Long-term follow-up for patients treated with CS-101
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
This study is checking how safe and effective the CS-101 treatment is for people who had it before, by following them for up to two years to see how they’re doing.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 5 (estimated) |
| Ages | 3 Years to 17 Years |
| Sex | All |
| Sponsor | Children's Hospital of Fudan University Academic / other |
| Locations | 1 site (Shanghai, Shanghai Municipality) |
| Trial ID | NCT06479616 on ClinicalTrials.gov |
What this trial studies
This study evaluates the long-term safety and efficacy of CS-101 in participants who previously received this treatment in an earlier study. Participants will be monitored for up to two years following their last follow-up visit after the CS-101 infusion. The aim is to gather comprehensive data on the effects and outcomes of the treatment over an extended period.
Who should consider this trial
Good fit: Ideal candidates are individuals who have previously received the CS-101 infusion in the related study.
Not a fit: Patients who have not received the CS-101 infusion will not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the long-term effects of CS-101, potentially improving treatment strategies for beta-thalassemia.
How similar studies have performed: While this study builds on previous research involving CS-101, the long-term follow-up aspect is relatively novel and aims to provide new insights.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * 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 Exclusion Criteria: * There are no exclusion criteria
Where this trial is running
Shanghai, Shanghai Municipality
- Children's Hospital of Fudan University — Shanghai, Shanghai Municipality, China (Recruiting)
Study contacts
- Principal investigator: Xiaowen Zhai, M.D. — Children's Hospital of Fudan University
- Study coordinator: Xiaowen Zhai, M.D.
- Email: zhaixiaowendy@163.com
- Phone: +86-021-64931126
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.