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

A Phase 1/2a, First-in-human, Randomized, Double-blinded, Placebo-controlled, Dose-finding Study in Healthy Volunteers and Participants With Sickle Cell Disease to Evaluate the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics, pH and Food Effect, and Preliminary Efficacy of BMS-986470

Phase1; Phase2 Interventional Bristol-Myers Squibb · NCT06481306

This study is testing a new drug called BMS-986470 to see how safe it is and how well it works in both healthy people and those with sickle cell disease.

Quick facts

PhasePhase1; Phase2
Study typeInterventional
Enrollment184 (estimated)
Ages18 Years and up
SexAll
SponsorBristol-Myers Squibb Industry-sponsored
Locations19 sites (Birmingham, Alabama and 18 other locations)
Trial IDNCT06481306 on ClinicalTrials.gov

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.

Who should consider this trial

Good fit: Ideal candidates include healthy males and females not of childbearing potential, as well as individuals with a documented diagnosis of sickle cell disease.

Not a fit: Patients with other forms of anemia or those who do not meet the specific inclusion criteria for either cohort may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to improved treatment options for individuals with sickle cell disease.

How similar studies have performed: Other studies evaluating similar pharmacological approaches have shown promise, but this specific drug's efficacy is still being tested.

Eligibility criteria

Show full inclusion / exclusion 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.

Where this trial is running

Birmingham, Alabama and 18 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Anemia, Sickle CellHealthy Volunteers
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.