Authorised (with conditions)Phase II and Phase III (Integrated)Interventional

C5351004 - A Phase 2/3 Randomized, Multicenter Study of Osivelotor Administered Orally to Participants with Sickle Cell Disease and an Open-Label Pharmacokinetics Study in Pediatric Participants with Sickle Cell Disease

This research study, called C5351004, is looking into a new oral medicine called osivelotor for adults living with sickle cell disease. The main goal is to find out if this medicine can help increase the amount of healthy red blood cells (haemoglobin) in the body and reduce painful events called vaso-occlusive crises (VOCs). Researchers will also be checking how safe the medicine is and if there are any side effects. Participants will be given either the new medicine or a dummy pill (placebo). There's also a part of the study specifically for children to understand how the medicine moves through their bodies. This is an important step in finding better treatments for sickle cell disease.

At a glance

Status

Authorised (with conditions)

Phase

Phase II and Phase III (Integrated)

Sponsor

Global Blood Therapeutics Inc.

Enrolment target

Start

23 Jul 2024

Sickle Cell Disease

What is this study about?

This study, known as C5351004, is a significant research project for people with sickle cell disease. The main purpose is to test a new medicine called osivelotor, which is given as a tablet. Researchers want to understand if this medicine can help improve the health of red blood cells in people with sickle cell, which in turn could lead to fewer painful episodes (known as vaso-occlusive crises or VOCs) and a better quality of life. This particular study is combining two phases, Phase 2 and Phase 3, which means it's looking closely at both the safety of the medicine and how effective it is.

The study is divided into different parts. One part is for adults and will compare osivelotor to a placebo (a tablet that looks the same but contains no medicine). This helps researchers see if any benefits come directly from the new medicine. Another part is specifically for children with sickle cell disease. In this part, the focus is on understanding how the medicine is absorbed, used, and cleared from the body, and what effects it has on their red blood cells. This information is crucial for figuring out the right dose for younger patients.

Ultimately, researchers hope that osivelotor will prove to be a safe and effective new treatment option that can help people with sickle cell disease live healthier lives with fewer complications. By taking part in a study like this, patients are contributing important knowledge that could benefit many others in the future.

Key takeaways

This study is testing a new oral medicine, osivelotor, for sickle cell disease.
It aims to increase healthy red blood cells and reduce painful sickle cell crises.
Adults will compare the new medicine with a dummy pill (placebo).
There's also a part of the study focusing on children with sickle cell disease.
Participation involves regular clinic visits, blood tests, and health checks.
It's a chance to contribute to new treatments for sickle cell disease.

Who may be eligible?

This study is looking for adults who are 18 years old or older and have sickle cell disease. Both men and women can take part.

There will be specific health criteria that the doctors will need to check to make sure the study is right for you. They will review your medical history and current health to ensure it's safe for you to participate.

It’s important to discuss with your doctor whether this study is suitable for your individual health situation. They can explain all the details and help you understand if you meet the specific requirements.

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 18 years old or older?
Do you have a confirmed diagnosis of sickle cell disease?
Are you able to take medicines by mouth (tablets)?
Are you willing and able to attend regular clinic appointments?
Are you prepared to provide blood samples as required?

Answer every question to see your result.

What does participation involve?

Taking part in this study will involve regular visits to the clinic over a period of time. You will be given either the new medicine, osivelotor, or a placebo (a dummy pill) to take by mouth during the adult part of the study. For the children's part, they will receive the study medicine.

During your visits, you can expect to have blood tests to check your blood cell levels and how the medicine is affecting your body. You might also have physical examinations, heart checks (ECGs), and provide information about any changes in your health or symptoms you experience. The doctors and nurses will be there to support you and explain everything clearly throughout the study. The total duration of your participation will be discussed with you, but for some parts, it could be up to 48 weeks with follow-up appointments.

Potential risks and benefits

Participating in any clinical trial involves both potential benefits and potential risks. You might benefit from receiving a new medicine before it’s widely available, which could improve your health conditions. However, the medicine may not work for everyone, or it might cause side effects. These side effects could be mild or, in rare cases, more serious. All known risks will be explained to you in detail before you decide to take part. You will also be closely monitored by medical staff throughout the study. Remember, your participation is completely voluntary, and you have the right to withdraw from the study at any time, for any reason, without affecting your usual medical care.

Locations (2)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Germany
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Unverified
France

Common questions

What is sickle cell disease?

Sickle cell disease is a lifelong inherited blood disorder where your red blood cells are shaped like a crescent moon or sickle. These cells can block blood flow, causing pain, infection, and damage to organs.

What is 'osivelotor'?

Osivelotor is the name of the new medicine being tested in this study. It's an oral tablet designed to help improve red blood cell health in people with sickle cell disease.

What is a 'placebo'?

A placebo is a tablet that looks exactly like the study medicine but doesn't contain any active drug. It's used to compare the effects of the new medicine to see if it's truly effective.

Will I know if I'm getting the real medicine or the placebo?

In the adult part of the study, neither you nor the study team will know who is getting the real medicine and who is getting the placebo. This is to ensure the results are accurate and unbiased.

What are 'vessel-occlusive crises' (VOCs)?

VOCs are sudden, very painful episodes that can happen in people with sickle cell disease. They occur when sickled red blood cells block small blood vessels, stopping blood flow to parts of the body.