Clinical trial NCT06685536: A Long-term Follow-up Study in Participants Who Received CS-101
Official title: A Long-term Follow-up Study Evaluating the Safety and Efficacy of Subjects With β-thalassemia Treated With Base-edited Autologous Hematopoietic Stem Cell (CS-101) Transplantation
Study type: OBSERVATIONAL
Overall status: ENROLLING_BY_INVITATION
Conditions: Beta-Thalassemia
Lead sponsor: CorrectSequence Therapeutics Co., Ltd
Sponsor class: INDUSTRY
Collaborators: name: First Affiliated Hospital of Guangxi Medical University
First posted: 2024-11-12
Enrollment: 5
Sex: ALL
Minimum age (years): 6
Maximum age (years): 35
Standard ages: CHILD
ADULT
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Subject with β-thalassemia treated with CS-101 infusion in last study

Brief 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 -101-06 (NCT06024876),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 -101-06 (NCT06024876). Subjects in the CS-101-06 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) followup visit after treatment with CS-101 Injection.

Eligibility criteria: Inclusion Criteria:

* 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 study

Exclusion Criteria:

* There are no exclusion criteria

Primary outcomes:
measure: Frequency and severity of SAEs and CS-101 related AES as assessed by CTCAE v5.0
timeFrame: Through 15 years post CS-101 infusion
measure: New malignancies and hematologic disorders
timeFrame: Through 15 years post CS-101 infusion
measure: Occurrence of all-cause death
timeFrame: Through 15 years post CS-101 infusion
measure: Occurrence of achieving transfusion independence for at least 12 consecutive months
timeFrame: Through 15 years post CS-101 infusion
Secondary outcomes:
measure: Change in fetal hemoglobin(HbF) concentration over time
timeFrame: Through 15 years post CS-101 infusion
measure: Change in total hemoglobin(Hb) concentration over time
timeFrame: Through 15 years post CS-101 infusion
measure: Chimerism level in Peripheral blood Proportion of alleles with intended genetic modification in
timeFrame: Through 15 years post CS-101 infusion
measure: Chimerism level in bone marrow Proportion of alleles with intended genetic modification in
timeFrame: Through 15 years post CS-101 infusion
measure: Change in F-Cell concentration over time
timeFrame: Through 15 years post CS-101 infusion
Other outcomes:
Locations:
city: Nanning
country: China
facility: The First Affiliated Hospital of Guangxi Medical University
state: Guangxi
Study officials:
affiliation: First Affiliated Hospital of Guangxi Medical University
name: Yongrong Lai, M.D.
role: PRINCIPAL_INVESTIGATOR
References: