International Registry and Biorepository for TMA(Thrombotic Microangiopathy)

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

Study Overview

• Status: Terminated
• Conditions: Thrombotic Thrombocytopenic Purpura; Hemolytic Uremic Syndrome; Thrombotic Microangiopathy

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 Type: Observational
• Enrollment (Actual): 6

Contacts and Locations

Study Locations

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

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 months to 18 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Pediatric patients, 6 months - 18 years

Description

Inclusion Criteria:

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

Exclusion Criteria:

• None

Study Plan

How is the study designed?

Design Details

• Observational Models: Case-Only
• Time Perspectives: Prospective

Number of groups / cohorts

4

Cohorts and Interventions

Group / Cohort
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 the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Determine epidemiology and outcomes of the various forms of TMA	Ongoing
Determine genetic causes of TMA	Ongoing

Secondary Outcome Measures

Outcome Measure	Time Frame
Initiate clinical trials in TMA	In the future

Collaborators and Investigators

• Sponsor: Northwell Health
• Collaborators: Children's Hospital of Philadelphia; National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Icahn School of Medicine at Mount Sinai; University of Utah; University of Virginia; Stollery Children's Hospital; Mario Negri Institute for Pharmacological Research; Montreal Children's Hospital of the MUHC
• Investigators: Principal Investigator: Howard Trachtman, MD, Schneider Children's Hospital

Publications and helpful links

Helpful Links

• Study is listed at Clinical Trials tab

Study record dates

Study Major Dates

• Study Start: January 1, 2007
• Primary Completion (Actual): October 1, 2011

Study Registration Dates

• First Submitted: January 2, 2008
• First Submitted That Met QC Criteria: January 11, 2008
• First Posted (Estimate): January 14, 2008

Study Record Updates

• Last Update Posted (Estimate): June 19, 2013
• Last Update Submitted That Met QC Criteria: June 18, 2013
• Last Verified: June 1, 2013

More Information

Terms related to this study

• Keywords: Thrombotic microangiopathy (TMA); Hemolytic uremic syndrome (HUS); Thrombotic thrombocytopenic purpura (TTP)
• Additional Relevant MeSH Terms: Pathologic Processes; Cardiovascular Diseases; Immune System Diseases; Kidney Diseases; Urologic Diseases; Hematologic Diseases; Hemorrhage; Anemia; Blood Coagulation Disorders; Skin Manifestations; Thrombocytopenia; Blood Platelet Disorders; Thrombophilia; Anemia, Hemolytic; Uremia; Vascular Diseases; Purpura; Purpura, Thrombocytopenic; Purpura, Thrombotic Thrombocytopenic; Thrombotic Microangiopathies; Hemolysis; Hemolytic-Uremic Syndrome
• Other Study ID Numbers: DK71221; DK R21-71221