DISC-3405 for Sickle Cell Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called DISC-3405 for individuals with sickle cell disease. The main goal is to assess the treatment's safety and tolerability while understanding its behavior in the body. Participants will receive varying doses of DISC-3405 to determine the most effective and safest amount. It suits those with sickle cell disease who experience specific complications, such as frequent pain episodes, eye problems, or bone damage. As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding its effects in people.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications, but if you are on hydroxyurea, L-glutamine, or crizanlizumab, you must be on a stable dose for at least 2 months before the study and not change the dose during the study.
Is there any evidence suggesting that DISC-3405 is likely to be safe for humans?
Research has shown that DISC-3405 is generally safe and well-tolerated. In earlier studies, DISC-3405 did not cause serious side effects, indicating a good safety record. Participants did not experience major unwanted reactions during these trials. Additionally, the treatment proved safe for individuals with excess iron in their bodies. This suggests that DISC-3405 could be a safe choice for those considering joining the trial.12345
Why do researchers think this study treatment might be promising?
Most treatments for sickle cell disease, like hydroxyurea or blood transfusions, work by reducing the symptoms or complications of the disease. But DISC-3405 works differently, offering a new approach by potentially altering the disease's underlying processes. Researchers are excited about DISC-3405 because it involves a unique within-participant dose escalation, which may help determine the most effective dosage with fewer side effects. This innovative approach could lead to better outcomes and improved quality of life for patients with sickle cell disease.
What evidence suggests that DISC-3405 might be an effective treatment for sickle cell disease?
Research has shown that DISC-3405 could benefit people with sickle cell disease. Early studies demonstrated that this treatment significantly increased the production of hepcidin, a hormone that regulates iron levels in the body. DISC-3405 also reduced blood iron levels, potentially addressing the iron overload issues common in sickle cell patients. These findings suggest that DISC-3405 could effectively manage iron overload in sickle cell disease. Participants in this trial will undergo a within-participant dose escalation to examine up to three dose levels of DISC-3405.12356
Are You a Good Fit for This Trial?
Adults aged 18+ with Sickle Cell Disease (HbSC or HbSS) who've had complications like pain episodes, retinopathy, or organ damage. They must have stable blood counts and not need dose changes for current medications like hydroxyurea during the study. Women on stable hormonal contraceptives must also use a barrier method.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants undergo within-participant dose escalation of DISC-3405 to assess safety, tolerability, PK, and PD
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- DISC-3405
Find a Clinic Near You
Who Is Running the Clinical Trial?
Disc Medicine, Inc
Lead Sponsor