Urine, DNA and Clinical Information Collection From Patients With Alport Nephropathy.

Urine, DNA and Retrospective Clinical Information Collection From Patients With Alport Nephropathy.

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT03074357

Enrollment

Registered

2017-03-08

Start date

2017-04-04

Completion date

2018-03-23

Last updated

2018-11-09

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Alport Nephropathy

Brief summary

This biomarker study is a follow-up to CPLATFRM2201. The goal of CBASICHR0005 is to collect another urine sample, interval clinical information, and an optional DNA sample from as many of the original 80 patients as possible. This new information will transform the data obtained in PLATFRM2201 from a cross-section to a temporal profile, which will (a) further enable the identification of biomarkers predictive of faster progression, and (b) satisfy the FDA's recommendation to perform natural history studies in rare diseases.

Interventions

None listed

Study design

Observational model

OTHER

Time perspective

RETROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

5 Years to No maximum

Healthy volunteers

Yes

Inclusion criteria

• Able to communicate well with the investigator, to understand and comply with the requirements of the study and able to provide written informed consent (parent or legal guardian for pediatric and adolescent subjects), which must be obtained before any assessment is performed. Alport Subject-Specific Inclusion Criteria: * Participation in CPLATFRM2201 * Physically able to provide a single first-morning urine sample of at least 30 mL (one ounce). Alport patients who have initiated dialysis therapy since participation in CPLATFRM2201 are exempt from this criterion and may still participate in this study. Healthy Volunteer-Specific Inclusion Criteria: • Male and Female Volunteers (may include healthy siblings of Alport patients) aged 5 to 15 years.

Exclusion criteria

Alport Subject-Specific

Design outcomes

Primary

Measure	Time frame	Description
Urine levels of biomarkers - corrected for urine creatinine	Day 1	Urine levels of biomarkers, corrected for urine creatinine, in Alport subjects stratified by magnitude of proteinura.

Secondary

Measure	Time frame	Description
Urine level of biomarkers - corrected for urine creatinine	Day 1	Urine levels of biomarkers, corrected for urine creatinine, in healthy subjects.

Countries

United States

Outcome results