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

Completed

• Conditions: Alport Nephropathy

• Registration Number: NCT03074357
• Lead Sponsor: Novartis Pharmaceuticals

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2017-02-23
• Study First Submitted QC: 2017-03-03
• Study First Posted: 2017-03-08
• Study Start: 2017-04-04
• Primary Completion: 2018-03-23
• Study Completion: 2018-03-23
• Status Verified: 2018-11
• Last Update Submitted: 2018-11-08
• Last Update Posted: 2018-11-09

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 68

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.

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Exclusion Criteria

Alport Subject-Specific Exclusion Criteria:

• None

Healthy Volunteer-Specific Exclusion Criteria:

• Use of investigational drugs at the time of enrollment, or within 30 days or 5 halflives of enrollment, whichever is longer; or longer if required by local regulations, and for any other limitation of participation in an investigational trial based on local regulations.
• Known history of one of more of (a) hematuria (gross or microscopic), (b)audiological deficits, (c) fixed (non-orthostatic) proteinuria, or (d) reduced renal filtration function with serum creatinine (Cr) above the upper limit of the normal age-specific reference range, on at least 2 prior occasions (serum Cr need not be measured specifically for eligibility).
• Any other clinically significant underlying medical conditions as judged by the PI.

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
Urine level of biomarkers - corrected for urine creatinine	Day 1	Urine levels of biomarkers, corrected for urine creatinine, in healthy subjects.

Trial Locations

Locations (1)

Novartis Investigative Site; 🇺🇸 Minneapolis, Minnesota, United States