Paroxysmal Nocturnal HemoglobinuriaNCT01412047
Paroxysmal Nocturnal Hemoglobinuria Human Anti-Human Antibodies Study
An observational study of Paroxysmal Nocturnal Hemoglobinuria and PNH, run by Alexion Pharmaceuticals, Inc. at 14 sites in 10 countries.
Trial summary
How does long term treatment with Soliris affect HAHA in PNH patients? Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria, PNH
- How many people
- 75 participants
- Where
- 14 sitesin 10 countries
- Study dates
- Mar 2012to 30 Jun 2013
- 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: Patients with PNH previously enrolled in study E05-001, who have a eculizumab naive serum sample available for comparison, and who are currently receiving treatment with commerical Soliris.
Who may be able to join
- PNH patients who previously participated in study E05-001;
- Patients who have an eculizumab naive serum sample for comparison;
- Patients who currently used commerical Soliris;
- Patients who are willing and able to gie written informed consent.
Who cannot join
- There are no exclusion criteria for this study.
Trial locations and contacts
14 sites in 10 countries.
14 sites in 10 countries
United States3 sites
University Park Hematology Oncology
Englewood, Colorado, 80113
Maine Cancer Center of Medicine
Scarborough, Maine, 04074
Cleveland Clinic Florida
Weston, Florida, 33331
Germany2 sites
Universitatsklinikum Essen
Essen
Institut fur Klinische Transfusionmedizin und Immungenetick
Ulm
Italy2 sites
Azienda Ospediliera Universitatia Careggi
Florence
Universita degli Studi di Napoli
Naples
About this study
Official title: A Post Marketing, Multi-Center, Trial to Measure Human Anti-Human Antibodies (HAHA) to Soliris (Eculizumab) in Patients With Paroxysmal Nocturnal Hemoglobinuria
How does long term treatment with Soliris affect HAHA in PNH patients?
Read the detailed description
To determine the long term effect of Soliris (eculizumab) treatment on the presence of human anti-human antibodies (HAHA) in Paroxysmal Nocturnal Hemoglobinuria (PNH) patients.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria, PNH
- Keywords
- Paroxysmal Nocturnal Hemoglobinuria, PNH, Human Anti-Human Antibody, HAHA, Soliris, eculizumab
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What is being measured
The primary outcome is the main question the study is designed to answer.
To summarize the proportion of patients with neutralizing HAHA antibodies.
Measured: Six (6) months
Secondary outcomes (2)
To summarize the proportion of patients with non-neutralizing HAHA antibodies.
Measured: Six (6) months
To summarize the proportion of patients with increased hemolysis in setting of neutralizing HAHA antibodies.
Measured: Six (6) months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Aug 2011 (estimated)
- Study startMar 2012
- Primary completionJun 2013
- Study completion30 Jun 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal HemoglobinuriaStudies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The sponsor's other registered studies.
- 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
- Other
- Time perspective
- Prospective
- Enrolment
- 75 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Camille Bedrosian, MD, Study director, Alexion Pharmaceuticals, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01412047
- Sponsor's protocol ID
- M07-003
- Status last verified
- Oct 2017
- First submitted
- 5 Aug 2011
- Last updated
- 18 Oct 2017
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Oct 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Post Marketing, Multi-Center, Trial to Measure Human Anti-Human Antibodies (HAHA) to Soliris (Eculizumab) in Patients With Paroxysmal Nocturnal Hemoglobinuria
- ClinicalTrials.gov ID
- NCT01412047
- Acronym / sponsor ID
- M07-003
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria, PNH
- Keywords
- Paroxysmal Nocturnal Hemoglobinuria, PNH, Human Anti-Human Antibody, HAHA, Soliris, eculizumab
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 75 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- Mar 2012
- Primary completion
- Jun 2013 (actual)
- Study completion
- 30 Jun 2013 (actual)
- First posted
- 8 Aug 2011 (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.
How does long term treatment with Soliris affect HAHA in PNH patients?
Detailed description
To determine the long term effect of Soliris (eculizumab) treatment on the presence of human anti-human antibodies (HAHA) in Paroxysmal Nocturnal Hemoglobinuria (PNH) patients.
Study design
- Observational model
- Other
- Time perspective
- Prospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with PNH previously enrolled in study E05-001, who have a eculizumab naive serum sample available for comparison, and who are currently receiving treatment with commerical Soliris.
Key inclusion criteria (4 listed)
- PNH patients who previously participated in study E05-001;
- Patients who have an eculizumab naive serum sample for comparison;
- Patients who currently used commerical Soliris;
- Patients who are willing and able to gie written informed consent.
Key exclusion criteria (1 listed)
- There are no exclusion criteria for this study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
To summarize the proportion of patients with neutralizing HAHA antibodies.
Time frame: Six (6) months
2 secondary outcomes
To summarize the proportion of patients with non-neutralizing HAHA antibodies.
Time frame: Six (6) months
To summarize the proportion of patients with increased hemolysis in setting of neutralizing HAHA antibodies.
Time frame: Six (6) months
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 18 Oct 2017.
- Individual participant dataSharing planned: No
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Not yet recruitingPhase 3
A Study of Efficacy and Safety of FXS6837 in Anti-C5 Treated PNH Patients.
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Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
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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.
Camille Bedrosian, MD study director
Alexion Pharmaceuticals, Inc.
14 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Paroxysmal Nocturnal Hemoglobinuria and PNH, run by Alexion Pharmaceuticals, Inc. at 14 sites in 10 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 4 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 18 Oct 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United States, Germany, Italy, Australia, Belgium and 5 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?
To summarize the proportion of patients with neutralizing HAHA antibodies. (time frame: Six (6) months). 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 18 Oct 2017.