C. Clinical trial Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
  More Information

No publications provided

Responsible Party: Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
ClinicalTrials.gov Identifier: NCT00211042     History of Changes
Other Study ID Numbers: CR004393, EPO-IMU-301
Study First Received: September 13, 2005
Last Updated: April 29, 2013
Health Authority: Thailand: Food and Drug Administration

Keywords provided by Johnson & Johnson Pharmaceutical Research & Development, L.L.C.:
Pure red-cell aplasia
Anemia
Red blood cells
Epoetin alfa (Eprex)
Anti-erythropoietin antibodies
Recombinant human erythropoietin

Additional relevant MeSH terms:
Red-Cell Aplasia, Pure
Anemia
Hematologic Diseases
Epoetin alfa
Hematinics
Hematologic Agents
Pharmacologic Actions
Therapeutic Uses

ClinicalTrials.gov processed this record on November 20, 2014