Hemoglobinuria, ParoxysmalPhase 3NCT00122304
Study of Safety in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Treated With Eculizumab
A Phase 3 study testing eculizumab in people with Hemoglobinuria, Paroxysmal, run by Alexion Pharmaceuticals, Inc. at 48 sites in 13 countries.
Trial summary
The primary objective is to evaluate the safety of eculizumab in patients with transfusion-dependent hemolytic PNH Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hemoglobinuria, Paroxysmal
- What is being tested?
- eculizumab
- How many people
- 85 participants
- Where
- 48 sitesin 13 countries
- Study dates
- Dec 2004to Nov 2006
- Phase
- Phase 3
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- PNH > 6 months
- Type III PNH red blood cell (RBC) clone by flow cytometry >10%
- At least one transfusion in the past 2 years but no more than 3 transfusions in the past 12 months; or personal beliefs that preclude the use of transfusion with severe hemolytic PNH
- Lactate dehydrogenase (LDH) >1.5 x upper limit of normal
- Must avoid conception
- Willing and able to give written informed consent
Who cannot join
- Platelet count of <30,000/mm3
- Absolute neutrophil count <500/ul
- Active bacterial infection
- Hereditary complement deficiency
- History of bone marrow transplantation
- Participation in any other investigational drug trial or exposure to other investigational agent, device or procedure within 30 days
- Pregnant, breast-feeding, or intending to conceive
- History of meningococcal disease
Trial locations and contacts
48 sites in 13 countries.
48 sites in 13 countries
United States19 sites
Johns Hopkins University Medical Center
Baltimore, Maryland, 21205
National Heart, Lung, and Blood Institute, National Institutes of Health
Bethesda, Maryland, 20892
Massachusetts General Hospital
Boston, Massachusetts, 02114
Roswell Park Cancer Institute
Buffalo, New York, 14263
The Cleveland Clinic Foundation
Cleveland, Ohio, 44195
Duke University Medical Center, Division Cell Therapy, Heme Malignancies Program
Durham, North Carolina, 27710
Hartford Hospital, Cancer Clinical Research Office
Hartford, Connecticut, 06102
Indiana University Cancer Pavilion
Indianapolis, Indiana, 46202
Norris Comprehensive Cancer Center
Los Angeles, California, 90033
NYU Clinical Cancer Center
New York, New York, 10016
Memorial Sloan Kettering Cancer Center
New York, New York, 10017
Presbyterian Medical Center, PENN Comprehensive Hemophilia & Thrombosis Program
Philadelphia, Pennsylvania, 19104
OHSU Center for Hematologic Malignancies
Portland, Oregon, 97239
Mayo Clinic, Division of Hematology
Rochester, Minnesota, 55905
University of Utah, Hematology-BMT Department
Salt Lake City, Utah, 84132
Washington University Medical Center, Department of Internal Medicine/Division of Hematology
St Louis, Missouri, 63110
Stanford University Medical Center, Division of Hematology
Stanford, California, 94305
Cooper University Hospital, Cooper Cancer Institute
Voorhees Township, New Jersey, 08043
Cleveland Clinic Florida, Dept. of Clinical Research
Weston, Florida, 33331
Australia5 sites
Royal Melbourne Hospital, Dept. of Clinical Haematology & Medical Oncology
Parkville, 3050
The Royal Perth Hospital, Dept. of Haematology/Level 2
Perth, Western Australia, 6000
Royal North Shore Hospital, Haematology Department
Saint Leonard, New South Wales, 2065
The Queen Elizabeth Hospital, Haematology/Oncology Department
Woodville South, South Australia, 5011
Princess Alexandra Hospital, Oncology Haematology Radiation Department
Woolloongabba, Queensland, 4102
Italy5 sites
Azienda Ospedaliera Universitaria Careggi, Dipartimento di area critica medico-chirurgica
Florence, 50139
Ospedale San Martino, Dept. of Hematology
Genova, I-16132
Ospedale Maggiore di Milano, Divisione di Ematologia
Milan, 35-20122
Universita degli Studi di Napoli, Divisione di Ematologia, Azienda Universitar Policlinico
Naples, 5-80131
Ospedale San Bortolo, Divisione di Ematologia
Vicenza, 37-36100
Germany4 sites
Universitatsklinikum Essen, Zentrum fur Innere Medizin
Essen, D-45147
Medizinische Hochschule Hannover, Abt. Hamatologie/Onkologie, Zentrum fur Innere Medizin
Hanover, D-30625
Universitatskliniken des Saarlandes, Innere Medizin 1
Homburg/Saar, D-66421
Institut fur Klinische Transfusionmedizin und Immungenetik, Abtlg. Transfusionmedizin des Univer. Ulm
Ulm, D-89081
Spain3 sites
Hospital Clinic i Provincial, Servicio de Hematologia
Barcelona, 08036
Hospital Universitario Germans Trias I Pujol, Servicio de Hematologia
Barcelona, 08916
Hospital De La Paz, Servicio de Hematologia
Madrid, 28046
United Kingdom3 sites
Leeds General Infirmary, D Floor Brotherton Wing
Leeds, LS1 3EX
St. Georges Hospital, Department of Haematology
London, 17 OQT
Royal Cornwall Hospital
Truro, Cornwall, TR1 3LJ
Canada2 sites
University of Alberta, Cross Cancer Institute
Edmonton, Alberta, T6G 2B7
London Regional Cancer Centre, Clinical Research Unit Room C3080
London, Ontario, N6A 4L6
Sweden2 sites
Stockholm South Hospital, Division of Hematology
Stockholm, 118-83
University Hospital, Dept. of Haematology
Uppsala, SE-751
France1 site
Hopital Saint-Louis, Centre d'investigation Clinique
Paris, Cedex, 10 75475
Ireland1 site
St. James Hospital, Haematology Dept., Cancer Clinical Trial Office
Dublin, 8
Netherlands1 site
UMC St. Radboud, Department of Hematology
Nijmegen, 6525 GA
Switzerland1 site
Kantonsspital Basel, Abteilung fuer Haematologie
Basel, CH-4031
About this study
Official title: Safety in Hemolytic PNH Patients Treated With Eculizumab: a Multi-Center Open-Label Research Design Study
The primary objective is to evaluate the safety of eculizumab in patients with transfusion-dependent hemolytic PNH
- Conditions
- Hemoglobinuria, Paroxysmal
- Being tested
- eculizumab
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
eculizumabDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
Acceptable safety (adverse events [AEs], labs, electrocardiograms [ECGs], vital signs)
Primary surrogate of efficacy endpoint is hemolysis measured by LDH area under the curve.
Secondary outcomes (2)
Hemolysis measured by the change of LDH from baseline;
Quality of Life
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jul 2005 (estimated)
- Study startDec 2004
- Study completionNov 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemoglobinuria, ParoxysmalStudies 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
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 85 participants
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00122304
- Sponsor's protocol ID
- SHEPHERD
- Other IDs
- C04-002
- Status last verified
- Feb 2007
- First submitted
- 19 Jul 2005
- Last updated
- 21 Feb 2007
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Feb 2007. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety in Hemolytic PNH Patients Treated With Eculizumab: a Multi-Center Open-Label Research Design Study
- ClinicalTrials.gov ID
- NCT00122304
- Acronym / sponsor ID
- SHEPHERD
- Conditions
- Hemoglobinuria, Paroxysmal
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 85 participants
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- Dec 2004
- Study completion
- Nov 2006
- First posted
- 22 Jul 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 primary objective is to evaluate the safety of eculizumab in patients with transfusion-dependent hemolytic PNH
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- PNH > 6 months
- Type III PNH red blood cell (RBC) clone by flow cytometry >10%
- At least one transfusion in the past 2 years but no more than 3 transfusions in the past 12 months; or personal beliefs that preclude the use of transfusion with severe hemolytic PNH
- Lactate dehydrogenase (LDH) >1.5 x upper limit of normal
- Must avoid conception
Key exclusion criteria (8 listed)
- Platelet count of <30,000/mm3
- Absolute neutrophil count <500/ul
- Active bacterial infection
- Hereditary complement deficiency
- History of bone marrow transplantation
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 |
|---|---|---|
| eculizumab | Drug | – |
Primary outcome
Acceptable safety (adverse events [AEs], labs, electrocardiograms [ECGs], vital signs)
Primary surrogate of efficacy endpoint is hemolysis measured by LDH area under the curve.
2 secondary outcomes
Hemolysis measured by the change of LDH from baseline;
Quality of Life
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 21 Feb 2007.
Related trials for Hemoglobinuria, Paroxysmal
Enrolling by invitationObservational study
An Observational Post-Authorization Safety and Effectiveness Study of Ravulizumab in Adult Patients Aged 18 and Older Diagnosed With Paroxysmal Nocturnal Hemoglobinuria Using Multicenter Electronic Medical Records in South Korea
Hemoglobinuria, Paroxysmal
RecruitingPhase 2
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
RecruitingPhase 3
A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works
Paroxysmal Nocturnal Hemoglobinuria
Not yet recruitingPhase 3
A Study of Efficacy and Safety of FXS6837 in Anti-C5 Treated PNH Patients.
Paroxysmal Nocturnal Hemoglobinuria
RecruitingPhase 2
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
Severe Aplastic Anemia (SAA), Hypo-Plastic Myelodysplastic Syndrome (MDS)
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.
The record lists no contact. Use the official record to reach the sponsor.
48 sites in 13 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing eculizumab in people with Hemoglobinuria, Paroxysmal, run by Alexion Pharmaceuticals, Inc. at 48 sites in 13 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 6 inclusion and 8 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 21 Feb 2007. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 48 sites in United States, Australia, Italy, Germany, Spain and 8 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.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Acceptable safety (adverse events [AEs], labs, electrocardiograms [ECGs], vital signs). The study lists 2 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 21 Feb 2007.