Pure Red-cell AplasiaNCT00211042
A Study of Patients With Pure Red Cell Aplasia Associated With Recombinant Human Erythropoietin Treatment
An observational study of Pure Red-cell Aplasia, run by Johnson & Johnson Pharmaceutical Research & Development, L.L.C. at 21 sites in 8 countries.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Pure Red-cell Aplasia
- How many people
- 52 participants
- Where
- 21 sitesin 8 countries
- Study dates
- Feb 2004to Dec 2006
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Participants diagnosed with Pure Red Cell Aplasia (PRCA)
Who may be able to join
- 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
Who cannot join
- Participants who are not fulfilling the inclusion criteria
Trial locations and contacts
21 sites in 8 countries.
21 sites in 8 countries
United Kingdom9 sites
Study site
Birmingham
Study site
Chelmsford
Study site
Edinburgh
Study site
London
Study site
Manchester
Study site
Santander N/A
Study site
Telford
Study site
Valencia
Study site
Westcliff-on-Sea
Sweden4 sites
Study site
Karlshamn
Study site
Linköping
Study site
Stockholm
Study site
Trollhättan
About this study
Official title: Observational, Multicenter Study of Subjects With Pure Red Cell Aplasia Associated With r-HuEPO Treatment
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.
Read the 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.
- Conditions
- Pure Red-cell Aplasia
- Being tested
- No intervention
- Keywords
- Pure red-cell aplasia, Anemia, Red blood cells, Epoetin alfa (Eprex), Anti-erythropoietin antibodies, Recombinant human erythropoietin
- Medical subject headings
- Red-Cell Aplasia, Pure, Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No interventionOther
This is an observational study. No medication will be given to the participants. Participants will receive standard-of-care treatment from their individual physicians.
| Study group | Role | What they receive |
|---|---|---|
| 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). Receives: Other: No intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with Pure Red Cell Aplasia (PRCA) outcome (Initial observation phase)
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.
Measured: Up to 24 months after the date of loss of efficacy
Number of participants with pure red cell aplasia outcome (Extended observation phase)
Participants remaining anti-epoetin alfa positive 24 months after loss of efficacy will enter in the extended observation period.
Measured: Up to 2 years after the enrollment in the extended observation phase
Overall clinical outcome of pure red cell aplasia (Initial observation phase)
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.
Measured: Up to 24 months after the date of loss of efficacy
Overall clinical outcome of pure red cell aplasia (Extended observation phase)
Measured: Up to 2 years after the enrollment in the extended observation phase
Secondary outcomes (3)
Different treatment modalities with pure red cell aplasia outcome (Initial observation phase)
Measured: Up to 24 months after the date of loss of efficacy
Different treatment modalities with pure red cell aplasia outcome (Extended observation phase)
Measured: Up to 2 years after the enrollment in the extended observation phase
Risk factors for Loss of Efficacy (LOE) and pure red cell aplasia (PRCA) outcome
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.
Measured: Period between LOE date and date of enrollment in the study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Sep 2005 (estimated)
- Study startFeb 2004
- Study completionDec 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pure Red-cell AplasiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 52 participants (actual)
Sponsor and investigators
- Lead sponsor
- Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Johnson & Johnson Pharmaceutical Research and Development, L. L. C. Clinical trial, Study director, Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00211042
- Sponsor's protocol ID
- CR004393
- Other IDs
- EPO-IMU-301
- Status last verified
- Apr 2013
- First submitted
- 13 Sep 2005
- Last updated
- 30 Apr 2013
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 30 Apr 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Observational, Multicenter Study of Subjects With Pure Red Cell Aplasia Associated With r-HuEPO Treatment
- ClinicalTrials.gov ID
- NCT00211042
- Acronym / sponsor ID
- CR004393
- Conditions
- Pure Red-cell Aplasia
- Keywords
- Pure red-cell aplasia, Anemia, Red blood cells, Epoetin alfa (Eprex), Anti-erythropoietin antibodies, Recombinant human erythropoietin
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 52 participants (actual)
- Lead sponsor
- Johnson & Johnson Pharmaceutical Research & Development, L.L.C.Industry (pharma, biotech, devices)
- Study start
- Feb 2004
- Study completion
- Dec 2006 (actual)
- First posted
- 21 Sep 2005 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pure Red Cell Aplasia (PRCA) | – | Other: No interventionThis 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). |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Participants diagnosed with Pure Red Cell Aplasia (PRCA)
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (1 listed)
- Participants who are not fulfilling the inclusion criteria
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| No interventionThis is an observational study. No medication will be given to the participants. Participants will receive standard-of-care treatment from their individual physicians. | Other | Pure Red Cell Aplasia (PRCA) |
Primary outcome
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
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
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
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
3 secondary outcomes
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
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
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Apr 2013.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Johnson & Johnson Pharmaceutical Research and Development, L. L. C. Clinical trial study director
Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
21 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Pure Red-cell Aplasia, run by Johnson & Johnson Pharmaceutical Research & Development, L.L.C. at 21 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 Apr 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 21 sites in United Kingdom, Sweden, Brazil, Germany, Canada and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Apr 2013.