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 between the ages of 12 and 35 years old.
• People who are willing to have a bone marrow sample taken (a procedure called a myelogram/bone marrow aspiration).
• People who have been diagnosed with a specific type of sickle cell disease called HbSS, confirmed by blood protein testing and genetic analysis.
• People with a documented history or current evidence of severe sickle cell disease, shown by at least one of the following: three or more painful crises requiring a hospital stay within the past two years while on hydroxyurea or transfusion treatment; one serious lung complication (called acute chest syndrome) that required an intensive care unit stay; at least two episodes of that lung complication, including one that happened while on hydroxyurea; at least two episodes of painful, prolonged erections lasting more than three hours in the past year, or frequent shorter episodes occurring at least once a week while on sickle cell treatment; or a specific finding on a heart scan suggesting possible high blood pressure in the lungs, without confirmed pulmonary hypertension (confirm with trial site).
• People whose sickle cell disease has not responded well enough to hydroxyurea treatment after at least three months, shown by continued hospital admissions for pain, the need for blood transfusions to maintain safe blood levels, or a lung complication despite appropriate care.
• People who score at least 60% on a standard scale used to measure their ability to carry out daily activities (confirm with trial site).
• People who are sexually active and are willing to use two forms of contraception together for at least 12 months after receiving the treatment.
• People who are able to give their own consent, or, for younger participants, whose guardians are able to provide consent on their behalf.
• People who are affiliated with the French social security system (confirm with trial site).

Who may not be able to join:

• People who have a brother or sister whose tissue type is a close enough match to donate bone marrow.
• People whose bone marrow sample shows certain chromosome or genetic abnormalities that are considered risky by the specialist medical team following a dedicated review meeting.
• People who have had low white blood cell counts, low levels of a specific type of white blood cell (neutrophils), or low platelet counts in the 90 days before the stem cell collection process, unless caused by a specific blood-cleaning procedure or a recent viral infection.
• People with abnormal blood clotting test results or a known significant bleeding disorder.
• People who carry two copies of a specific genetic deletion linked to a condition called alpha-thalassemia, which could create risks after gene therapy.
• People who have a known allergy or sensitivity to any of the medicines used in the trial, including plerixafor, busulfan, or anti-inflammatory drugs.
• People who have previously received gene therapy.
• People whose liver enzyme levels (ALT or AST) are more than three times the upper limit of normal, based on tests done within the six months before the screening visit.
• People with severe iron build-up in the liver detected by MRI, or suspected liver scarring (cirrhosis) confirmed by biopsy, based on tests done within six months before the screening visit.
• People whose kidneys are not filtering blood well enough, based on a measured kidney function test result below a certain threshold, within six months before the screening visit.
• People whose heart is not pumping effectively (below 40% on a heart function scan) or who have significant abnormalities on a heart rhythm test, within six months before the screening visit.
• People who have had a stroke that left significant lasting effects on brain function (confirm with trial site for scoring details).
• People with a confirmed narrowing of blood vessels in the brain related to sickle cell disease, who are also already on a program of regular blood transfusions to keep a certain level of sickle cell blood below 30% (confirm with trial site).
• People who have scarring changes in the lungs AND significantly reduced lung capacity AND reduced ability of the lungs to transfer oxygen, all measured at a stable point in their health.
• People with confirmed high blood pressure in the lungs, detected by a specific heart procedure (right heart catheterisation).
• People who test positive for certain serious viral infections including HIV, Hepatitis C, HTLV-1, active Hepatitis B, active CMV, or active parvovirus B19, confirmed by blood testing.
• People who are pregnant or currently breastfeeding.
• People who currently have or have previously had cancer, except for certain fully treated non-melanoma skin cancers.
• People who have an immediate family member with a confirmed or suspected hereditary cancer syndrome.
• People with a significant psychiatric condition that could seriously affect their ability to take part in the study.
• People who have previously had a stem cell transplant that was unsuccessful.
• People with any significant active infection at the time of screening.
• People who have taken part in another clinical trial involving an experimental drug within the 30 days before screening.
• People who, in the opinion of the trial's medical team, have any condition that could increase safety risks or make it difficult to follow the trial's procedures.

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