International Registry and Biorepository for TMA(Thrombotic Microangiopathy)

Sponsor
Northwell Health (Other)
Overall Status
Terminated
CT.gov ID
NCT00593229
Collaborator
Children's Hospital of Philadelphia (Other), Mario Negri Institute for Pharmacological Research (Other), Icahn School of Medicine at Mount Sinai (Other), University of Virginia (Other), Montreal Children's Hospital of the MUHC (Other), University of Utah (Other), Stollery Children's Hospital (Other), National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) (NIH)
6
1

Study Details

Study Description

Brief Summary

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

Study Type:
Observational
Actual Enrollment :
6 participants
Observational Model:
Case-Only
Time Perspective:
Prospective
Official Title:
An Observational Study of All Forms of Thrombotic Microangiopathy in Pediatric Patients
Study Start Date :
Jan 1, 2007
Actual Primary Completion Date :
Oct 1, 2011

Arms and Interventions

Arm Intervention/Treatment
3

Familial atypical HUS

4

Thrombotic thrombocytopenic purpura (TTP)

1

Severe diarrhea-associated hemolytic uremic syndrome (D+HUS)

2

Non-familial atypical HUS

Outcome Measures

Primary Outcome Measures

  1. Determine epidemiology and outcomes of the various forms of TMA [Ongoing]

  2. Determine genetic causes of TMA [Ongoing]

Secondary Outcome Measures

  1. Initiate clinical trials in TMA [In the future]

Eligibility Criteria

Criteria

Ages Eligible for Study:
6 Months to 18 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
Yes
Inclusion Criteria:
  • Severe HUS, familial or non-familial atypical HUS, TTP
Exclusion Criteria:
  • None

Contacts and Locations

Locations

Site City State Country Postal Code
1 Schneider Chldren's Hospital New Hyde Park New York United States 11040

Sponsors and Collaborators

  • Northwell Health
  • 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)

Investigators

  • Principal Investigator: Howard Trachtman, MD, Schneider Children's Hospital

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

Publications

None provided.
Responsible Party:
Northwell Health
ClinicalTrials.gov Identifier:
NCT00593229
Other Study ID Numbers:
  • DK71221
  • DK R21-71221
First Posted:
Jan 14, 2008
Last Update Posted:
Jun 19, 2013
Last Verified:
Jun 1, 2013

Study Results

No Results Posted as of Jun 19, 2013