プロピオン酸血症患者を対象としたmRNA-3927のオープンラベル試験

基本情報

NCT ID

NCT04159103

ステータス

募集中

試験のフェーズ

第1/第2相

試験タイプ

介入

目標被験者数

77

治験依頼者名

ModernaTX, Inc.

概要

This 3-part, Phase 1/2 study is designed to characterize the safety, tolerability, and pharmacological activity (as assessed by biomarker measurements) and to determine the selected dose of mRNA-3927 in participants with genetically confirmed propionic acidemia (PA). After establishing a dose with an acceptable safety and pharmacodynamic (PD) response for participants ≥1 year of age in Part 1, participants will be enrolled in Part 2 (which will serve as the pivotal study) to allow for determination of the efficacy, safety, and PD of mRNA-3927. Part 3 will evaluate the safety, efficacy and PD response of mRNA-3927 in infants (\<1 year of age).

対象疾患

介入

依頼者（Sponsor）

実施施設 (3)