Paroxysmal Hemoglobinuria, NocturnalPhase 3NCT00122317
Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH)
A Phase 3 study testing eculizumab in people with Paroxysmal Hemoglobinuria, Nocturnal, run by Alexion Pharmaceuticals, Inc. at 40 sites in 12 countries.
Trial summary
The purpose of this study is to evaluate the long-term 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
- Paroxysmal Hemoglobinuria, Nocturnal
- What is being tested?
- eculizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 187 participants
- Where
- 40 sitesin 12 countries
- Study dates
- May 2005to Oct 2008
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Patients who have fully completed the TRIUMPH, SHEPHERD, or X03-001 studies
- TRIUMPH patients who have discontinued receiving investigational drug prior to the last visit of the study due to lack of efficacy or exacerbation of symptoms of PNH and have completed all monthly safety and efficacy procedures
- Patient must be willing and able to give written informed consent
- Patient must avoid conception during the trial
Who cannot join
- Patients who have terminated early from the SHEPHERD or X03-001 studies
- Patients who have terminated early from the TRIUMPH study due to an adverse event
- Female who is pregnant, breast feeding, or intending to conceive during the course of the study
- Any condition that could increase the patient's risk by participating in the study or could confound the outcome of the study
Trial locations and contacts
40 sites in 12 countries.
40 sites in 12 countries
United States14 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
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
Indianapolis University Cancer Center
Indianapolis, Indiana, 46202
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 & Thombosis Program
Philadelphia, Pennsylvania, 19104
Mayo Clinic, Divison of Hematology
Rochester, Minnesota, 55902
Washington University, Department of Internal Medicine/Division of Hematology
St Louis, Missouri, 63110
Stanford University Medical Center, Division of Hematology
Stanford, California, 94305
Cleveland Clinic Florida, Department of Clinical Research
Weston, Florida, 33331
Australia5 sites
Royal Melbourne Hospital, Department of Clinical Haematology & Medical Oncology
Parkville, 3050
The Royal Perth Hospital, Department of Haematology/Level 2
Perth, Western Australia, 6000
Royal North Shore Hospital, Haematology Department
Saint Leonards, 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, Department 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 Immunogenetik, Abtlg. Transfusionmedizin des Univ. 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
Canada2 sites
University of Alberta, Cross Cancer Institute
Edmonton, Alberta, T6G 2B7
London Regional Cancer Centre, Clinical Research Unit Room C3080
London, Ontario, N6A4L6
United Kingdom2 sites
Leeds General Infirmary, D Floor Brotherton Wing
Leeds, LS1 3EX
St. George's Hospital, Department of Haematology
London, SW17OQT
France1 site
Hopital Saint Louis, Centre d'investigation Clinique
Paris, Cedex, 10 75475
Netherlands1 site
UMC St. Radboud, Department of Hematology
Nijmegen, GA, 6525
Sweden1 site
Stockholm South Hospital, Division of Hematology
Stockholm, 118-83
About this study
Official title: Open Label Extension Study of Eculizumab in Patients With Transfusion Dependent PNH
The purpose of this study is to evaluate the long-term safety of eculizumab in patients with transfusion dependent hemolytic PNH.
Read the detailed description
An open-label extension study to evaluate long-term safety of eculizumab in PNH patients who had completed the TRIUMPH (C04-001), SHEPHERD (C04-002), and X03-001 studies.
- Conditions
- Paroxysmal Hemoglobinuria, Nocturnal
- Being tested
- eculizumab
- Keywords
- transfusion dependent, paroxysmal nocturnal hemoglobinuria, hemolytic
- Medical subject headings
- Hemoglobinuria, Paroxysmal, Hemolysis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
eculizumabDrug
Also called Soliris
| Study group | Role | What they receive |
|---|---|---|
| Eculizumab | Experimental | 600 mg intravenous infusion every week x 4 then 900 mg iv every two weeks Receives: Drug: eculizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-emergent Adverse Events
Measured: From time of consent to a maximum of 2.5 years of study treatment
Secondary outcomes (3)
Hemolysis as Measured by Change From Baseline in LDH Area Under the Curve
Measured: From time of first infusion through 24 months of study treatment
Quality of Life as Measured by FACIT-Fatigue Scale Change From Baseline
The FACIT-Fatigue scale is a collection of quality of life questionnaires pertaining to the management of fatigue symptoms due to a chronic illness. The FACIT-Fatigue is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function over the preceding 7 days. Patients score each item on a 5-point scale: 0 (Not at all) to 4 (Very much). Total scores range from 0 to 52, with higher score indicating better quality of life.
Measured: From time of first infusion through 24 months of study treatment
Incidence of Thrombosis After Eculizumab Infusion
Thrombosis was defined as occurrence of major adverse vascular events
Measured: From time of first ever dose through last dose (up to 24 months of study treatment)
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 startMay 2005
- Primary completionSep 2008
- Study completionOct 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Paroxysmal Hemoglobinuria, NocturnalStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 187 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00122317
- Sponsor's protocol ID
- E05-001
- Status last verified
- Feb 2018
- First submitted
- 20 Jul 2005
- Last updated
- 13 Mar 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
Hillmen P, Muus P, Duhrsen U, Risitano AM, Schubert J, Luzzatto L, Schrezenmeier H, Szer J, Brodsky RA, Hill A, Socie G, Bessler M, Rollins SA, Bell L, Rother RP, Young NS. Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria. Blood. 2007 Dec 1;110(12):4123-8. doi: 10.1182/blood-2007-06-095646. Epub 2007 Aug 16.
PubMed 17702897
Study at a glance
From the registry record, last updated by the sponsor on 13 Mar 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open Label Extension Study of Eculizumab in Patients With Transfusion Dependent PNH
- ClinicalTrials.gov ID
- NCT00122317
- Acronym / sponsor ID
- E05-001
- Conditions
- Paroxysmal Hemoglobinuria, Nocturnal
- Keywords
- transfusion dependent, paroxysmal nocturnal hemoglobinuria, hemolytic
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 187 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- May 2005 (actual)
- Primary completion
- Sep 2008 (actual)
- Study completion
- Oct 2008 (actual)
- First posted
- 22 Jul 2005 (estimated)
- Results
- Posted 5 Jun 2017
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 evaluate the long-term safety of eculizumab in patients with transfusion dependent hemolytic PNH.
Detailed description
An open-label extension study to evaluate long-term safety of eculizumab in PNH patients who had completed the TRIUMPH (C04-001), SHEPHERD (C04-002), and X03-001 studies.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Eculizumab | Experimental | Drug: eculizumab600 mg intravenous infusion every week x 4 then 900 mg iv every two weeks |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Patients who have fully completed the TRIUMPH, SHEPHERD, or X03-001 studies
- TRIUMPH patients who have discontinued receiving investigational drug prior to the last visit of the study due to lack of efficacy or exacerbation of symptoms of PNH and have completed all monthly safety and efficacy procedures
- Patient must be willing and able to give written informed consent
- Patient must avoid conception during the trial
Key exclusion criteria (4 listed)
- Patients who have terminated early from the SHEPHERD or X03-001 studies
- Patients who have terminated early from the TRIUMPH study due to an adverse event
- Female who is pregnant, breast feeding, or intending to conceive during the course of the study
- Any condition that could increase the patient's risk by participating in the study or could confound the outcome of the study
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 |
|---|---|---|
| eculizumabAlso: Soliris | Drug | Eculizumab |
Primary outcome
Incidence of Treatment-emergent Adverse Events
Time frame: From time of consent to a maximum of 2.5 years of study treatment
3 secondary outcomes
Hemolysis as Measured by Change From Baseline in LDH Area Under the Curve
Time frame: From time of first infusion through 24 months of study treatment
Quality of Life as Measured by FACIT-Fatigue Scale Change From Baseline
Time frame: From time of first infusion through 24 months of study treatment
Incidence of Thrombosis After Eculizumab Infusion
Time frame: From time of first ever dose through last dose (up to 24 months of study treatment)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Mar 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 17702897Hillmen P, Muus P, Duhrsen U, Risitano AM, Schubert J, Luzzatto L, Schrezenmeier H, Szer J, Brodsky RA, Hill A, Socie G, Bessler M, Rollins SA, Bell L, Rother RP, Young NS. Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria. Blood. 2007 Dec 1;110(12):4123-8. doi: 10.1182/blood-2007-06-095646. Epub 2007 Aug 16.
- Individual participant dataSharing planned: No
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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.
40 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 Paroxysmal Hemoglobinuria, Nocturnal, run by Alexion Pharmaceuticals, Inc. at 40 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 4 inclusion and 4 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 13 Mar 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 40 sites in United States, Australia, Italy, Germany, Spain 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?
Yes. Results are posted in the registry (first posted 5 Jun 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Incidence of Treatment-emergent Adverse Events (time frame: From time of consent to a maximum of 2.5 years of study treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00122317, which also lists 1 linked publication.
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 13 Mar 2018; AzTrials last refreshed its copy on 8 Nov 2026.