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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 being developed for hereditary thrombotic thrombocytopenic purpura (TTP), a rare blood disorder. It is an investigational therapy studied in a Phase 1 dose escalation trial in patients with this condition. It is not yet approved for commercial use.
Recombinant ADAMTS13 is being developed by Takeda Pharmaceutical Company Limited, which trades under the ticker TAK. The company sponsored a Phase 1 clinical trial of the therapy in patients with hereditary thrombotic thrombocytopenic purpura.
Recombinant ADAMTS13 is in Phase 1 clinical development. One Phase 1 trial has been completed, and no active trials are currently listed. The therapy remains investigational and has not been approved by the FDA for hereditary thrombotic thrombocytopenic purpura.
Recombinant ADAMTS13 has been studied in one completed Phase 1 trial, NCT02216084, titled a Phase 1 Dose Escalation, Single Dose Study to Assess Safety and Pharmacokinetics of BAX930 in Hereditary Thrombotic Thrombocytopenic Purpura. The trial enrolled 16 patients across the United States, Austria, Germany, Japan, Poland, Switzerland, and the United Kingdom.
Yes, BAX930 is an alternative name for recombinant ADAMTS13. The Phase 1 trial NCT02216084 refers to the investigational therapy as BAX930, while the drug is also known as recombinant ADAMTS13. Both names refer to the same Takeda development program for hereditary thrombotic thrombocytopenic purpura.