184 Participants Needed

BMS-986470 for Sickle Cell Disease

Recruiting at 29 trial locations
Fl
BC
Overseen ByBMS Clinical Trials Contact Center www.BMSClinicalTrials.com
Age: 18+
Sex: Any
Trial Phase: Phase 1 & 2
Sponsor: Bristol-Myers Squibb
Must be taking: Hydroxyurea
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment, BMS-986470, to evaluate its safety and effectiveness for people with sickle cell disease and healthy volunteers. Sickle cell disease can cause pain and other serious health issues. The trial seeks healthy individuals and those with sickle cell disease who have experienced multiple painful episodes, known as vaso-occlusive crises, in the past year. Participants will help researchers understand the treatment's effects and how the body processes it. As a Phase 1 trial, this research focuses on understanding the treatment's function in people, offering participants the opportunity to be among the first to receive it.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, if you are receiving regular blood transfusions, you may not be eligible to participate.

Is there any evidence suggesting that BMS-986470 is likely to be safe for humans?

Research shows that BMS-986470 is being tested for safety in treating sickle cell disease. In earlier studies, most participants tolerated the treatment well, with no reports of serious side effects. Some experienced mild issues like headaches and nausea, common with many treatments, which did not halt the study.

The treatment remains in the early testing phase, so researchers continue to assess its safety. While initial results are encouraging, more research is needed to fully understand its safety profile. The continuation of the study suggests confidence in its potential safety for patients.12345

Why do researchers think this study treatment might be promising for sickle cell disease?

Most treatments for sickle cell disease, like hydroxyurea and blood transfusions, aim to manage symptoms and prevent complications. But BMS-986470 is unique because it targets sickle cell disease at the root by aiming to modify the underlying causes of the disease rather than just treating symptoms. Researchers are excited about this treatment because it could potentially reduce the frequency of painful episodes and improve overall quality of life for patients. This new approach offers hope for a more effective and long-lasting impact compared to existing therapies.

What evidence suggests that BMS-986470 might be an effective treatment for sickle cell disease?

Research shows that BMS-986470, which participants in this trial may receive, is designed to help people with sickle cell disease by increasing levels of fetal hemoglobin (HbF). Fetal hemoglobin can prevent red blood cells from becoming sickle-shaped, a major issue in this disease. While researchers are still learning about its effectiveness in humans, this treatment modifies certain proteins to boost HbF production. Early results from other studies suggest it might be beneficial, but more research is needed to confirm its efficacy for sickle cell disease.12567

Who Is on the Research Team?

BS

Bristol-Myers Squibb

Principal Investigator

Bristol-Myers Squibb

Are You a Good Fit for This Trial?

This trial is for healthy individuals and those with sickle cell disease. Participants must meet specific health criteria to join, but the exact inclusion and exclusion details are not provided.

Inclusion Criteria

I have Sickle Cell Disease with a specific genotype.
My organs are functioning normally.
Cohort A: BMI of 18.0 to 32.0 kg/m^2
See 4 more

Exclusion Criteria

I have had severe pain crises or lung-related complications.
Cohort B: Creatinine clearance < 60 mL/min/1.72m2
Cohort A: Any significant medical condition or condition confounding data interpretation
See 4 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive BMS-986470 to evaluate safety, tolerability, pharmacokinetics, and pharmacodynamics

4 weeks
Multiple visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

22 weeks
Regular visits (in-person)

What Are the Treatments Tested in This Trial?

Interventions

  • BMS-986470
Trial Overview The study is testing BMS-986470's safety, how it affects the body (pharmacokinetics/dynamics), its behavior in different pH levels and after food intake, as well as its initial effectiveness in treating sickle cell disease compared to a placebo.
How Is the Trial Designed?
5Treatment groups
Experimental Treatment
Group I: Cohort B Part 2Experimental Treatment1 Intervention
Group II: Cohort B Part 1Experimental Treatment2 Interventions
Group III: Cohort A Part 3Experimental Treatment2 Interventions
Group IV: Cohort A Part 2Experimental Treatment2 Interventions
Group V: Cohort A Part 1Experimental Treatment2 Interventions

Find a Clinic Near You

Who Is Running the Clinical Trial?

Bristol-Myers Squibb

Lead Sponsor

Trials
2,731
Recruited
4,127,000+
Headquarters
New York City, USA
Known For
Oncology & Cardiovascular
Top Products
Eliquis, Opdivo, Revlimid, Orencia
Christopher Boerner profile image

Christopher Boerner

Bristol-Myers Squibb

Chief Executive Officer since 2023

PhD in Business Administration from the Haas School of Business, University of California, Berkeley; BA in Economics and History from Washington University in St. Louis

Deepak L. Bhatt profile image

Deepak L. Bhatt

Bristol-Myers Squibb

Chief Medical Officer since 2024

MD from Yale University; MSc in Clinical Epidemiology from the University of Pennsylvania

Citations

Trial ID CA230-1019 | NCT06481306 - BMS Clinical TrialsTrial ID CA230-1019 | NCT06481306. A Study to Evaluate BMS-986470 in Healthy Volunteers and Participants With Sickle Cell Disease.
Study on the Safety and Effects of BMS-986470 for Patients ...This clinical trial investigates the safety and effects of BMS-986470, a film-coated tablet, for patients with Sickle Cell Disease.
A drug, BMS-986470, to treat sickle cell disease (SCD)BMS-986470 is a small molecule cereblon E3 ligase modulator (CELMoD) that doctors think will increase fetal hemoglobin (HbF).
Bristol-Myers Squibb's Promising Study on BMS-986470 ...The study aims to assess the safety, tolerability, and preliminary efficacy of BMS-986470, a potential treatment for sickle cell disease, ...
BMS-986470 for Sickle Cell DiseaseThe purpose of this study is to evaluate the safety and tolerability, pharmacokinetics and pharmacodynamics, pH and food effect, and preliminary efficacy of ...
Bristol Myers Squibb's Presentations at ASH 2024 ...... BMS-986470 for the treatment of sickle cell disease; Multiple preclinical analyses evaluating potential first-in-class CELMoD agent BMS-986397 targeting ...
BMS-986470 | ZBTB7A/WIZ Dual Molecular Glue DegraderBMS-986470 has potent γ-globin induction activity. BMS-986470 can be used for sickle cell disease (SCD) research. Molecular Weight. 523.54.
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