Clinical trial NCT00210977: A Study of Patients Treated With Erythropoietin Receptor Agonists for Anemia Who Developed Anti-erythropoietin Antibodies
Official title: An Observational Study of Subjects Treated With Erythropoietin Receptor Agonists for Anemia Who Developed Anti-erythropoietin Antibodies
Study type: OBSERVATIONAL
Overall status: WITHDRAWN
Conditions: Anemia
Keywords: Anemia
Erythropoietin
Epoetin alfa
Erythropoietin receptor agonist (ERA) therapy
Anti-erythropoietin antibodies (anti-EPO Ab)
Recombinant human erythropoietin
Pure red cell aplasia
Darbepoetin
Lead sponsor: Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
Sponsor class: INDUSTRY
First posted: 2005-09-21
Enrollment: 0
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants with positive or borderline serum anti erythropoietin antibody titer and who are responding to any erythropoietin receptor agonist (ERA) therapy for any cause of anemia

Brief summary: The purpose of this study is to assess the presence of anti erythropoietin antibodies (anti EPO Ab) in participants responding to any erythropoietin receptor agonist (ERA) therapy to treat or prevent anemia without loss of effectiveness to see if they will develop pure red cell aplasia or loss of effectiveness to recombinant erythropoietin and to measure the duration of effectiveness of ERA therapy.

Detailed description: This is an observational (study in which the investigators/physicians observe the participant's data and measure their outcomes), international, multicenter (study conducted at multiple sites), cohort (group of individuals with similar characteristics) study. The study consists of prestudy phase, observational phase (2 years), and follow-up phase (1 year). Approximately 50 participants who already receiving ERA therapy for anemia will be observed in this study. In the observational phase, participants' erythropoietin antibody status (positive or negative), clinical progress, treatment for anemia or prevention of anemia, and outcome will be monitored. During this study, enrolled participants will continue to receive standard-of-care treatment for their disease from their individual investigators as before enrollment to this study. Safety evaluations will include assessment of adverse events which will be monitored throughout the study. The total duration of the study will be 3 years.

Eligibility criteria: Inclusion Criteria:

* Receiving any ERA therapy for any cause of anemia and having responded to the ERA therapy for at least 3 months
* Positive or borderline serum for antibodies to erythropoietin identified by serum radioimmunoprecipitation
* Maintained a stable hemoglobin within a 3-month period that has not decreased more than 2 g/dL in any 1-month period and with no increase in red blood cell transfusion requirement
* An ERA maintenance dose that has not increased more than 50 percentages from initial effective dose
* If blood count information is available, must have a count of reticulocytes (immature red blood cells) greater than or equal to 30 million/L

Exclusion Criteria:

* Stem cell or bone marrow transplantation
* Treatment with medication that decreases the ability of the immune system to function normally, within the last 3 months
* Participants who have shown a loss of effectiveness to ERA therapy, defined as participants who initially responded to treatment for anemia \[a rise in hemoglobin which lasted for a minimum of 3 months\] followed by an unexplained decrease in hemoglobin (greater than or equal to 2 g/dL) within a 1-month period

Primary outcomes:
measure: Number of Participants With Presence of Anti-Erythropoietin Antibodies (anti-EPO Ab)
timeFrame: Up to 2 years
measure: Duration of Effectiveness of Erythropoietin Receptor Agonist Therapy
timeFrame: Up to 2 years
Secondary outcomes:
measure: Concentration of Plasma Anti Erythropoietin Antibody
timeFrame: Up to 2 years
measure: Factors That Forecast Progression to Loss of Effectiveness
timeFrame: Up to 2 years
measure: Factors That Forecast Progression to Suspected Pure Red Cell Aplasia
timeFrame: Up to 2 years
measure: Factors That Forecast Progression to Confirmed Pure Red Cell Aplasia
timeFrame: Up to 2 years
measure: Number of Participants With Adverse Events
timeFrame: Up to 3 years
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: