デュシェンヌ型筋ジストロフィーの男児におけるPF-06252616の安全性を評価するための非盲検延長試験

基本情報

NCT ID

NCT02907619

ステータス

中止

試験のフェーズ

第2相

試験タイプ

介入

目標被験者数

59

治験依頼者名

Pfizer

概要

This study is an open-label extension to protocol B5161002 and will provide an assessment of the long term safety, efficacy, pharmacodynamics and pharmacokinetics of intravenous dosing of PF 06252616 in boys with Duchenne muscular dystrophy. Approximately 105 eligible subjects will be assigned to receive a monthly individualized maximum tolerated dose based on their tolerability profile/data from B5161002. This study will not contain a placebo comparator. Subjects will undergo safety evaluations (Laboratory, cardiac monitoring, physical exams, x-ray, MRI), functional capacity evaluations (4 stair climb, range of motion, strength testing, Northstar Ambulatory Assessment, upper limb functional testing, six minute walk test and pulmonary function tests) and pharmacokinetic testing.

対象疾患

介入

依頼者（Sponsor）

実施施設 (2)