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New treatment option for Sickle Cell Disease
Official title CS-206 in Patients With Sickle Cell Disease
ClinicalTrials.gov ID: NCT06565026
What this study is testing
What is CS-206?
CS-206 is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for sickle cell disease.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 12 to 35
You may be able to join if
- Participants must be between 12 to 35 years old (inclusive). Participants or their legal guardians (for participants below 18 years old) must provide...
- 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.
- Acute pain crisis: requiring a visit to a medical facility and administration of pain medications (opioids or intravenous NSAIDs) or red blood cell...
You likely can't join if
- 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).
- people who have received or are receiving luspatercept treatment within 3 months prior to screening.
- people who have previously received any gene therapy for the disease.
- people with a fully matched related donor who are already scheduled for allogeneic hematopoietic stem cell transplantation.
- More than 10 unplanned hospitalizations or emergency visits within 12 months prior to screening, which the investigator believes are related to...
See the full eligibility criteria
- 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.
- Acute pain crisis: requiring a visit to a medical facility and administration of pain medications (opioids or intravenous NSAIDs) or red blood cell transfusions.
- Acute chest syndrome: defined by the presence of a new pulmonary infiltrate on a chest X-ray, associated with pneumonia-like symptoms, including chest pain, fever, or respiratory distress.
- Priapism lasting more than 2 hours and necessitating a visit to a medical facility for intervention.
- Stroke or transient ischemic attack (TIA): confirmed by imaging studies (e.g., MRI or CT scan), including silent stroke, and overt stroke leading to neurological deficits lasting \>24 hours.
- Presence of red cell alloimmunization (\>2 antibodies) and the need for ongoing chronic transfusions.
- Participants who have failed, not tolerated, refused the standard of care for Sickle Cell Disease (SCD), or are unable to access the standard of care due to the availability
- Other situations deemed appropriate for hematopoietic stem cell transplantation according to the sickle cell anemia treatment guidelines, as determined by the investigator.
- Laboratory Parameters:
- Documented Hemoglobin S (HbS) level ≥30% of total hemoglobin (Hb) concentration prior to transfusion.
- HbF at screening \< 20%
- Participants must have a Karnofsky Performance Status (KPS for participants above 16 years old, inclusive) or Lansky Play-Performance Scale (LPPS for participants below 16 years old) score of ≥70, indicating sufficient...
- Willing to comply with the protocol requirements, use contraception as required, attend regular follow-up visits, and cooperate with examinations.
- 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).
- people who have received or are receiving luspatercept treatment within 3 months prior to screening.
- people who have previously received any gene therapy for the disease.
- people with a fully matched related donor who are already scheduled for allogeneic hematopoietic stem cell transplantation.
- More than 10 unplanned hospitalizations or emergency visits within 12 months prior to screening, which the investigator believes are related to significant chronic pain rather than acute pain crisis (VOC).
- Severe liver dysfunction:
- Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \>3× the upper limit of normal (ULN) or:
- International Normalized Ratio (INR) \>1.5× ULN
- Severe renal impairment (creatinine clearance \<30 mL/min/1.73 m²) are excluded.
- people with HIV, cytomegalovirus (CMV), Epstein-Barr virus (EBV), or Treponema pallidum infection during the screening period; those with active HBV or HCV infection; or known tuberculosis or parasitic infection, etc...
- Deemed unsuitable for autologous hematopoietic stem cell transplantation procedures as determined by the investigator.
- Other situations deemed unsuitable for this study as determined by the investigator.
The study team makes the final eligibility decision.
Where it's taking place
- Nanning, Guangxi, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 12 years to 35 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Nanning, Guangxi, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.