CA2301019: Phase 1/2a Study of BMS-986470 in Healthy and Sickle Cell Participants
Research type
Research Study
Full 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
IRAS ID
1011581
Contact name
GSM-CT Representative
Contact email
Sponsor organisation
Bristol-Myers Squibb Services Unlimited Company
Eudract number
2023-510283-12
Clinicaltrials.gov Identifier
Research summary
This study is testing a new drug called BMS-986470 to see if it is safe and effective for people with Sickle Cell Disease (SCD). SCD is a genetic disorder that causes red blood cells to become abnormally shaped, causing them to be more rigid and likely to stick together leading to pain and other health problems. This oral drug aims to increase foetal haemoglobin (HbF), which can help reduce these symptoms and improve the quality of life for people with SCD by reducing pain and preventing long-term damage.
The study has two groups: healthy volunteers and participants with SCD; by including both groups, researchers will gather important information on the drug's safety, how it behaves in the body, and its effects. Initially, healthy volunteers will help find the best dose of the drug that is also safe. Then, this dose will be tested in people with SCD to see how safe and how well it works. This second part of the trial is the one which is being offered to patients in the UK.
Participants must be healthy, over 18, with no upper age limit for SCD. People with SCD need to have specific types of SCD, such as the most common form (HbSS), also call sickle cell anaemia, or a combination of sickle cell and beta-thalassemia (HbSβ0 or HbSβ+) and have experienced a certain number of pain crises in the past year.
Participants will go through screening, treatment, and follow-up stages. Participants will receive different doses of BMS-986470 or a placebo to find the best dose and see if the drug has the potential to reduce SCD complications. The study will take place at multiple locations, lasting up to 182 days, depending on the assigned study group, with Screening (up to 28 days), On-treatment (28 or126 days), and Follow-up (28 days).
The study is funded by Bristol-Myers Squibb and is important for patients, the public, researchers, and doctors as it could lead to better treatment options for SCD.
REC name
Wales REC 2
REC reference
25/WA/0075
Date of REC Opinion
20 May 2025
REC opinion
Further Information Favourable Opinion