CS-206 in Patients With Sickle Cell Disease
ConditionSickle Cell Disease
Trial statusRecruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age12-35
SponsorCorrectSequence Therapeutics Co., Ltd
About this trial
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 injection in treating sickle cell disease.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Participants must be between 12 to 35 years old (inclusive). Participants or their legal guardians (for participants below 18 years old) must provide written informed consent before any study-related procedures.
Participants must have a Documented βS/βS, βS/β0 or βS/β+ genotype.
Participants must have at least one of the following conditions
At least 2 occurrences of any of the following events within 2 years prior to screening.
Disqualifiers
Female participants who are pregnant, breastfeeding, or planning pregnancy during the study period are excluded.
Participation in another investigational drug trial within 30 days prior to screening or within 5 half-lives (whichever is longer).
Subjects who have received or are receiving luspatercept treatment within 3 months prior to screening.
Subjects who have previously received any gene therapy for the disease.
Trial design
Design model
Single group
Treatments tested in this trial
CS-206
GeneticAutologous CD34+ hematopoietic stem cell suspension modified by in vitro base editing technique
Treatment groups
5 Participants
are divided into 1 treatment groupGroup A: CS-206Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
AEs(Adverse Events) and SAEs(Serious Adverse Events) after CS-101 infusion
Frequency and severity of adverse events(AEs）as assessed by CTCAE(Common Terminology Criteria for Adverse Events)v5.0
Time frame
From signing informed consent to 24 months post-CS-206 infusion2
Incidence of transplant-related mortality
Incidence of transplant-related mortality(Transplant-related mortality events defined as deaths assessed by the investigator as potentially transplant-related)
Time frame
From baseline to 100 days and 12 months post-CS-206 infusion3
Time to neutrophil engraftment
Time to neutrophil engraftment is defined as first day of 3 consecutive measurements of absolute neutrophil count≥0.5×10\^9/L on three different days.
Time frame
Up to 24 months post-CS-206 infusion4
Time to platelet engraftment
Time to platelet engraftment is defined as first day of 3 consecutive measurements of absolute platelet count≥20×10\^9/L on three different days and without platelet transfusion.
Time frame
Up to 24 months post-CS-206 infusionSecondary outcomes
1
Free from hospitalization due to severe vaso-occlusive crises for 12 consecutive months(HF12)
Time frame
starting 60 days after the last red blood cell transfusion up to 24 months2
Free from severe VOCs for 9 consecutive months (VF9)
Free from severe vaso-occlusive crises (VOCs) for 9 consecutive months (VF9)
Time frame
starting 60 days after the last red blood cell transfusion up to 24 months3
Annualized incidence of severe vaso-occlusive crises (VOC)
Time frame
starting 60 days after the last red blood cell transfusion up to 24 months4
Annualized incidence of hospitalization due to severe vaso-occlusive crises
Time frame
starting 60 days after the last red blood cell transfusionSponsors and contacts
Click on the lead sponsor to view all of their trials.
CorrectSequence Therapeutics Co., Ltd
Lead sponsor
First Affiliated Hospital of Guangxi Medical University
Collaborator
Source ClinicalTrials.gov