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Sickle Cell Disease Clinical Trial in Duarte CA
NCT04091737 | Phase 1 | Interventional
Have you or your loved ones been diagnosed with Sickle Cell Disease?
If so, you may be eligible for a clinical trial.
Have you or your loved ones been diagnosed with Sickle Cell Disease? If so, you may be eligible for a clinical trial.
Where you'll go
Details for the study
Treatments and/or Procedures
Inclusion Criteria: - Diagnosis of sickle cell disease with the homozygous HbSS or an HbSβ thalassemia variant (ie, HbSβ0 thalassemia or HbSβ+ thalassemia) genotype, confirmed by hemoglobin studies. - Fetal hemoglobin (HbF) ≤ 15%. - Severe sickle cell disease symptomatology, defined as any one or more of the following: 1. ≥ 2 episodes of acute chest syndrome in the last 2 years. 2. ≥ 3 episodes of severe pain events requiring a visit to a medical facility and treatment with opioids in the last 2 years. 3. > 2 episodes of recurrent priapism in the last 2 years. 4. Red-cell alloimmunization (> 2 antibodies) during long-term transfusion therapy (lifetime history). 5. Chronic transfusions for primary or secondary prophylaxis (lifetime history). 6. Trans-thoracic echocardiograph evidence of tricuspid valve regurgitant jet velocity ≥ 2.7 m/sec (lifetime history). 7. Clinically significant neurologic event (eg, ischemic stroke) or any neurological deficit lasting > 24 hours. Exclusion Criteria: - Hypoxanthine-guanine phosphoribosyl transferase (HPRT) deficiency. - Thiopurine S-methyltransferase (TPMT) deficiency. - Alpha thalassemia. - Serum ferritin ≥ 2500 ng/mL. - Inadequate bone marrow function, defined as at least 1 of the following: 1. Absolute neutrophil count < 1000/µL. 2. Platelet count < 120,000/µL.