CS-101 in Patients With Sickle Cell Disease
- Conditions
- Sickle Cell Disease
- Registration Number
- NCT06565026
- Lead Sponsor
- CorrectSequence Therapeutics Co., Ltd
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- Not specified
Inclusion Criteria:<br><br> - Participants must be between 12 to 35 years old (inclusive). Participants or their<br> legal guardians (for participants below 18 years old) must provide written informed<br> consent before any study-related procedures.<br><br> - Participants must have a Documented ßS/ßS, ßS/ß0 or ßS/ß+ genotype.<br><br> - Participants must have at least one of the following conditions<br><br> 1. At least 2 occurrences of any of the following events within 2 years prior to<br> screening.<br><br> 1. Acute pain crisis: requiring a visit to a medical facility and<br> administration of pain medications (opioids or intravenous NSAIDs) or red<br> blood cell transfusions.<br><br> 2. Acute chest syndrome: defined by the presence of a new pulmonary<br> infiltrate on a chest X-ray, associated with pneumonia-like symptoms,<br> including chest pain, fever, or respiratory distress.<br><br> 3. Priapism lasting more than 2 hours and necessitating a visit to a medical<br> facility for intervention.<br><br> 4. Stroke or transient ischemic attack (TIA): confirmed by imaging studies<br> (e.g., MRI or CT scan), including silent stroke, and overt stroke leading<br> to neurological deficits lasting >24 hours.<br><br> 2. Presence of red cell alloimmunization (>2 antibodies) and the need for ongoing<br> chronic transfusions.<br><br> 3. Participants who have failed, not tolerated, refused the standard of care for<br> Sickle Cell Disease (SCD), or are unable to access the standard of care due to<br> the availability<br><br> 4. Other situations deemed appropriate for hematopoietic stem cell transplantation<br> according to the sickle cell anemia treatment guidelines, as determined by the<br> investigator.<br><br> - Laboratory Parameters:<br><br> 1. Documented Hemoglobin S (HbS) level =30% of total hemoglobin (Hb) concentration<br> prior to transfusion.<br><br> 2. HbF at screening < 20%<br><br> - Participants must have a Karnofsky Performance Status (KPS for participants above 16<br> years old, inclusive) or Lansky Play-Performance Scale (LPPS for participants below<br> 16 years old) score of =70, indicating sufficient functional status to undergo the<br> intervention.<br><br> - Willing to comply with the protocol requirements, use contraception as required,<br> attend regular follow-up visits, and cooperate with examinations.<br><br>Exclusion Criteria:<br><br> - Female participants who are pregnant, breastfeeding, or planning pregnancy during<br> the study period are excluded.<br><br> - Participation in another investigational drug trial within 30 days prior to<br> screening or within 5 half-lives (whichever is longer).<br><br> - Subjects who have received or are receiving luspatercept treatment within 3 months<br> prior to screening.<br><br> - Subjects who have previously received any gene therapy for the disease.<br><br> - Subjects with a fully matched related donor who are already scheduled for allogeneic<br> hematopoietic stem cell transplantation.<br><br> - More than 10 unplanned hospitalizations or emergency visits within 12 months prior<br> to screening, which the investigator believes are related to significant chronic<br> pain rather than acute pain crisis (VOC).<br><br> - Severe liver dysfunction:<br><br> 1. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >3× the<br> upper limit of normal (ULN) or:<br><br> 2. International Normalized Ratio (INR) >1.5× ULN<br><br> - Severe renal impairment (creatinine clearance <30 mL/min/1.73 m²) are excluded.<br><br> - Subjects with HIV, cytomegalovirus (CMV), Epstein-Barr virus (EBV), or Treponema<br> pallidum infection during the screening period; those with active HBV or HCV<br> infection; or known tuberculosis or parasitic infection, etc. Excludes subjects with<br> stable hepatitis B (HBV-DNA negative) after treatment and those cured of hepatitis C<br> (HCV-RNA negative). Known active bacterial, viral, or fungal infections.<br><br> - Deemed unsuitable for autologous hematopoietic stem cell transplantation procedures<br> as determined by the investigator.<br><br> - Other situations deemed unsuitable for this study as determined by the investigator.
Not provided
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method AEs(Adverse Events) and SAEs(Serious Adverse Events) after CS-101 infusion;Incidence of transplant-related mortality;Time to neutrophil engraftment;Time to platelet engraftment;All-cause mortality;Free from severe VOCs for 12 consecutive months (VF12)
- Secondary Outcome Measures
Name Time Method Free from hospitalization due to severe vaso-occlusive crises for 12 consecutive months(HF12);Free from severe VOCs for 9 consecutive months (VF9);Annualized incidence of severe vaso-occlusive crises (VOC);Annualized incidence of hospitalization due to severe vaso-occlusive crises;HbF (fetal hemoglobin) level in blood samples;Proportion of edited alleles in peripheral blood leukocytes and bone marrow cells, and persistence and chimerism kinetics evaluation