A Phase 1b Study of the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DISC-3405 in Participants With Sickle Cell Disease
Disc Medicine, Inc
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.
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Aged 18 years or older at the time of signing the informed consent form (ICF). 2. Male or female study participants with SCD HbSC or HbSS. 3. 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. 4. Hgb ≥7.0 g/dL during…
Interventions
- DrugDISC-3405
DISC-3405 is administered subcutaneously.
Locations (4)
- Julie KanterBirmingham, Alabama
- Emory UniversityAtlanta, Georgia
- Innovative Hematology - Indiana Hemophilia & Thrombosis CenterIndianapolis, Indiana
- Mount Sinai HospitalNew York, New York