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Recombinant ADAMTS13 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Recombinant ADAMTS13 | EXPERIMENTAL | The study is comprised of 3 dose cohorts and two dose escalation steps \[Cohort 1: 3 subjects; Cohort 2: 3 subjects; Cohort 3: 8 subjects\]. Subjects will be enrolled and dosed sequentially. Subjects will be recruited to the next dose level only after short-term safety has been demonstrated and reviewed by an independent Data Monitoring Committee (DMC) at the preceding dose level. The first 2 subjects in any cohort will be ≥ 18 years of age. The effects of the investigational product on vital signs, hematology, and clinical chemistry parameters (up to 96 ± 2 hrs blood sampling timepoint) will determine short-term safety. The DMC will recommend whether to proceed with the study or in case of a safety concern recommend remedial actions and/or to discontinue the study. Subject participation will continue until 28 ± 3 days after infusion of the investigational product. Subject participation in one Dose Cohort (1-3) is expected to be approximately 6-8 weeks. |
| Name | Type | Description |
|---|---|---|
| Recombinant ADAMTS13 | DRUG | rADAMTS13 (a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13) is a lyophilized formulation for intravenous injection. The lyophilized rADAMTS13 is reconstituted with sterile water for injection. Subjects will receive an intravenous injection with rADAMTS13 at a dose of either 5 U/kg bodyweight (Cohort 1), or 20 U/kg bodyweight (Cohort 2), or 40 U/kg bodyweight (Cohort 3). |
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 de...
Recombinant ADAMTS13 is an investigational therapy being developed for hereditary Thrombotic Thrombocytopenic Purpura (TTP), a rare blood disorder. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.
Recombinant ADAMTS13 is being developed by Takeda Pharmaceutical Company Limited, which trades under the ticker TAK. The drug is in Phase 1 clinical development for hereditary Thrombotic Thrombocytopenic Purpura (TTP).
Recombinant ADAMTS13 is in Phase 1 clinical development. It is an investigational drug for hereditary Thrombotic Thrombocytopenic Purpura (TTP) and has not yet been approved by regulatory authorities.
Recombinant ADAMTS13 has one completed Phase 1 trial, NCT02216084, which was a dose escalation, single dose study to assess safety and pharmacokinetics in hereditary Thrombotic Thrombocytopenic Purpura (TTP). The trial enrolled 16 participants across the United States, Austria, Germany, Japan, Poland, Switzerland, and the United Kingdom.
Recombinant ADAMTS13 is also known as BAX930. The Phase 1 clinical trial NCT02216084 evaluated BAX930 in patients with hereditary Thrombotic Thrombocytopenic Purpura (TTP).