Not medical advice. This website is not medical advice, a diagnosis, a match to a trial, or a recommendation to join a study. Listings are plain-English rewrites of public ClinicalTrials.gov records. Whether a site is still enrolling, and whether you qualify, can only be confirmed by the study team and your clinician. Source registry: ClinicalTrials.gov. Editorial rules: methodology.
Phase 1: testing DISC-3405 for Sickle Cell Disease
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.
Phase 1: the first tests in people. The main question is safety and dose, not whether it cures the condition. Groups are small. Side effects are watched closely.
Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.
What the registry says happens
If you join, the listed intervention(s) are DISC-3405. The registry lists allocation as na. 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.
They plan to enroll about 24 people at 11 sites. Taking part typically means taking a study drug (or dummy pill) plus check-ins.
Official title (unrewritten)
A Phase 1b Study of the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DISC-3405 in Participants With Sickle Cell Disease
Interventions: DISC-3405
Primary outcome (registry wording): Safety and tolerability of DISC-3405 administration in participants with SCD
Who it is for (from inclusion criteria)
Ages 18 Years to any
Male or female study participants with SCD HbSC or HbSS
Hgb ≥7.0 g/dL during Screening. The first 2 participants must have an Hgb ≥9 g/dL
Normal alpha globin gene screen
Absolute reticulocyte count or % reticulocyte count \>1.5 × upper limit of normal (ULN) during Screening
TSAT ≥15% at Screening
Ferritin ≥50 ng/mL for HbSC or ≥100 ng/mL for HbSS (ferritin must be \<1000 ng/mL at Screening)
For participants taking hydroxyurea, L-glutamine, or crizanlizumab, stable dose for at least 2 months prior to Screening and with no anticipated need for dose adjustments during the study
Who the registry excludes
Participants who are receiving regularly scheduled blood (RBC) transfusion therapy or phlebotomy or have received RBC transfusion or phlebotomy within 60 days of Screening
Hospitalized for VOC or other sickle cell related complication within 14 days of Screening
Participants with clinically significant bacterial, fungal, parasitic, or viral infection
Active HIV, hepatitis B, or C. A positive hepatitis or HIV result should be discussed between the Investigator and Sponsor prior to enrollment
Significant renal dysfunction, evidenced by estimated glomerular filtration rate of \<60 mL/min/1.73 m2 at the Screening visit, as assessed locally
Hepatic dysfunction characterized by alanine aminotransferase (ALT) \>2.5 × ULN
This study is not a treatment protocol and no experimental treatments are involved. Study participants may be seen as needed for clinical, translational and basic research studies, or as medically indicated.
This study will collect representative blood samples from healthy children and adults and from children and adults who have unique red blood cell features that are related to sickle cell disease. Sickle cell disease is a blood disease that limits the ability of red blood cells to carry oxygen throughout the body.
Background: Some people with the same disorder on a genetic level have more complications than others. Researchers want to look for a link between the PKLR gene and sickle cell disease (SCD) symptoms.
The sponsor posted status “Recruiting” on ClinicalTrials.gov at last ingest. Sites can pause independently. Confirm with the official record and the listed contact.
What phase is NCT07187973?
Phase 1. Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.
Can PlainTrials tell me if I qualify for NCT07187973?
No. The inclusion and exclusion lists on this page are readable extracts of the public eligibility block. Only the study team can screen you.