A Safety and Efficacy Study Evaluating CS-101 in Subjects With β-Thalassemia Major (NCT06291961) | Clinical Trial Compass
CompletedPhase 1
A Safety and Efficacy Study Evaluating CS-101 in Subjects With β-Thalassemia Major
China9 participantsStarted 2024-04-18
Plain-language summary
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 in treating patients with β-thalassemia major anemia.
Who can participate
Age range
12 Years – 35 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* 12 to 35 years old(inclusive) male or female subjects at the time of informed consenting. For minors, their legal representative is required to sign the informed consent form, besides, if the subjects aged 8 years or older, they should provide a signed and dated.
* Diagnosis of β-thalassemia major.
* Generally in good condition, Karnofsky performance score≥60 points for subjects≥16 years old, or Lansky Play-Performance score≥60 points for subjects under 16 years old.
* For female subjects of childbearing potential: use effective contraceptive measures from the start of screening and agree to continue using such measures for contraception throughout the study
* For male subjects who have a potential ability to father a child: use condoms or other methods continuously from the start of mobilization to ensure effective contraception for sexual partners during the study period
Exclusion Criteria:
* Treatment with other investigational medications or other experimental interventions 30 days prior to signing informed consent or within 6 half-lives of the drug, whichever is longer
* Subjects who have received or are receiving thalidomide and/or Luspatercept in the past 6 months before screening
* Previously received allogeneic hematopoietic stem cell transplantation or gene(edited) therapy
* Subjects have available related fully matching donors and are eligible and prepared for allogeneic hematopoietic stem cell transplantation
* Patients with coexisting α-t…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
AEs(Adverse Events) and SAEs(Serious Adverse Events) after CS-101 infusion
Timeframe: From signing informed consent to 12 months post-CS-101 infusion
2
Overall survival rate
Timeframe: Up to 12 months post-CS-101 infusion
3
Proportion of subjects with engraftment
Timeframe: Within 42 days post-CS-101 infusion
4
Time to neutrophil engraftment
Timeframe: Up to 12 months post-CS-101 infusion
5
Time to platelet engraftment
Timeframe: Up to 12 months post-CS-101 infusion
6
Incidence of transplant-related mortality
Timeframe: From baseline to 100 days post-CS-101 infusion
7
Change in fetal hemoglobin(HbF) concentration over time