Clinical trial NCT00211042: A Study of Patients With Pure Red Cell Aplasia Associated With Recombinant Human Erythropoietin Treatment
Official title: Observational, Multicenter Study of Subjects With Pure Red Cell Aplasia Associated With r-HuEPO Treatment
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Pure Red-cell Aplasia
Keywords: Pure red-cell aplasia
Anemia
Red blood cells
Epoetin alfa (Eprex)
Anti-erythropoietin antibodies
Recombinant human erythropoietin
Lead sponsor: Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
Sponsor class: INDUSTRY
First posted: 2005-09-21
Enrollment: 52
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants diagnosed with Pure Red Cell Aplasia (PRCA)

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.

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 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

Primary outcomes:
measure: Number of participants with Pure Red Cell Aplasia (PRCA) outcome (Initial observation phase)
timeFrame: Up to 24 months after the date of loss of efficacy
measure: Number of participants with pure red cell aplasia outcome (Extended observation phase)
timeFrame: Up to 2 years after the enrollment in the extended observation phase
measure: Overall clinical outcome of pure red cell aplasia (Initial observation phase)
timeFrame: Up to 24 months after the date of loss of efficacy
measure: Overall clinical outcome of pure red cell aplasia (Extended observation phase)
timeFrame: Up to 2 years after the enrollment in the extended observation phase
Secondary outcomes:
measure: Different treatment modalities with pure red cell aplasia outcome (Initial observation phase)
timeFrame: Up to 24 months after the date of loss of efficacy
measure: Different treatment modalities with pure red cell aplasia outcome (Extended observation phase)
timeFrame: Up to 2 years after the enrollment in the extended observation phase
measure: Risk factors for Loss of Efficacy (LOE) and pure red cell aplasia (PRCA) outcome
timeFrame: Period between LOE date and date of enrollment in the study
Other outcomes:
Locations:
city: Santos
country: Brazil
city: Sao Paulo
country: Brazil
city: Saskatoon
country: Canada
state: Saskatchewan
city: Darmstadt
country: Germany
city: Hann. Münden
country: Germany
city: Tvnsberg
country: Norway
city: Bloemfontein
country: South Africa
city: Karlshamn
country: Sweden
city: Linköping
country: Sweden
city: Stockholm N/A
country: Sweden
city: Trollhättan
country: Sweden
city: N/a N/a
country: Thailand
city: Birmingham
country: United Kingdom
city: Chelmsford
country: United Kingdom
city: Edinburgh
country: United Kingdom
city: London
country: United Kingdom
city: Manchester
country: United Kingdom
city: Santander N/A
country: United Kingdom
city: Telford
country: United Kingdom
city: Valencia
country: United Kingdom
city: Westcliff-On-Sea
country: United Kingdom
Study officials:
affiliation: Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
name: Johnson & Johnson Pharmaceutical Research and Development, L. L. C. Clinical trial
role: STUDY_DIRECTOR
References: