A Phase 1/2a, First-in-human Study to Evaluate BMS-986470 in Healthy Volunteers and Participants with Sickle Cell Disease

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Bristol-Myers Squibb Services Unlimited Company

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Hemic and Lymphatic Diseases [C15]

Trial duration

12 Jun 2025 → ongoing

Decision date (initial)

2025-05-16

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

External identifiers

EU CT number

2023-510283-12-00

WHO UTN

U1111-1301-6753

ClinicalTrials.gov

NCT06481306

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacodynamic, Safety, Others, Pharmacokinetic

To check if BMS-986470 is safe and well-tolerated when given alone to healthy participants and participants with sickle cell disease. To find out the recommended phase 2 dose (RP2D) of BMS-986470 in participants with sickle cell disease.

Secondary objectives 3

1. To characterize the plasma pharmacokinetic (PK) of BMS-986470 following one or multiple dose oral administration in healthy volunteers and participants with SCD
2. To characterize the pharmacodynamic (PD) profile and preliminary efficacy of BMS-986470 related to changes in total Hb and Hb fractions from baseline
3. To assess additional preliminary efficacy of BMS-986470

Conditions and MedDRA coding

Sickle Cell Disease

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Participants must be at least 18 years of age or older with a documented diagnosis of SCD with genotype HbSS, HbSβ0 -thal, or HbSβ+ -thal; ≥ 4 VOCs within the previous 12 months or ≥ 2 VOCs within the previous 6 months
2. Participant has failed, is unable to tolerate, or is not a candidate for approved therapies for SCD therapies
3. Participants must have the following laboratory values: Hb ≥ 5.5 and ≤ 12 g/dL (males) or ≥ 5.5 and ≤ 10.6 g/dL (females), absolute neutrophil ≥ 1500/μL , Platelet ≥ 100 × 103 /μL, absolute reticulocyte > 100 × 103 /μL or > 50 × 103 /μL if taking hydroxyurea.

Exclusion criteria 3

1. 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 drug or any VOC requiring ≥ 24 hours of hospital admission within 30 days prior to the first dose of study drug.
2. Participants with any episode of acute chest syndrome within the last 6 months prior to the first dose.
3. 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 participanting in this study.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Incidence of adverse events (AEs), serious adverse events (SAEs), AEs meeting protocol-defined dose limiting toxicity (DLT) criteria, AEs leading to discontinuation, and death.

Secondary endpoints 2

1. A summary of the exposure levels of the study drug as well as changes in levels of different types of hemoglobin including fetal and sickle forms of hemoglobin.
2. The study will also check whether other markers connected to sickle cell disease are improving

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 3

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Bristol-Myers Squibb Services Unlimited Company

Sponsor organisation

Bristol-Myers Squibb Services Unlimited Company

Address

Plaza 254 Blanchardstown Corporate Park 2

City

Dublin 15

Postcode

D15 T867

Country

Ireland

Scientific contact point

Organisation

Bristol-Myers Squibb Services Unlimited Company

Contact name

GSM-CT

Public contact point

Organisation

Bristol-Myers Squibb Services Unlimited Company

Contact name

GSM-CT

Third parties 13

Locations

1 EU/EEA country · 5 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Oversight and notifications

Regulatory notifications under CTR Articles 38, 52, 53, 54 and 77

Unexpected events 2 · Art. 53 CTR

Note: SUSARs are reported via EudraVigilance, not CTIS — events shown here are CTIS-public notifications only.

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 11 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications