New pill shows promise for rare bleeding disorder
NCT ID NCT03395210
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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Beth Israel Deaconess Medical Center- Site Number : 1099
Boston, Massachusetts, 02215, United States
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Bleeding and Clotting Disorders Institute- Site Number : 1087
Peoria, Illinois, 61614-2868, United States
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Investigational Site Number : 101
Clayton, Victoria, 3168, Australia
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Investigational Site Number : 102
Woolloongabba, Queensland, 4102, Australia
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Investigational Site Number : 103
Perth, Western Australia, 6005, Australia
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Investigational Site Number : 104
Sydney, New South Wales, 2139, Australia
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Investigational Site Number : 105
Canberra, Australian Capital Territory, 2605, Australia
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Investigational Site Number : 106
Parkville, Victoria, 3050, Australia
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Investigational Site Number : 1161
Toronto, Ontario, M5B 1W8, Canada
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Investigational Site Number : 1162
Montreal, Quebec, H3A 1A1, Canada
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Investigational Site Number : 211
Varna, 9010, Bulgaria
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Investigational Site Number : 213
Pleven, 5800, Bulgaria
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Investigational Site Number : 214
Sofia, 1431, Bulgaria
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Investigational Site Number : 431
Brno, 62500, Czechia
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Investigational Site Number : 432
Prague, 12808, Czechia
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Investigational Site Number : 433
Hradec Králové, 50005, Czechia
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Investigational Site Number : 434
Ostrava - Poruba, 70852, Czechia
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Investigational Site Number : 541
Grålum, 1714, Norway
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Investigational Site Number : 542
Bergen, N-5021, Norway
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Investigational Site Number : 727
Rotterdam, 3015 GD, Netherlands
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Investigational Site Number : 728
The Hague, 2545 CH, Netherlands
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Investigational Site Number : 980
London, London, City of, W12 0HS, United Kingdom
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Investigational Site Number : 981
Leicester, Leicestershire, LE1 5WW, United Kingdom
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Investigational Site Number : 983
London, London, City of, E1 2ES, United Kingdom
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Investigational Site Number : 984
Birmingham, B15 2GW, United Kingdom
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Massachusetts General Hospital Cancer Center- Site Number : 1092
Boston, Massachusetts, 02114-2603, United States
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Mid Michigan Medical Center- Site Number : 1086
Midland, Michigan, 48670, United States
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New York Presbyterian Hospital/Weill Cornell Medical Center- Site Number : 1097
New York, New York, 10021, United States
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Pitt County Memorial Hospital- Site Number : 1095
Greenville, North Carolina, 27834, United States
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RCCA MC LLC- Site Number : 1091
Bethesda, Maryland, 20817-1915, United States
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Seattle Cancer Care Alliance Site Number : 1098
Seattle, Washington, 98109-4405, United States