New pill shows promise for rare bleeding disorder

NCT ID NCT03395210

Completed Disease control Sponsor: Principia Biopharma, a Sanofi Company Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested an experimental oral drug called rilzabrutinib in 86 adults with relapsed immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, raising bleeding risk. The drug aims to block a key immune signal to help raise platelet counts. The trial looked at safety and how well the drug worked at different doses.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: Rilzabrutinib (an oral BTK inhibitor)
What this could lead to: If successful, this could offer a new oral treatment option for people with relapsed immune thrombocytopenia who have few alternatives.
What could go wrong: This is a relatively small, early-phase study (Phase 1/2) with no placebo comparison. The results may not hold up in larger trials, and side effects are still being evaluated.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

Purpura, Thrombocytopenic, Idiopathic thrombocytopenia due to immune destruction

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

IMMUNE THROMBOCYTOPENIA

Contacts and locations

Locations

Beth Israel Deaconess Medical Center- Site Number : 1099

Boston, Massachusetts, 02215, United States
Bleeding and Clotting Disorders Institute- Site Number : 1087

Peoria, Illinois, 61614-2868, United States
Investigational Site Number : 101

Clayton, Victoria, 3168, Australia
Investigational Site Number : 102

Woolloongabba, Queensland, 4102, Australia
Investigational Site Number : 103

Perth, Western Australia, 6005, Australia
Investigational Site Number : 104

Sydney, New South Wales, 2139, Australia
Investigational Site Number : 105

Canberra, Australian Capital Territory, 2605, Australia
Investigational Site Number : 106

Parkville, Victoria, 3050, Australia
Investigational Site Number : 1161

Toronto, Ontario, M5B 1W8, Canada
Investigational Site Number : 1162

Montreal, Quebec, H3A 1A1, Canada
Investigational Site Number : 211

Varna, 9010, Bulgaria
Investigational Site Number : 213

Pleven, 5800, Bulgaria
Investigational Site Number : 214

Sofia, 1431, Bulgaria
Investigational Site Number : 431

Brno, 62500, Czechia
Investigational Site Number : 432

Prague, 12808, Czechia
Investigational Site Number : 433

Hradec Králové, 50005, Czechia
Investigational Site Number : 434

Ostrava - Poruba, 70852, Czechia
Investigational Site Number : 541

Grålum, 1714, Norway
Investigational Site Number : 542

Bergen, N-5021, Norway
Investigational Site Number : 727

Rotterdam, 3015 GD, Netherlands
Investigational Site Number : 728

The Hague, 2545 CH, Netherlands
Investigational Site Number : 980

London, London, City of, W12 0HS, United Kingdom
Investigational Site Number : 981

Leicester, Leicestershire, LE1 5WW, United Kingdom
Investigational Site Number : 983

London, London, City of, E1 2ES, United Kingdom
Investigational Site Number : 984

Birmingham, B15 2GW, United Kingdom
Massachusetts General Hospital Cancer Center- Site Number : 1092

Boston, Massachusetts, 02114-2603, United States
Mid Michigan Medical Center- Site Number : 1086

Midland, Michigan, 48670, United States
New York Presbyterian Hospital/Weill Cornell Medical Center- Site Number : 1097

New York, New York, 10021, United States
Pitt County Memorial Hospital- Site Number : 1095

Greenville, North Carolina, 27834, United States
RCCA MC LLC- Site Number : 1091

Bethesda, Maryland, 20817-1915, United States
Seattle Cancer Care Alliance Site Number : 1098

Seattle, Washington, 98109-4405, United States

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