Beta-ThalassemiaNCT06685536
A Long-term Follow-up Study in Participants Who Received CS-101
An observational study of Beta-Thalassemia, run by CorrectSequence Therapeutics Co., Ltd 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 -101-06 (NCT06024876),No investigational drug product will be administered in the study. Read more about this study
- Who is it for?
- Ages 6 to 35 years
- Conditions the study is for
- Beta-Thalassemia
- What is being tested?
- Safety and efficacy assessments
- How many people
- 5 participants
- Where
- 1 siteChina
- Study dates
- 18 Jan 2025to Jul 2039
- Phase
- Observational
- 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 6 to 35 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Subject with β-thalassemia treated with CS-101 infusion in last study
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 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
The First Affiliated Hospital of Guangxi Medical University
Nanning, Guangxi
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 -101-06 (NCT06024876),No investigational drug product will be administered in the study.
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 -101-06 (NCT06024876). Subjects in the CS-101-06 study will be entered into long-term follow-up of this study up to 15 years post-infusion at the completion of the last (12-month) followup visit after treatment with CS-101 Injection.
- Conditions
- Beta-Thalassemia
- Being tested
- Safety and efficacy assessments
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Safety and efficacy assessmentsOther
Safety evaluations,disease-specific assessments,and assessments to monitor for long-term complications of autologous transplant
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: Through 15 years post CS-101 infusion
New malignancies and hematologic disorders
Based on ICD-11
Measured: Through 15 years post CS-101 infusion
Occurrence of all-cause death
Measured: Through 15 years post CS-101 infusion
Occurrence of achieving transfusion independence for at least 12 consecutive months
Measured: Through 15 years post CS-101 infusion
Secondary outcomes (5)
Change in fetal hemoglobin(HbF) concentration over time
Measured: Through 15 years post CS-101 infusion
Change in total hemoglobin(Hb) concentration over time
Measured: Through 15 years post CS-101 infusion
Chimerism level in Peripheral blood Proportion of alleles with intended genetic modification in
Measured: Through 15 years post CS-101 infusion
Chimerism level in bone marrow Proportion of alleles with intended genetic modification in
Measured: Through 15 years post CS-101 infusion
Change in F-Cell concentration over time
Measured: Through 15 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 posted12 Nov 2024
- Study start18 Jan 2025
- Primary completionJul 2039 (estimated)
- Study completionJul 2039 (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.
- 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
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 5 participants (estimated)
Sponsor and investigators
- Lead sponsor
- CorrectSequence Therapeutics Co., Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- First Affiliated Hospital of Guangxi Medical University
- Responsible party
- Sponsor
- Investigators
- Yongrong Lai, M.D., Principal investigator, First Affiliated Hospital of Guangxi Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06685536
- Sponsor's protocol ID
- CS-101-13
- Status last verified
- Feb 2026
- First submitted
- 7 Nov 2024
- Last updated
- 10 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Feb 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
- NCT06685536
- Acronym / sponsor ID
- CS-101-13
- Conditions
- Beta-Thalassemia
- Study type
- Observational
- Recruitment status
- Enrolling by invitation
- Enrolment
- 5 participants (estimated)
- Lead sponsor
- CorrectSequence Therapeutics Co., LtdIndustry (pharma, biotech, devices)
- Collaborators
- First Affiliated Hospital of Guangxi Medical University
- Study start
- 18 Jan 2025 (actual)
- Primary completion
- Jul 2039 (estimated)
- Study completion
- Jul 2039 (estimated)
- First posted
- 12 Nov 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 -101-06 (NCT06024876),No investigational drug product will be administered in the study.
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 -101-06 (NCT06024876). Subjects in the CS-101-06 study will be entered into long-term follow-up of this study up to 15 years post-infusion at the completion of the last (12-month) followup visit after treatment with CS-101 Injection.
Study design
- Observational model
- Other
- Time perspective
- Prospective
Who may qualify
- Ages
- 6 to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Subject with β-thalassemia treated with CS-101 infusion in last study
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 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 |
|---|---|---|
| Safety and efficacy assessmentsSafety evaluations,disease-specific assessments,and assessments to monitor for long-term complications of autologous transplant | Other | – |
Primary outcome
Frequency and severity of SAEs and CS-101 related AES as assessed by CTCAE v5.0
Time frame: Through 15 years post CS-101 infusion
CommonTerminology Criteria for Adverse Events#CTCAE#has 5 levels of AE determination, increasing in severity as the level increases
New malignancies and hematologic disorders
Time frame: Through 15 years post CS-101 infusion
Based on ICD-11
Occurrence of all-cause death
Time frame: Through 15 years post CS-101 infusion
Occurrence of achieving transfusion independence for at least 12 consecutive months
Time frame: Through 15 years post CS-101 infusion
5 secondary outcomes
Change in fetal hemoglobin(HbF) concentration over time
Time frame: Through 15 years post CS-101 infusion
Change in total hemoglobin(Hb) concentration over time
Time frame: Through 15 years post CS-101 infusion
Chimerism level in Peripheral blood Proportion of alleles with intended genetic modification in
Time frame: Through 15 years post CS-101 infusion
Chimerism level in bone marrow Proportion of alleles with intended genetic modification in
Time frame: Through 15 years post CS-101 infusion
Change in F-Cell concentration over time
Time frame: Through 15 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 10 Feb 2026.
- Individual participant dataSharing planned: No
Related trials for Beta-Thalassemia
Active, not recruiting
Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.
Transfusion-dependent Beta-Thalassemia
RecruitingObservational study
Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
Transfusion-dependent Beta-Thalassemia, Gene Therapy
Not yet recruiting
Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector
Beta-Thalassemia
RecruitingPhase 1/2
β-Thalassemia Treatment With KL003 Cell Injection
Transfusion-dependent Beta-Thalassemia
Active, not recruitingPhase 3
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
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.
Yongrong Lai, M.D. principal investigator
First Affiliated Hospital of Guangxi Medical University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Beta-Thalassemia, run by CorrectSequence Therapeutics Co., Ltd 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: enrolling by invitation, as of the sponsor's last update on 10 Feb 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?
The record lists no contact. The official record names the sponsor, who can be asked directly. 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: Through 15 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 10 Feb 2026.