8 patients with β-thalassemia tracked for years after one-time cell therapy
NCT ID NCT06717932
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study follows 8 people with β-thalassemia who already received a one-time treatment called CS-101, which modifies their own blood cells to produce fetal hemoglobin. No new drug is given. Researchers will monitor them for years to see if they stay free of blood transfusions and check for any long-term side effects.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- CS-101 (a cell therapy that modifies the patient's own blood cells to produce fetal hemoglobin)
- What this could lead to
- If successful, this could show that a single treatment with CS-101 allows people with β-thalassemia to live without needing regular blood transfusions.
- What could go wrong
- This is a very small follow-up study (8 people) with no new treatment given. It only looks at those who already received CS-101, so results may not apply to everyone. Long-term risks are still unknown.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Participants
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About 8 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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May 2025
- Expected to finish
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Nov 2039
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
Who is studied
Subject with β-thalassemia treated with CS-101 infusion in CS-101-01 study
- Ages
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12 to 35 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * 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 Exclusion Criteria: * There are no exclusion criteria
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Get notified about this study
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
The full official record for this study. This one lists no contact details, but it is the first place any would appear.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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The First Affiliated Hospital of Guangxi Medical University
Nanning, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a single stem cell infusion rewrite the code of beta thalassemia?
- A gentler transplant may cure sickle cell and thalassemia — can the body accept donor cells?
- How does a blood disorder drug perform in everyday practice?
- Newborn screening study aims to catch rare diseases at birth
- New stem cell transplant aims to ease severe blood disorders
- Gene-Editing breakthrough offers hope for blood disorder patients