8 patients with β-thalassemia tracked for years after one-time cell therapy

NCT ID NCT06717932

ENROLLING_BY_INVITATION Disease control Sponsor: CorrectSequence Therapeutics Co., Ltd Source: ClinicalTrials.gov ↗

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.

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Conditions

The condition(s) this trial relates to.

beta thalassemia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

BETA-THALASSEMIA

Contacts and locations

Locations

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.