Thrombotic MicroangiopathyNCT00593229
International Registry and Biorepository for TMA(Thrombotic Microangiopathy)
An observational study of Thrombotic Microangiopathy and Hemolytic Uremic Syndrome, run by Northwell Health at 1 site in United States.
Trial summary
This registry will collect clinical data and store biosamples (seru, plasma, urine, and DNA) annually from pediatric patients with thrombotic mcroangiopathy Read more about this study
- Who is it for?
- Ages 6 months to 18 yearsHealthy volunteers can take part
- Conditions the study is for
- Thrombotic Microangiopathy, Hemolytic Uremic Syndrome, Thrombotic Thrombocytopenic Purpura
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2007to Oct 2011
- 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 6 months to 18 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Study population: Pediatric patients, 6 months - 18 years
Who may be able to join
- Severe HUS, familial or non-familial atypical HUS, TTP
Who cannot join
- None
Trial locations and contacts
1 site in 1 country.
United States1 site
Schneider Chldren's Hospital
New Hyde Park, New York, 11040
About this study
Official title: An Observational Study of All Forms of Thrombotic Microangiopathy in Pediatric Patients
This registry will collect clinical data and store biosamples (seru, plasma, urine, and DNA) annually from pediatric patients with thrombotic mcroangiopathy
Read the detailed description
Children age, 6 months - 18 years, are eligible if they have one of the following categories of thrombotic microangiopathy (TMA):(1) severe D+HUS; (2) non-familial atypical HUS; (3) familial atypical HUS; or (4) TTP
Patients are seen intially and annually thereafter
Biosamples are collected for storage annually.
- Conditions
- Thrombotic Microangiopathy, Hemolytic Uremic Syndrome, Thrombotic Thrombocytopenic Purpura
- Keywords
- Thrombotic microangiopathy (TMA), Hemolytic uremic syndrome (HUS), Thrombotic thrombocytopenic purpura (TTP)
- Medical subject headings
- Thrombotic Microangiopathies, Hemolytic-Uremic Syndrome, Purpura, Thrombotic Thrombocytopenic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 3 | Familial atypical HUS | |
| 4 | Thrombotic thrombocytopenic purpura (TTP) | |
| 1 | Severe diarrhea-associated hemolytic uremic syndrome (D+HUS) | |
| 2 | Non-familial atypical HUS |
What is being measured
The primary outcome is the main question the study is designed to answer.
Determine epidemiology and outcomes of the various forms of TMA
Measured: Ongoing
Determine genetic causes of TMA
Measured: Ongoing
Secondary outcomes (1)
Initiate clinical trials in TMA
Measured: In the future
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jan 2008 (estimated)
- Study startJan 2007
- Primary completionOct 2011
Why it stopped: study stopped at this site, as investigator left the institution
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thrombotic MicroangiopathyStudies of the same condition, open and under way.
- More trials run by Northwell HealthThe 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
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Northwell Health
- Sponsor type
- University, hospital or other
- Collaborators
- Children's Hospital of Philadelphia, Mario Negri Institute for Pharmacological Research, Icahn School of Medicine at Mount Sinai, University of Virginia, Montreal Children's Hospital of the MUHC, University of Utah, Stollery Children's Hospital, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- Responsible party
- Sponsor
- Investigators
- Howard Trachtman, MD, Principal investigator, Schneider Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00593229
- Sponsor's protocol ID
- DK71221
- Other IDs
- DK R21-71221
- Status last verified
- Jun 2013
- First submitted
- 2 Jan 2008
- Last updated
- 19 Jun 2013
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Jun 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Observational Study of All Forms of Thrombotic Microangiopathy in Pediatric Patients
- ClinicalTrials.gov ID
- NCT00593229
- Acronym / sponsor ID
- DK71221
- Conditions
- Thrombotic Microangiopathy, Hemolytic Uremic Syndrome, Thrombotic Thrombocytopenic Purpura
- Keywords
- Thrombotic microangiopathy (TMA), Hemolytic uremic syndrome (HUS), Thrombotic thrombocytopenic purpura (TTP)
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: study stopped at this site, as investigator left the institution
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Northwell HealthUniversity, hospital or other
- Collaborators
- Children's Hospital of Philadelphia, Mario Negri Institute for Pharmacological Research, Icahn School of Medicine at Mount Sinai, University of Virginia, Montreal Children's Hospital of the MUHC, University of Utah, Stollery Children's Hospital, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- Study start
- Jan 2007
- Primary completion
- Oct 2011 (actual)
- First posted
- 14 Jan 2008 (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 registry will collect clinical data and store biosamples (seru, plasma, urine, and DNA) annually from pediatric patients with thrombotic mcroangiopathy
Detailed description
Children age, 6 months - 18 years, are eligible if they have one of the following categories of thrombotic microangiopathy (TMA):(1) severe D+HUS; (2) non-familial atypical HUS; (3) familial atypical HUS; or (4) TTP
Patients are seen intially and annually thereafter
Biosamples are collected for storage annually.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| 3 | – | –Familial atypical HUS |
| 4 | – | –Thrombotic thrombocytopenic purpura (TTP) |
| 1 | – | –Severe diarrhea-associated hemolytic uremic syndrome (D+HUS) |
| 2 | – | –Non-familial atypical HUS |
Who may qualify
- Ages
- 6 months to 18 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
- Study population
- Pediatric patients, 6 months - 18 years
Key inclusion criteria (1 listed)
- Severe HUS, familial or non-familial atypical HUS, TTP
Key exclusion criteria (1 listed)
- None
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
Determine epidemiology and outcomes of the various forms of TMA
Time frame: Ongoing
Determine genetic causes of TMA
Time frame: Ongoing
1 secondary outcome
Initiate clinical trials in TMA
Time frame: In the future
Results and safety
The study was stopped early (reason given: study stopped at this site, as investigator left the institution). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Jun 2013.
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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.
Howard Trachtman, MD principal investigator
Schneider Children's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Thrombotic Microangiopathy and Hemolytic Uremic Syndrome, run by Northwell Health at 1 site in United States.
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: stopped early, as of the sponsor's last update on 19 Jun 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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 was stopped early (reason given: study stopped at this site, as investigator left the institution). Results are not available in the source record.
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?
Determine epidemiology and outcomes of the various forms of TMA (time frame: Ongoing). 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 19 Jun 2013.