Study on the Safety and Effects of BMS-986470 for Patients with Sickle Cell Disease

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What is this study about?

This clinical trial is focused on studying Sickle Cell Disease, a condition that affects the red blood cells, causing them to become misshapen and break down. The study will evaluate a new treatment called BMS-986470, which is taken as a film-coated tablet. The purpose of the study is to determine if BMS-986470 is safe and well-tolerated when given to both healthy individuals and those with sickle cell disease. Additionally, the study aims to find the best dose of BMS-986470 for future research in participants with sickle cell disease.

The study will involve participants taking the medication and being monitored for any side effects or adverse events. Some participants will receive a placebo, which is a substance with no active medication. The study will also look at how the body processes the drug and its effects on different types of hemoglobin, including fetal and sickle forms. Researchers will check if there are improvements in other markers related to sickle cell disease.

Participants will be involved in the study for a period of time, during which they will take the medication and undergo various tests and assessments. The study will help gather important information about the safety and potential benefits of BMS-986470 for people with sickle cell disease, contributing to the development of new treatment options for this condition.

1 initial assessment

Upon joining the study, an initial assessment will be conducted to confirm eligibility. This includes reviewing medical history and current health status.

Blood tests will be performed to ensure that blood levels meet the study’s requirements.

2 medication administration

Participants will receive the study medication, BMS-986470, in the form of a film-coated tablet.

The medication is taken orally as directed by the study team. Dosage and frequency will be determined based on the study phase and individual response.

3 monitoring and follow-up

Regular follow-up visits will be scheduled to monitor health and response to the medication.

During these visits, blood tests and other assessments will be conducted to track any changes in health and to measure the levels of different types of hemoglobin.

4 evaluation of safety and tolerability

The study will evaluate the safety and tolerability of BMS-986470 by monitoring for any adverse events or side effects.

Participants are encouraged to report any unusual symptoms or health changes during the study.

5 completion of study participation

At the end of the study period, a final assessment will be conducted to evaluate overall health and the effects of the medication.

Participants will receive information on the study’s findings and any next steps if applicable.

Who Can Join the Study?

Participants must be at least 18 years old.
Participants must have a documented diagnosis of Sickle Cell Disease (SCD) with specific genotypes: HbSS, HbSβ0-thalassemia, or HbSβ+-thalassemia.
Participants must have experienced at least 4 Vaso-Occlusive Crises (VOCs) in the past 12 months or at least 2 VOCs in the past 6 months. VOCs are painful episodes caused by blocked blood flow.
Participants must have tried and failed, cannot tolerate, or are not suitable for approved therapies for SCD.
Participants must have certain blood test results:
- Hemoglobin (Hb) levels between 5.5 and 12 g/dL for males, or between 5.5 and 10.6 g/dL for females. Hemoglobin is a protein in red blood cells that carries oxygen.
- Absolute neutrophil count of at least 1500/μL. Neutrophils are a type of white blood cell important for fighting infections.
- Platelet count of at least 100,000/μL. Platelets help with blood clotting.
- Absolute reticulocyte count greater than 100,000/μL, or greater than 50,000/μL if taking hydroxyurea. Reticulocytes are young red blood cells.

Who Cannot Join the Study?

Participants who do not have Sickle Cell Disease cannot join the study.
Participants who are not within the specified age range cannot join the study.
Participants who are part of a vulnerable population cannot join the study.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Hopital Necker Enfants Malades	Paris	France
Aix Marseille University	Marseille	France

Other Sites

Site Name	City	Country
Institut De Cancerologie Strasbourg Europe	STRASBOURG, Alsace	France
Hrockib Hpisy Mqsfle &qumvrz 1 rts Ghdygtm Ebbxps	Creteil	France
Hpbdctwi Uzbkngfwfkqjny Saeffnhzms &yoxtcc Hztzsxk df Hvklffpelvi	STRASBOURG, Alsace	France

Trial status

Country	Status	Recruitment Start
France	Recruiting	26.05.2025

Trial locations

Investigated drugs:

BMS-986470

BMS-986470 is a new medication being tested to see if it is safe and can be tolerated by people. The study is looking at how the body processes this medication and how it affects the body. It is being tested in both healthy people and those with sickle cell disease, a condition that affects the red blood cells. The goal is to find out the best dose to use in future studies for people with sickle cell disease. This medication is still in the early stages of research, so it is important to understand how it works and if it can help people with this condition.

Investigated diseases:

Sickle cell disease

Sickle Cell Disease – Sickle Cell Disease is a genetic disorder that affects the red blood cells, causing them to become rigid and shaped like a crescent or sickle. These abnormally shaped cells can block blood flow in small blood vessels, leading to pain and potential organ damage. The disease is characterized by episodes of pain, known as sickle cell crises, which can vary in frequency and severity. Over time, the repeated blockages and reduced blood flow can lead to complications such as anemia, infections, and damage to organs like the spleen and liver. The progression of the disease can vary widely among individuals, with some experiencing more frequent and severe symptoms than others. The condition is lifelong and typically diagnosed in early childhood.

Trial ID:

2023-510283-12-00

Protocol code:

CA230-1019

NCT ID:

NCT06481306

Trial Phase:

Phase I and Phase II (Integrated) – First administration to humans

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Study on the Safety and Effects of BMS-986470 for Patients with Sickle Cell Disease

What is this study about?

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