ATYPICAL HEMOLYTIC UREMIC SYNDROME (AHUS)NCT01755429
To Characterize the Safety and Tolerability of Eculizumab in Two Japanese aHUS Patients
An observational study of ATYPICAL HEMOLYTIC UREMIC SYNDROME (AHUS), run by Alexion Pharmaceuticals, Inc. at 2 sites in Japan.
Trial summary
This protocol is intended to formally collect data on the treatment of aHUS with eculizumab in Japanese patients. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- ATYPICAL HEMOLYTIC UREMIC SYNDROME (AHUS)
- How many people
- 2 participants
- Where
- 2 sitesJapan
- Study dates
- May 2012to Jul 2012
- 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: The two identified Japanese patients with aHUS for which eculizumab treatment was initiated in 2011
Who may be able to join
- Patient and/or legal guardian must be willing and able to give written informed consent and id the patient is not an adult and able to read and write, the patient is willing to give written informed assent
- Eculizumab treatment initiated as personal importation for aHUS in 201
Who cannot join
- Not Applicable
Trial locations and contacts
2 sites in 1 country.
Japan2 sites
Shinshu University School of Medicine
Matsumoto
Tokyo Medical and Dental University
Tokyo
About this study
Official title: A Research Study to Describe the Safety and Efficacy of Eculizumab in Japanese Patients With Atypical Hemolytic Uremic Syndrome (aHUS). An Assessment of Two Case Studies
This protocol is intended to formally collect data on the treatment of aHUS with eculizumab in Japanese patients.
- Medical subject headings
- Atypical Hemolytic Uremic Syndrome
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in platelet count from baseline
Measured: 26 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Dec 2012 (estimated)
- Study startMay 2012
- Primary completionMay 2012
- Study completionJul 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ATYPICAL HEMOLYTIC UREMIC SYNDROME (AHUS)Studies 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
- Time perspective
- Retrospective
- Enrolment
- 2 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
- NCT01755429
- Sponsor's protocol ID
- C11-004J
- Status last verified
- Feb 2012
- First submitted
- 4 Dec 2012
- Last updated
- 24 Dec 2012
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Dec 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Research Study to Describe the Safety and Efficacy of Eculizumab in Japanese Patients With Atypical Hemolytic Uremic Syndrome (aHUS). An Assessment of Two Case Studies
- ClinicalTrials.gov ID
- NCT01755429
- Acronym / sponsor ID
- C11-004J
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- May 2012
- Primary completion
- May 2012 (actual)
- Study completion
- Jul 2012 (actual)
- First posted
- 24 Dec 2012 (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.
This protocol is intended to formally collect data on the treatment of aHUS with eculizumab in Japanese patients.
Study design
- Time perspective
- Retrospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- The two identified Japanese patients with aHUS for which eculizumab treatment was initiated in 2011
Key inclusion criteria (2 listed)
- Patient and/or legal guardian must be willing and able to give written informed consent and id the patient is not an adult and able to read and write, the patient is willing to give written informed assent
- Eculizumab treatment initiated as personal importation for aHUS in 201
Key exclusion criteria (1 listed)
- Not Applicable
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
Change in platelet count from baseline
Time frame: 26 weeks
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 24 Dec 2012.
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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.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of ATYPICAL HEMOLYTIC UREMIC SYNDROME (AHUS), run by Alexion Pharmaceuticals, Inc. at 2 sites in Japan.
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 2 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 24 Dec 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Japan. 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?
Change in platelet count from baseline (time frame: 26 weeks). 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 24 Dec 2012.