Actively Recruiting
A Phase 1b, Open-Label Study of DISC-3405 in Participants With Sickle Cell Disease (SCD)
Led by Disc Medicine, Inc · Updated on 2026-05-12
24
Participants Needed
5
Research Sites
90 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
A Phase 1b, Open-Label Study of DISC-3405 in Participants With Sickle Cell Disease (SCD)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Aged 18 years or older at the time of signing the informed consent form
- Male or female participants with sickle cell disease HbSC or HbSS
- Diagnosed with SCD-related complications such as 1-10 episodes of vaso-occlusive crisis in the past 12 months, sickle cell retinopathy, silent cerebral infarct, avascular necrosis, sensorineural hearing loss, or specific episodes of priapism, hepatic or splenic sequestration, or infarct within the last 12 months
- Hemoglobin level of at least 7.0 g/dL during screening (first 2 participants must have at least 9 g/dL)
- Normal alpha globin gene screen
- Absolute or percentage reticulocyte count greater than 1.5 times the upper limit of normal during screening
- Transferrin saturation of at least 15% at screening
- Ferritin level at least 50 ng/mL for HbSC or 100 ng/mL for HbSS and less than 1000 ng/mL at screening
- Stable dose of hydroxyurea, L-glutamine, or crizanlizumab for at least 2 months prior to screening with no planned dose changes
- If male, not vasectomized for at least 6 months, agrees to use highly effective double birth control methods with female partner(s) of childbearing potential during and 120 days after study drug
- If female, either postmenopausal, surgically sterile, or agrees to use highly effective birth control methods during and 120 days after study drug
- Negative pregnancy test for females of childbearing potential prior to dosing
- Able to understand study aims, procedures, and provide informed consent
- Able to comply with all study procedures
You will not qualify if you...
- Receiving regular blood transfusions or phlebotomy or received these within 60 days of screening
- Hospitalized for vaso-occlusive crisis or other SCD complications within 14 days of screening
- Clinically significant bacterial, fungal, parasitic, or viral infection
- Active HIV, hepatitis B, or C infection unless approved after discussion
- Significant kidney dysfunction with estimated glomerular filtration rate below 60 mL/min/1.73 m2
- Liver dysfunction with alanine aminotransferase greater than 2.5 times upper limit of normal
- Any acute chest syndrome episode in the last 6 months
- Prior or planned stem cell transplant or gene therapy
- Unstable or worsening heart or lung disease within 6 months prior to screening
- History of invasive cancers within last 5 years except certain cured cancers
- Major surgery within 8 weeks before screening or incomplete recovery
- Known allergy or anaphylaxis to study drug components or related substances
- History of alcohol dependence or excessive use
- Other medical or psychiatric conditions that increase risk or interfere with study
- Pregnant or breastfeeding females
- Participation in another experimental therapy study within 30 days before screening
- History of transient ischemic attack or stroke unless approved after consultation
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 5 locations
1
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35294
Actively Recruiting
2
Emory University
Atlanta, Georgia, United States, 30322
Actively Recruiting
3
Augusta University
Augusta, Georgia, United States, 30912
Actively Recruiting
4
Innovative Hematology - Indiana Hemophilia & Thrombosis Center
Indianapolis, Indiana, United States, 46260
Actively Recruiting
5
Mount Sinai Hospital
New York, New York, United States, 10029
Actively Recruiting
Research Team
D
Disc Medicine Clinical Trials
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
1
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