A Study to Evaluate BMS-986470 in Healthy Volunteers and Participants With Sickle Cell Disease
This research study is looking at a new medication called BMS-986470. It's being tested in two groups: healthy volunteers and people who have sickle cell disease. For all participants, the main goals are to make sure the medicine is safe and well-tolerated, and to understand how it behaves in the body. For those with sickle cell disease, the study also aims to see if this new medicine could help improve symptoms, such as painful crises. The study is in its early stages (Phases 1 and 2), meaning it's one of the first times this medicine is being given to people.
At a glance
What is this study about?
Imagine you or someone you know lives with sickle cell disease (SCD), a condition where red blood cells are shaped like crescent moons instead of round discs. These oddly shaped cells can block tiny blood vessels, leading to severe pain, called a vaso-occlusive crisis (VOC), and other health problems. Researchers are always looking for new ways to help manage this condition and improve the lives of people with SCD.
This study, called "A Study to Evaluate BMS-986470 in Healthy Volunteers and Participants With Sickle Cell Disease," is exploring a new medicine named BMS-986470. The study has two main parts. One part involves healthy volunteers who don't have sickle cell disease. Their involvement helps researchers understand how the new medicine works in the body and what effects it might have, before it's given to people who are unwell. The other part involves people living with sickle cell disease.
For the healthy volunteers, the focus is entirely on safety and understanding how the body handles the medicine. For people with sickle cell disease, the study will also look at whether the medicine helps reduce symptoms like painful crises. This is an early-stage study, meaning it's one of the first times this particular medicine has been given to people. The information gathered will be crucial for deciding if this medicine has potential as a future treatment for sickle cell disease.
Key takeaways
- This study is testing a new medicine (BMS-986470) for sickle cell disease.
- It involves both healthy volunteers and people with sickle cell disease.
- The main goals are to check the medicine's safety, how it works, and if it helps with sickle cell symptoms.
- This is an early-stage clinical trial (Phases 1 and 2).
- Participation involves clinic visits, assessments, and taking the study medication or a placebo.
- You can withdraw from the study at any time.
Who may be eligible?
To join this study, you need to be at least 18 years old. There are two groups of participants: healthy volunteers and people with sickle cell disease.
If you're a healthy volunteer, you'll need to be in general good health, as confirmed by a doctor, and have a body weight that falls within a certain range. Women who are able to have children won't be able to join this group. You also shouldn't have any major medical conditions or planned surgeries.
If you have sickle cell disease, you'll need to have a confirmed diagnosis (types HbSS, HbSβ0-thal, or HbSβ+-thal) and have experienced a certain number of painful crises (at least 4 in the last year, or 2 in the last 6 months). Your general health should be good enough to take part in a study, and your blood test results, like haemoglobin and white blood cell counts, must be within specific limits. You also shouldn't have any other significant medical conditions that could make it unsafe to participate.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you aged 18 or older?
- Do you have a diagnosis of sickle cell disease (HbSS, HbSβ0-thal, or HbSβ+-thal) or are you a healthy adult?
- If you have sickle cell disease, have you had at least 4 painful crises in the last year, or 2 in the last 6 months?
- Are you generally in good health, as assessed by a doctor?
- Are you willing and able to attend all required study appointments?
- If you are a woman of childbearing potential, are you prepared to use effective contraception if required for the study?
What does participation involve?
Taking part in this study means you would likely have several visits to the study clinic. During these visits, the study team would perform various assessments. This includes physical examinations, checking your vital signs (like blood pressure and heart rate), taking blood and urine samples, and performing heart checks (ECGs). You'll be given the study medication, which could be BMS-986470, a placebo (a dummy medicine with no active ingredients), or famotidine (a common heartburn medicine used as a comparison). You won't know which one you're receiving. The study involves regular follow-up to monitor your health and how you respond to the treatment. The total duration of your participation in the study would be explained in detail by the study team.
Potential risks and benefits
Locations (19)
- University of Alabama at BirminghamVerified postcodeBirmingham, United States· Recruiting
- University of California San Diego - La JollaVerified postcodeLa Jolla, United States· Recruiting
- UCSF Benioff Children's Hospital OaklandVerified postcodeOakland, United States· Recruiting
- Yale-New Haven HospitalVerified postcodeNew Haven, United States· Recruiting
- Winship Cancer Institute of Emory UniversityVerified postcodeAtlanta, United States· Recruiting
- Local Institution - 0034Verified postcodeChicago, United States· Not yet recruiting
- Local Institution - 0001Verified postcodeLenexa, United States· Active not recruiting
- Local Institution - 0024Verified postcodeBoston, United States· Not yet recruiting
- Boston Medical CenterVerified postcodeBoston, United States· Recruiting
- Thomas Jefferson University - Medicine/GI and HepatologyVerified postcodePhiladelphia, United States· Recruiting
- Local Institution - 0032Verified postcodePittsburgh, United States· Not yet recruiting
- Inova Schar Cancer InstituteVerified postcodeFairfax, United States· Recruiting
Common questions
What is sickle cell disease?
Sickle cell disease is a blood disorder where red blood cells become crescent-shaped, causing pain and other health issues.
What is a placebo?
A placebo is an inactive 'dummy' medicine that looks like the real treatment but contains no active ingredients. It helps researchers compare results.
Why are healthy volunteers included in the study?
Healthy volunteers help researchers understand how the new medicine works in the body and if it's safe before it's given to people with the disease.
Can I leave the study if I decide to?
Yes, you have the right to withdraw from the study at any time, for any reason, without it affecting your regular medical care.
What are 'Phases 1 and 2' of a study?
These are early stages of drug development. Phase 1 focuses on safety and how the medicine works in the body, while Phase 2 looks at effectiveness and dose.
How to find out more
BMS Clinical Trials Contact Center www.BMSClinicalTrials.com
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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