A Long-term Follow-up Study in Participants Who Received CS-101
ConditionBeta-Thalassemia
Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age3-17
SponsorChildren's Hospital of Fudan University
About this trial
This is a study to evaluate the long-term safety and efficacy of CS-101 in participants who received CS-101 in study CS -101-03 (NCT06065189)
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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 IIT study
Disqualifiers
There are no exclusion criteria
Trial design
Design model
Single group
Treatments tested in this trial
CS-101
GeneticAutologous CD34+ hematopoietic stem cell suspension modified by in vitro base editing technique
Treatment groups
5 Participants
are divided into 1 treatment groupGroup A: long term follow upExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
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
Time frame
Signing of informed consent up to 2 years post CS-101 infusion2
Occurrence of all-cause death
Time frame
Signing of informed consent up to 2 years post CS-101 infusion3
New malignancies and hematologic disorders
Based on ICD-11
Time frame
Signing of informed consent up to 2 years post CS-101 infusion4
Occurrence of achieving transfusion independence for at least 12 consecutive months
Time frame
From 3 months after last RBC transfusion up to 2 years post CS-101 infusionSecondary outcomes
1
Change in fetal hemoglobin(HbF) concentration over time
Time frame
up to 2 years post-CS-101 infusion2
Change in total hemoglobin(Hb) concentration over time
Time frame
up to 2 years post-CS-101 infusion3
Chimerism level in Peripheral blood and bone marrow Proportion of alleles with intended genetic modification in peripheral blood leukocytes and bone marrow over time
Proportion of alleles with intended genetic modification in peripheral blood leukocytes and bone marrow over time
Time frame
up to 2 years post-CS-101 infusionSponsors and contacts
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Source ClinicalTrials.gov