Clinical trial Recruiting at 1 site Interventional Ages 3 to 17

Long-term follow-up for patients treated with CS-101

Official title: 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

Run by Children's Hospital of Fudan University · NCT06479616 · Data updated 2026-11-12

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

Study typeInterventional
Ages3 Years to 17 Years
SexAll
Enrollment5 (estimated)
SponsorChildren's Hospital of Fudan University
Academic / other
Locations1 site (Shanghai, Shanghai Municipality)
StatusRecruiting
StartedJune 18, 2024
Main results expectedDecember 2026
Study endsJune 2027
Registry record updatedOctober 26, 2026
Trial IDNCT06479616 on ClinicalTrials.gov

Is this trial a fit for you?

Likely a good fit

Ideal candidates are individuals who have previously received the CS-101 infusion in the related study.

Probably not a fit

Patients who have not received the CS-101 infusion will not benefit from this study.

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

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 gone: 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.

Who can join: the full rules

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 it's running

  • Children's Hospital of Fudan University
    Shanghai, Shanghai Municipality, China
    Recruiting

Study contacts

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

How to take part

  1. Review the eligibility criteria above with your treating physician.
  2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
  3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.
Last reviewed 2026-11-14 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.