Actively Recruiting
Phase 1b Study of DISC-3405 Safety and Effects in Adults With Sickle Cell Disease
Led by Disc Medicine, Inc · Updated on 2026-07-30
24
Participants Needed
8
Research Sites
17 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are conducting an open-label, multicenter study to evaluate DISC-3405 in adults with sickle cell disease SCD. The study examines up to three different dose levels of DISC-3405 to assess its safety, tolerability, pharmacokinetics PK, and pharmacodynamics PD. This Phase 1b trial focuses on participants with SCD, including those with certain complications related to the disease. In this study, DISC-3405 is given by subcutaneous injection in a within-participant dose-escalation design, meaning each participant may receive increasing doses over time. The study examines up to three dose levels sequentially to evaluate how the body processes the drug and how the drug affects blood markers related to SCD. The treatment period can last up to 36 weeks, during which safety and PD measures are closely monitored. Participants will be involved throughout the treatment period with regular assessments including blood tests to measure hemoglobin, hematocrit, reticulocyte count, red blood cell count, lactate dehydrogenase, bilirubin levels, and drug concentrations in blood. Safety and tolerability are primary concerns, with follow-up evaluations up to 36 weeks. Participants must comply with study procedures and provide informed consent before enrollment, ensuring they understand the studys aims and requirements.
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 the informed consent form
- Male or female participants with sickle cell disease HbSC or HbSS
- Diagnosed with 1-10 vaso-occlusive crises in the past 12 months or other specified SCD complications
- 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 during screening
- Stable dose of hydroxyurea, L-glutamine, or crizanlizumab for at least 2 months prior to screening if taking these medications
- Male participants 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 after the study
- Female participants must be postmenopausal, surgically sterile, or agree to use highly effective birth control methods during and after the study
- Negative pregnancy test for females of childbearing potential prior to dosing
- Able to understand and provide informed consent
- Able to comply with all study procedures
You will not qualify if you...
- Receiving regular blood transfusion or phlebotomy therapy or received such treatment 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 without prior discussion with investigator and sponsor
- Significant kidney dysfunction (eGFR less than 60 mL/min/1.73 m2)
- Liver dysfunction with ALT greater than 2.5 times the upper limit of normal
- Acute chest syndrome episode within 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 cancer within 5 years except certain localized cancers
- Major surgery within 8 weeks before screening or incomplete recovery
- History of allergic reaction to study drug ingredients or anaphylaxis to any food or drug
- History of alcohol dependence or excessive alcohol use
- Other medical or psychiatric conditions judged to increase risk or prevent participation
- Conditions or medications that would interfere with interpreting study results or recent major psychiatric exacerbations
- Pregnant or breastfeeding females
- Participation in other experimental therapy studies within 30 days of screening
- History of transient ischemic attack or stroke requiring consultation with sponsor
Research Team
D
Disc Medicine Clinical Trials
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