Phase 1 Dose Escalation, Single Dose Study to Assess Safety and Pharmacokinetics of BAX930 in Hereditary Thrombotic Thrombocytopenic Purpura (TTP)

Inclusion Criteria: * Subject is between 12 and 65 years of age, inclusive. (The first 2 subjects in any cohort will be ≥ 18 years of age.) * Subject and/or legally authorized representative has provided written informed consent. * Subject has a documented diagnosis of severe hereditary ADAMTS13 deficiency, defined as 1) confirmed by genetic testing, documented in patient history or at screening, and 2) ADAMTS13 activity \< 6%, documented in patient history or at screening. NOTE: In patients receiving prophylactic therapy with fresh frozen plasma (FFP) or other ADAMTS13 containing products, the levels of plasma ADAMTS13 activity may exceed 6% at screening. * Cryoprecipitate, FFP, or other ADAMTS13 containing products interfering with ADAMTS13 PK have to be paused at least 10 days prior to infusion of the investigational product. * The subject is not displaying any severe TTP symptoms at screening. Patients presenting with minor, but stable laboratory abnormalities (LDH not higher than 3 times the upper limit of normal; platelet count not lower than 100,000 per μl) at screening may be enrolled. * Subjects ≥18 years of age have a Karnofsky score ≥ 60%, and subjects \< 18 years of age have a Lansky score ≥ 70%. * Subject is hepatitis C virus negative (HCV-) as confirmed by antibody or polymerase chain reaction (PCR) testing; HCV positive (HCV+) subjects are eligible for inclusion if their disease is chronic but stable. * If female of childbearing potential, subject presents with a negative serum pregnancy test and agrees to employ adequate birth control measures for the duration of the study. * Subject is willing and able to comply with the requirements of the protocol. Exclusion Criteria: * Subject has been diagnosed with any other TTP-like disorder (for example, microangiopathic hemolytic anemia), including acquired TTP. * Subject has known hypersensitivity to hamster proteins or other components of the investigational product. * Subject has a medical history or presence of a functional neutralizing ADAMTS13 inhibitor at screening. * Subject has a medical history of immunological disorders, excluding seasonal allergic rhinitis/conjunctivitis/mild asthma, food allergies or animal allergies. * Subject has a medical history of hematological disorders, in particular systemic lupus erythematosus, amyloidosis, antiphospholipid antibody syndrome, vasculitis, other hemolytic anemia, disseminated intravascular coagulation, and systemic scleroderma. * Subject has a history of significant neurological events, such as major stroke, indicating that a relapse might have severe consequences, as judged by the investigator. * Subject is HIV positive with an absolute CD4 count \< 200/mm3. * Subject has been diagnosed with a cardiovascular disease \[New York Heart Association (NYHA) classes 3-4\]. * Subject is scheduled to undergo elective surgery during study participation. * Subject has been diagnosed with severe liver disease, as evidenced by, but not limited to, any of the following: serum ALT 3 times the upper limit of normal, international normalized ratio (INR) \> 1.5, hypoalbuminemia, portal vein hypertension (e.g. presence of otherwise unexplained splenomegaly, history of esophageal varices). * Subject has been diagnosed with severe glomerular disease, with gross proteinuria and a serum creatinine level ≥ 2.5 mg/dL. * Subject has been treated with an immunomodulatory drug, in case of corticoids with an equivalent to hydrocortisone greater than 10 mg /day, excluding topical treatment (e.g. ointments, nasal spray), within 30 days prior to enrollment. * Subject has a history of drug and/or alcohol abuse within the last 6 months prior to study enrollment. * Subject has a life expectancy of less than 3 months. * Subject is identified by the investigator as being unable or unwilling to cooperate with study procedures. * Subject is a family member or employee of the investigator. * Subject suffers from a mental condition rendering him/her unable to understand the nature, scope and possible consequences of the study and/or evidence of an uncooperative attitude. * If female, subject is pregnant or lactating at the time of study enrollment. * Subject has participated in another clinical study involving an investigational product or device within 30 days prior to study enrollment. * Subject is scheduled to participate in another clinical study involving an investigational product or device during the course of this study.