Evaluating BMS-986470 in healthy individuals and those with sickle cell disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study aims to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of BMS-986470 in both healthy volunteers and participants diagnosed with sickle cell disease. It will also investigate the effects of pH and food on the drug, as well as its preliminary efficacy. Participants will be divided into two cohorts: healthy individuals and those with specific genotypes of sickle cell disease. The study will involve administering either the drug or a placebo to evaluate outcomes.

Why it matters

Who can join: the full rules

Inclusion Criteria:

Cohort A:

* 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.
* Body mass index (BMI) of 18.0 to 32.0 kg/m2, inclusive. BMI = weight (kg)/\[height (m)\]2 as measured at screening.
* 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:

* Participants with a documented diagnosis of sickle cell disease (SCD) with genotype HbSS, HbSβ0-thal, or HbSβ+-thal.
* For Cohort B Part 1 only: Participants with ≥ 4 vaso-occlusive crises (VOCs) within the previous 12 months or ≥ 2 VOCs within the previous 6 months. For Cohort B Part 2 only: Participants with ≥ 2 VOCs and ≤ 15 VOCs within the previous 12 months.
* Participant has an Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.
* Participants must have the following laboratory values:

  i) Hemoglobin ≥ 5.5 and ≤ 12 g/dL (males) or ≥ 5.5 and ≤ 10.6 g/dL (females). ii) Absolute neutrophil count ≥ 1500/μL. iii) Platelet count ≥ 100 × 10\^3/μL. iv) Absolute reticulocyte count \> 100 × 10\^3/μL or \> 50 × 10\^3/μL if taking hydroxyurea.

Exclusion Criteria:

Cohort A:

* Any significant medical condition or any condition that confounds the ability to interpret data from the study.
* 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.
* Any major surgery or planned surgery (except GI surgery) within 12 weeks of the first study intervention administration.

Cohort B:

* 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.
* For Cohort B Part 1 only: 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.
* For Cohort B Part 1 only: participants with any episode of acute chest syndrome within the last 6 months prior to the first dose of study intervention.
* Creatinine clearance (CrCl) \< 60 mL/min/1.72m2 using Chronic Kidney Disease Epidemiology (CKD-EPI) equation.

Cohort A and B:

* 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.

Where it's running

• University of Alabama at Birmingham
  Birmingham, Alabama, United States Recruiting
• University of California San Diego - La Jolla
  La Jolla, California, United States Recruiting
• UCSF Benioff Children's Hospital Oakland
  Oakland, California, United States Recruiting
• Yale-New Haven Hospital
  New Haven, Connecticut, United States Recruiting
• Winship Cancer Institute of Emory University
  Atlanta, Georgia, United States Recruiting
• Boston Medical Center
  Boston, Massachusetts, United States Recruiting

Study contacts

• Study coordinator: BMS Clinical Trials Contact Center www.BMSClinicalTrials.com
• Email: Clinical.Trials@bms.com
• Phone: 855-907-3286

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.