This is a phase 3, prospective, multicenter, randomized, double-blinded, placebo-controlled withdrawal study comparing the efficacy of ADV7103 versus placebo in preventing the development of metabolic acidosis defined by serum bicarbonate levels in pediatric (6 months to \< 18 years of age) and adult (18 to 65 years of age) subjects with primary Distal Renal Tubular Acidosis (dRTA).
The study will target enrolling at least 4 subjects in each of the following age groups: 6 months - 23 months; 2-11 years, and ≥ 12 years. Subjects will be in the study for up to 21 weeks. After screening and enrollment, subjects will participate in an 8-12 week open label period where there dose of ADV7103 will be titrated to effect, then continued for the remainder of the open-label period. Periodic measurements of bicarbonate and potassium levels will be collected during this period. Following the open-label period, subjects will enter a 6-day randomized withdrawal period. A follow-up period up to four weeks on re-established therapy completes the trial. Subjects can elect to return to their previous standard of care regimen after completing the withdrawal period or will have the opportunity to subsequently enter an open-label extension study.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
PREVENTION
Masking
QUADRUPLE
Enrollment
3
Each dose of ADV7103 contains a fixed ratio of 1/3 of ADV7103-CK (potassium citrate) and 2/3 of ADV7103-BK (potassium bicarbonate) based on the mass of active substances. The strength is 6.44 (± 10 %) mEq/g of ADV7103 (alkalinizing power).
Placebo is a combination of 2 mm green coated lactose granules and 2 mm white coated lactose granules to be taken by mouth. Each dose of placebo contains a fixed ratio of 1/3 of green granules and 2/3 of white granules.
Children's Hospital Colorado
Aurora, Colorado, United States
University of South Florida Pediatric Infectious Disease
Tampa, Florida, United States
Emory Children's Center
Atlanta, Georgia, United States
J.W. Riley Hospital for Children/Indiana University
Mean change in blood bicarbonate levels
Compare the efficacy of ADV7103 versus placebo in preventing metabolic acidosis, defined as 2 consecutive serum bicarbonate levels \< 18 mEq/L for subjects ≥ 4 years old and \< 17 mEq/L for subjects \< 4 years old, during the Withdrawal Period
Time frame: 6 days
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Indianapolis, Indiana, United States
Montefiore Medical Center
The Bronx, New York, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
University of Alberta
Edmonton, Alberta, Canada
The Hospital for Sick Children
Toronto, Ontario, Canada