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

Phase 1

Recruiting

• Conditions: Healthy Volunteers; Anemia, Sickle Cell
• Interventions: Drug: BMS-986470; Drug: Placebo

• Registration Number: NCT06481306
• Lead Sponsor: Bristol-Myers Squibb

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-06-25
• Study First Submitted QC: 2024-06-25
• Study First Posted: 2024-07-01
• Study Start: 2024-07-17
• Status Verified: 2025-02
• Last Update Submitted: 2025-02-18
• Last Update Posted: 2025-02-19
• Primary Completion: 2027-01-06
• Study Completion: 2027-11-16

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 184

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Cohort A Part 3	BMS-986470	-
Cohort B Part 2	BMS-986470	-
Cohort A Part 2	BMS-986470	-
Cohort B Part 1	Placebo	-
Cohort A Part 2	Placebo	-
Cohort A Part 1	BMS-986470	-
Cohort A Part 1	Placebo	-
Cohort B Part 1	BMS-986470	-

Primary Outcome Measures

Name	Time	Method
Number of deaths	Up to Day 154
Number of participants with serious adverse events (SAEs)	Up to Day 154
Number of participants with AEs leading to discontinuation	Up to Day 154
Number of participants with AEs meeting protocol-defined Dose Limiting Toxicity (DLT) criteria	Up to Day 154
Number of participants with adverse events (AEs)	Up to Day 154

Secondary Outcome Measures

Name	Time	Method
Change from baseline in total hemoglobin (Hb)	Up to Day 154	Cohort A Part 2, Cohort B Parts 1 and 2
Change from baseline in markers of red blood cell (RBC) lysis: total Hb	Up to Day 154	Cohort B
Change from baseline in markers of RBC lysis: haptoglobin	Up to Day 154	Cohort B
Number of participants achieving HbF ≥ 20%	Up to Day 154	Cohort B
Change from baseline in markers of RBC lysis: reticulocyte percentage of RBCs	Up to Day 154	Cohort B
Change from baseline in markers of RBC lysis: lactate dehydrogenase (LDH)	Up to Day 154	Cohort B
Change from baseline in markers of RBC lysis: absolute reticulocyte count	Up to Day 154	Cohort B
Change from baseline in markers of RBC lysis: schistocyte percentage of RBCs	Up to Day 154	Cohort B
Number of participants achieving HbF ≥ 30%	Up to Day 154	Cohort B
Number of participants achieving HbF ≥ 10%	Up to Day 154	Cohort B
Change from baseline in markers of RBC lysis: absolute schistocyte count	Up to Day 154	Cohort B
Time of maximum observed plasma concentration (Tmax)	Up to Day 28	Cohort A Parts 1 and 2 and Cohort B Part 1
Change from baseline in total Hb fractions: sickle Hb (HbS)	Up to Day 154	Cohort B
Change from baseline in markers of RBC lysis: aspartate aminotransferase (AST)	Up to Day 154	Cohort B
Maximum observed plasma concentration (Cmax)	Up to Day 28	Cohort A Parts 1 and 2 and Cohort B Part 1
Change from baseline in total Hb fractions: adult Hb (HbA)	Up to Day 28	Cohort A Part 2
Area under the concentration-time curve (AUC)	Up to Day 28	Cohort A Parts 1 and 2 and Cohort B Part 1
Dose proportionality of BMS-986470 for Cmax and AUC	Up to Day 28	Assessed by using the slope of a statistical linear relationship between the ln-transformed PK parameters AUC and Cmax and the ln-transformed dose will be fitted by using power model
Change from baseline in total Hb fractions: fetal Hb (HbF)	Up to Day 154	Cohort A Part 2, Cohort B Parts 1 and 2
Change from baseline in markers of RBC lysis: total bilirubin	Up to Day 154	Cohort B
Change from baseline in markers of RBC lysis: indirect bilirubin	Up to Day 154	Cohort B

Trial Locations

Locations (16)

Local Institution - 0025; 🇫🇷 Strasbourg, Alsace, France

Local Institution - 0008; 🇺🇸 Birmingham, Alabama, United States

Local Institution - 0021; 🇺🇸 La Jolla, California, United States

Local Institution - 0003; 🇺🇸 Oakland, California, United States

Local Institution - 0022; 🇺🇸 New Haven, Connecticut, United States

Local Institution - 0017; 🇺🇸 Atlanta, Georgia, United States

ICON; 🇺🇸 Lenexa, Kansas, United States

Local Institution - 0024; 🇺🇸 Boston, Massachusetts, United States

Local Institution - 0016; 🇺🇸 Boston, Massachusetts, United States

Local Institution - 0007; 🇺🇸 Philadelphia, Pennsylvania, United States

Local Institution - 0013; 🇺🇸 Fairfax, Virginia, United States

Local Institution - 0014; 🇺🇸 Richmond, Virginia, United States

Local Institution - 0015; 🇫🇷 Marseille, Bouches-du-Rhône, France

Local Institution - 0023; 🇫🇷 Paris, France

Local Institution - 0006; 🇬🇧 London, London, City Of, United Kingdom

Local Institution - 0005; 🇬🇧 Leeds, United Kingdom