Beta-ThalassemiaNCT06717932
A Follow-up Study to Evaluate the Long-term Safety and Efficacy in Participants Who Received CS-101 Injection
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 -101-01 (NCT06291961),No investigational drug product will be administered in the study. Read more about this study
- Who is it for?
- Ages 12 to 35 years
- Conditions the study is for
- Beta-Thalassemia
- How many people
- 8 participants
- Where
- 1 siteChina
- Study dates
- 15 May 2025to Nov 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 12 to 35 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Subject with β-thalassemia treated with CS-101 infusion in CS-101-01 study
Who may be able to join
- Participants must have received CS-101 infusion in CS-101-01 study
- 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
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.
About this study
Official title: A Long-term Follow-up Study Evaluating the Safety and Efficacy of Subjects With β-thalassemia Treated With 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 -101-01 (NCT06291961),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 -101-01 (NCT06291961). Subjects in the CS-101-01 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) follow up visit after treatment with CS-101 Injection.
- Conditions
- Beta-Thalassemia
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Safety and efficacy assessments | 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
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 posted5 Dec 2024
- Study start15 May 2025
- Primary completionNov 2039 (estimated)
- Study completionNov 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
- 8 participants (estimated)
Sponsor and investigators
- Lead sponsor
- CorrectSequence Therapeutics Co., Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06717932
- Sponsor's protocol ID
- CS-101-05
- Status last verified
- Feb 2026
- First submitted
- 1 Dec 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 CS-101 Transplantation
- ClinicalTrials.gov ID
- NCT06717932
- Acronym / sponsor ID
- CS-101-05
- Conditions
- Beta-Thalassemia
- Study type
- Observational
- Recruitment status
- Enrolling by invitation
- Enrolment
- 8 participants (estimated)
- Lead sponsor
- CorrectSequence Therapeutics Co., LtdIndustry (pharma, biotech, devices)
- Study start
- 15 May 2025 (actual)
- Primary completion
- Nov 2039 (estimated)
- Study completion
- Nov 2039 (estimated)
- First posted
- 5 Dec 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 -101-01 (NCT06291961),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 -101-01 (NCT06291961). Subjects in the CS-101-01 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) follow up visit after treatment with CS-101 Injection.
Study design
- Observational model
- Other
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Safety and efficacy assessments | – | –Safety evaluations,disease-specific assessments,and assessments to monitor for long-term complications of autologous transplant |
Who may qualify
- Ages
- 12 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 CS-101-01 study
Key inclusion criteria (2 listed)
- Participants must have received CS-101 infusion in CS-101-01 study
- 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
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.
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
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
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A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
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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.
The record lists no contact. Use the official record to reach the sponsor.
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 3 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.