A Phase 3, Double-blind, Placebo-controlled, Randomized Withdrawal Study to Evaluate the Efficacy and Safety of SYNB1934 in Patients 12 Years and Older with PKU (SYNPHENY-3)

Date Added
October 3rd, 2023
PRO Number
Pro00127030
Researcher
Neena Champaigne

List of Studies

Keywords
Genetics
Summary

The primary study objective in DEP is to assess the percentage change in blood Phe level.
The secondary objectives in Period 1 are to assess the absolute change in Phe level; to assess the safety and tolerability of SYNB1934; to assess the proportion of patients with a ≥ 20% reduction in blood Phe concentration; and, to assess proportion of patients achieving blood Phe ≤ 360 μmol/L.
Exploratory objectives in DEP are to determine the responder population for RWP, assess the absolute and relative change in blood Phe concentration; and to assess the proportion of patients achieving a blood Phe ≤ 600 μmol/L.

Institution
MUSC
Recruitment Contact
Annie Glenn
8437927965
glennju@musc.edu



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