A Phase 1b, Open-Label Study of DISC-3405 in Participants With Sickle Cell Disease (SCD) (NCT07187973) | Clinical Trial Compass
RecruitingPhase 1
A Phase 1b, Open-Label Study of DISC-3405 in Participants With Sickle Cell Disease (SCD)
United States24 participantsStarted 2026-01-06
Plain-language summary
This is an open-label, multicenter, within-participant dose-escalation study examining up to 3 dose levels of DISC-3405 and will assess the safety, tolerability, PK, and PD of DISC 3405 in participants with sickle cell disease.
Who can participate
Age range18 Years
SexALL
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Inclusion criteria
✓. Aged 18 years or older at the time of signing the informed consent form (ICF).
✓. Male or female study participants with SCD HbSC or HbSS.
✓. Participants who have been diagnosed with any of the following SCD-related complications: between 1-10 episodes of VOC in the past 12 months, any history of sickle cell related retinopathy, silent cerebral infarct, avascular necrosis, sensorineural hearing loss; or at least 1 episode of priapism, hepatic sequestration, splenic sequestration, or splenic infarct within the last 12 months as assessed locally.
✓. Hgb ≥7.0 g/dL during Screening. The first 2 participants must have an Hgb ≥9 g/dL.
✓. Normal alpha globin gene screen.
✓. Absolute reticulocyte count or % reticulocyte count \>1.5 × upper limit of normal (ULN) during Screening.
✓. TSAT ≥15% at Screening.
✓. Ferritin ≥50 ng/mL for HbSC or ≥100 ng/mL for HbSS (ferritin must be \<1000 ng/mL at Screening).
Exclusion criteria
✕. Participants who are receiving regularly scheduled blood (RBC) transfusion therapy or phlebotomy or have received RBC transfusion or phlebotomy within 60 days of Screening.
✕. Hospitalized for VOC or other sickle cell related complication within 14 days of Screening.
✕. Participants with clinically significant bacterial, fungal, parasitic, or viral infection.
What they're measuring
1
Safety and tolerability of DISC-3405 administration in participants with SCD
✕. Active HIV, hepatitis B, or C. A positive hepatitis or HIV result should be discussed between the Investigator and Sponsor prior to enrollment.
✕. Significant renal dysfunction, evidenced by estimated glomerular filtration rate of \<60 mL/min/1.73 m2 at the Screening visit, as assessed locally.