Multi-center Controlled Clinical Trials in Alport Syndrome-A Feasibility Study

NCT01696253 | Completed

• 1 other identifier: 1206M15384
• Study Type: observational
• Target: 360
• Locations: 1 country
• Sites: 1

Brief Summary

Over the past 30 years much has been learned about the molecular genetics and natural history of familial forms of hematuria. However, enhanced understanding of these conditions has yet to generate effective therapies for Alport syndrome(AS), the form of familial hematuria associated with end-stage renal disease. Males with AS inevitably develop end-stage kidney failure, with a 50% likelihood of dialysis or kidney transplantation by age 25 years. There is no proven treatment for AS, although studies in animals have suggested several promising potential therapies. Pharmacological or biological treatments that might delay or prevent the development of kidney failure exist, but need to be evaluated through clinical trials. Researchers interested in implementing clinical trials in AS will face several challenges, the foremost of which is the relative rarity of the disease, necessitating aggressive efforts to identify and recruit potential subjects for multi-center collaborative clinical trials. The Alport Syndrome Research Collaborative (ARC) was established in 2009 as a partnership of the Alport Syndrome Treatments and Outcomes Registry (ASTOR), the European Alport Registry and centers of AS research in Canada, China and France with the objective of testing potential treatments to delay or prevent terminal renal failure in people with AS. In this feasibility study the five ARC centers will interrogate existing AS registries and databases, and monitor accrual of new AS cases over an 18-month period, in order to quantify subjects in the disease categories of interest. As part of this project we will examine the utility of urinary uromodulin excretion as a marker of kidney injury and potential trial endpoint in AS clinical trials. Our goals are to (1) demonstrate that participating centers have access to sufficient numbers of males and females with AS to populate adequately-powered clinical trials focused on two clinical targets, microalbuminuria and overt proteinuria, and (2) to test the hypothesis that in males with AS urinary uromodulin excretion decreases as albuminuria and proteinuria increase and that uromodulin offers an independent and insightful measure of renal fibrosis and response to therapy.

Conditions

Alport Syndrome

Outcome Measures

Primary Outcomes (1)

• Multi-center Controlled Clinical Trials in Alport Syndrome-A Feasibility Study

  The primary outcome to be measured is the number of subjects with Alport syndrome who would meet eligibility criteria for an anticipated clinical treatment trial. Power calculations indicate that the trial would require recruitment of 90 total subjects, stratified into 4 groups based on urine albumin and protein excretion.

  2 years

Secondary Outcomes (1)

• Multi-center Controlled Clinical Trials in Alport Syndrome-A Feasibility Study

  2 years

Study Arms (2)

Subjects at risk for Alport sydrome

Newly identified subjects with Alport syndrome

Eligibility Criteria

• Age: 1 Year+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

A family is eligible for this multi-center feasibility study if a firm diagnosis of AS in a family member has been made by skin biopsy, kidney biopsy, or molecular genetic analysis. In order to enhance identification of subjects with early stage AS (normalbuminuria or MA) and subjects with untreated proteinuria, investigators at each recruitment site will contact all known AS families in their databases, seeking at-risk children whose disease status is unknown. The family populations to be queried at each site are estimated in the table below:

Sponsors & Collaborators

• University of Minnesota: lead
• University of Utah: collaborator
• Peking University First Hospital: collaborator
• University of Toronto: collaborator
• University of Göttingen: collaborator
• Hôpital Necker-Enfants Malades: collaborator

Study Sites (1)

University Of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Related Links

• Alport Registry

Biospecimen

Retention: SAMPLES WITHOUT DNA

First morning clean catch urine sample.

MeSH Terms

Conditions

Nephritis, Hereditary

Condition Hierarchy (Ancestors)

Urogenital Abnormalities; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Nephritis; Kidney Diseases; Urologic Diseases; Male Urogenital Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Collagen Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases

Study Officials

• Clifford E Kashtan, MD

  University of Minnesota

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: OTHER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 20, 2012
• First Posted: September 28, 2012
• Study Start: September 1, 2012
• Primary Completion: August 1, 2014
• Study Completion: December 1, 2017
• Last Updated: March 22, 2018

Record last verified: 2018-03

Locations

US (1)