Registry for Patients With Wilms' Tumor Suppressor Gene 1... | NCT01252901

Studies
Registry for Patients With Wilms' Tumor Suppressor Gene 1 (WT1) Mutation Associated Diseases

NCT01252901

Sponsor

Universitätsklinikum Hamburg-Eppendorf

Status

Completed

Last Update Posted

May 28, 2015Estimated

Enrollment

52Actual

Phase

Not provided

Conditions

Denys-Drash Syndrome
Frasier Syndrome
Nephrotic Syndrome
Wilms Tumor

Interventions

Not provided

Countries

Germany

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

May 2015

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Oct 2010

Primary Completion Date

Nov 2013Actual

Completion Date

Jun 2014Actual

First Submitted Date

Dec 1, 2010

First Submission Date that Met QC Criteria

Dec 2, 2010

First Posted Date

Dec 3, 2010Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

May 27, 2015

Last Update Posted Date

May 28, 2015Estimated

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

The Wilms' tumor suppressor gene 1 (WT1) encodes for a transcription factor which plays an important role during urogenital development. Patients carrying a WT1 germline mutation show symptoms like proteinuria, Wilms tumors, genital malformations and kidney failure. Milder variants are possible and classification is not always possible. In this registry we are collecting detailed clinical data of affected individuals to establish genotype-phenotype correlations with the greater goal to optimize patient care.

Detailed Description

The Wilms' tumor suppressor gene 1 (WT1) encodes for a transcription factor which plays an important role during urogenital development. Patients carrying a WT1 germline mutation show symptoms like proteinuria, Wilms tumors, genital malformations and kidney failure, which usually occur in early childhood. Milder variants are possible and classification as one of the three rare syndromes associated with WT1 mutations (Denys-Drash syndrome, Frasier syndrome or WAGR syndrome) is not always possible. In this registry we are collecting detailed clinical data of affected individuals to establish genotype-phenotype correlations with the greater goal to optimize patient care.

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

52Actual

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Germline mutation in WT1 gene

Exclusion Criteria:

none

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Patients with WT1 mutation

Sampling Method

Probability Sample

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Anja Lehnhardt, MD	Universitätsklinikum Hamburg-Eppendorf	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Universitätskrankenhaus Hamburg-Eppendorf		Hamburg			Germany

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
15150775	Background	Royer-Pokora B, Beier M, Henzler M, Alam R, Schumacher V, Weirich A, Huff V. Twenty-four new cases of WT1 germline mutations and review of the literature: genotype/phenotype correlations for Wilms tumor development. Am J Med Genet A. 2004 Jun 15;127A(3):249-57. doi: 10.1002/ajmg.a.30015.
16439601	Background

ID	Term
D030321	Denys-Drash Syndrome
D052159	Frasier Syndrome
D009404	Nephrotic Syndrome
D009396	Wilms Tumor
D011507	Proteinuria

ID	Term
D018193	Neoplasms, Complex and Mixed
D009370	Neoplasms by Histologic Type
D009369	Neoplasms
D007680	Kidney Neoplasms