Active not recruitingPHASE3INTERVENTIONAL

Study of Two Doses of Crizanlizumab Versus Placebo in Adolescent and Adult Sickle Cell Disease Patients

This study is investigating a new medication called crizanlizumab. It's for teenagers and adults with sickle cell disease who have experienced painful episodes, known as vaso-occlusive crises (VOCs), which needed a hospital visit. We are testing two different doses of crizanlizumab against a placebo (a dummy medicine with no active drug) to see how well they work and if they are safe. Some participants might already be taking other sickle cell medicines like hydroxyurea/hydroxycarbamide, and this study will look at crizanlizumab alongside those. The main goal is to find out if crizanlizumab can help reduce the number of painful crises and improve life for people with sickle cell disease.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Novartis Pharmaceuticals

Enrolment target

255

Start

26 Jul 2019

Estimated completion

23 Nov 2026

Sickle Cell Disease (SCD)

What is this study about?

This study is called STAND, and it's looking into a new medicine called crizanlizumab for people with sickle cell disease. Sickle cell disease can cause very painful episodes, known as vaso-occlusive crises (VOCs), which often require a trip to the doctor or hospital. The main aim of this study is to see if crizanlizumab can help prevent or reduce these painful crises.

Researchers are comparing two different doses of crizanlizumab (5 mg/kg and 7.5 mg/kg) against a 'placebo.' A placebo contains no active medicine and is used to see if the new medicine really makes a difference. Participants will be given one of the two crizanlizumab doses or the placebo. The study will look carefully at whether the new medicine is effective in reducing the number of painful crises and also if it's safe to use.

This is a 'Phase 3' study, which means the medicine has already been tested in smaller groups and is now being tested in a larger group of people to confirm its effects and safety before it can be considered for wider use. It's also a 'double-blind' study, meaning neither the participants nor their doctors will know whether they are receiving crizanlizumab or the placebo. This helps make sure the results are fair and unbiased. Participants will continue any other sickle cell treatments they are already on.

Key takeaways

Tests a new medicine (crizanlizumab) for sickle cell disease.
Aims to reduce painful sickle cell crises.
Compares two doses of the medicine against a dummy pill (placebo).
Open to teenagers and adults with sickle cell disease who have had painful crises.
Participants continue their regular sickle cell treatments if already on them.
It's a large-scale study (Phase 3) to confirm safety and effectiveness.

Who may be eligible?

This study is looking for volunteers aged 12 and older who have been diagnosed with sickle cell disease. To join, you must have experienced at least two painful sickle cell crises (VOCs) that needed medical attention in the last year.

If you're already taking medicines like hydroxyurea (also known as hydroxycarbamide) or L-glutamine, you need to have been on a steady dose for at least three months. If you're not taking these medicines, you can still join, but you must not have taken them for at least six months before starting the study. The study wants to make sure that the painful crises you've experienced were despite your current treatment, showing you need more help with your symptoms.

Before you can start, you'll need to sign a consent form, which means you understand what the study involves and agree to take part. Researchers will also check your health to make sure the study is a good fit for you.

Quick self-check

Are you 12 years old or older?
Do you have a confirmed diagnosis of sickle cell disease?
Have you had at least two painful sickle cell crises that needed medical help in the last year?
If you take hydroxyurea or L-glutamine, have you been on a steady dose for at least 3 months?
If you don't take these medicines, have you not taken them for at least 6 months?
Are you able to attend regular study appointments?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

Taking part in this study means you'll be given either crizanlizumab or a placebo (a dummy medicine). You will receive this treatment regularly. You will also have regular check-ups with doctors and nurses, which will involve giving blood samples, having your health monitored, and discussing any symptoms you experience. The study will track how many painful crises you have and how well you feel.

These visits will happen over a period of time, and it's important that you can attend all scheduled appointments. You'll be asked about your health, any pain you're having, and how you're feeling overall. The total duration for each participant is not specified in detail here, but these types of studies typically involve ongoing treatment and follow-up for a year or more. You'll continue taking any regular sickle cell medicines you were on before the study started.

Potential risks and benefits

Participating in this study might offer some potential benefits, such as receiving a new treatment that could reduce your painful sickle cell crises. However, there's also a chance you could receive the placebo, which won't directly treat your condition. Like all medicines, crizanlizumab can have side effects, which the study team will monitor closely. You'll be given detailed information about these. It's really important to remember that joining a study is always your choice, and you are free to leave at any time, for any reason, without it affecting your medical care.

Locations (59)

Childrens Healthcare of Atlanta
Atlanta, United States
Boston Medical Center
Boston, United States
Levine Cancer Insitute Carolinas Healthcare System
Charlotte, United States
Univ of Tenn Health Sciences Ctr
Memphis, United States
U of TX Health Science Ct
Houston, United States
Novartis Investigative Site
Brussels, Belgium
Novartis Investigative Site
Brussels, Belgium
Novartis Investigative Site
Edegem, Belgium
Novartis Investigative Site
Salvador, Brazil
Novartis Investigative Site
Belém, Brazil
Novartis Investigative Site
Recife, Brazil
Novartis Investigative Site
Rio de Janeiro, Brazil

+47 more sites — see the official record for the full list.

Common questions

What is crizanlizumab?

Crizanlizumab is a new study medicine being tested to see if it can reduce painful crises (VOCs) in people with sickle cell disease.

What is a 'placebo'?

A placebo is a dummy medicine that looks just like the real medicine but contains no active drug. It helps researchers see if the real medicine is truly effective.

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

No, neither you nor your study doctor will know if you're receiving crizanlizumab or the placebo. This is called 'double-blind' and helps make the study fair.

Can I still take my usual sickle cell medicines?

Yes, you will continue your usual sickle cell medicines (like hydroxyurea) as prescribed by your doctor while taking part in this study.

What exactly is a 'painful crisis' for this study?

For this study, a painful crisis (VOC) means severe pain not easily explained otherwise, that requires you to seek medical help and receive strong pain relief, or conditions like acute chest syndrome.