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.
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
- 否
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.
