Summary

Eligibility
for people ages 18 years and up (full criteria)
Healthy Volunteers
healthy people welcome
Location
at UCSD UCSF
Dates
study started
study ends around

Description

Summary

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.

Official Title

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

Keywords

Anemia, Sickle Cell, Healthy Volunteers, Sickle Cell Anemia, Famotidine, BMS-986470

Eligibility

You can join if…

Open to people ages 18 years and up

  • 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/m2, 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:.

  1. Hemoglobin ≥ 5.5 and ≤ 12 g/dL (males) or ≥ 5.5 and ≤ 10.6 g/dL (females).
  2. Absolute neutrophil count ≥ 1500/μL.
  3. Platelet count ≥ 100 × 103/μL.
  4. Absolute reticulocyte count > 100 × 103/μL or > 50 × 103/μL if taking hydroxyurea.

You CAN'T join if...

  • 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.

Locations

  • University of California San Diego - La Jolla accepting new patients
    La Jolla California 92037 United States
  • UCSF Benioff Children's Hospital Oakland accepting new patients
    Oakland California 94609 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Bristol-Myers Squibb
Links
BMS Clinical Trial Information BMS Clinical Trial Patient Recruiting
ID
NCT06481306
Phase
Phase 1/2 research study
Study Type
Interventional
Participants
Expecting 184 study participants
Last Updated