International Registry and Biorepository for TMA(Thrombotic Microangiopathy)

This study is enrolling participants by invitation only.

First Received on January 2, 2008. Last Updated on July 9, 2010 History of Changes

Sponsor:	North Shore Long Island Jewish Health System
Collaborators:	Children's Hospital of Philadelphia Mario Negri Institute for Pharmacological Research Mount Sinai School of Medicine 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)
Information provided by:	North Shore Long Island Jewish Health System
ClinicalTrials.gov Identifier:	NCT00593229

Purpose

This registry will collect clinical data and store biosamples (seru, plasma, urine, and DNA) annually from pediatric patients with thrombotic mcroangiopathy

Condition
Thrombotic Microangiopathy Hemolytic Uremic Syndrome Thrombotic Thrombocytopenic Purpura

Study Type:	Observational
Study Design:	Observational Model: Case-Only Time Perspective: Prospective
Official Title:	An Observational Study of All Forms of Thrombotic Microangiopathy in Pediatric Patients

Resource links provided by NLM:

Genetics Home Reference related topics: atypical hemolytic-uremic syndrome factor V Leiden thrombophilia hemophilia MYH9-related disorder thrombotic thrombocytopenic purpura

U.S. FDA Resources

Further study details as provided by North Shore Long Island Jewish Health System:

Primary Outcome Measures:

Determine epidemiology and outcomes of the various forms of TMA [ Time Frame: Ongoing ] [ Designated as safety issue: No ]
Determine genetic causes of TMA [ Time Frame: Ongoing ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Initiate clinical trials in TMA [ Time Frame: In the future ] [ Designated as safety issue: Yes ]

Biospecimen Retention: Samples With DNA

Serum, plasma, urine, and DNA

Estimated Enrollment:	60
Study Start Date:	January 2007

Groups/Cohorts
3 Familial atypical HUS
4 Thrombotic thrombocytopenic purpura (TTP)
1 Severe diarrhea-associated hemolytic uremic syndrome (D+HUS)
2 Non-familial atypical HUS

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.

Eligibility

Ages Eligible for Study:	6 Months to 18 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	Yes
Sampling Method:	Non-Probability Sample

Study Population

Pediatric patients, 6 months - 18 years

Criteria

Inclusion Criteria:

Severe HUS, familial or non-familial atypical HUS, TTP

Exclusion Criteria:

None

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00593229

Locations

United States, New York
Schneider Chldren's Hospital
New Hyde Park, New York, United States, 11040

Sponsors and Collaborators

North Shore Long Island Jewish Health System

Children's Hospital of Philadelphia

Mario Negri Institute for Pharmacological Research

Mount Sinai School of Medicine

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)

Investigators

Principal Investigator:

Howard Trachtman, MD

Schneider Children's Hospital

More Information

Additional Information:

Study is listed at Clinical Trials tab This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party:	Howard Trachtman, Feinstein Institute for Medical Research of North Shore-LIJ Health System
ClinicalTrials.gov Identifier:	NCT00593229 History of Changes
Other Study ID Numbers:	DK71221, DK R21-71221
Study First Received:	January 2, 2008
Last Updated:	July 9, 2010
Health Authority:	United States: Federal Government United States: Institutional Review Board

Keywords provided by North Shore Long Island Jewish Health System:

Thrombotic microangiopathy (TMA)
Hemolytic uremic syndrome (HUS)
Thrombotic thrombocytopenic purpura (TTP)

Additional relevant MeSH terms:

Hemolytic-Uremic Syndrome
Purpura
Purpura, Thrombocytopenic
Purpura, Thrombotic Thrombocytopenic
Thrombosis
Vascular Diseases
Azotemia
Hemolysis
Thrombotic Microangiopathies
Uremia
Kidney Diseases
Urologic Diseases
Anemia, Hemolytic

Anemia
Hematologic Diseases
Thrombocytopenia
Blood Platelet Disorders
Blood Coagulation Disorders
Hemorrhage
Pathologic Processes
Skin Manifestations
Signs and Symptoms
Immune System Diseases
Thrombophilia
Embolism and Thrombosis
Cardiovascular Diseases

ClinicalTrials.gov processed this record on May 22, 2012