Hemoglobinuria, ParoxysmalPhase 3NCT00122330
Study Using Eculizumab in Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients
A Phase 3 study testing eculizumab in people with Hemoglobinuria, Paroxysmal, run by Alexion Pharmaceuticals, Inc. at 43 sites in 12 countries.
Trial summary
The primary objective is to evaluate the safety and efficacy of eculizumab in transfusion dependent patients with 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
- 75 participants
- Where
- 43 sitesin 12 countries
- Study dates
- Oct 2004to Jan 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
- Must have required at least 4 transfusions in the past 12 months
- PNH type III red blood cell (RBC) clone by flow cytometry of >10%
- Lactate dehydrogenase (LDH) level > 1.5 x upper limit of normal
- Platelet count > 100,000/mm3
- Patient taking erythropoietin must be on a stable dose for at least 26 weeks
- Patient taking immunosuppressants must be on a stable dose for at least 26 weeks
Show all 11Show fewer
- Patient taking corticosteroids must be on a stable dose for at least 4 weeks
- Patient taking coumadin must be at a stable INR for at least 4 weeks
- Patient taking iron supplements or folic acid must be on a stable dose for 4 weeks
- Willing and able to give written informed consent
- Must avoid conception
Who cannot join
- Mean hemoglobin level prior to transfusion over the previous 12 months is >10.5 gm/dl
- Absolute neutrophil count <500/ul
- Active bacterial infection
- Hereditary complement deficiency
- Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedures within 30 days
- Pregnant, breast-feeding, or intending to conceive
- History of meningococcal disease
- History of bone marrow transplantation
Trial locations and contacts
43 sites in 12 countries.
43 sites in 12 countries
United States16 sites
Johns Hopkins University Medical Center
Baltimore, Maryland, 21205
National Heart, Blood, and Lung Institute, National Institutes of Health
Bethesda, Maryland, 20892
Massachusetts General Hospital
Boston, Massachusetts, 02114
Cleveland Clinic Foundation
Cleveland, Ohio, 44195
City of Hope National Medical Center, Div. of Hematology & Bone Marrow Transplant
Duarte, California, 91010-3000
Duke University Health System, Division of Cell Therapy, Heme Malignancies Program
Durham, North Carolina, 27710
Hartford Hospital, Cancer Clinical Research Office
Hartford, Connecticut, 06102
Indiana University Cancer Pavilion, Div. of Hematology-Oncology, Hematological Malignancy Program/Immunology
Indianapolis, Indiana, 46202
University of California at Los Angeles
Los Angeles, California, 90024
NYU Clinical Cancer Center
New York, New York, 10016
Memorial Sloan Kettering Cancer Center
New York, New York, 10021
Presbyterian Medical Center, PENN Comprehensive Hemophilia & Thrombosis Program
Philadelphia, Pennsylvania, 19104
Scripps Cancer Center
San Diego, California, 92121
Washington University School of Medicine, Dept. of Internal Medicine/Division of Hematology
St Louis, Missouri, 63110-1093
Stanford University Medical Center, Division of Hematology
Stanford, California, 94305-5821
Cleveland Clinic, Dept. of Clinical Research
Weston, Florida, 33331
Germany5 sites
Universitatsklinikum Essen, Zentrum fur Innere Medizin
Essen, D-45147
Universitatsklinik Greifswald, Innere Medizin C - Hamato-Onkologie
Greifswald, D-17487
Medizinische Hochschule Hannover, Abt. Hamatologie/Onkologie, Zentrum fur Innere Medizin
Hanover, D-30625
Saarland University Medical School, Internal Medicine 1
Homburg/Saar, D-66421
Institut fur Klinische Transfusionmedizin und Immungenetik, Abtlg. Transfusionsmedizin des Universitatskinikums Ulm
Ulm, D-89081
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
Universitar degli Studi di Napoli, Divisione di Ematologia, Azienda Universitar Policlinico
Naples, 5-80131
Ospedale San Bortolo, Divisione di Ematologia
Vicenza, 37-36100
Australia4 sites
Royal Melbourne Hospital, Dept. of Clinical Haematology & Medical Oncology
Parkville, Victoria, 3050
The Royal Perth Hosptial, Department of Haematology/Level 2
Perth, Western Australia, 6000
Queen Elizabeth Hospital, Dept. of Haematology
Woodville South, South Australia, 5011
Princess Alexandra Hospital, Oncology Haematology Radiation Department
Woolloongabba, Queensland, 4102
United Kingdom4 sites
Belfast City Hospital, Dept. of Haematology C Floor
Belfast, BT9 7AB
Leeds General Infirmary, D Floor Brotherton Wing
Leeds, LS1 3EX
St. George's Hospital, Department of Haematology
London, SW17 OQT
Royal Cornwall Hospital, Haematology Dept.
Truro, Cornwall, TR1 3LJ
France2 sites
Hospital De L'Hotel Dieu, Hematologie et Oncologie Medicale
Cedex, Paris, 04 75181
Hopital Saint-Louis, Centre G. Hayem-Secteur Bleu Porte 7, Laboratoire De Pathologie/Greffe de Moelle
Cedex, Paris, 10 75475
Sweden2 sites
Lund University Hospital, Department of Internal Medicine, Section of Hematology
Lund, SE-22185
Umea University Hospital, Dept. of Internal Medicine 3, Section for Hematology
Umeå, 90185
Canada1 site
University of Alberta, Cross Cancer Institute
Edmonton, Alberta, T6G 2B7
Switzerland1 site
Universitatsklinik Basel, Division of Hematology
Basel, CH, 4031
About this study
Official title: Randomized, Double-Blind, and Placebo-Controlled Study Using Eculizumab in Transfusion Dependent PNH Patients
The primary objective is to evaluate the safety and efficacy of eculizumab in transfusion dependent patients with 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
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 startOct 2004
- Study completionJan 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- 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
- Early clinical test
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Enrolment
- 75 participants
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00122330
- Sponsor's protocol ID
- TRIUMPH
- Status last verified
- Nov 2006
- First submitted
- 18 Jul 2005
- Last updated
- 4 Dec 2006
- Results posted
- No
Publications
Hillmen P, Young NS, Schubert J, Brodsky RA, Socie G, Muus P, Roth A, Szer J, Elebute MO, Nakamura R, Browne P, Risitano AM, Hill A, Schrezenmeier H, Fu CL, Maciejewski J, Rollins SA, Mojcik CF, Rother RP, Luzzatto L. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2006 Sep 21;355(12):1233-43. doi: 10.1056/NEJMoa061648.
PubMed 16990386Hill A, Rother RP, Wang X, Morris SM Jr, Quinn-Senger K, Kelly R, Richards SJ, Bessler M, Bell L, Hillmen P, Gladwin MT. Effect of eculizumab on haemolysis-associated nitric oxide depletion, dyspnoea, and measures of pulmonary hypertension in patients with paroxysmal nocturnal haemoglobinuria. Br J Haematol. 2010 May;149(3):414-25. doi: 10.1111/j.1365-2141.2010.08096.x. Epub 2010 Mar 8.
PubMed 20230403
Study at a glance
From the registry record, last updated by the sponsor on 4 Dec 2006. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized, Double-Blind, and Placebo-Controlled Study Using Eculizumab in Transfusion Dependent PNH Patients
- ClinicalTrials.gov ID
- NCT00122330
- Acronym / sponsor ID
- TRIUMPH
- Conditions
- Hemoglobinuria, Paroxysmal
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 75 participants
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- Oct 2004
- Study completion
- Jan 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 and efficacy of eculizumab in transfusion dependent patients with hemolytic PNH.
Study design
- Primary purpose
- Early clinical test
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Must have required at least 4 transfusions in the past 12 months
- PNH type III red blood cell (RBC) clone by flow cytometry of >10%
- Lactate dehydrogenase (LDH) level > 1.5 x upper limit of normal
- Platelet count > 100,000/mm3
- Patient taking erythropoietin must be on a stable dose for at least 26 weeks
Key exclusion criteria (8 listed)
- Mean hemoglobin level prior to transfusion over the previous 12 months is >10.5 gm/dl
- Absolute neutrophil count <500/ul
- Active bacterial infection
- Hereditary complement deficiency
- Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedures within 30 days
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
Not reported in the source record.
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 4 Dec 2006.
- Results publication: PubMed 16990386Hillmen P, Young NS, Schubert J, Brodsky RA, Socie G, Muus P, Roth A, Szer J, Elebute MO, Nakamura R, Browne P, Risitano AM, Hill A, Schrezenmeier H, Fu CL, Maciejewski J, Rollins SA, Mojcik CF, Rother RP, Luzzatto L. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2006 Sep 21;355(12):1233-43. doi: 10.1056/NEJMoa061648.
- Derived from this study: PubMed 20230403Hill A, Rother RP, Wang X, Morris SM Jr, Quinn-Senger K, Kelly R, Richards SJ, Bessler M, Bell L, Hillmen P, Gladwin MT. Effect of eculizumab on haemolysis-associated nitric oxide depletion, dyspnoea, and measures of pulmonary hypertension in patients with paroxysmal nocturnal haemoglobinuria. Br J Haematol. 2010 May;149(3):414-25. doi: 10.1111/j.1365-2141.2010.08096.x. Epub 2010 Mar 8.
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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.
The record lists no contact. Use the official record to reach the sponsor.
43 sites in 12 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 43 sites in 12 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 11 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 4 Dec 2006. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 43 sites in United States, Germany, Italy, Australia, United Kingdom and 7 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.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00122330, which also lists 2 linked publications.
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 4 Dec 2006; AzTrials last refreshed its copy on 6 Nov 2026.