ムコ多糖症I型（MPS I）患者におけるアルデュラザイム®補充療法の研究

基本情報

NCT ID

NCT00258011

ステータス

完了

試験のフェーズ

第3相

試験タイプ

介入

目標被験者数

3

治験依頼者名

Sanofi

概要

This is a multi-center, open label, study conducted to evaluate the safety of laronidase administered by intravenous drip infusion in Japanese patients with MPS I disease. Following baseline evaluation, patients will receive weekly infusions of JC0498 at an intravenous dose of 100 units/kg. Patient safety will be monitored continuously throughout the trial. In addition, the effects of JC0498 treatment in this patient population will be assessed by periodically evaluating aspects of MPS I disease in patients at scheduled intervals over the duration of the trial. Since patients may be eligible for the trial if they have received JC0498, a portion of the data may be captured retrospectively and recorded onto the case report forms (CRFs). This study represents the first good clinical practice (GCP) effort to characterize MPS I in the Japanese population and evaluate the effects of JC0498 on disease manifestations.

対象疾患

介入

依頼者（Sponsor）

実施施設 (2)