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:

• A person who has given informed consent (or whose legal guardian has given consent), and who has been registered in the required safety monitoring program for this study
• People between the ages of 12 and 40 years old
• People whose general physical functioning meets a certain minimum level, as assessed by their medical team (confirm with trial site)
• People who have a confirmed diagnosis of sickle cell disease and are considered at high risk for serious complications, based on at least one of the following:
  • A history of stroke or any neurological problem lasting more than 24 hours, or an abnormal result on a special blood flow test in the brain
  • One or more episodes of a serious lung complication called acute chest syndrome in the two years before joining, even after receiving supportive treatments like asthma therapy or a medicine called hydroxyurea
  • One or more severe pain crises per year in the two years before joining, even after receiving supportive treatments like pain management plans or hydroxyurea
  • Repeated episodes of a painful condition called priapism that required medical treatment
  • Bone damage in two or more joints, even after receiving supportive care
  • Having needed eight or more blood transfusions per year for more than one year to prevent serious complications
  • A heart scan result showing abnormal blood flow through a specific heart valve at a certain speed
• People who have a related donor whose tissue type closely matches theirs on at least 8 out of 10 tested markers
• People whose liver function blood test results are within acceptable ranges, tested within 30 days before starting the study
• People whose kidney function is at or above a certain level, tested within 30 days before starting
• People whose blood clotting test results are within acceptable ranges (or, if taking blood thinners, within the appropriate treatment range), tested within 30 days before starting
• People whose heart pumping function meets a minimum level, tested within 28 days before starting
• People whose lung function tests meet a minimum level, or — if unable to do those tests — whose blood oxygen level is above a certain point on room air, tested within 28 days before starting
• People who test negative for HIV, hepatitis C, active hepatitis B, and syphilis, tested within 28 days before starting
• People who meet all required infectious disease testing standards set by the institution and applicable regulations
• Women who could become pregnant must have a negative pregnancy test before starting
• People who are able and willing to use effective birth control or avoid sexual activity that could lead to pregnancy throughout the study and for at least six months after the last treatment dose

For the stem cell donor, additional criteria apply:

• The donor must be 60 years old or younger
• The donor must be in good general health as confirmed by a medical review
• The donor must have completed a full set of required infectious disease blood tests
• Female donors who could become pregnant must have a negative pregnancy test within the required timeframes before donation
• The donor must be informed about the research nature of the study and have signed a consent form

Who may not be able to join:

• People who have previously had a stem cell transplant of any kind
• People who are currently taking part in another clinical trial or receiving other cancer-related treatments such as chemotherapy, biological therapy, or radiation
• People who have had a serious allergic reaction to medicines that are similar to those used in this study
• People who currently have an active cancer diagnosis, other than certain common types of skin cancer
• People with a significant medical, neurological, or psychiatric condition that, in the lead doctor's opinion, would make it unsafe or too difficult to go through the transplant process
• People who currently have an active infection requiring antibiotic treatment
• Women who are pregnant or breastfeeding
• People with any other condition that the investigator believes would make participation unsafe
• People who, in the investigator's opinion, may not be able to follow all required study procedures

For the stem cell donor, additional reasons a donor may not be eligible include:

• The donor has an active infection
• The donor has a medical or physical condition that would make it unsafe or unlikely for them to tolerate the donation process — whether that involves a procedure to collect cells from the blood or a surgical procedure to collect bone marrow
• The donor has factors that increase their risk of complications from the donation process
• Female donors who are breastfeeding or who are unwilling to use adequate birth control
• The donor tests positive for HIV
• The donor has previously received radiation therapy

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