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

NCT06481306 | Recruiting Phase 1 FDA Drug

• 3 other identifiers: CA230-10192023-510283-12U1111-1301-6753
• Study Type: interventional
• Target: 184
• Locations: 3 countries
• Sites: 19

Brief 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.

Conditions

Anemia, Sickle Cell; Healthy Volunteers

Outcome Measures

Primary Outcomes (5)

• Number of participants with adverse events (AEs)

  Up to 26 months

• Number of participants with serious adverse events (SAEs)

  Up to 26 months

• Number of participants with AEs meeting protocol-defined Dose Limiting Toxicity (DLT) criteria

  Up to 26 months

• Number of participants with AEs leading to discontinuation

  Up to 26 months

• Number of deaths

  Up to 26 months

Secondary Outcomes (19)

• Maximum observed plasma concentration (Cmax)

  Up to Day 28

• Area under the concentration-time curve (AUC)

  Up to Day 28

• Time of maximum observed plasma concentration (Tmax)

  Up to Day 28

• Dose proportionality of BMS-986470 for Cmax and AUC

  Up to Day 28

• Change from baseline in total hemoglobin (Hb)

  Up to 26 months

Study Arms (5)

Cohort A Part 1

EXPERIMENTAL

Drug: BMS-986470; Drug: Placebo

Cohort A Part 2

EXPERIMENTAL

Drug: BMS-986470; Drug: Placebo

Cohort A Part 3

EXPERIMENTAL

Drug: BMS-986470; Drug: Famotidine

Cohort B Part 1

EXPERIMENTAL

Drug: BMS-986470; Drug: Placebo

Cohort B Part 2

EXPERIMENTAL

Drug: BMS-986470

Interventions

BMS-986470 DRUG

Specified dose on specified days

Cohort A Part 1; Cohort A Part 2; Cohort A Part 3; Cohort B Part 1; Cohort B Part 2

Placebo DRUG

Specified dose on specified days

Cohort A Part 1; Cohort A Part 2; Cohort B Part 1

Famotidine DRUG

Specified dose on specified days

Cohort A Part 3

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• \- 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.

You may not qualify 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.

Sponsors & Collaborators

• Bristol-Myers Squibb: lead

Study Sites (19)

University of Alabama at Birmingham

Birmingham, Alabama, 35233, United States

RECRUITING

University of California San Diego - La Jolla

La Jolla, California, 92037, United States

RECRUITING

UCSF Benioff Children's Hospital Oakland

Oakland, California, 94609, United States

RECRUITING

Yale-New Haven Hospital

New Haven, Connecticut, 06510, United States

RECRUITING

Winship Cancer Institute of Emory University

Atlanta, Georgia, 30322, United States

RECRUITING

Local Institution - 0034

Chicago, Illinois, 60612, United States

NOT YET RECRUITING

Local Institution - 0001

Lenexa, Kansas, 66219, United States

ACTIVE NOT RECRUITING

Local Institution - 0024

Boston, Massachusetts, 02114, United States

NOT YET RECRUITING

Boston Medical Center

Boston, Massachusetts, 02118, United States

RECRUITING

Thomas Jefferson University - Medicine/GI and Hepatology

Philadelphia, Pennsylvania, 19107, United States

RECRUITING

Local Institution - 0032

Pittsburgh, Pennsylvania, 15213, United States

NOT YET RECRUITING

Inova Schar Cancer Institute

Fairfax, Virginia, 22031, United States

RECRUITING

Virginia Commonwealth University (VCU) Medical Center

Richmond, Virginia, 23298, United States

RECRUITING

Institut de cancérologie Strasbourg Europe (ICANS)

Strasbourg, Alsace, 67033, France

RECRUITING

Assistance Publique Hôpitaux de Marseille - Hôpital de la Timone

Marseille, 13385, France

RECRUITING

Hôpital Universitaire Necker Enfants Malades

Paris, 75015, France

RECRUITING

University Hospitals Sussex NHS Foundation Trust

East Sussex, Brighton And Hove, BN2 1ES, United Kingdom

RECRUITING

King's College Hospital

London, London, City of, SE5 9RL, United Kingdom

RECRUITING

Local Institution - 0005

Leeds, LS9 7TF, United Kingdom

NOT YET RECRUITING

Related Links

• BMS Clinical Trial Information
• BMS Clinical Trial Patient Recruiting

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

Famotidine

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Thiazoles; Sulfur Compounds; Organic Chemicals; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Officials

• Bristol-Myers Squibb

  Bristol-Myers Squibb

  STUDY DIRECTOR

Central Study Contacts

BMS Clinical Trials Contact Center www.BMSClinicalTrials.com

CONTACT

855-907-3286; Clinical.Trials@bms.com

First line of the email MUST contain NCT # and Site #.

CONTACT

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 25, 2024
• First Posted: July 1, 2024
• Study Start: July 17, 2024
• Primary Completion (Estimated): January 6, 2027
• Study Completion (Estimated): November 16, 2027
• Last Updated: March 17, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR
• Time Frame: See Plan Description
• Access Criteria: See Plan Description

Locations

GB (3); US (13); FR (3)