Sickle Cell DiseasePhase 1NCT07187973
A Phase 1b, Open-Label Study of DISC-3405 in Participants With Sickle Cell Disease (SCD)
A Phase 1 study testing DISC-3405 in people with Sickle Cell Disease, run by Disc Medicine, Inc at 11 sites in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- DISC-3405
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 11 sitesUnited States
- Study dates
- 6 Jan 2026to Oct 2027
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
- Normal alpha globin gene screen.
- Absolute reticulocyte count or % reticulocyte count >1.5 × upper limit of normal (ULN) during Screening.
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- 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.
- If male, not vasectomized for at least 6 months, with female sexual partner(s) of childbearing potential, agrees he and partner will use double methods of the following highly effective methods of birth control (described below) from the first dose of randomized study drug until 120 days after the last administration of study drug and must not donate sperm during their study participation:
- Stable hormonal contraceptive (≥3 months; female partner) in conjunction with a barrier method (eg, condom [male or female] or diaphragm).
- Intrauterine device, in place for at least 3 months (female partner) in conjunction with a barrier method (eg, condom [male or female] or diaphragm).
- Surgically sterile by hysterectomy, bilateral oophorectomy, or bilateral tubal ligation (female partner) in conjunction with a barrier method (eg, condom [male or female] or diaphragm).
- If female, then EITHER postmenopausal, defined as at least 12 months natural, spontaneous amenorrhea, 6 months of spontaneous amenorrhea with serum follicle-stimulating hormone (FSH) >40 mIU/mL at Screening, or at least 6 weeks following surgical menopause (bilateral oophorectomy with or without hysterectomy); surgically sterile, OR agree to use 1 of the following highly effective methods of birth control on Day 1 (or earlier) and for at least 120 days after the last administration of study drug:
- Stable hormonal contraceptive (≥3 months) in conjunction with a barrier method (eg, condom [male or female] or diaphragm).
- Intrauterine device, in place for at least 3 months in conjunction with a barrier method (eg, condom [male or female] or diaphragm).
- Tubal ligation or single male partner with vasectomy in conjunction with a barrier method (eg, condom [male or female] or diaphragm).
- Negative pregnancy test (females of childbearing potential) prior to dosing.
- Able to understand the study aims, procedures, and requirements, and provide written informed consent.
- Able to comply with all study procedures.
Who cannot join
- 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.
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- Any episode of ACS in the last 6 months.
- Prior or planned hematopoietic stem cell transplant or gene therapy.
- History of unstable or deteriorating cardiac or pulmonary disease within 6 months prior to Screening.
- History of invasive malignancies within the last 5 years, except localized cured prostate cancer and cervical cancer, or other malignancies deemed acceptable by the Sponsor.
- Major surgery within 8 weeks before Screening or incomplete recovery from any previous surgery.
- A history or known allergic reaction to any IP excipients or history of anaphylaxis to any food or drug.
- History of alcohol dependence or excessive alcohol consumption, as assessed by the Investigator.
- Other medical or psychiatric condition or laboratory finding not specifically noted above that, in the judgment of the Investigator or Sponsor, would put the participant at an unacceptable risk or otherwise preclude the participant from participating in the study.
- Condition or concomitant medication that would confound the ability to interpret clinical, clinical laboratory, including a major psychiatric condition that has had an exacerbation or required hospitalization in the last 6 months.
- If female, pregnant or breastfeeding.
- Participation in any other clinical protocol or investigational study that involves administration of experimental therapy and/or therapeutic devices within 30 days of Screening.
- Participants with a history of transient ischemic attack or stroke may be considered in consultation with Sponsor.
Trial locations and contacts
11 sites in 1 country. Contact the study team or a site near you to ask about taking part.
11 sites in 1 country
United States11 sites, 11 recruiting
- Recruiting
Emory University
Atlanta, Georgia, 30322
Sonjile James, 404-778-6470, sonjile.james@emory.edu
Brittney Schwebach, 470-588-6670, brittney.schwebach@emory.edu
- Recruiting
Augusta University
Augusta, Georgia, 30912
Latanya Bowman, lbowman@augusta.edu
Sharon Jones, 706-721-2172, sjones5@augusta.edu
- Recruiting
University of Alabama at Birmingham
Birmingham, Alabama, 35294
Emily Warner, 205-996-1735, ewarner@uabmc.edu
Emily Burke, 205-996-2205, eduke@uabmc.edu
- Recruiting
University of Illinois Hospital and Health Sciences System
Chicago, Illinois, 60612
Hassan Taif, 773-676-4204, tohassan@uic.edu
Lani Krauz, LIgnacio@uic.edu
- Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Kara McClure, 513-803-7271, kara.mcclure@cchmc.org
Amy Shova, 513-803-1917, Amy.Shova@cchmc.org
- Recruiting
Duke University Medical Center
Durham, North Carolina, 27708
Mary Beth Summers, 919-613-4657, mary.summers@duke.edu
Lindsey Ford, lindsey.ford@duke.edu
- Recruiting
Brody School of Medicine at East Carolina University
Greenville, North Carolina, 27834
Kristin Wooten, 252-744-3617, wootenk16@ecu.edu
Jerry Awasume, awasumej24@ecu.edu
- Recruiting
Innovative Hematology - Indiana Hemophilia & Thrombosis Center
Indianapolis, Indiana, 46260
Nehal Sheth, 317-871-0000, nsheth@inheme.org
Neelam Thrukal, nthukral@inheme.org
- Recruiting
Mount Sinai Hospital
New York, New York, 10029
Mitali Pradhan, 201-423-3585, mitali.pradhan@mountsinai.org
Jordan Bellis, 646-668-6642, jordan.bellis@mountsinai.org
- Recruiting
University of Pittsburgh Medical Center (UPMC)
Pittsburgh, Pennsylvania, 15213
Manzoor Mohideen, 412-648-6466, mohideenm@upmc.edu
Laura De Castro, MD
- Recruiting
University of California San Francisco (UCSF) Medical Center
San Francisco, California, 94143
Cyrus Bascon, 510-428-3885 ext. 6953, Cyrus.bascon@ucsf.edu
Robert Hagar, MD
About this study
Official title: A Phase 1b Study of the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DISC-3405 in Participants With 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.
- Conditions
- Sickle Cell Disease
- Being tested
- DISC-3405
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DISC-3405Drug
DISC-3405 is administered subcutaneously.
| Study group | Role | What they receive |
|---|---|---|
| Within-participant dose escalation | Experimental | This is an open-label, multicenter, within-participant dose-escalation study examining up to 3 dose levels of DISC-3405. Receives: Drug: DISC-3405 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Up to 36 weeks
Secondary outcomes (14)
Pharmacodynamic (PD) properties of DISC-3405 in participants with SCD by change from baseline for hemoglobin (Hgb)
Measured: Up to 36 weeks
Pharmacodynamic (PD) properties of DISC-3405 in participants with SCD by change from baseline for hematocrit (HCT)
Measured: Up to 36 weeks
Pharmacodynamic (PD) properties of DISC-3405 in participants with SCD by change from baseline for reticulocyte count
Measured: Up to 36 weeks
Pharmacodynamic (PD) properties of DISC-3405 in participants with SCD by change from baseline for Red Blood Cell Count (RBC)
Measured: Up to 36 weeks
Pharmacodynamic (PD) properties of DISC-3405 in participants with SCD by change from baseline for Lactate Dehydrogenase (LDH)
Measured: Up to 36 weeks
Pharmacodynamic (PD) properties of DISC-3405 in participants with SCD by change from baseline for bilirubin (direct and indirect)
Measured: Up to 36 weeks
Area under the plasma concentration versus time curve (AUCt,) following the first dose
Measured: Up to 36 weeks
Area under the plasma concentration versus time curve extrapolated to infinity (AUC∞) following the first dose
Measured: Up to 36 weeks
Maximum plasma concentration (Cmax) following the first dose
Measured: Up to 36 weeks
Time to maximum plasma concentration after drug administration (Tmax)
Measured: Up to 36 weeks
Elimination half-life (T1/2el) following the first dose
Measured: Up to 36 weeks
Apparent clearance (CL/F) following the first dose
Measured: Up to 36 weeks
Pre-dose trough concentration (Ctrough) after repeating doses
Measured: Up to 36 weeks
Apparent volume of distribution corrected for bioavailability (Vd/F)
Measured: Up to 36 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Sep 2025
- Study start6 Jan 2026
- Primary completionJun 2027 (estimated)
- Study completionOct 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Model description
- Up to 2 cohorts of participants are planned: * Cohort A will enroll 12 participants with HbSC or HbSS. At least one-third of participants must be HbSC, and at least one-third of participants must be HbSS. * Cohort B is optional and will enroll up to 12 participants with HbSC or HbSS, administering previously studied or lesser dose levels and regimens
- Masking
- None (open label)
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Disc Medicine, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07187973
- Sponsor's protocol ID
- DISC-3405-102
- Status last verified
- May 2026
- First submitted
- 8 Sep 2025
- Last updated
- 5 Oct 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No