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A Phase 1b Study of the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DISC-3405 in Participants With Sickle Cell Disease
Led by Disc Medicine, Inc · Updated on 2026-06-04
24
Participants Needed
7
Research Sites
17 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating DISC-3405, a drug being studied in adults with sickle cell disease (SCD). This open-label, multicenter study focuses on examining up to three dose levels of DISC-3405 to assess its safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) in participants with SCD. The study is led by Disc Medicine, Inc and aims to better understand how this drug behaves and affects blood markers in people with this condition. Participants will receive DISC-3405 administered by subcutaneous injection in a within-participant dose escalation design. The study will explore up to three different doses of the drug to observe how participants respond to each level. This approach allows researchers to evaluate safety and drug effects at increasing dose amounts. The study lasts up to 36 weeks and includes multiple assessments during this time. Throughout the study, participants will undergo regular monitoring of safety and tolerability as the primary outcome. Researchers will also measure changes in blood-related markers like hemoglobin, hematocrit, reticulocyte count, red blood cell count, lactate dehydrogenase, and bilirubin. Pharmacokinetic measures such as drug concentration over time, maximum plasma concentration, elimination half-life, and clearance will also be tracked. Participants must be able to comply with study procedures, including providing informed consent and attending all visits during the trial period.
CONDITIONS
Brief 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 informed consent
- Male or female participants with sickle cell disease HbSC or HbSS
- Diagnosed with 1-10 episodes of vaso-occlusive crisis in past 12 months or history of specific sickle cell complications
- Hemoglobin level of 7.0 g/dL or higher during screening (first 2 participants must have 9 g/dL or higher)
- Normal alpha globin gene screen
- Absolute or percent reticulocyte count greater than 1.5 times upper limit of normal during screening
- Transferrin saturation of 15% or higher at screening
- Ferritin level 50 ng/mL or higher for HbSC or 100 ng/mL or higher for HbSS but less than 1000 ng/mL at screening
- Stable dose of hydroxyurea, L-glutamine, or crizanlizumab for at least 2 months prior to screening if taking these medications
- Males not vasectomized for at least 6 months with female partners of childbearing potential must agree to use two highly effective birth control methods during and 120 days after study
- Females must be postmenopausal, surgically sterile, or agree to use one highly effective birth control method plus barrier method from day 1 through 120 days after study
- Negative pregnancy test for females of childbearing potential prior to dosing
- Able to understand study aims and provide written informed consent
- Able to comply with all study procedures
You will not qualify if you...
- Receiving regularly scheduled red blood cell transfusions or phlebotomy or received these within 60 days of screening
- Hospitalized for vaso-occlusive crisis or other sickle cell complication within 14 days of screening
- Clinically significant bacterial, fungal, parasitic, or viral infection
- Active HIV, hepatitis B, or C infection unless discussed and approved by investigator and sponsor
- Significant kidney dysfunction with eGFR less than 60 mL/min/1.73 m2
- Liver dysfunction with ALT greater than 2.5 times upper limit of normal
- Acute chest syndrome episode in last 6 months
- Prior or planned stem cell transplant or gene therapy
- History of unstable or worsening heart or lung disease in last 6 months
- 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 drugs
- History of alcohol dependence or excessive use
- Other medical or psychiatric conditions that increase risk or interfere with study
- Conditions or medications that interfere with interpreting clinical results or recent psychiatric hospitalizations
- Pregnant or breastfeeding females
- Participation in other investigational studies within 30 days
- History of transient ischemic attack or stroke considered only with sponsor consultation
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 36 weeks
Participants receive escalating doses of DISC-3405 administered subcutaneously to evaluate safety and pharmacodynamics.
Visits scheduled according to dose escalation protocol
Trial Site Locations
Total: 7 locations
1
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35294
Actively Recruiting
2
Emory University
Atlanta, Georgia, United States, 30322
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3
Augusta University
Augusta, Georgia, United States, 30912
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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
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6
Duke University Medical Center
Durham, North Carolina, United States, 27708
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7
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
Research Team
D
Disc Medicine Clinical Trials
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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