A Study to Observe the Number of Patients Who Develop Pure Red Cell Aplasia (PRCA, a Rare Form of Anemia) While Receiving Epoetin Alfa or Other Recombinant Erythropoietins

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
ClinicalTrials.gov Identifier:
NCT00211029
First received: September 13, 2005
Last updated: June 29, 2015
Last verified: June 2015
  Purpose

The purpose of this study is to record the number of participants with chronic renal disease who are receiving treatment with epoetin alfa or other recombinant erythropoietins who develop pure red cell aplasia (PRCA, a rare form of anemia) during the study period.


Condition Intervention Phase
Red-Cell Aplasia, Pure
Drug: No Intervention
Phase 4

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: An Active Safety Surveillance Plan to Prospectively Monitor the Incidence of Pure Red Cell Aplasia (PRCA) Among Patients Receiving Epoetin Alfa Therapy or Other Recombinant Erythropoietins

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) [ Time Frame: Up to 2 years ] [ Designated as safety issue: No ]
    The PRCA is the suppression of erythropoiesis with little or no abnormality of leukocyte or platelet production.

  • Number of Participants With Positive Serum Erythropoietin (EPO) Antibodies Development Associated With Different Routes of Administration [ Time Frame: Up to 2 years ] [ Designated as safety issue: No ]
    Antibodies are immunoglobulin molecules having a specific amino acid sequence by virtue of which they interact only with the antigen (or a very similar shape) that induced their synthesis in cells of the lymphoid series.

  • Relationship between EPO Antibodies and PRCA [ Time Frame: Up to 2 years ] [ Designated as safety issue: No ]
    Natural history of EPO antibodies will be examined and its relationship to PRCA will be checked.


Secondary Outcome Measures:
  • Number of Participants With Pure Red Cell Aplasia (PRCA) Associated With Different Routes of Administration [ Time Frame: Every 3 Months up to up to 2 years ] [ Designated as safety issue: No ]
    The PRCA is the suppression of erythropoiesis with little or no abnormality of leukocyte or platelet production.

  • Change From Baseline in Number of Participants With PRCA Over Time [ Time Frame: Baseline and Month 3, 6, 9, 12, 15, 18, 21, 24 ] [ Designated as safety issue: No ]
    The PRCA is the suppression of erythropoiesis with little or no abnormality of leukocyte or platelet production.

  • Number of Participants With Pure Red Cell Aplasia (PRCA) and non-PRCA [ Time Frame: Baseline and Month 3, 6, 9, 12, 15, 18, 21, 24 ] [ Designated as safety issue: No ]
    Data will be collected from PRCA cases and non-PRCA cases to assist with identification of risk factors for PRCA such as demographic profile and duration of exposure to epoetin alfa or other recombinant erythropoietins.


Enrollment: 4670
Study Start Date: January 2003
Study Completion Date: July 2005
Primary Completion Date: July 2005 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
Participants Receiving Epoetin Alfa or Another Erythropoietin
Participants will not receive any intervention in this study. Participants with chronic renal disease receiving treatment with epoetin alfa or other recombinant erythropoietins will be prospectively observed to monitor incidence of pure red cell aplasia and/or antibodies to erythropoietin. Participants will receive standard-of-care treatment for their chronic renal (or other) disease from their individual Investigators.
Drug: No Intervention
Participants will not receive any intervention in this study. Participants with chronic renal disease receiving treatment with epoetin alfa or other recombinant erythropoietins will be prospectively observed to monitor incidence of pure red cell aplasia and/or antibodies to erythropoietin. Participants will receive standard-of-care treatment for their chronic renal (or other) disease from their individual Investigators.

Detailed Description:

This is a prospective (observation of a population for a sufficient number of persons over a sufficient number of years to generate incidence or mortality rates subsequent to the selection of the study group), observational (clinical study in which participants may receive diagnostic, therapeutic, or other types of interventions, but the investigator does not assign participants to specific interventions) and cohort study. Study specific information will be collected from participants with chronic renal disease who are receiving recombinant erythropoietin and their treatment course will be followed for up to 2 years and participants will not receive any intervention in this study. Upon entering the study, information will be collected regarding disease history and recombinant erythropoietin treatment. Every 3 months thereafter, progress information will be collected, including recombinant erythropoietin treatment, number of red blood cells and presence of any signs of pure red cell aplasia (PRCA) development. The results of this study will be combined with a similar study (EPO-IMU-402) that also collects blood samples to observe the number of patients who develop antibodies to erythropoietin. Participants discontinuing erythropoietin will be followed only for an additional 12 months from the time erythropoietin is discontinued. Participants will receive standard-of-care treatment for their chronic renal disease from their individual Investigators. Participants will be primarily observed prospectively for PRCA. The study hypothesis is that these data will provide a framework for the expansion of the patient population to estimate the incidence of PRCA associated with the use of epoetin alfa or other recombinant erythropoietins for other therapeutic indications.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Participants with chronic renal disease receiving treatment with epoetin alfa or other recombinant erythropoietins.

Criteria

Inclusion Criteria:

  • Male or female patients of legal age to give consent according to local standards
  • Patients must be receiving or about to receive treatment with an erythropoietin as part of a pre-existing treatment plan for their chronic renal (or other) disease
  • Physicians treating the patients must agree in advance to document any appearance of pure red cell aplasia (PRCA) such that information and details of the case may be reported to regulatory agencies (with patient identification protected)
  • If required by local ethics committees, patients must give consent to permit the collection of de-identified personal data for the specific purpose of this study

Exclusion Criteria:

  • Patients who are unable to complete future follow-up visits
  • Patients who when entering the study have any of the following symptoms of pure red cell aplasia (PRCA): Loss of effectiveness of erythropoietin or low number of immature red blood cells while on erythropoietin as shown by a blood test or bone marrow examination
  • Patients with a history of PRCA or loss of effectiveness with erythropoietin at the time of enrollment into the study
  • Patients whose anemia did not respond to previous treatment with an erythropoietin
  • Patients with a history of antibodies to erythropoietin prior to entering the study
  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: NCT00211029

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

Additional Information:
No publications provided

Responsible Party: Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
ClinicalTrials.gov Identifier: NCT00211029     History of Changes
Other Study ID Numbers: CR004405, EPO-IMU-401
Study First Received: September 13, 2005
Last Updated: June 29, 2015
Health Authority: United States: Institutional Review Board

Keywords provided by Johnson & Johnson Pharmaceutical Research & Development, L.L.C.:
Anemia, Hemoglobin, Red Blood Cells, Epoetin alfa
Epoetin
erythropoietin
Red Cell Aplasia, Pure Red Cell Aplasia
Chronic Renal Failure
Kidney Failure

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 July 28, 2015