Phase 1 Sickle Cell Disease Trial, Recruiting
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- You have been diagnosed with sickle cell anemia (a specific type called Hgb SS or SB° Thalassemia) with a high level of the abnormal hemoglobin S in your blood.
- You have had a stroke or a neurological problem (such as weakness or other symptoms) lasting more than 24 hours, OR a brain blood flow test showed dangerously high readings (above 200 m/s).
- You have had one or more episodes of a serious lung complication called acute chest syndrome in the past 2 years, even while receiving supportive treatments like asthma therapy or hydroxyurea.
- You have had one or more severe pain crises per year in the past 2 years, even while receiving supportive treatments like pain management plans or hydroxyurea.
- You have had repeated episodes of painful, prolonged erections (priapism) that required medical treatment.
- You have had bone damage in two or more joints despite receiving supportive care.
- You have been receiving regular blood transfusions (8 or more per year for over a year) to prevent sickle cell complications.
- A heart ultrasound showed a specific finding related to a heart valve (tricuspid valve) that suggests possible high blood pressure in the lungs (confirm with trial site).
- You are between 1 and 30 years old.
- You are willing and able to use reliable contraception before and for 6 months after the study, if there is any chance you could become pregnant or father a child.
- You are able to understand and sign a consent form (or a parent/guardian can do so on your behalf if you are a child).
- You have a related family donor who is a half-match (haploidentical) for a specific set of immune markers (HLA-A, B, C and DRB1).
- There is no fully matched sibling or fully matched unrelated donor available for you.
Who may not be able to join:
- You currently have an uncontrolled illness, or an active bacterial, viral, or fungal infection.
- You are currently taking part in another clinical trial or receiving other experimental treatments, chemotherapy, or radiation therapy.
- You have had a serious allergic reaction to medications similar to those used in this transplant program.
- You are currently pregnant.
- You have an active cancer (other than certain minor skin cancers).
- You have a medical, neurological, or psychiatric condition that would make it difficult to follow the treatment plan or safely undergo a transplant, in the opinion of the lead doctor.
- You have previously had a bone marrow or stem cell transplant of any kind.
- A fully matched related or unrelated donor is available for you.
- The study team has concerns that you may not be able to follow the safety monitoring requirements of the study.
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Principal Investigator: Anna B. Pawlowska, MD, City of Hope Medical Center
Phone: 626-218-8442
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
Primary endpoints
Rate of unacceptable adverse events that are defined as any of the following events that occur from start of pre-transplant immunosuppressive therapy to the first 100 days post HCT:
Can't join this trial?
Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.