Blood

Thrombotic Thrombocytopenic Purpura

Also known as TTP, ADAMTS13 deficiency, Moschcowitz disease

Thrombotic thrombocytopenic purpura is a life-threatening thrombotic microangiopathy caused by severe deficiency of the metalloprotease ADAMTS13, which normally cleaves ultra-large von Willebrand factor multimers; in acquired TTP this defic

ORPHA:54057 ↗Gene ADAMTS13Prevalence 2-6 per million per year for acquired TTPOnset Any age; acquired form peaks in young adults; congenital form presents in infancy or childhoodAcquired (immune-mediated) or congenital (Upshaw-Schulman syndrome)

97

studies recruiting now

as of 7 Sept 2026

556

studies registered in total

as of 7 Sept 2026

15

countries with a recruiting site

as of 7 Sept 2026

26 Aug 2026

most recent study posted

among recruiting studies

Recruiting trials

Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.

See all 97 recruiting studies

Where recruiting studies are running

Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.

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About Thrombotic Thrombocytopenic Purpura

Thrombotic thrombocytopenic purpura is a life-threatening thrombotic microangiopathy caused by severe deficiency of the metalloprotease ADAMTS13, which normally cleaves ultra-large von Willebrand factor multimers; in acquired TTP this deficiency results from autoantibodies against ADAMTS13, while congenital TTP (Upshaw-Schulman syndrome) is caused by biallelic ADAMTS13 mutations. Unopposed ultra-large VWF multimers promote widespread platelet microthrombi in the microcirculation, causing thrombocytopenia, microangiopathic hemolytic anemia, and ischemic organ injury. Without prompt treatment with therapeutic plasma exchange, mortality approaches 90%.

Common clinical features

Thrombocytopenia with platelet counts often below 20,000/uLMicroangiopathic hemolytic anemia with schistocytes on peripheral smearFluctuating neurological symptoms including confusion, headache, and seizuresFeverRenal impairment with elevated creatinineFatigue and pallor from hemolysisElevated LDH and indirect bilirubinCardiac involvement including troponin elevation in severe cases

From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.

Treatments being studied

3 approved treatments and 5 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.

Approved: Apadamtase Alfa (Adzynma)Approved: Caplacizumab (Cablivi)Approved: Cinaxadamtase Alfa
Phase 3Magnesium Sulfate Anhydrous (Magnesium sulfate anhydrous component of suprep bowel prep kit)
Phase 2/3Aspirin (8-hour bayer)
Phase 2/3Methylprednisolone (Medrol)
Phase 2/3Rituximab (Blitzima)
Phase 2Danazol (Danazol)

Before you apply

Things trial teams commonly ask about for Thrombotic Thrombocytopenic Purpura. Not eligibility rules; those are set by each study.

  • ADAMTS13 activity level below 10% with a detectable inhibitor or anti-ADAMTS13 IgG confirms acquired TTP and is required for most immunotherapy and novel agent trials; bring your acute episode laboratory results.
  • Caplacizumab use during acute episode and number of prior acute TTP episodes (relapses) are important eligibility variables for trials of rituximab combinations and novel immunosuppressants.
  • Congenital TTP (Upshaw-Schulman syndrome) patients with confirmed ADAMTS13 mutations may qualify for separate recombinant ADAMTS13 replacement trials distinct from acquired TTP studies.

Related conditions

Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).