Phase 1 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:

• You are between 18 and 24 years old (up to age 24 years and 11 months).
• You have been diagnosed with sickle cell disease with one of these specific genetic types: Hb SS, Hb SB0, or Hb SB+.
• You have had either 2 or more sickle cell-related pain crises or serious complications (such as acute chest syndrome, priapism, or splenic sequestration) per year over the past 2 years, OR you have been receiving regular red blood cell transfusions (at least 8 in the past 12 months) — with some exceptions related to stroke prevention (confirm with trial site).
• You have tried hydroxyurea therapy but it did not work for you, you could not tolerate it, or you chose not to take it.
• Your doctor has determined that you are healthy enough to undergo a stem cell transplant using your own cells.
• If you are a woman who could become pregnant, you must agree to use an approved form of birth control from the start of treatment until at least 6 months after receiving the therapy.
• If you are male, you must agree to use effective birth control from the start of treatment until at least 6 months after receiving the therapy.
• You are willing to take part in a long-term follow-up study after this trial ends.

Who may not be able to join:

• You have a brother or sister whose tissue type (HLA) is a match and who is willing and able to donate stem cells for a transplant.
• Your general health and ability to carry out daily activities is below a certain level, as measured by a standard medical scoring tool (confirm with trial site).
• You are currently pregnant (confirmed by a pregnancy test taken within 14 days before enrolling).
• You are currently breastfeeding.
• You have had a serious bacterial, viral, or fungal infection in the past month that is not under control or is getting worse.
• You have been diagnosed with Hepatitis B or Hepatitis C.
• You have tested positive for HIV or HTLV (a type of virus affecting blood cells).
• You have a history of stroke.
• Certain liver function blood test results are above acceptable levels — though some exceptions may apply depending on the cause (confirm with trial site).
• Your heart is not pumping blood effectively enough, based on a heart ultrasound (echocardiogram).
• Your kidneys are not working well enough, based on a standard kidney function measurement.
• Your lungs are not working well enough, based on breathing tests or oxygen levels.
• You have previously had a stem cell transplant or gene therapy.
• You have been diagnosed with liver cirrhosis, significant liver scarring, or active liver inflammation.
• You currently have or have had a cancer, a blood cell disorder called myelodysplasia, abnormal chromosomes, or a problem with your immune system.
• You have a history of a serious bleeding disorder.
• You have had a procedure on the blood vessels of the brain within the past 6 months, including a surgery for a condition called moyamoya.
• You have untreated moyamoya disease, or have moyamoya disease that your doctor believes puts you at risk of bleeding.
• A genetic blood test has found a concerning change in your genes that could be linked to blood cancers.
• You have a known allergy, intolerance, or serious reaction to certain medications used in this trial (plerixafor, motixafortide, busulfan, or ingredients in the study drug).
• You are currently in another clinical trial using an experimental drug, or have been in one recently (within about 30 days or longer, depending on the drug).
• You have developed antibodies against red blood cell proteins (alloimmunization), and your doctor is concerned there may not be enough compatible blood available for you throughout the study.

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