A Phase II, Single-Blind, Randomised, Placebo-Controlled Trial to Study the Efficacy and Safety of Anti-von Willebrand Factor Nanobody Administered as Adjunctive Treatment to Patients With Acquired Thrombotic Thrombocytopenic Purpura

Study Status

Open to Enrollment

Study Description

The purpose of this study is to determine if anti-von Willebrand factor Nanobody (also called ALX-0081) is effective in treating patients with Acquired Thrombotic Thrombocytopenic  Purpura (TTP) who require treatment with plasma exchange (PE). In addition, the study will help determine if ALX-0081 is safe at the doses and schedule in this study and has few side effects.

Study participants will be randomized (selected randomly, like a flip of a coin) to receive either ALX-0081 or a placebo (an inactive substance that does not contain medication) as adjunctive therapy to plasma exchange.

Participants will receive a first infusion of ALX-0081 or placebo prior to starting PE. The first PE will be followed by a subcutaneous injection of the ALX-0081 or placebo. Subsequently, patients will receive daily subcutaneous injections of ALX-0081 or placebo following each PE session for the duration of PE, and then once daily for 30 days following the last PE.

Disease Status and/or Stage

Thrombotic Thrombocytopenic Purpura (TPP)



Key Eligibility

  • Men and women age 18 and older
  • Diagnosed with Thrombotic Thrombocytopenic Purpura (TPP)
  • Needing plasma exchange (PE)
  • Detailed eligibility reviewed when you contact the study team

Principal Investigator

Maria DeSancho, MD


  • June Greenberg, RN
  • (212) 746-2651
  • [email protected]
  • Non-Malignant Hematology Medical Practice
  • (646) 962-2065

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Contact Us

For general inquiries, or if you need assistance finding a study, please contact:

Robert Hagerty
Subject Recruitment Manager
Tel: (646) 962-9340
[email protected]

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