Active not recruitingPHASE3INTERVENTIONAL

Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)

This study looked at a new medication called rilzabrutinib for adults and teenagers who have a condition called Immune Thrombocytopenia (ITP). ITP is a blood disorder where your body doesn't have enough platelets, making you more likely to bruise or bleed. The study compared rilzabrutinib to a dummy pill (placebo) to see if it could help increase platelet counts. Participants took either rilzabrutinib or the placebo twice a day. The main goal was to find out if this new medicine is safe and effective in helping people with ITP maintain healthy platelet levels. The study involved regular check-ups and lasted up to 60 weeks for each participant, including a period where neither the patient nor the doctor knew who was getting the actual medicine.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Principia Biopharma, a Sanofi Company

Enrolment target

232

Start

14 Dec 2020

Estimated completion

26 Aug 2026

Immune Thrombocytopenia

What is this study about?

This study was designed to investigate a new medication called rilzabrutinib for people living with a blood condition known as Immune Thrombocytopenia (ITP). ITP is a condition where your body's immune system mistakenly attacks and destroys your own platelets. Platelets are tiny blood cells that help your blood to clot, so having too few can lead to easy bruising, bleeding, or other more serious problems.

Researchers hoped to find out if rilzabrutinib could help increase and maintain stable platelet counts for patients whose ITP hasn't responded well to other treatments or has come back after treatment. By improving platelet levels, the medication might help reduce the risk of bleeding episodes and improve the quality of life for those with ITP. This type of study, called a "Phase 3" trial, is a crucial step in understanding if a new medication is safe and effective enough to be considered for wider use.

The study compared rilzabrutinib to a "placebo," which is a dummy pill that looks just like the study medicine but contains no active drug. This comparison helps scientists determine if any improvements seen are genuinely due to the new medication or if they could have happened by chance. This is a common and important practice in medical research to get clear answers about new treatments.

Key takeaways

This study tested a new medicine, rilzabrutinib, for ITP.
It included both adults and teenagers with persistent or chronic ITP.
Participants received either the new medicine or a dummy pill (placebo).
The study lasted up to 60 weeks, with options for longer treatment.
It aimed to improve platelet counts and reduce bleeding risk.

Who may be eligible?

To be able to take part in this study, adults needed to have had ITP for at least three months, and teenagers (aged 12 to younger than 18) needed to have had it for at least six months. Participants also had to have consistently low platelet counts, specifically below 30,000 per microliter, even after trying some standard ITP treatments.

There were also certain health checks participants needed to pass, like having healthy kidney and liver function, and a good level of hemoglobin (which carries oxygen in your blood). Both men and women needed to agree to use effective birth control if they were able to have children. Also, participants or their guardians needed to be able to understand and sign consent forms.

You wouldn't be able to join the study if your ITP was caused by another medical condition, if you were pregnant or breastfeeding, or if you had certain types of cancer or had received a blood transfusion recently. These checks help make sure the study gathers the most accurate information and that it is as safe as possible for all involved.

Quick self-check

Do I have ITP that has lasted for more than 3 months (adults) or 6 months (teenagers)?
Are my platelet counts consistently low (below 30,000)?
Have I tried other ITP treatments without lasting success?
Am I able to use effective birth control if needed?
Do I understand and agree to the study commitment?

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

What does participation involve?

If you decided to join this study, it would last for up to 60 weeks, which is about 14 months, from start to finish. This period included an initial screening phase lasting up to 4 weeks to check if you met all the requirements. After that, you would enter a "blinded" treatment period lasting between 12 and 24 weeks. This means neither you nor your doctor would know if you were receiving rilzabrutinib or the placebo.

Following the blinded period, everyone who continued in the study moved into an "open-label" period for 28 weeks. During this time, everyone knew they were receiving rilzabrutinib. After finishing the medication, there was a 4-week follow-up period to check on your health. For adults, there was also an option to continue treatment in a long-term extension period for up to another 12 months. Teenagers also had a similar option for an extended period of treatment.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. A potential benefit of this study was that you might receive a new medication, rilzabrutinib, which could improve your platelet count and reduce your risk of bleeding. However, you might also receive the placebo, which would not have any direct treatment effect on your ITP during the blinded phase. Potential risks could include side effects from the medication, which would be carefully monitored by the study team. Full details of any known side effects would be explained before you join. Remember, your participation is completely voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (155)

University of Southern California_Investigational Site Number 84024
Los Angeles, United States
UCSF Benioff Children's Hospital San Francisco_Investigational Site Number 84020
San Francisco, United States
Lundquist Institute for Biomedical Innovation at Harbor UCLA Medical Center_Investigational Site Number 84037
Torrance, United States
The Oncology Institute of Hope and Innovation_Investigational Site Number 84031
Whittier, United States
Children's Hospital Colorado_Investigational Site Number 84025
Aurora, United States
IMMUNOe International Research Centers_Investigational Site Number 84028
Centennial, United States
ASCLEPES Research Centers_Investigational Site Number 84023
Weeki Wachee, United States
Children's Healthcare of Atlanta_Investigational Site Number 84034
Atlanta, United States
Rush University Medical Center_Investigational Site Number 84029
Chicago, United States
University of Louisville - James Graham Brown Cancer Center_Investigational Site Number 84033
Louisville, United States
Massachusetts General Hospital Site Number : 84038
Boston, United States
Montefiore Medical Center_Investigational Site Number 84032
The Bronx, United States

+143 more sites — see the official record for the full list.

Common questions

What is ITP?

ITP stands for Immune Thrombocytopenia. It's a blood condition where your immune system attacks and destroys your platelets, which are important for blood clotting. This can lead to easy bruising or bleeding.

What is rilzabrutinib?

Rilzabrutinib is the name of the new medication being tested in this study. It's designed to help increase the number of platelets in people with ITP.

What is a 'placebo'?

A placebo is a 'dummy' pill that looks, tastes, and is given in the same way as the study medicine, but it doesn't contain any active drug. It helps researchers compare the real medicine's effects.

How long would I be in the study?

The study for each participant could last up to 60 weeks (about 14 months), including screening, treatment periods, and follow-up. Some participants might have the option to join a longer treatment period.

Can I leave the study at any time?

Yes, your participation is completely voluntary. You can choose to leave the study at any time, for any reason, without affecting your regular medical care.