A Study to Evaluate BMS-986470 in Healthy Volunteers and Participants With Sickle Cell Disease

Launched by BRISTOL-MYERS SQUIBB · Jun 25, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a new medication called BMS-986470 to see how safe it is and how well it works for people with sickle cell disease and healthy volunteers. The researchers want to understand how the drug behaves in the body, its effects on the body, and how food might influence its effectiveness.

To participate, healthy individuals must be between 18 and 65 years old and meet certain health criteria, such as having a body mass index (BMI) between 18 and 32. People with sickle cell disease can join if they have had recent episodes of pain crises and meet specific health measurements. Participants can expect to undergo tests and monitoring while receiving the medication, and they will be helping researchers learn more about this potential new treatment for sickle cell disease. It's important to note that there are specific health conditions that may exclude someone from participating to ensure safety during the study.

Gender

ALL

Eligibility criteria

• • Inclusion Criteria
• • - Cohort A.
• • i) Healthy male and female (who are not of childbearing potential) participants, as determined by the investigator based on medical history and other determinations. Females not of childbearing potential must have been amenorrhoeic for at least 12 months without an alternative medical cause and have follicle-stimulating hormone (FSH) levels of at least 40 IU/L or have undergone a hysterectomy, bilateral oophorectomy, or bilateral salpingectomy.
• • ii) Body mass index (BMI) of 18.0 to 32.0 kg/m\^2, inclusive. BMI = weight (kg)/ (height \[m\])\^2 as measured at screening.
• • iii) No evidence of organ dysfunction or any clinically significant deviation from normal in physical examination, vital signs, ECG, or clinical laboratory assessments beyond what is consistent with the target population.
• • - Cohort B.
• • i) Participants with a documented diagnosis of Sickle Cell Disease (SCD) with genotype HbSS, HbSβ0-thal, or HbSβ+-thal.
• • ii) Participants with ≥ 4 vaso-occlusive crises (VOCs) within the previous 12 months or ≥ 2 VOCs within the previous 6 months.
• • iii) Participant has an Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.
• • iv) Must have the following laboratory values:.
• • A. Hemoglobin ≥ 5.5 and ≤ 12 g/dL (males) or ≥ 5.5 and ≤ 10.6 g/dL (females).
• • B. Absolute neutrophil count ≥ 1500/μL.
• • C. Platelet count ≥ 100 × 10\^3/μL.
• • D. Absolute reticulocyte count \> 100 × 10\^3/μL or \> 50 × 10\^3/μL if taking hydroxyurea.
• • Exclusion Criteria
• • - Cohort A.
• • i) Any significant medical condition or any condition that confounds the ability to interpret data from the study.
• • ii) Participant has any condition, including the presence of laboratory abnormalities, that places the participant at unacceptable risk if the participant was to participate in the study.
• • iii) Any major surgery or planned surgery (except GI surgery) within 12 weeks of the first study intervention administration.
• • - Cohort B.
• • i) Participants with any condition, including significant acute or chronic medical illness, active or uncontrolled infection, or the presence of laboratory abnormalities, that places participants at unacceptable risk if participating in this study.
• • ii) Participants with more than 6 severe VOCs defined as VOCs requiring ≥ 24 hours of hospital admission within 12 months prior to the first dose of study intervention or any VOC requiring ≥ 24 hours of hospital admission within 30 days prior to the first dose of study intervention.
• • iii) Participants with any episode of acute chest syndrome within the last 6 months prior to the first dose of study intervention.
• • iv) Creatinine clearance (CrCl) \< 60 mL/min/1.72m2 using Chronic Kidney Disease Epidemiology (CKD-EPI) equation
• • Cohort A and B.
• • i) Participant is receiving regularly scheduled RBC or platelet transfusions or has received a RBC transfusion within 28 days and a platelet transfusion within 14 days prior to starting treatment with BMS-986470.
• • Other protocol-defined Inclusion/Exclusion criteria apply.