A Long-term Follow-up Study Evaluating the Safety and Efficacy of Subjects With β-thalassemia Treated With Base-edited Autologous Hematopoietic Stem Cell (CS-101) Transplantation
Status: Recruiting
Location: See location...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Not Applicable
SUMMARY
This is a study to evaluate the long-term safety and efficacy of CS-101 in participants who received CS-101 in study CS -101-03 (NCT06065189)
Eligibility
Participation Requirements
Sex: All
Minimum Age: 3
Maximum Age: 17
Healthy Volunteers: f
View:
• Participants (or his or her legally appointed and authorized representative or guardian) must sign and date informed consent form (ICF) and, where applicable, an assent form
• Participants must have received CS-101 infusion in last IIT study
Locations
Other Locations
China
Children's Hospital of Fudan University
RECRUITING
Shanghai
Contact Information
Primary
Xiaowen Zhai, M.D.
zhaixiaowendy@163.com
+86-021-64931126
Backup
Zifeng Li, M.S.
zfli18@fudan.edu.cn
+86-13920704768
Time Frame
Start Date: 2024-06-18
Estimated Completion Date: 2027-06
Participants
Target number of participants: 5
Treatments
Experimental: long term follow up
All participants who complete CS-101-03(NCT0606518) studie after CS-101 infusion will be asked to participate in this long-term follow-up study.
Related Therapeutic Areas
Sponsors
Leads: Children's Hospital of Fudan University