Beta-ThalassemiaNCT06479616
A Long-term Follow-up Study in Participants Who Received CS-101
A study testing CS-101 in people with Beta-Thalassemia, run by Children's Hospital of Fudan University at 1 site in China.
Trial 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) Read more about this study
- Who is it for?
- Ages 3 to 17 years
- Conditions the study is for
- Beta-Thalassemia
- What is being tested?
- CS-101
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteChina
- Study dates
- 18 Jun 2024to Jun 2027
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- There are no exclusion criteria
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Children's Hospital of Fudan University
Shanghai, Shanghai Municipality, 201102
Zifeng Li, M.S., +86-13920704768, zfli18@fudan.edu.cn
About this study
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
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)
Read the detailed description
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) .
Subjects in the CS-101-03 study will be entered into long-term follow-up of this study up to 2 years post-infusion at the completion of the last (6-month) follow-up visit after treatment with CS-101 Injection.
- Conditions
- Beta-Thalassemia
- Being tested
- CS-101
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CS-101Genetic
Autologous CD34+ hematopoietic stem cell suspension modified by in vitro base editing technique
| Study group | Role | What they receive |
|---|---|---|
| long term follow up | Experimental | 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. Receives: Genetic: CS-101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency and severity of SAEs and CS-101 related AES as assessed by CTCAE v5.0
CommonTerminology Criteria for Adverse Events（CTCAE）has 5 levels of AE determination, increasing in severity as the level increases
Measured: Signing of informed consent up to 2 years post CS-101 infusion
Occurrence of all-cause death
Measured: Signing of informed consent up to 2 years post CS-101 infusion
New malignancies and hematologic disorders
Based on ICD-11
Measured: Signing of informed consent up to 2 years post CS-101 infusion
Occurrence of achieving transfusion independence for at least 12 consecutive months
Measured: From 3 months after last RBC transfusion up to 2 years post CS-101 infusion
Secondary outcomes (3)
Change in fetal hemoglobin(HbF) concentration over time
Measured: up to 2 years post-CS-101 infusion
Change in total hemoglobin(Hb) concentration over time
Measured: up to 2 years post-CS-101 infusion
Chimerism level in Peripheral blood and bone marrow Proportion of alleles with intended genetic modification in peripheral blood leukocytes and bone marrow over time
Proportion of alleles with intended genetic modification in peripheral blood leukocytes and bone marrow over time
Measured: up to 2 years post-CS-101 infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jun 2024
- Study start18 Jun 2024
- Primary completionDec 2026 (estimated)
- Study completionJun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Beta-ThalassemiaStudies of the same condition, open and under way.
- More trials run by Children's Hospital of Fudan UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Fudan University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Xiaowen Zhai, M.D., Principal investigator, Children's Hospital of Fudan University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06479616
- Sponsor's protocol ID
- CS-101-04
- Status last verified
- Oct 2026
- First submitted
- 24 Jun 2024
- Last updated
- 26 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06479616
- Acronym / sponsor ID
- CS-101-04
- Conditions
- Beta-Thalassemia
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 5 participants (estimated)
- Lead sponsor
- Children's Hospital of Fudan UniversityUniversity, hospital or other
- Study start
- 18 Jun 2024 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Jun 2027 (estimated)
- First posted
- 28 Jun 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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)
Detailed description
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) .
Subjects in the CS-101-03 study will be entered into long-term follow-up of this study up to 2 years post-infusion at the completion of the last (6-month) follow-up visit after treatment with CS-101 Injection.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| long term follow up | Experimental | Genetic: CS-101All participants who complete CS-101-03(NCT0606518) studie after CS-101 infusion will be asked to participate in this long-term follow-up study. |
Who may qualify
- Ages
- 3 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- 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
Key exclusion criteria (1 listed)
- There are no exclusion criteria
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CS-101Autologous CD34+ hematopoietic stem cell suspension modified by in vitro base editing technique | Genetic | long term follow up |
Primary outcome
Frequency and severity of SAEs and CS-101 related AES as assessed by CTCAE v5.0
Time frame: Signing of informed consent up to 2 years post CS-101 infusion
CommonTerminology Criteria for Adverse Events（CTCAE）has 5 levels of AE determination, increasing in severity as the level increases
Occurrence of all-cause death
Time frame: Signing of informed consent up to 2 years post CS-101 infusion
New malignancies and hematologic disorders
Time frame: Signing of informed consent up to 2 years post CS-101 infusion
Based on ICD-11
Occurrence of achieving transfusion independence for at least 12 consecutive months
Time frame: From 3 months after last RBC transfusion up to 2 years post CS-101 infusion
3 secondary outcomes
Change in fetal hemoglobin(HbF) concentration over time
Time frame: up to 2 years post-CS-101 infusion
Change in total hemoglobin(Hb) concentration over time
Time frame: up to 2 years post-CS-101 infusion
Chimerism level in Peripheral blood and bone marrow Proportion of alleles with intended genetic modification in peripheral blood leukocytes and bone marrow over time
Time frame: up to 2 years post-CS-101 infusion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Oct 2026.
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Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
Transfusion-dependent Beta-Thalassemia, Gene Therapy
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Xiaowen Zhai, M.D. principal investigator
Children's Hospital of Fudan University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing CS-101 in people with Beta-Thalassemia, run by Children's Hospital of Fudan University at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 26 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Xiaowen Zhai, M.D. (+86-021-64931126, zhaixiaowendy@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Frequency and severity of SAEs and CS-101 related AES as assessed by CTCAE v5.0 (time frame: Signing of informed consent up to 2 years post CS-101 infusion). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Oct 2026.