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临床试验/NCT05154851
NCT05154851No Longer Available不适用

An Expanded Access IND to Evaluate the Safety and Efficacy of Autologous HBadMSCs for the Treatment of a Single Pediatric Patient With Congenital Muscular Dystrophy.

Hope Biosciences Research Foundation1 个研究点 分布在 1 个国家开始时间: 2021年12月13日最近更新:
适应症

试验速览

阶段
不适用
状态
No Longer Available
试验地点
1

研究概览

简要总结

This individual patient expanded access IND is requested for a patient diagnosed with LMNA-related congenital muscular dystrophy (L-CMD). In this expanded access, the patient will receive the investigational product through 14 intravenous infusions, followed by Follow-Up visit and an End of Study.

详细描述

This Expanded Access includes an up to 28 days screening period, a 44-week Treatment Period, and a 52-week Safety Follow-up Period. All periods are associated with evaluations and procedures that must be performed at specific time points.

-Screening

At the Screening Visit (Visit 1), the study's participant information will be collected by delegated personnel to evaluate trial eligibility. The following information is required to determine eligibility:

  • Parent Permission before any trial-related activities.
  • Collection of demographic information, such as age, race, ethnicity, date of birth, gender, and relevant medical and surgical history.
  • Collection of Medical History and concomitant medications, including relevant information about study participants' past and present health.
  • Inclusion and Exclusion criteria evaluation.
  • Measurement of vital signs includes respiratory rate, body temperature, blood pressure, pulse rate, oxygen saturation, and weight and height measurement.
  • Blood chemistry, Coagulation panel, Inflammatory Markers, & Hematology.
  • Physical examination by Principal Investigator.

Within 28 days of the Visit 1 - Screening, the principal investigator must decide the participant's eligibility. Once the principal investigator has confirmed the subject's eligibility, the subject will be scheduled to receive Infusion 1.

研究设计

研究类型
Expanded Access

入排标准

性别
All
接受健康志愿者

入选标准

  • Subject diagnosed with LCMNA congenital muscular dystrophy.
  • Subject must have banked his stem cells at Hope Biosciences LLC.

排除标准

  • Subject has any active infection requiring medications.
  • The subject has any known coagulation anomalies.

研究者

申办方类型
Industry
责任方
Sponsor

研究点 (1)

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