Paroxysmal Nocturnal HemoglobinuriaPhase 2NCT01194804
E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients
A Phase 2 study testing Eculizumab in people with Paroxysmal Nocturnal Hemoglobinuria, run by Alexion Pharmaceuticals, Inc..
Trial summary
The objective of this study was to assess the long-term safety and efficacy of eculizumab in hemolytic PNH patients who completed the 4-week screening and 12-week treatment period of the C07-001 study. In addition, pharmacokinetic and pharmacodynamic assessments of eculizumab were conducted. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria
- 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
- 27 participants
- Where
- No sites listed yet
- Study dates
- Apr 2008to Mar 2011
- Phase
- Phase 2
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients must have fully completed C07-001 study 12-week treatment period
Who cannot join
- Patients who terminated early from the C07-001 study
About this study
Official title: Phase II Clinical Study of Eculizumab (h5G1.1-mAb) in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients: Extension Study to C07-001 Protocol
The objective of this study was to assess the long-term safety and efficacy of eculizumab in hemolytic PNH patients who completed the 4-week screening and 12-week treatment period of the C07-001 study. In addition, pharmacokinetic and pharmacodynamic assessments of eculizumab were conducted.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Being tested
- Eculizumab
- Keywords
- Hemolytic Paroxysmal Nocturnal Hemoglobinuria, Eculizumab, PNH
- 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
Also called Soliris
Each vial contains 30 mL of 10 mg/mL eculizumab; dose of 900 mg intravenous every 14 days.
| Study group | Role | What they receive |
|---|---|---|
| Eculizumab | Experimental | Treatment with eculizumab for patients with PNH who have successfully completed the C07-001 protocol Receives: Drug: Eculizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Lactate Dehydrogenase
Measured: 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)
Secondary outcomes (4)
Change From Baseline in FACIT-Fatigue Scale Total Score
The FACIT-Fatigue scale, Version 4.0, 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: 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)
Change From Baseline in PNH Red Blood Cell (RBC) Count
Measured: 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)
Change From Baseline in Number of Units of Packed RBCs Transfused
Baseline is defined as the number of units transfused in 3 months prior to baseline
Measured: 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)
Change From Baseline in Plasma Free Hemoglobin
Measured: 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Sep 2010 (estimated)
- Study startApr 2008
- Primary completionSep 2010
- Study completionMar 2011
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 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- CMIC Co, Ltd. Japan
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01194804
- Sponsor's protocol ID
- E07-001
- Status last verified
- Sep 2019
- First submitted
- 30 Aug 2010
- Last updated
- 17 Sep 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Kanakura Y, Ohyashiki K, Shichishima T, Okamoto S, Ando K, Ninomiya H, Kawaguchi T, Nakao S, Nakakuma H, Nishimura J, Kinoshita T, Bedrosian CL, Ozawa K, Omine M. Long-term efficacy and safety of eculizumab in Japanese patients with PNH: AEGIS trial. Int J Hematol. 2013 Oct;98(4):406-16. doi: 10.1007/s12185-013-1404-y. Epub 2013 Aug 11.
PubMed 23934275
Study at a glance
From the registry record, last updated by the sponsor on 17 Sep 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Clinical Study of Eculizumab (h5G1.1-mAb) in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients: Extension Study to C07-001 Protocol
- ClinicalTrials.gov ID
- NCT01194804
- Acronym / sponsor ID
- E07-001
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Keywords
- Hemolytic Paroxysmal Nocturnal Hemoglobinuria, Eculizumab, PNH
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 27 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Collaborators
- CMIC Co, Ltd. Japan
- Study start
- Apr 2008
- Primary completion
- Sep 2010 (actual)
- Study completion
- Mar 2011 (actual)
- First posted
- 3 Sep 2010 (estimated)
- Results
- Posted 12 Jul 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The objective of this study was to assess the long-term safety and efficacy of eculizumab in hemolytic PNH patients who completed the 4-week screening and 12-week treatment period of the C07-001 study. In addition, pharmacokinetic and pharmacodynamic assessments of eculizumab were conducted.
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: EculizumabTreatment with eculizumab for patients with PNH who have successfully completed the C07-001 protocol |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (1 listed)
- Patients must have fully completed C07-001 study 12-week treatment period
Key exclusion criteria (1 listed)
- Patients who terminated early from the C07-001 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: SolirisEach vial contains 30 mL of 10 mg/mL eculizumab; dose of 900 mg intravenous every 14 days. | Drug | Eculizumab |
Primary outcome
Change From Baseline in Lactate Dehydrogenase
Time frame: 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)
4 secondary outcomes
Change From Baseline in FACIT-Fatigue Scale Total Score
Time frame: 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)
Change From Baseline in PNH Red Blood Cell (RBC) Count
Time frame: 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)
Change From Baseline in Number of Units of Packed RBCs Transfused
Time frame: 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)
Change From Baseline in Plasma Free Hemoglobin
Time frame: 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Sep 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 23934275Kanakura Y, Ohyashiki K, Shichishima T, Okamoto S, Ando K, Ninomiya H, Kawaguchi T, Nakao S, Nakakuma H, Nishimura J, Kinoshita T, Bedrosian CL, Ozawa K, Omine M. Long-term efficacy and safety of eculizumab in Japanese patients with PNH: AEGIS trial. Int J Hematol. 2013 Oct;98(4):406-16. doi: 10.1007/s12185-013-1404-y. Epub 2013 Aug 11.
- 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.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Eculizumab in people with Paroxysmal Nocturnal Hemoglobinuria, run by Alexion Pharmaceuticals, Inc..
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 1 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 17 Sep 2019. Status can change between updates, so confirm with the study contact before making plans.
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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 12 Jul 2019). 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?
Change From Baseline in Lactate Dehydrogenase (time frame: 52 weeks (includes 12 weeks of eculizumab treatment in the parent study and 40 weeks in the extension study)). 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 NCT01194804, 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 17 Sep 2019.