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New treatment option for Sickle Cell Disease
Official title A Phase 1b, Open-Label Study of DISC-3405 in Participants With Sickle Cell Disease (SCD)
ClinicalTrials.gov ID: NCT07187973
What this study is testing
What is DISC-3405?
DISC-3405 is an investigational medicine, given as an injection under the skin, being studied as a potential treatment for sickle cell disease.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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: an early, usually small safety study
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- 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...
- Hgb ≥7.0 g/dL during Screening. The first 2 participants must have an Hgb ≥9 g/dL.
- Normal alpha globin gene screen.
You likely can't join if
- Participants who are receiving regularly scheduled blood (RBC) transfusion therapy or phlebotomy or have received RBC transfusion or phlebotomy...
- 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.
See the full eligibility criteria
- 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...
- 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.
- 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...
- 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...
- 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.
- 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.
- 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...
- 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...
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Birmingham, Alabama, United States
- Atlanta, Georgia, United States
- Augusta, Georgia, United States
- Indianapolis, Indiana, United States
- New York, New York, United States
- Durham, North Carolina, United States
- Cincinnati, Ohio, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Birmingham, Alabama, United States; Atlanta, Georgia, United States; Augusta, Georgia, United States; Indianapolis, Indiana, United States; New York, New York, United States; Durham, North Carolina, United States and 1 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.