軟骨無形成症の小児におけるレシフェルセプトの安全性、忍容性および有効性に関する研究

基本情報

NCT ID

NCT04638153

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

60

治験依頼者名

Pfizer

概要

Approximately 63 participants will be randomized to one of three doses to receive Recifercept either * Low Dose * Medium Dose * High Dose Participants will will attend the clinic at baseline and at Day 1, 4, 8, 15, 29 \& then Month 2, 3 6, 9 \& 12. Assessments include safety, blood sampling, physical examination, vital signs, anthropometric body measurements \& patient/caregiver quality of life questionnaires Participants will received treatment with Recifercept for 12 months. All participants who complete the study and in the opinion of the investigator, continue to have a positive risk:benefit profile, will be offered to enroll into an open-label extension (OLE) study. A PK cohort will include 12 participants who will randomly receive a single dose of 3 mg/kg of Phase 2 study (process 1c) formulation and a single dose of 3 mg/kg of the proposed Phase 3 (process 2) study formulation in a cross over study. Dose of the cohort could be changed due to emerging safety and efficacy data in the study.

対象疾患

介入

依頼者（Sponsor）

実施施設 (2)