Subjects diagnosed with hereditary thrombotic thrombocytopenic purpura (TTP) MedDRA version: 18.0 Level: LLT Classification code 10043562 Term: Thrombocytopenic purpura, thrombotic System Organ Class: 100000004851
Conditions
Interventions
Product Name: Recombinant A Disintegrin and Metalloproteinase with Thrombospondin Type-1 Motifs 13
Product Code: BAX930
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Propos
Sponsors
Baxalta Innovations GmbH
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: -Subject is between 12 and 65 years of age, inclusive. The first two subjects in any Cohort will be = 18 years of age. -The subject has a documented diagnosis of severe hereditary ADAMTS13 deficiency -Cryoprecipitate, FFP, or other ADAMTS13 containing products interfering with ADAMTS13 PK have to be paused at least 10 days prior infusion of the IP. -The subject is not displaying any severe TTP symptoms at screening. -Subjects =18 years of age have a Karnofsky score = 60% and subjects =65 years) yes F.1.3.1 Number of subjects for this age range 1
Exclusion criteria
Exclusion criteria: -The subject has been diagnosed with any other TTP-like disorder (for eg, microangiopathic hemolytic anemia), including acquired TTP. -The subject has a medical history or presence of a functional neutralizing ADAMTS13 inhibitor at screening. -The subject has a medical history of immunological disorders, excluding seasonal allergic rhinitis/conjunctivitis/mild asthma, food allergies or animal allergies. -The 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. -The 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. -The subject has been diagnosed with a cardiovascular disease [New York Heart Association (NYHA) classes 3-4]. -The 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, INR > 1.5, hypoalbuminemia, portal vein hypertension (e.g. presence of otherwise unexplained splenomegaly, history of esophageal varices). -The subject has been diagnosed with severe glomerular disease, with gross proteinuria and a serum creatinine level = 2.5 mg/dL. -If female, subject is pregnant or lactating at the time of study enrollment. For the entire list of inclusion criteria refer to protocol section 9.2
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety of BAX930 following single infusions at doses of 5, 20, and 40 U/kg BW, including the occurrence of adverse events (serious and non-serious) and formation of binding and inhibitory antibodies to BAX 930.;Secondary Objective: -To evaluate the pharmacokinetics of BAX 930 following single infusions of rADAMTS13 at doses of 5, 20, and 40 U/kg BW -To evaluate the effect of BAX930 on plasma VWF levels and multimeric patterns ;Primary end point(s): Occurrence of adverse events (serious and non-serious), including the incidence of binding and inhibitory antibody formation, occurring up to 28 ± 3 days after the last investigational product infusion. ;Timepoint(s) of evaluation of this end point: At specified timepoints following IP infusion and at study completion (28 ± 3 days after the last investigational product infusion)in each dose cohort. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): -Standard pharmacokinetic parameters for ADAMTS13 activity and ADAMTS13:Ag after single infusions of rADAMTS13 in Dose Cohorts 1, 2 and 3; -Measurement of plasma VWF:RCo, VWF:Ag and VWF structure analysis prior to and following a single infusion of rADAMTS13. ;Timepoint(s) of evaluation of this end point: At specified timepoints following IP infusion and at study completion (28 ± 3 days after the last investigational product infusion) in each dose cohort. | — |
Countries
Austria, Germany, Japan, Poland, United Kingdom, United States
Contacts
Public ContactManfred Rieger
Baxalta Innovations GmbH
Outcome results
None listed