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

NCT03074357 | Completed

• 1 other identifier: CBASICHR0005
• Study Type: observational
• Target: 68
• Locations: 1 country
• Sites: 1

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.

Conditions

Alport Nephropathy

Outcome Measures

Primary Outcomes (1)

• Urine levels of biomarkers - corrected for urine creatinine

  Urine levels of biomarkers, corrected for urine creatinine, in Alport subjects stratified by magnitude of proteinura.

  Day 1

Secondary Outcomes (1)

• Urine level of biomarkers - corrected for urine creatinine

  Day 1

Eligibility Criteria

• Age: 5 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The study population will be males and females comprised of up to 80 Alport subjects who participated in CPLATFRM2201 study as well as up to 20 pediatric and adolescent healthy volunteers (including healthy siblings of Alport subjects) that have met all other eligibility criteria.

You may qualify if:

• 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.
• 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.
• Male and Female Volunteers (may include healthy siblings of Alport patients) aged 5 to 15 years.

You may not qualify if:

• None
• 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.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (1)

Novartis Investigative Site

Minneapolis, Minnesota, 55455, United States

Location

Biospecimen

Retention: SAMPLES WITH DNA

Urine Saliva for DNA

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 23, 2017
• First Posted: March 8, 2017
• Study Start: April 4, 2017
• Primary Completion: March 23, 2018
• Study Completion: March 23, 2018
• Last Updated: November 9, 2018

Record last verified: 2018-11

Locations

US (1)