RightFit
RecruitingPhase 1 / Phase 2

A Study to Evaluate BMS-986470 in Healthy Volunteers and Participants 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

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
18 Years and older
Sex
All sexes
Healthy volunteers
Accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests BMS-986470 in healthy volunteers and people with sickle cell disease. It looks at safety, side effects, how the body handles the drug, and how the drug affects the body. It also studies the effects of food and acid levels (pH), and early signs that the drug works.

The purpose of this study is to evaluate the safety and tolerability, pharmacokinetics and pharmacodynamics, pH and food effect, and preliminary efficacy of BMS-986470 in healthy volunteers and participants with sickle cell disease.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Group A: Healthy males and females who cannot become pregnant. Females must have had no periods for at least 12 months without another medical cause, plus an FSH hormone level of at least 40 IU/L; or have had their womb, both ovaries, or both fallopian tubes removed.
  • Group A: Body mass index (BMI), a measure of weight compared with height, must be 18 to 32.
  • Group A: No signs of organ problems or important abnormal results on exams, heart tests, blood pressure and other vital signs, or lab tests.
  • Group B: A confirmed diagnosis of sickle cell disease of type HbSS, HbSβ0-thal, or HbSβ+-thal.
  • Group B: Part 1 requires at least 4 blocked-blood-flow pain crises in the past 12 months, or at least 2 in the past 6 months. Part 2 requires 2 to 15 crises in the past 12 months.
  • Group B: Fully active, or able to walk and do light work but limited in hard physical activity.
  • Group B: Hemoglobin, which carries oxygen in blood, must be 5.5–12 g/dL for males or 5.5–10.6 g/dL for females. Neutrophils, a type of infection-fighting cell, must be at least 1,500/μL.
  • Group B: Platelets, which help blood clot, must be at least 100,000/μL. Reticulocytes, or young red blood cells, must be above 100,000/μL, or above 50,000/μL if taking hydroxyurea.

Who can't join

  • Group A: A major health problem or a condition that makes study results hard to understand.
  • Group A: Any condition or abnormal lab result that makes joining the study too risky.
  • Group A: Major surgery or planned surgery within 12 weeks of the first study treatment, except surgery on the digestive system.
  • Group B: Any condition, serious short-term or long-term illness, active or uncontrolled infection, or abnormal lab result that makes joining too risky.
  • Group B, Part 1 only: More than 6 severe pain crises requiring a hospital stay of at least 24 hours in the 12 months before the first dose.
  • Group B, Part 1 only: Acute chest syndrome, a serious lung problem linked to sickle cell disease, in the 6 months before the first dose.
  • Group B: A kidney filtering test result below 60 mL/min/1.72 m², calculated using the CKD-EPI formula.
  • Both groups: Regular red blood cell or platelet transfusions, or a red blood cell transfusion within 28 days and a platelet transfusion within 14 days before starting BMS-986470. Other study entry rules also apply.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

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.

Study sites (19)

AL

CA

CT

GA

IL

KS

MA

MD

MO

NC

OH

PA

SC

VA