Phase 2 Sickle Cell Disease Trial, Recruiting

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• People who have been diagnosed with sickle cell disease (SCD) with one of these specific genetic types: βS/βS, βS/β0, or βS/β+.
• People who are between 2 and 50 years old at the time of signing consent.
• People who weigh at least 6 kg.
• People who have a reasonable level of general physical functioning, measured by a standard performance scale (Karnofsky score of 60 or above for those aged 16 and over, or Lansky score of 60 or above for those under 16).
• People who have been treated and regularly monitored for their sickle cell disease at a medical centre that kept detailed records for at least the past 24 months.
• People who have had at least 4 painful vaso-occlusive episodes (VOEs — episodes where blood flow is blocked, causing pain or other complications) in the 24 months before signing consent, while receiving appropriate supportive care such as a pain management plan.
• People who have either tried the medication hydroxyurea (HU) in the past and it did not work well enough, or who were unable to continue taking it due to intolerance.
• People of childbearing potential (both female and male) who agree to use one highly effective method of contraception from the screening stage until at least 6 months after receiving the treatment.
• People (or their parent/legal guardian, where applicable) who are able to provide written informed consent to participate in the study.

Who may not be able to join:

• People for whom a stem cell transplant from a matched related donor is considered medically appropriate by the treating doctor, and where such a donor is available.
• People with severe blood vessel disease in the brain, including any history of stroke, certain abnormal blood flow results in brain scans, significant narrowing or blockage of brain blood vessels, or a condition called Moyamoya disease detected on screening scans.
• People who test positive for HIV-1 or HIV-2, hepatitis B, hepatitis C, HTLV-1 (a type of virus), or active syphilis.
• People with a clinically significant, active infection caused by bacteria, viruses, fungi, or parasites.
• People with advanced liver disease, such as liver cirrhosis, active hepatitis, significant scarring of the liver, or high levels of iron in the liver (unless a liver biopsy shows no serious liver damage).
• People whose bone marrow is not functioning adequately, shown by low levels of certain blood cells (neutrophils or platelets) on blood tests.
• People who have medical reasons they cannot receive the medications used in this trial, including plerixafor (used to mobilise stem cells), busulfan (used in the conditioning process), or other required medications — including known allergies to any of these.
• People who need blood-thinning (anticoagulation) treatment during the period from conditioning through to the early recovery phase after the transplant.
• People who are unable to receive red blood cell transfusions.
• People who have previously received a stem cell transplant from a donor.
• People who have previously received gene therapy.
• People who have or have had a cancer or immune system disorder, except for certain previously treated, non-life-threatening skin cancers that have been fully cured.
• People who have an immediate family member with a known or suspected hereditary cancer syndrome.
• People who are pregnant, breastfeeding, or plan to become pregnant from screening until at least 6 months after receiving the treatment.
• People who have any other medical condition that would make them unsuitable for a stem cell transplant, as determined by the treating doctor.
• People who have participated in another clinical trial involving an experimental drug within 30 days before screening.
• People who have a chromosomal abnormality or genetic mutation that the investigator considers may increase the risk of developing certain blood cancers (MDS or AML).
• People who have genetic mutations that result in the loss of function of two or more alpha-globin genes (a gene involved in making haemoglobin).

Important: Always verify eligibility with the trial site directly before applying.