Clinical trial NCT00211029: 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
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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Red-Cell Aplasia, Pure
Keywords: Anemia, Hemoglobin, Red Blood Cells, Epoetin alfa
Epoetin
erythropoietin
Red Cell Aplasia, Pure Red Cell Aplasia
Chronic Renal Failure
Kidney Failure
Lead sponsor: Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
Sponsor class: INDUSTRY
First posted: 2005-09-21
Enrollment: 4670
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants with chronic renal disease receiving treatment with epoetin alfa or other recombinant erythropoietins.

Brief summary: 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.

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 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

Primary outcomes:
measure: Number of Participants With Pure Red Cell Aplasia (PRCA)
timeFrame: Up to 2 years
measure: Number of Participants With Positive Serum Erythropoietin (EPO) Antibodies Development Associated With Different Routes of Administration
timeFrame: Up to 2 years
measure: Relationship between EPO Antibodies and PRCA
timeFrame: Up to 2 years
Secondary outcomes:
measure: Number of Participants With Pure Red Cell Aplasia (PRCA) Associated With Different Routes of Administration
timeFrame: Every 3 Months up to up to 2 years
measure: Change From Baseline in Number of Participants With PRCA Over Time
timeFrame: Baseline and Month 3, 6, 9, 12, 15, 18, 21, 24
measure: Number of Participants With Pure Red Cell Aplasia (PRCA) and non-PRCA
timeFrame: Baseline and Month 3, 6, 9, 12, 15, 18, 21, 24
Other outcomes:
Locations:
Study officials:
affiliation: Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
name: Johnson & Johnson Pharmaceutical Research & Development, L.L. C. Clinical Trial
role: STUDY_DIRECTOR
References: