A Phase 1b, Open-Label Study of DISC-3405 in Participants With Sickle Cell Disease (SCD)
ConditionSickle Cell Disease
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18+
SponsorDisc Medicine, Inc
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Aged 18 years or older at the time of signing the informed consent form (ICF).
Male or female study participants with SCD HbSC or HbSS.
Participants who have been diagnosed with any of the following SCD-related complications: between 1-10 episodes of VOC in the past 12 months, any history of sickle cell related retinopathy, silent cerebral infarct, avascular necrosis, sensorineural hearing loss; or at least 1 episode of priapism, hepatic sequestration, splenic sequestration, or splenic infarct within the last 12 months as assessed locally.
Hgb ≥7.0 g/dL during Screening. The first 2 participants must have an Hgb ≥9 g/dL.
Disqualifiers
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.
Trial design
Design model
Sequential
Treatments tested in this trial
DISC-3405
DrugDISC-3405 is administered subcutaneously.
Treatment groups
24 Participants
are divided into 1 treatment groupGroup A: Within-participant dose escalationExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Safety and tolerability of DISC-3405 administration in participants with SCD
Proportion of participants with treatment-emergent adverse events (TEAEs), changes in vital signs, changes in physical examinations, changes in electrocardiograms (ECGs), and changes in clinical laboratory results
Time frame
Up to 36 weeksSecondary outcomes
1
Pharmacodynamic (PD) properties of DISC-3405 in participants with SCD by change from baseline for hemoglobin (Hgb)
Time frame
Up to 36 weeks2
Pharmacodynamic (PD) properties of DISC-3405 in participants with SCD by change from baseline for hematocrit (HCT)
Time frame
Up to 36 weeks3
Pharmacodynamic (PD) properties of DISC-3405 in participants with SCD by change from baseline for reticulocyte count
Time frame
Up to 36 weeks4
Pharmacodynamic (PD) properties of DISC-3405 in participants with SCD by change from baseline for Red Blood Cell Count (RBC)
Time frame
Up to 36 weeksSponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov