Urine, DNA and Clinical Information Collection From Patients With Alport Nephropathy.
| Status: | Completed |
|---|---|
| Conditions: | Renal Impairment / Chronic Kidney Disease |
| Therapuetic Areas: | Nephrology / Urology |
| Healthy: | No |
| Age Range: | 5 - Any |
| Updated: | 11/11/2018 |
| Start Date: | April 4, 2017 |
| End Date: | March 23, 2018 |
Urine, DNA and Retrospective Clinical Information Collection From Patients With Alport Nephropathy.
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.
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.
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 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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